2 min read

Why I Joined Medable: Diversity & Drive

By Jared Klingeisen on Jun 3, 2021 3:44:58 PM

Since I joined Medable 3 years ago a lot has changed, but many of the reasons I joined still hold true today and continue to evolve as we grow. To highlight a few of them: 

Mission Driven Mindset 

We spend a lot of our lives working. I wanted to work somewhere that my tireless work ethic and effort could help others. At Medable our mission is to enable effective therapies to reach patients faster through better clinical trials. If we do our jobs well there can be less suffering and better health. This is why we do what we do, and it’s why I enjoy waking up and going to work every day. It’s empowering and rewarding to work with colleagues that share this same drive and passion. 

Diverse Perspectives 

I’m a firm believer that people make a company. In order for us to disrupt and evolve we have to think differently. As a result our team is made up of incredible people from different industries, companies, countries and walks of life. Those different perspectives and backgrounds help us to look at and resolve problems in a new and often better way. For example, our Patient Advisory Council provides us the direct perspective of patients, family members and researchers that are fighting the diseases we hope to help cure. 

Our diverse teams span across the globe, and the unique perspectives each person brings help us better serve our partners and patients, create opportunities for more diversity and inclusion in clinical trials, and keep us laser focused on our mission.

Entrepreneurial Opportunity 

One of the things I often hear in interviews is that people want to work somewhere where their thoughts, ideas and suggestions are heard and respected. Time and again I hear stories of companies where suggestions for improvement take so long to go “up the chain'' that they don’t make a timely impact. Here we strive to constantly “uplevel” our organization and encourage everyone to share their thoughts and drive improvement on a daily basis. Together we collaborate to build a company and a solution that we are all proud to be a part of. 

At Medable, we continue to look for people who think differently to join our diverse and ever-evolving team. I think Michelle (our CEO) said it best, “If you love learning, are humble but confident, empathetic yet unrelenting in the pursuit of goals, are inspired to be the best teammate and fiercest opponent - and, most importantly - are dedicated to the mission of getting effective therapies to patients faster Medable wants you.”

View our open positions: https://www.medable.com/company/careers

Topics: decentralized trials life sciences Innovation Diversity Inclusion
1 min read

Why I Joined Medable: Advancing the DCT Movement

By Andrew Mackinnon on Apr 30, 2021 1:09:41 PM

Throughout my career in clinical research, encompassing time within big pharma, small biotech and large CRO, the increasing expectations that we place on both sites and patients and the additional burden that this creates has always been something that I have been careful to consider and endeavor to minimise. During my involvement in one of the very early decentralised clinical trials (DCTs), and observing the benefits this approach brought, the spark in me was set and I knew that this was an area I wanted to work in.

Having been introduced to Medable through a previous colleague of mine and after having met our CEO, Michelle Longmire, who passionately explained her vision for the company and what she wanted to achieve it was clear that Medable was going to be at the absolute forefront of this paradigm shift in how clinical research was conducted. I immediately knew that this was the right company for me to join so that I could play a part in this movement. 

Joining Medable in early January 2020, the dramatic acceleration in the uptake of DCTs in response to the COVID19 pandemic was about to start. Little did I know what a wild ride we were all in for, but also what an amazing experience it would be working alongside the most passionate, dedicated and focused colleagues as we looked to implement new patient recruitment, patient management and data collection methods into both ongoing and new studies. 

For me, Medable fulfils the most important aspects of job satisfaction. I’m able to observe the daily impact my work is having in tangibly reducing the burden of clinical trials and improving access to clinical trials to a broader base of the global population. Medable now has the privileged position of being the market leader in the DCT landscape and this gives me the opportunity to work at the cutting edge of DCTs alongside incredible people throughout the industry. Being part of changing this industry, simplifying clinical research and improving access to clinical trials and bringing medicines to patients quicker is truly the most satisfying role I’ve had in my career and I can’t wait to see what tomorrow brings.

6 min read

Call Centers and Clinical Trials

By Allison Kalloo, MPH on Apr 16, 2021 7:59:52 AM

If I were not personally and professionally involved in clinical trials myself— if I lacked the experience and navigational knowledge to fall back on, and if I did not possess the wherewithal and determination to pursue answers to my questions— there is no way I would ever participate in clinical trials. I don’t have the patience. It feels cumbersome and is burdensome.

 Why?

 Like many of you, my experiences with external call centers have been underwhelming at best. Unfortunately, this includes my perspective as an advocate for enhancing diversity in clinical trials, as well as on my own behalf as a patient/participant. The obstacles encountered have led to a host of frustrations. (Here is what has been routinely missing with themes in bold):

  1. What’s the 411? Finding a number to call in the first place!
  2. Anybody there? Getting hold of a live human and instead, reaching a recording from an answering service requesting that caller’s leave their name and contact information for a callback that may or may not happen
  3. Just answer my question. Being able to get study-specific answers because the answering service is set up to only refer callers to the caller’s local clinical site for more information (and again reaching the coordinator’s voicemail at the site)
  4. Am I in or out? Being able to get pre-screened on the first try (first call) rather than at some later time
  5. Taking my sweet time. Being able to speak with someone who was 1) knowledgeable about the study in question, but 2) also courteous, engaging, and not in a rush to get a caller off the phone so they could move on to the next one
  6. Are you speaking from experience? Feeling comfortable and connecting with operators who demonstrate authentic interest and concern about the human component of participating (rather than merely the mechanics of enrolling patients)

 From the outside looking in, call center operations provide precious little encouragement to participate in the way of customer service, and too few early encounters people can genuinely feel good about. The most apropos comparison that comes to mind is trying to get through to the DMV, and that’s being generous.

Once, there was a time that I was placing a call to a clinical site to make some preliminary inquiries on behalf of a patient. ClinicalTrials.gov had listed the Principle Investigator as the point of contact. He actually answered the phone with impatience and outright hostility, annoyed that I had called, demanding to know why I was calling him, and how I had gotten his number. It was horrifying. I followed up and did not let it end there. But I digress.

 As a rule, it is entirely too difficult for the average person to locate studies or the call centers that support them. It is too difficult to identify and to grasp the inclusion/exclusion criteria (due to the medical jargon) that would precipitate making such a call. And it is too difficult to speak with a live person who can clarify early clues as to what’s involved and on which a caller will base decisions about participating.

The call center must be viewed as an integral component of the clinical trial experience. Not ancillary to it. From the end-user perspective, call centers have routinely been so problematic that I have come to believe that— done poorly— they actually do more harm than good.

The fact of the matter is that call centers often provide the first impression of any study. If attention to detail is lacking, call centers can actually turn candidates off before they’re even screened. If callers feel as if their time and interest are taken for granted... if callers don’t feel respected and heard during the call, this brief encounter can become the last and will be an indictment of the industry and clinical trials as a whole.

To emphasize this point, it is in response to these experiences that we began providing a call center feature for our Clinical Ambassador clients to enhance patient recruitment when we were contracted to provide. We understand first-hand how critical it is to connect with people on more than a superficial, mechanical level. We recognized just how much this first encounter can be their introduction to the clinical trial process, sets the stage for the patient experience, and for many people of color in particular, is a critical building block of trust.

The importance of offering expanded hours of operation cannot be overstated. Among underrepresented patient populations, a significant percentage of the communities we seek to engage happen to work second and third shifts. These callers can encounter strict rules at work regarding the use of their cell phones and where personal calls can take place. Being cognizant and accommodating regarding the times that people are able to reach out is important also.

We train our Ambassadors to be familiar with the parameters of specific studies and to be ready to have real conversations about what it’s like to participate in a clinical trial— from the patient perspective. We have used our call center to help manage expectations so that otherwise viable candidates don’t get inaccurate impressions about what’s involved and get frustrated or feel otherwise unprepared for study participation and drop out.

Should callers choose to enroll in a study, those who have opted in have access to Ambassadors who stand ready to act as their advocates and on whom they can call every step along their participation journey. And not only do our Ambassadors assist callers by pre-screening for contracted clinical trials, we also perform study searches for “outside” studies when the I/E criteria of the original trial are not a suitable match for them and have a list of additional resources ready to share.

The rapport created from the get-go means that callers also refer members of their families, coworkers, and community members to the call center. Our contacts have reached out to us to mitigate logistics such as transportation and daycare when they have technical difficulties with their diaries (we have helped troubleshoot issues with tech support as needed), when they have concerns about an upcoming procedure, and even when they wanted to report not being treated well during their last visit. Peace of mind and someone to speak to means the difference between someone staying in a study to completion versus being lost to follow-up.

Site staff and typical call centers have neither the time nor the finesse to hold hands with participants. Clinical Ambassador enjoys being relied upon to be a reassuring voice for our callers. We make a point of letting them know that they can discuss anything related to their participation with us and that they will get nothing but straight-talk in return. We also escalate issues to clinical site administrators on participants’ behalf so that sites can have the opportunity to address problems as early as possible before patients get so frustrated that they drop out.

At Clinical Ambassador, our remit is addressing disparities in participation and advocating on behalf of equitable access and representation in clinical trials. In the interest of cultural sensitivity, our boutique call center takes the time to nurture relationships and allow interpersonal dialogues that address issues ranging from basic education about clinical trials to historical distrust to specifics on a study-by-study basis. As a result, communities of color who experience disproportionate disease burden and underrepresentation in clinical trials in parallel come to better understand participation in the context of diversity, equity, and inclusion.

Being responsive to a broader spectrum of participating candidates and allowing all to feel greater comfort about the unknown— in what can effectively be a foreign environment— is critical. The best call centers are those prepared to field queries from callers with a range of familiarity, that only hire operators who blend being personable with professionalism, and those that can be most accommodating to callers with physical/health or technological limitations.

Of course, there are call centers set up to collect information about patient experience and side effects in particular. But if patients find that their call center encounters have been more mechanical and impersonal and less than pleasant to that point, potential data about drug reactions will likely be incomplete at best because it may go unreported.

The take-home message is that there must be attention paid to “the small things.” In my experience, “real people” respond most favorably and are more likely to enroll (or remain) in a clinical trial when they feel confident in the people and the process, in what’s expected of them, and when they have a solid understanding of what they can expect from those involved.

From my experience, people who use call centers are interested in knowing whether they qualify, they desire a better understanding of study requirements, may need help troubleshooting technical issues, and just need reassurance that they can trust what they’ve agreed to.

Bottom line:

If any call center is not prepared to implement exemplary customer service, to be accommodating and to meet callers “where they are” regarding their knowledge, attitudes, and beliefs, and if they are not actively pursuing the balance between the technology of decentralized clinical trials and humanizing the patient experience, then don’t bother.

 

Allison Kalloo, MPH

 

 

5 min read

Let's Make it a Universal Language: Clinician Reported Outcomes

By Dr. Reem Yunis, PhD on Apr 14, 2021 2:37:39 PM

In prior posts, we noted that COAs play a significant role in regulatory submissions. In  this post we will look closely into the clinician reported outcomes (ClinROs) type.

Medicine is both science and art, and as such it is subjective in nature, as those who practice it have their own perception of the disease and how their patients are responding to the prescribed treatment. Because approvals of regulatory submissions take into consideration patient health outcomes in regard to effectiveness and safety of the new product (or repurposed product), clinical outcome assessments determined by clinicians must follow the scientific method more than the art! Clinical assessments utilizing ClinROs must be carried out by a healthcare professional with specific training in the area in which the rating/assessment is being determined to establish confidence and to ensure reliability in the rating, especially when utilized to determine the benefits and risks of a medical intervention.

The path to having a meaningful and validated ClinRO can be long and it must follow a rigorous scientific process. The FDA has put forth guidelines and workshops to provide clarity and guidance around clinical assessment outcomes (COAs) in clinical trials. Early on, these activities were mostly focused around developing and implementing patient reported outcomes (PROs) leaving some ambiguity around the development of ClinROs. Luckily, the The Professional Society for Health Economics and Outcomes Research (ISPOR)-task force stepped in to provide a framework for developing ClinROs. 

“The explicit objective in following good measurement practices is to minimize error in measurement of the concept of interest and achieve clarity in the interpretation of study results when evaluating treatment benefit.“ ISPOR task force (2017)

The ClinRO must be well defined in addressing a health concept, measurable, consistent in its interpretation, and with minimum inter-rater variability. In contrast to PROs, patient perspective in developing ClinROs is not central. Involving clinicians in the design and development of ClinROs is, however, essential. Their professional expertise, understanding of the disease and the nuances of treatment and outcomes provide critical information to the validity of ClinROs. 

The development of ClinROs generally follows the PRO development process and falls under 4 major steps. When developing ClinROs to be used as endpoints in clinical trials additional input needs to be considered.  

Major steps in developing ClinROs

There are three main types of ClinROs:

Readings. These are assessments where well-trained clinicians use their professional judgement to determine the results after reading tests such as pathology reports, mammography, MRI, x-ray, etc. The decision is presented in a dichotomous manner such as Yes/No and Present/Absent. 

Patient input is not needed when developing Reading ClinROs. Concept of measurement, context of use, and confirmation of measurement steps must be well defined, while the content validity step is irrelevant in the development of this type of ClinRO.

Ratings. Clinicians use scales or instruments to rate the clinical assessment similar to patients using PRO instruments. These scales can be continuous or categorical measures with at least 3 levels. The knowledge and expertise of the administering clinician for Rating ClinROs is paramount to the validity of the results. Examples of such ClinROs include the Montreal Cognitive Assessment (MoCA), Karnofsky score, and Psoriasis Area and Severity Index (PASI). The development of Rating ClinROs must follow all four development steps and take into consideration inter-rater variability (see below) which can greatly impact sample size and the iterative development processes.

Clinician Global Assessments. This type of ClinRO concerns the clinician global impression and the clinician global impression of change, and it depends heavily on the professional judgement of the clinician, their knowledge of the disease, the treatment modalities, the patient’s health status, and the methods by which the health status is being assessed. This falls more under the art side of medicine than the science, and therefore when used in clinical trials, they are usually used as secondary or exploratory measures for evaluating the effectiveness of an investigational intervention. Their effectiveness could be enhanced by using them in combination with biomarkers, PROs, etc. It is essential to consider the context of use and measurement properties, which must be well defined, when developing this type of ClinROs.

ClinRO as endpoints

ClinROs are considered surrogate endpoints because they provide insight on and can indirectly measure how the patient feels or functions. However, like all other COAs, when being considered as endpoints, ClinROs must meet the fundamental requirements for an informative measure that addresses how patients feel, function, or survive. Because of that, the FDA encourages the use of PROs as primary and secondary endpoints in clinical trials because they directly provide measures on the patient’s feelings and function. This has proven to be valuable and successful in regulatory submissions. On the other hand, the use of ClinROs as primary or secondary endpoints continues to lag behind. One could argue this is because of the complexity of the clinical evaluation and outcome determination that include, understanding the disease mechanism, disease presentation and manifestation, comorbidities, environmental factors as well as the clinician’s own perception of the disease and the patient. This complex evaluation could lead to greater variability in a measured outcome between different clinicians, what is referred to as inter-rater variability. The ISPOR task force therefore has formulated the steps for developing and validating ClinROs factoring in their potential as study endpoints covered in their 2017 article. 

Steps in developing ClinROs as endpoints

Remote ClinRO

The pandemic has rapidly pushed the healthcare and pharmaceutical industries to adopt remote and digital tools to continue providing care and monitoring their patients. Regulatory groups have also provided greater flexibility in how healthcare and clinical trial services can be provided. In the last year alone, there has been a boom in remote patient monitoring technologies in healthcare and clinical trials such as televisit, ePRO, eConsent. This evolution is welcome and provides greater opportunities to innovate and foster more remote assessments that reduce patient burdens related to travel and time off of work for clinic visits, and more likely allows for better healthcare access and increased diversity in clinical trials. 

Could ClinROs also ride the wave and be completed remotely? Is that feasible for at least a portion of them? Yes, ClinROs utilize scales, but still require patient-clinician interaction and input from these stakeholders is important. What do investigators and clinicians think about conducting remote clinical assessments? How confident are they in their reliability? At a minimum, ClinROs will have to follow a rigorous testing and validation when being converted to remote assessments. We look forward to including the scientific method to enable robust and rigorous validation to foster the move to remote  digital ClinRO options!   

Resources

Powers JH. III,  Howard K,  Saretsky T, et al. Patient-Reported Outcome Assessments as Endpoints in Studies in Infectious Diseases. 2016 Clinical Infectious Diseases, 63(2):S52–S56.

Powers JH. III, Patrick DL , Walton MK, et al. Clinician-Reported Outcome Assessments of Treatment Benefit: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force. 2017 Value in Health, 20:2-14.

Topics: Hybrid Trials clinical trials Decentralized eCOA ePRO
2 min read

Why I Joined Medable: Ambitious, Knowledgeable, & Passionate People

By Amanda Inks on Apr 13, 2021 6:28:54 AM

   My career in quality assurance began in a Viral Pilot Plant working on Phase I and Phase II drug substance. This was the first exposure I had to the clinical trials and the importance of Quality Assurance. I fell in love with Clinical Research and Product Development! There is so much work and so many people that go into the development lifecycle to get safe and effective therapies on the market. My interest in Clinical Research is what lead me to join Medable. At the time Medable was a small company with a big dream.

   I was encouraged to apply to the Quality Assurance role at Medable and coming from big Pharma companies like Novartis and Novo Nordisk I didn’t know what to expect from this up and coming company. Once my foot was in the door, I was met with some of the most ambitious, hardworking, and knowledgeable people I have ever worked with. Medable takes its mission to get effective therapies to patients very seriously, with a passion that seems like it is able to move mountains.

Screenshot 2021-04-06 at 17.21.11

   My role at Medable has provided me with the opportunity to learn and grow with the company. Medable is pioneering a new path to flip the clinical trial world on its axis and give the patients some possession of the process using technology. It is exciting to be brought into some of the novel discussions and decisions that shape the way we work. The expression “where there is a will there is a way” definitely applies to Michelle and her mission. In the current state of the world, I think everyone has realized how important it is to be able to provide effective treatments to patients quickly.

   Medable’s unwavering determination and common goal keep the individuals at Medable held together as a community even though we are a remote workforce. The opportunities at Medable have not only allowed me to grow professionally but also personally. I feel empowered to educate others on the importance of clinical trials and Medable’s mission. I know when I go to sleep, I am working towards a patient getting an effective treatment faster and that makes a difference. Medable has made a difference in my life, maybe it will make a difference in yours one day.

If you are interested in joining our diverse, motivated team, view our open positions here: https://www.medable.com/company/careers

3 min read

Wrestling with the Monster That is COVID-19

By Joan Venticinque, PAC member on Apr 8, 2021 7:55:47 AM

It all started with a headache

Twice I have heard the words, “You have cancer.” They brought fear, anxiety, shame and guilt. Cancer patients often question whether they brought the illness upon themselves. Did I eat the wrong food? Was I too stressed out? Did I not exercise enough? 

When I received the call that I was positive for COVID-19 those same emotions came quick and fierce. How did I get it? Did I not use the hand gel religiously? Who have I exposed?

It started with a severe headache and chill I couldn’t get rid of. Upon first appearance of these symptoms, I wasn’t thinking COVID-19 as the cause; maybe just a migraine and the cold weather were the culprits. The next day fever, body aches, fatigue and joint pain were added to the list of symptoms. I was still questioning if this was COVID-19 as I went to the drive-through testing site. Most of us think COVID-19 will not happen to us. Then it does, and when that denial falls apart it is frightening.

By January 2021 we all heard the horror stories of what a monster COVID-19 could be. These personal accounts would incite fear in anyone. Would today be the day I start having trouble breathing? Would I have to go to the hospital, and would I make it out alive? Would I suffer from Long-COVID?

Monoclonal antibodies

As it turns out, getting a COVID-19 test at a major research hospital is a golden ticket to cutting-edge treatment. The day after I tested positive, a research team called to tell me I was eligible for monoclonal antibody treatment and asked if I was interested. Even though it was still a treatment under FDA emergency use authorization, I jumped on it. It was a way to increase my odds of staying out of the hospital. 

The next day, I drove alone to the hospital and waited outside for the nurse to come and get me. I was taken to a private room and soon the IV was in and I watched as those antibodies dripped into my vein. I was fearful of side-effects, but I was more afraid of COVID-19 taking me on a longer unpleasant ride. As I lay there, I was watching a live Zoom presentation on ‘COVID-19 and the Immune Response’ from the Precision Medicine World Conference. Ironic, I thought as I lay there getting immunotherapy for COVID-19. My second thought was just how lucky I was. I live in the right place and knew the right questions to ask and had the time and ability to get the therapy. I thought about the millions of COVID-19 patients who did not have the access I did, and how unfair it was that this therapy was out of reach for so many people. COVID-19 has laid bare the inadequacies of the US healthcare system, and how the populations affected the most from COVID-19 don’t have access to the care they need.

 It came for my family

COVID-19 didn’t just affect me physically and mentally, but socially as well. I had an incredible amount of support when I was diagnosed with cancer. COVID-19 is a much different monster. You are isolated not just from friends and co-workers, but from family. There are no reassuring hugs four times a day. The fear of how bad it might get are thoughts that are hard to drive away when you are alone.

Turns out I wasn’t alone for long because the big scary monster had come for my family, just as it has for millions of others. My 2.5-year-old asymptomatic grandson tested positive a week after me. Three days later his dad, my son, tested positive. The feelings of guilt weighed heavily on me. Did I give this to them? We had decided when COVID-19 started we would keep our family bubble together and safe. We were all being so careful. How did this happen? My son and grandson moved in with me and my diabetic husband moved out. My daughter-in-law was now alone with her 3-month-old. For the next 3.5 weeks we all lived our separate COVID-19 lives. Finally, after almost a month at “Nonni’s COVID Camp” my son and grandson went home. I don’t think I have ever been more exhausted in my life. Chasing and playing with a 2.5-year-old while trying to recover from COVID-19 is not on the doctor-ordered recovery plan.

The Hunger Games

I have recovered, with some lingering taste issues, fatigue, and COVID-19 brain fog. Those emotions of fear, shame and guilt are still there but I am working on them. I have read that some side-effects get better after a vaccine. Because I had the monoclonal antibody treatment, it was recommended I wait for 90 days to get my vaccine. Now that the eligibility for vaccines has opened to younger age groups, I can’t find any available appointments. Living in a state with 39 million people and trying to get a vaccine is like the Hunger Games. But at least I am still around to play, and the monster didn’t win.

 

4 min read

March Pulse Check on Decentralized Clinical Trials - Overcoming DCT Implementation and Planning Obstacles

By David Swanger on Apr 1, 2021 1:26:24 PM

Throughout 2021 we’ll take a look at the latest developments in the realm of Decentralized Clinical Trials. Read this monthly roundup to discover the latest trends, insights, and modalities of decentralized clinical trials.

Over the past year, we at Medable have seen tremendous growth in Decentralized Clinical Trials (DCT’s). In 2020 DCT’s moved from edge case to main case. And as we look back on the first three months of 2021, it’s clear that there is tremendous interest across life science organizations to adopt DCT’s on a widespread level.

Essentially, the question life science organizations are most often asking when it comes to DCT’s is no longer: “Why DCT’s?” 

Today the question is “How?”

How do study teams work together to implement, deploy, and manage a DCT? How do they customize it for their specific treatment, protocol design, and therapeutic area? 

And once digging into specifics several other questions emerge such as:

Can we decentralize studies into therapies with higher toxicity profiles, such as those in oncology? How do we ensure older participants are able to successfully use eCOA and DCT technologies? Can we use a remote eCOA and DCT approach without the reliance on multiple vendors and disjointed solutions? 

In March at Medable, we focused on demystifying how to overcome DCT Implementation and Planning Obstacles. 

On March 8th, 2021, leaders from Medable, Janssen, J&J, PPD, Mallinckrodt Pharmaceuticals, and UCB led a Reuters featured webinar focused on “Overcoming Perceived DCT and eCOA Adoption Hurdles.” You can view the webinar here.

Earlier on March 4th, Medable’s Ali Holland helped attendees navigate how to utilize a single platform for virtually all sensors and wearables, as they continue to become hallmarks of DCT’s. While these sensors and wearables can bring additional quality of life, disease state, and demographic data to complement traditional primary endpoints, managing them in a disparate manner can introduce complexity and risk to a study. To learn more, you can view the webinar here.

Finally, on March 29th as part of Decentralized Clinical Trials Digital week, we delivered the opening keynote of the event delivering a webinar:

“Decentralized Trials Have Arrived: How to Successfully Implement Decentralized Trials.” You can view the webinar here.

 

Medable in the News in March 2021

During March, Medable was also featured in several news articles with a focus on highlighting how different stakeholders in the clinical trial process can successfully adapt to a Decentralized Clinical Trial approach. These articles focused on several key stakeholders, including study teams, patients and legal teams.

Clinical Trials and Tribulations

Our own Parag Vaish, Chief Product Officer, sits down with The Medicine Maker’s Maryam Mahdi to talk about pharma’s adoption rate of new clinical trial technologies, including DCT’s

Medable Advisory Council Works to Amplify Patient Voice, Inclusivity

Medable Patient Advisory Council (PAC) member Allison Kalloo, and our own Reem Yunis, Head of DEI at Medable were interviewed by Outsourcing Pharma on how we can work together to bring diversity, equity, and inclusion to modern trials.

As Clinical Trials Move Off-Site, Legal Teams Prepare for New Challenges

Andrea Sanchez, Vice President, Legal & SaMD at Medable covers the changing legal landscape, as more trials at home create new types of legal challenges

International Women’s Month

March also marked International Women’s Month. During March, Medable founder and CEO Dr. Michelle Longmire sat down with Made By Women, a podcast about women who are transforming the world in unique ways. In the conversation with host Kim Azzarelli, Michelle shared her journey to entrepreneurship and how women can support each other’s ventures.

You can view the blog and listen to the podcast here!

https://blog.medable.com/womens-history-month-ceo-michelle-longmire-explores-medables-quest-for-discovery-on-made-by-women-podcast

Why We Joined Medable? And Why You Should Too.

Curious as to how and why people are joining Medable? In March Anushka Gupta, a Data Scientist, and Snadeep Jutla, a Digital Project Manager, both shared their excitement as to why they joined Medable, and why you should too. 

You can read them here!

Looking Forward

As March turns to April, we at Medable will continue to help sponsors, CRO’s, sites, and patients to successfully navigate their transition to Decentralized Clinical Trials (DCT’s). In April we’ll focus on how a DCT Platform approach enables each clinical trial to establish bespoke workflows, blending technology-enabled services to suit the protocol, sites and patients. This results in accelerated recruitment, increased patient retention and quicker progress to database lock. We are the first platform to enable the entire patient journey to be digitized from recruitment to closeout, across activities both remote and in-person.  Our platform is architected for ease of integrations, such that we partner with best-in-class wearables/devices and integrate the set-up, training and data flow into the patient and site-facing workflows where possible. This enables Medable to offer the most flexible decentralized clinical trial platform allowing sponsors to scale up and down their hybrid capabilities to suit their unique studies.

If you’d like to see the Medable Platform in action, sign up for a demo.







2 min read

Bias and Decentralized Trials: How to Avoid Selection Bias Through Proactive Action

By Mary Costello on Apr 1, 2021 10:10:09 AM

Avoiding bias in clinical research has been an important concern of drug makers worldwide and is part of what guides oversight on every level.  As we move into an era of opportunity to expand clinical trial inclusion via the power of technology, we will also need to ensure that we don’t create the conditions for bias as a result of access to devices or wifi.

What do I mean by that statement?  Successful decentralized trials will rely heavily on a reliable internet signal capable of transmitting data on a regular basis.  Much of the new technology offered to support DCTs comes with the convenience of collecting data through patients’ laptops or phones, which provides an ease of participation that did not exist in traditional RCT models.  However, these interactions with patients presuppose that they will have phones capable of working with apps (iPhones or Androids), or that they own a laptop and that they have a consistent and reliable internet signal.

It is heartening to see a national conversation turn in the direction of diversity and outline guidance and the necessity of expanding clinical research access to a broader community.  But project planning that fails to account for the variety of scenarios that exist today – especially in some of our lower income communities -- will mean that by default, these same communities that are at the forefront of the conversation will continue to get left behind. To prevent this, project planning must proactively introduce ways of provisioning devices and ensuring access to a strong signal as a key part of widening the inclusion opportunities for all patients.

Sites must be at the forefront of this planning. They will need to understand what challenges may exist and be provided with the support and budget to remove any technical barriers that may prevent participation or continuation in a study. While this may seem like a fairly straight-forward task, there is a danger in not considering the breadth of challenges facing some patients. For example, consider the experience of one site where, at the height of COVID, participants in a trial without home access to adequate wifi were asked to travel to their local library, daily, to upload their diary details.  Yes, that was in the midst of a pandemic.  Realizing the difficulty in this, the site asked for hotspot support but the sponsor did not want to allow this additional request.  The result? Many of the participants dropped out, depleting the numbers and introducing systematic bias in the sample. 

I would encourage sites to begin collecting data that will help gauge the technical situation of their patients.  This could be as simple as emails, whether participants are able and willing to communicate via text, what type of phone they have, whether they have wifi at home, and, importantly,  asking them to rank their comfort level with technology.  Having this information at the ready will help with pre planning, device support, and other critical information that will help assess their ability to support the study while creating a budget to do so.  For research  sites with an associated healthcare practice,  EMRs may already have collected some of this information, providing them with a useful first step in planning.  

In summary: If we know that today’s trials have inherent bias, then we must make sure future trials include everyone, from all types of communities and all types of situations.

 

3 min read

Women’s History Month: CEO Michelle Longmire Explores Medable’s Quest for Discovery on “Made by Women” Podcast

By Big Valley, on behalf of Medable on Mar 30, 2021 7:17:39 PM

Women’s History Month has been filled with amazing stories about women’s accomplishments across all walks of life. Unfortunately, women entrepreneurs continue to face an uphill climb. While there are 114% more women entrepreneurs now than 20 years ago, still less than 3 percent of all venture funding goes to women entrepreneurs. That’s especially ironic since private tech companies led by women reportedly achieve 35% higher ROI

Medable founder and CEO Dr. Michelle Longmire recently sat down with Made By Women, a podcast about women who are transforming the world in unique ways. In the conversation with host Kim Azzarelli, Michelle shared her journey to entrepreneurship and how women can support each other’s ventures. 

 

Know Your Strengths & Lean Into Them

Michelle always knew she had a passion for science—inspired in part by her mother (also a scientist)—and was “always inspired by how discovery could transform the world.” 

However, it wasn’t until her time at Stanford that Michelle knew she would be an entrepreneur. While she was working in rare disease research at Stanford, she saw gaps and inefficiencies in how clinical trials were happening. Michelle wanted to use her love of science, rare disease and desire to help patients. She saw that research needed to happen quicker, with less cost and fewer hurdles to trial execution. 

It was that knowledge and empathy for patients that drove her to see how research could be done differently, which led to development of new systems to make clinical trials accessible to nearly everyone and easy to use. 

 

Find Your Way

While less than 3 percent of venture funding goes to women-led startups, Michelle has been able to defy the odds—raising $136 million to date. Michelle attributes the success in part to a good support system and a lot of tenacity.

When Medable began, Michelle was building it on the side. When she wasn’t practicing medicine, she was attending hackathons and learning how to build a startup. She encourages listeners to keep pushing forward, even if they hear no a thousand times. Since it’s harder for women and minorities to get funding, Michelle stressed the importance of leaning on existing support networks for knowledge and potential funding.

Based on Michelle’s vision for clinical trials, along with a couple of talented developers and an initial outside investment of $50,000, she set Medable in motion. She tells listeners: “Starting your own company is freeing. Nothing can prepare you for it, but you’ve just gotta do it.”

 

Support Other Women

Michelle says the tech industry—and every other industry—needs more women entrepreneurs. Part of what will make this possible is through supporting other women. And it can’t be just in good times. When you see another woman entrepreneur struggling, Michelle says you must lift them up. The community is small but strong and growing.

What started out as Michelle’s side hustle and desire to help people with rare diseases, Medable’s decentralized trials platform now has over one million users. 

Michelle leaves listeners with this invaluable advice: “If there’s something you’re passionate about, and you’re thinking about starting your own company, go for it! And before you know it, you’ll have made more progress than you had even originally imagined.”

You can listen to the full podcast episode here, where Michelle shares more about her journey from clinical research to technology CEO. 

 

Topics: clinical trials womens history month
2 min read

Technology for Clinical Trials: What can be Learned From the COVID-19 Vaccine Roll Out?

By Mary Costello on Mar 23, 2021 2:20:30 PM

By Mary Costello and Sina Mossayeb

Good news here!

The US has already achieved its target of 100 million Americans vaccinated in 100 days.  Cities, towns and states across the country have pulled off an amazing effort supported by huge teams of volunteers and healthcare professionals who have enlisted stadiums,clinics,mobile transport and public buildings to accomplish their goal. Despite a wide range of processes for rolling out the vaccine, there is one thing they have in common —the registration for the vaccine itself has been anything but simple.

Even “tech centers” such as San Francisco, New York City ,Boston and Austin, where user experience is part of day to day consideration, faced frustrating interfaces for vaccine registrations.  Nonetheless, resilience prevailed, with many of the first wave of eligible patients turning to their children, relatives, and neighbors for support. Tech savvy volunteers on NextDoor, for example, posted offers to help guide often despondent patients through that cumbersome process.

So, what can we learn from this effort?  

Obviously, the strong desire to be vaccinated is enough motivation to ensure that the end user soldiers through. 

But what about our industry? Clinical trials can be an even trickier experience, and studies have shown that frustration in the user experience will cause patients to pause treatment, or give up on participation completely.

Good tech is grounded in human centered design, which considers not only the desired outcome, but also the human journey that defines the experience. This design begins with empathy for end users, considering potential internal or external barriers to entry, their hopes and fears. And, in the best case scenario, it involves co-creating the experience alongside those who will be using the product. The practice is often referenced to as user experience design. Tech as a proposed better solution should support and uphold the experience, not become an impediment or aversion to the goal. 

A well-planned trial considers all the risks to successful study execution prior to finalizing a protocol. To get good data, one might consider making the experience human centered so that engagement increases, by way of a deeper understanding of the people in the trial.  Considerations should include patients’ age, living situations, transportation barriers, abilities, mindsets around health and health providers, support systems and more. For example, are the subjects you are trying to include fearful of hospitals and doctors? Beyond demographics, how do financial difficulties become blockers for participating in a trial or even considering one on a day to day basis?

Remember the registration for vaccines that we started with at the outset of this post? What if the font sizes were bigger. What if less information was on each page, and allow for bite size digestion? What if terminology was made simpler? What if there was an audio or video option to walk seniors through the process? What if there was an ability to hit a button to have a volunteer give you a ride (if you didn’t have someone in your life to help you)? These aren’t just features, these are human centered considerations to create a better experience, which in turn drive results.

The silver lining to every major challenge we face is that those same situations also turn into opportunities to incorporate learnings and continue to improve a more effective process.  Just as we will emerge from the pandemic with a heightened awareness of clinical needs and settings around the world, we will also be in a position to improve public health roll outs for the future. Here’s to the next 100 million and to using human centered design as our starting point.

2 min read

Highlights From Scope 2021, and the Clinical Trial Road Ahead

By Dan Horsey on Mar 17, 2021 12:44:02 PM

Those in the clinical trial industry understand that there are several barometers that can be used to gauge the overall state of the industry. Chief among these are the “big conferences” that occur each year, such as  SCOPE, J.P. Morgan week, and D.I.A.  

With another SCOPE behind us, we thought we’d take the time to summarize what we felt were some of the important industry trends and topics discussed in detail this year. 

 

An Increased Focus on Sensors in Trials

With the outside world still continuing to keep the majority of patients at home, a large focus at this year’s scope was on the promise and potential of sensors and wearables in the market. 

New ideas about the best ways to capture and use clinical trial data were bolstered by arguments showing the positive impact to drug development that comes from combining trial data with supplementary patient data. While the overall sentiment was positive and hopeful around the growing use of sensors, many cautioned that there would need to be a large concerted effort to centralize their deployment and standards of use with review boards and regulators worldwide. 

Presentation(s) of Note: 

Sensors and Wearables in a Decentralized World - Play back available now.
Ali Holland,Exec GM, Digital and Decentralized Solutions

Meeting all Endpoint Needs with a Single Platform
Geoff Gill, President, Shimmer Americas, Shimmer Research

 

The Growing World of Decentralized and Remote Trials

Continuing the sentiments of 2020, decentralization and remote trials continued to influence the conference at large. Many industry organizations touted their plans and capabilities when it came to administering trials at home, in-office, or anywhere in between. 

Furthermore, more companies were speaking to the general needs and circumstances that have led us towards decentralization. This includes not only COVID-19, but older research indicating how many patients, sites, and even sponsors are burdened and constrained by in-clinic only trials. 

Presentation(s) of Note: 

Scaling Decentralized Trials: Best Practices and Lessons Learned to Help Strengthen Patient-Centricity & Trial Delivery
Nagaraja (Sri) Srivatsan, Senior Vice President and Chief Digital Officer, Technology Solutions, IQVIA
Christopher Varner, Associate Director, Virtual Trials, IQVIA

 

The Effect of COVID-19 on Our Industry

For those who didn’t see this one coming, it only took a brief glance at this year’s agenda to know what would be a major talking point at this year’s SCOPE.

COVID-19 drove discussions pertaining to topics relevant across our entire industry. This included COVID as a driver for innovation, but also flexibility, speed, new ways of running trials, changing regulations, and the lasting legacy of clinical trials. 

Presentations of note:

Keynote Presentation: COVID-19: How an External Event Redoubled Janssen’s Culture of Innovation
Darren Weston, Vice President, Integrated Data Analytics & Reporting (IDAR), Janssen

 

Overall

As one could expect, several of the main themes of this year’s SCOPE were a direct result of our world in 2020. What SCOPE cemented, however, was that the “new norm” of doing things, one brought upon by COVID, is definitely here to stay.

5 min read

Wrestling with ‘Fabulous’ - When Cancer Goes Against Your Image

By Allison Kalloo, MPH on Mar 12, 2021 12:06:29 PM

Gloria was my surrogate mom and a role model. For a time.

                                   

Upon even casual consideration, it was so clear that her name was the perfect moniker for such a force of nature. But then she amplified it. Gloria claimed a nickname for herself that would live in infamy. If you knew her, it would be neither surprising nor a bridge too far. “The Fabulous One” and mom were best friends. My mother was fun in a dignified, more conservative way, while Gloria was flamboyantly “living out loud” and boldly unapologetic about taking up space in the world. Long before any of us had access to easy-peasy genetic testing, Gloria had claimed her African ancestry proudly and loudly, and just in case anyone was confused by her light-bright complexion. The fact that she performed in community theatre was such a natural extension of her personality that it was redundant. Gloria was a character. She never, ever left the house without looking like she had a date with destiny.

 Gloria wasn’t the life of the party, she was the party. Her go-to style was festive but regal. She was consistently decked out in generously flowing kaftans made of bright colored fabrics and usually in African or Indian motifs. She didn’t just walk, she pranced and glided and promenade. Whatever she happened to have on would gather around her, flappling and flowing in joyous movement while trying to keep up with her. Accessories were all but unnecessary, but she was never seen in public or in her own home without wearing a pair of earrings likely acquired from a festival which some local artisan had handcrafted. She believed in supporting fellow artists. In all the years I knew her, Gloria also wore prominent headwraps at least fifty percent of the time. When her head wasn’t covered, she rocked a short ‘do’ that was dramatic in itself at a time when most women were trying to emphasize length. Her style consistently included statement bangle bracelets that made noise. Her hands, that were in constant motion if not in a dramatic pose, were always adorned with at least one ring each.

 Gloria Davis Hill had a distinctive way of calling the house and asking to speak to mother. And it always brought a smile and a snicker. And her cadence was “extra” also, making certain that Dad or I would be. The fact that we recognized her voice was beside the point. “This is ‘The Fabulous One. Is your mother available?” I fondly remember Dad and I laughing at all the drama, The “extra.” But we all had come to relish her effervescent eccentricity. To quote one of my favorite song lyrics, she brought the sun out to light our lives up. I can dedicate that sentiment to both my mother and her best friend. And ironically, both of them were Black women who taken too soon in the prime of their lives. My mother’s health problem was chronic and spanned decades. Gloria’s disease was apparently known only to her and hit the rest of us like a bolt of lightning.

But, when the long distance phone call came from her daughter, I thought she was using gallows humor and making a morbid joke. I teased back and basically told her it was . Understand the fuller context of who Gloria was, but understand that I was quite literally waiting for her to arrive in New Haven by train. That day. I was to be picking her up from Union Station so that she could lead a series of cultural storytelling the following day during my portion of the International Festival of Arts & Ideas for which I had been chosen as an artist-in-residence. Gloria was slated to be a featured artist in my installation celebrating New Haven’s Sister Cities program. But Kimberly was not calling to say that her mother was running late. She was calling to say that “The Fabulous One” was dead.

 Wait.... WHAT!?!

 No, there hadn’t been an accident. It was not foul play. She asked me if I’d known that her mother had breast cancer. Because she hadn’t even told her daughters. Did I know?

 Wait...WHAT?!?

 There was an investigation opened as a result of someone (I forget who) finding her lifeless body. Medical officials quickly theorized based on visual examination that she died of breast cancer that had likely metastasized. It appeared that she had not sought treatment. It also appeared to have been the more aggressive form of invasive breast cancer. She had rebuked all forms of standard medicine, anyway. As the information evolved, it was clear that Gloria had not accepted what would be considered standard insurgery, radiation or traditional chemotherapy that would seem almost obligatory for anyone who loved life as much as she did, and who had as many loved ones in her tribe as Gloria did. It will always be impossible to reconcile her decision to suffer in silence. The pain she must have suffered as the masses in her breasts grew is absolutely unfathomable. The fact that she did it her way is an absolutely classic Gloria move.

 I was accustomed to Gloria using essential oils, burning sage and using natural aminos in her food. None of those things would have been red flags. A couple of months earlier— which would be the last time I was fortunate to in her presence— we had visited with her in DC and I went in for a heartfelt good-bye hug, I kind of noticed in that moment that one of her arms came across her chest but she hugged me back with the other. At the time, I assumed she was just adjusting the extra fabric of her kaftan. In hindsight, I think she instinctively blocked her chest to buffer the heightened pain she would have endured. In hindsight, that moment was both a “tell” and a profound metaphor. She loved me fiercely, but at the same time, was not about to let me in on this horrific secret. She loved all of us, but she could not bring herself to be seen as vulnerable or sick or dying.

 There is something to be said about doing things your own way. She would not have embraced any pity parties or fawning over her out of sympathy. Ironic, but I reckon that she was impatient with the appointments and side effects that would have been disruptive to not only her lifestyle and schedule, but her very persona itself. Having cancer did not fit in with her sense of self, and living out loud— And whether she was in the deepest denial or her own version of full acceptance will remain a mystery.

 The fact that Gloria did not receive treatment creates more questions. Was she under care but opting out of treatment? Did she lack insurance coverage and was her unwillingness really indicative of her lack of access? Rather than refusal, was it really because she lacked medical coverage? Despite her customary bravado, was she in fact intimidated by white coats and navigating the medical system? Was she cynical about medicine because she had lost her own mother as a child? Was she fearful because of historical abuses that went by the names of Tuskegee and Henrietta Lacks? Was she embarrassed because she felt she could have preemted a late diagnosis had she acted sooner? Was it all about not appearing weak or refusing to play “victim?” So many questions.

 Also this: Was the possibility of enrolling in a clinical trial ever brought up? I’m thinking not. All of the poignancy surrounding this tragedy makes me resolute. You will hear from me. Believe that. I applaud Gloria for maintaining her own agency and respect her for making her decisions without interference. But there comes a time when suffering in silence begs purpose. It will never be my way.

 I am eternally grateful for the beautiful friendship “The Fabulous One” granted my mother. I revere her for the love she showered on me. And the example she set for claiming your space and speaking up. I have also decided that martyrdom is not a good look or a desirable legacy. Denying loved ones the opportunity to know you more fully and in a truthful, balanced way. To rally to your aid. To feverishly help you explore treatment options. To identify patient support programs, to be supportive, and to navigate the unknown waters of clinical trials. To deny loved ones the ability to spend precious time with you and — to hold your hand and express our love for you— is not fabulous. Not in the least. I have learned from that, too.

 

Topics: clinical trials Oncology Cancer
3 min read

Device Buffets: How to select the right device for your clinical trial from the seemingly endless options

By Jared Klingeisen on Mar 10, 2021 12:19:09 PM

I love a good buffet as much as the next person, but the current COVID-19 environment is not the only time a buffet might be problematic; it can also prove challenging when selecting a device for your clinical trial. Buffets work well when abundance is the goal, but when you’re focused on a specific objective, the seemingly limitless options may become distracting, overindulgent, and ultimately leave your needs unmet. At Medable, we’ve seen our fair share of device buffets, and we want to help you avoid choosing something that’s been sitting under the heat lamp for too long. 

COVID-19 has accelerated the need for decentralized clinical trials and adoption of wearable devices for remote patient monitoring. Connected device data is helping transform healthcare across the world, and the benefits of this approach are well documented. If you’ve already decided that wearable devices are a great fit for your trial, but you’re not sure how to pick the right device out of a selection of hundreds of options, the information below may help. We actively canvas and assess the connected device market, and wanted to share the top factors our team reviews to determine our device catalogue.

Top 5 Factors to Consider when Selecting Connected Devices

Security: How is data captured, securely transmitted, and stored?

Accurate and secure data capture is of paramount importance. Finding a reliable device that can not only accurately capture the data, but securely and timely transfer that data to a compliant database is a must. If the device has the proper integration capabilities, it allows the device to seamlessly pass that data directly into a 21 CFR Part 11 or GDPR compliant database, like Medable’s, so it is never exposed to a security risk.

Some devices require their data be transmitted to their own database or application, which in turn may require a sponsor to qualify the connected device vendors' app and database as well. This can be costly and take a lot of time, and more importantly, some of these device vendors don’t have databases or applications that meet the strict storage and security requirements that this industry requires.

Usability: Is the device easy for a patient to set up and use? 

Selecting a device that’s intuitive for a patient to use is critical. If it’s difficult for a patient to use the device or upload the data from it, they likely won’t. Clear, step-by-step instructions should be included for patients to reference when they take measurements or for them to consult throughout the study. Even seemingly small things, like devices that require a cord to connect to a phone or to download data, can be a barrier for patients and result in incomplete datasets. 

The best solution is to integrate the device right into the ePRO solution they are already using on a daily/weekly basis for their trial, so the data automatically uploads during use. We’ve seen much higher compliance with this approach, and as a result it’s our standard set up. 

Dashboards: How is the data captured displayed and reported to patients or the study team? 

Many connected devices don’t actually show patients the data they capture. If you are collecting steps or blood pressure on a daily basis, and it's possible to share the data, patients prefer to see their data. Presenting this data (where applicable) can lead to higher engagement and adherence, adding an element of gamification and stickiness to your study for the participants.

Presenting daily data back to your patients can lead to higher engagement and adherence, adding an element of gamification and stickiness to your study.

Reports and dashboards for patients and study teams help with monitoring study progress. A valuable approach is to set alert ranges on the data that is collected, which can trigger notifications to the study team to proactively identify a patient that might need attention. Examples include setting triggers for blood pressure, heart rate, temperature, or weight changes. 

Regulatory: Is the device approved and cleared for use in the countries in your trial? 

Make sure the device you select has the proper approvals and certifications (examples: 510k cleared, medical grade, CE mark, etc) for how you intend to use it. Additionally, you will want to inquire whether there are country or region specific versions of the devices (some manufacturers standardize globally, while some create a device specifically catered to a region). Sometimes simple things, like providing the right charger/adapter for all countries that will use the device, are critical to successful use of the device in your trial.  

Support: Will support for the device exist throughout the duration of your trial?

Technology and devices iterate and change quickly, and during a long study, the manufacturer may not support a device for the duration of your study. In those cases, technical support for that particular device ceases to exist in the market.

Remote Patient Monitoring at Medable

At Medable, we continue to proactively research the market to identify reliable, easy to use devices to add to our library of tested and approved devices. If you’re not sure which device to use, we’d be happy to provide our insight and help you with your “buffet” selections. You can send us an email at sales@medable.com 

Topics: decentralized trials Hybrid Trials life sciences Innovation clinical trials
3 min read

Recommendations for Optimizing Televisits

By Mary Costello on Mar 9, 2021 11:09:40 AM

Like many of you, the past months for me have been learning about how to transfer what was my everyday normal life into online mediums.  Some have worked well, while others have fallen short. For instance, I liked virtual workout classes better than I would have expected. However,  a zoom or facetime call with my 86 year old father ends up feeling more sad than satisfying.  However, only this week did I finally experience my first health televisit, with my physician who wanted to discuss a medication change.  

Given that I have spent the last ten months working with sites interested and eager to introduce digital options into their clinical research realm, I felt this was an opportunity for me to observe how the process works in healthcare.

First of all – what worked?  

I was given instructions verbally over the phone by the nurse about three weeks prior on what to do, none of which I wrote down. But, I did remember the basic expectations! “Check that my registration with the on-line portal was up to date, download the app for the televisit and be ready ten to fifteen minutes prior to the appointment.”  

I was prompted the week before to confirm my visit via text. I did receive an email with a link to download the app, and I remembered my apple password without having to retrieve it, and my doctor called me exactly at the time of my appointment.  It was actually great! I know her, seeing her and having her undivided attention for twenty minutes with no waiting ahead of time or hassles of parking etc. went well!  If I had rated my satisfaction with the visit itself, I would have given it a ten, as I did not feel as though the level of care or attention was any way less than an in person visit would have been and the hassle factor was significantly lower.

What didn’t go as well? And, what were my takeaways when incorporating into research?  

I am not sure that the scheduling system / text reminder system was synchronized with the televisit.  I got a prompt on Sunday night reminding me about safe visit procedures on masking and ensuring I was COVID symptom free before coming to the office.  That was the first red flag.  Since I had also gotten the email asking me to download the app, I figured it was just what it turned out to be – two separate systems for communication, operating independently.  

On Monday morning, I saw a missed call from the doctor’s office.  When I returned it, the message said that due to inclement weather, the office was closed that day.  Austin had had its first snowstorm in about ten + years the night before.  There was no way to leave a message and if I chose to call their on call service for a non-emergency, the standard message warned me that I could incur a charge.  At this point, I am wondering whether my physician, since her office was closed, would expect me to reschedule and would take the day off.  

How did it go? 

She called right at the appointed time which had always stayed as a possibility for me.  The other communication ended up being distracting rather than downright annoying as we are still under Stage Five restrictions here. 

However, it did underscore for me that sites need to think about how they communicate and how they create automated reminders and confirmations.  Before implementing tech for a televisit, walk through how all your patient communications will go.  As it was my first televisit, it would have been quite helpful for the office to have a standard. In fact, it could look a bit like this. 

“How to prepare for your first televisit. Here is the link, here are the steps, here is a screenshot, etc.:

In essence, over communicate! 

If you can’t modify your reminder systems, then point out that a patient may receive a reminder making it seem as though they have an in person appointment, and advise them to ignore that message.

As we migrate to the new world order, we must remember that flexibility is here to stay and our goal should be to enhance that experience at every level! 

Topics: telemedicine TeleVisit
6 min read

A Tale of Two Caregivers: An Interview on Experiences, Sacrifice, Wellness, Clinical Trials, Pharma Support and More

By Jennifer McNary on Mar 5, 2021 10:41:36 AM

 

On Thursday, February 13, 2021, PAC members Jennifer McNary and Paul Kidwell sat down with Medable's Content Marketing Manager to discuss their lives and experiences as caregivers. 

As anyone who's dealt with a serious illness among their loved ones knows, the emotions that are experienced can be intense, intimate, and difficult to process. Thus, Medable would like to thank both Jenn and Paul for sharing some early and intimate details about the early hours of their caregiver experience in the preamble below, as well as their thoughts throughout the interview. 

You can find their stories in written form and video below.

Preamble

 

Words to Live by – from Jennifer McNary

When I learned at 21 years old that I was parenting two children with a terminal illness, I fought hard against the tragedy of the diagnosis. I struggled to maintain my own identity and not simply the mother of dying children.


Identities are fluid throughout our lives. We are children of our parents, brothers, sisters, wives, employees. Thus, no one role defines us and I do not want the role of caregiver to define me either. I have definitely at times been simply the “mom” in fact I still have to push back when teachers and doctors don’t bother to learn my name at all - and simply refer to me as “Mom”.

Self-preservation means that I also spend what I would consider a great deal of time on myself.

Whether its riding the Peloton, practicing yoga, reading or even binging on Netflix, I take great pride in caring for me, daily. I fly a lot- so in the words of every great flight attendant, put your own mask on first and then secure the masks of those who need assistance.

Words to live by

 

Difficult Conversations: A Parent Tells a Young Child About A Mother’s Serious Illness - by Paul Kidwell

All who have been diagnosed with a serious illness will tell you that the diagnosis, treatment, and patient journey is universally felt throughout an entire family and not just with the patient.

Part of this experience is the need to distill very complex medical information with each family member, no matter their level of understanding; a role often relegated to the care partner; or in my case the husband and father.

The idea of telling our son – who was 13 at the time - that his Mom had a progressive neurological condition stunk. We knew that he should know and it was up to us – actually up to me – to ensure that what and when I told him was understandable for such a young person. No time is perfect for this type of conversation so about a year after my wife’s diagnosis she and I decided that the timing was right. In particular, with a progressive disease there are too many medical appointments, medicines and necessary family conversations that begin to crop up that you just cannot hide or explain away. Even at a tender age, knowledge is KING and the lack thereof can cause uncertainty and fear. The content and the direction of these conversations is unique to each circumstance, but I have identified a few “rules of the road” to consider.

Think Before You Speak.
Plan this conversation in advance. Timing, location and content are all important. Maybe even talk to a mental health professional before.

Keep it Simple. The Simpler the Better
Tell them about the illness, its affect on the individual and what we are doing to make sure Mom gets better. Tell them to come to you with questions not Dr. Google.

Quality Time
Even though a patient at home takes precedent, a parent still needs to show love and affection towards the children. I have found that scheduled “kid-time” is important. Also, involve them in the care when possible.

All Feelings are Right
This time in a child’s life represents an emotional juggernaut and they will exhibit a myriad of emotions; including, anger, sadness, jealousy, worry, guilt. The usual suspects. All are real and nothing is out of bounds. If you cannot help them navigate these, seek the help of an outside mental health professional who specializes in child issues.

The Interview

 

Question 1: What Has Been Your Experience as a Caregiver?

 

Question 2: What is a zone of wellness, how do you establish one?

 

Question 3: What sacrifices have each of you had to make?

 

Question 4: What has been your experience with Pharma? What level of support is there for caregivers?

 

Question 5: What is your final advice for anyone in Pharma starting trials today?

 

3 min read

Recap: Industry Experts Discuss the Current and Future State of DCT - Insights from HCL Technologies' "Straight Talk" Session

By Dan Horsey on Mar 3, 2021 10:42:00 AM

On Thursday, February 24th, eight healthcare professionals from a coalition of clinical trial companies gathered on Twitter. Together, they were led through a series of questions about the impact, future, and value of decentralized clinical trials (DCTs) by HCL Technologies. 

Medable’s own Tyler Pugsley was one of eight professionals, answering questions from HCL and the live audience together in a new Twitter series known as “Straight Talk.” The blog below serves as a recap of key insights from their discussion.

Question 1 - How big a role will virtual/decentralized clinical trials play in future pharma R&D?

The group stated that virtual trials are here to stay now, having made a foothold in the industry prior to 2020, and becoming mainstream as a direct result of COVID. Thus, they will continue to play a vital role in the future of trials. 

When it came to discussing the role they will continue to play, many stated that DCT has a directly positive impact on trial access, as they expand the patient population pools out of specific geographic regions, and into the national level. Others discussed the benefits of passive data, and it’s combination with TeleHealth. Used appropriately, each has the ability to provide a clearer picture of a patient, their health, and how they’re progressing through a trial.

Finally, some highlighted the added benefits of speed, and reduced costs that come with the embrace and proliferation of services like TeleHealth, direct to patient labs, and more.

Question 2 - In what ways is the technology to support such clinical trials (DCTs) evolving?

The panel noted that technology is often a tool for removing barriers and improving communication with current solutions making trials more patient-centric, and opening access to anytime, anywhere clinical trial participation.

For instance, wearables have evolved to become smaller, less cumbersome, and better at passive data collection during the last few years. Other technologies have focused on closing the gaps between clinicians and patients, with real-time data helping clinicians understand how to facilitate ongoing patient engagement and manage health conditions.

Medable’s Tyler Puglsey, VP of Business Development and Growth laid out his vision for the future, as he believes the way we perceive patients will change over time to see them more as consumers of trials, and less participants.

Question 3 - How has COVID-19 changed the industry’s approach to adopting virtual/decentralized clinical trial platforms, instead of more traditional electronic data collection platforms (EDC)?

Here, many were quick to point out that COVID-19 seemed to remove many of the barriers towards DCT adoption almost overnight, noting that the pandemic highlighted what the industry could have looked like for some time.

Additionally, participants responded with the real world concerns brought on by the pandemic, noting that patients were unwilling to go onsite in many instances. The result? Televists, remote monitoring and more have become the “new normal” for many studies, as they provide an effective and safe way to interact with patients.

Question 4 - How can an organization transition to decentralized clinical trials at enterprise scale across its entire clinical research portfolio.

With this question, one panelist noted that the true barrier that must be overcome for enterprise scaling is culture. Focusing on technology alone, others suggested organizations begin by leaning into key components of DCT, such as online eConsent and eCOA that would allow for greater digital data collection.

Finally, some noted that these processes must be directly integrated into both the organization’s infrastructure and key processes in order to see the true benefits of data, workflow, and speed.

Question 5 - What long-term effect will such clinical trials (DCTs) have on drug development?

Many panelists noted that patient centered designs met with technology that increases convenience and mobility are here to stay. While many saw long-term gains in improving patient recruitment, engagement, and trial data. 

Others hoped to see the downfall of trial failure due to patient recruitment and dropout issues. 

With perhaps the clearest take on the industry’s future, our own Tyler Pugsley cut straight to the core of the issue, stating that “Let's break it down to the core objective: Speed to safe and effective drug approvals.”



5 min read

February Pulse Check on Decentralized Clinical Trials: Entry Points to DCT’s

By David Swanger on Mar 2, 2021 11:43:35 AM

Throughout 2021 we’ll take a look at the latest developments in the realm of Decentralized Clinical Trials. Read this monthly roundup to discover the latest trends, insights, and modalities of decentralized clinical trials.

This past February, our blog focused on the various entry points involved in moving trials to a fully Decentralized Clinical Trial (DCT). Although our industry is gaining familiarity with this new approach to clinical development, several questions remain. One of the most important we find our customers asking is “Where do I start?”. 

In February we dug into a couple of key entry points. The first has to do with electronic clinical outcome assessments (eCOA’s). The second has to do with enabling the voice of the patient.

Before we look at our recap, let’s first define Decentralized Clinical Trials (DCT’s) for those who have found this blog as their entry point in education. DCTs differ from traditional clinical trials by bringing the trial to the patient through a combination of technologies (eConsent, eCOA, TeleVisits, Sensors & Wearables), mobile/local healthcare providers, and enabling the voice of the patient. Thus, there are a variety of modalities that can be used as an entry point in decentralizing a trial, whether through consent, outcomes reporting, or site visits. Regardless of the point of entry, the solution must be tailored in a flexible and modular manner to fit the unique needs of an indication, therapy, or patient population.

This past month we published two blogs on how to successfully operationalize eCOA’s.
 

Our own Dr. Ingrid Oakley-Girvan began the month by delving into:

 The Language of COA’s

In the blog, Dr. Oakley-Girvan dives into the four categories of Clinical Outcome Assessments spanning Patient Reported Outcomes (PRO), Performance Outcomes (PerfO), Clinician Reported Outcomes (ClinRO), and Observer Reported Outcomes (ObsRO). She also examines the suitability of each outcome assessment for decentralization and how each can be measured remotely through various technologies and processes.

Next, our own Dr. Reem Yunis took a deeper look into the most popular form of eCOAs, Electronic Patient Reported Outcomes (ePRO):

I Want To Tell You in My Own Words: Patient Reported Outcomes

In the blog, Dr. Yunis charts the path our industry has taken from paper based patient reported outcomes to electronic. Important factors she highlights for consideration in the blog include the modality and device type a PRO is presented on as well as the challenges presented in achieving compliance. Both are factors which are heavily influenced by patient engagement and compliance.

In February we also continued to expand how we can give voice to the patients and caregivers that are critical to DCT adoption.
 

Over the past year Medable has been expanding it’s Patient Advisory Council and this February we announced the following:

Medable Expands Patient Advisory Council to Drive Patient-Centered Improvements in Clinical Trials

Our four new PAC members include:

Sumaira Ahmed: a marketing director at Brigham & Women’s Hospital in Boston. Shortly after being diagnosed with sero-negative Neuromyelitis Optica Spectrum Disorder (NMOSD) in 2014, she founded The Sumaira Foundation to raise global awareness and find a cure for NMO. 

Gaurav Dave, MD: an Associate Professor of Medicine, Associate Director of the Center for Health Equity Research, and Director of Abacus Evaluation Consulting at the University of North Carolina at Chapel Hill. 

Allison Kalloo, MPH: a patient recruitment specialist and the founder of Clinical Ambassador, iParticipate and CliniVIVRE aimed at expanding minority access and impacting diversity, equity and inclusion in clinical research. 

Paul Kidwell: a Boston-based public relations and patient advocacy consultant, providing media relations and patient engagement support for biopharmaceutical companies, and a rare disease podcaster. 

Additionally, several of our PAC members made important contributions to expanding the voice of the patient this past February including:
 

Wither the Caregiver. Phama and Healthcare’s New Best Friend 

In this blog Mr. Kidwell highlights some of the unsung heroes of our healthcare system: caregivers. He shares his personal experience of being thrust into the role. Just as “nobody expects to get sick until they do, for caregivers, this sentiment is exactly the same. The role and its inherent demands are never part of a person’s thinking”

Our own Sumaira Ahmed was also featured on a podcast:

Illuminating NMO: The Power of the Patient Voice

During the podcast, Ms Ahmed shares her story around Neuromyelitis optica and how she started a foundation that's mission is to amplify NMO patient voices and raise money for researching a cure.

Finally, February marked Black History Month and with it two of our patient advocates commemorated the life and legacy of Henrietta Lacks:

Getting Her Say: The Immortal Legacy of Henrietta Lacks

Allison Kalloo and Sumaira Ahmed investigated the legacy of Henrietta Lacks through different prisms, one focused on her impact on medical research and the other on how betraying public trust can have far-reaching and longstanding implications that we will continue to confront for some time.

Giving voice to the patient perspective will be critical to the ongoing success of Decentralized Clinical Trials (DCT’s). And we at Medable are honored and humbled by the work members of the Patient Advisory Council do every day to make these voices heard. Doing so is critical to entering DCT’s with the right perspective. 

As February turns to March, we at Medable will continue to look forward to how we can continue to operationalize DCT’s and help patients, sites, CRO’s, and sponsors to realize the full benefits of this powerful new modality of clinical research. In March we’ll turn our attention to overcoming implementation and planning obstacles related to DCT’s. 

Next week on March 8th, Medable is proud to participate in a Reuters webinar that will highlight how to:
 

Overcome Perceived DCT and eCOA Adoption Hurdles

 

Topics: decentralized trials PAC eCOA
1 min read

Why I Joined Medable - Smoother Sailing to the Clinical Future

By Anushka Gupta on Mar 1, 2021 1:18:12 PM

When medicine changes, the world changes: new treatments, vaccines, and therapies create tectonic shifts in global quality of life. But it’s not easy—the space of possible treatments is vast, and the steps we take to a solution can be uncertain, logistically difficult, and costly.

What if we could chart a better course through those stormy seas, and sail it with the efficiency, coordination, and speed brought to so many industries by the internet age? 

As a kid, I always wanted to help people; I thought being a doctor was the best way of achieving that. By my senior year of high school I’d drifted into engineering, but I kept my interest in healthcare throughout my undergrad.

It became personal when I contracted Multi-Drug Resistant Tuberculosis while on a trip for my Formula-1 team. I spent the second half of my undergrad in isolation, battling the infection with drugs so strong their side-effects left me perpetually confused, nauseous, and in pain. Some of them were experimental, with no fixed course. My parents became my caregivers and spent hours of each day sharing my difficulties.

It could’ve been easier, and shorter-lived, if I hadn’t at first been misdiagnosed. Instead, I had plenty of time—days; weeks—to think about how the whole process could be better.

Though my doctors suggested I take a year to rest, I applied for graduate school and moved to the states, where I worked on multiple projects applying machine learning to healthcare data. I was excited to find Medable because they seemed focused on solving many of the problems I had faced: optimizing the trial process for discovering new treatments, making them more accessible for everyone, and reducing the burden on patient caregivers. 

At Medable, I work with a diverse group of motivated people toward a common goal. I get to work on a bunch of projects, benefiting from the expertise of many teams. For a fresh graduate taking on her first job, Medable has been the perfect place to grow while working on things I enjoy. Even better, it’s work that matters and I can feel us breaking new ground.

View our open positions here: https://www.medable.com/company/careers 

Topics: decentralized trials careers Diversity
4 min read

I Want To Tell You In My Own Words: Patient Reported Outcomes

By Dr. Reem Yunis, PhD on Feb 25, 2021 1:17:09 PM

As patient's health and quality of life became an increasingly important component of treatment response, clinical outcome assessments, or COAs, became an important part of evaluating the safety and efficacy of medical products. In January we covered COAs and their use in general. This post will focus on patient reported outcomes (PROs).

What patients report directly about their health condition and disease symptoms (i.e., without the interpretation of a clinician) is very important and could influence the course and outcome of their treatment. In clinical trials, PROs can be utilized to determine the safety and efficacy of medical products. Because they are subjective in nature and are influenced by the patient’s perception of their own disease, PRO measures must be well defined and collected in a consistent and robust manner. Instruments intended to capture health outcomes directly from patients can be developed and validated with appropriate sample sizes and rigorous study design. Once tested in the population of interest, or multiple populations in some cases, and adequately validated according to scientific rigor, they can then be used alone or in combination with biomarkers in research studies or clinical trials.  PROs combined with biospecimens or digital biomarkers from connected devices can provide direct or indirect evidence not only for medical safety but also for efficacy in clinical trials in support of labeling claims.

The Road From Paper to Electronic Mode

The digital revolution wave extended how healthcare is captured and delivered. Clinical trial processes were no exception, and many remember the transition to EDC platforms to capture patient data in clinical trials. Naturally, paper-based health assessments rode the tide and electronic COA (eCOA) have become a mainstay in clinical trial operations.

The FDA encourages the use of electronic PROs (ePROs) whenever possible for the simple fact that paper PROs lack the timestamp and thus an inability to confirm when the data are entered by the patient. As we enable more remote clinical trial engagement, ePROs take on even greater significance.  However, there is much more to the migration of a PRO to an ePRO than simply making a digital copy. The transition must ensure that the patient responses are equivalent between paper and electronic formats. This is not a straight line as the equivalency may need to be tested in multiple populations and the extent of changes impact the work needed to switch to the electronic road.

According to the ISPOR task force guidance, when a paper PRO is delivered in a new modality such as in a mobile app and only minor modifications are needed (things like spacing, font, etc.) small groups of 5-10 patients may undergo cognitive debriefing and usability testing interviews. However, when more significant modifications are made to PROs (when digitizing them), the process of migration necessitates robust clinical study designs and statistical analyses. This guidance was published by the ISPOR task force in response to the release of the FDA guidance “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims” and is likely key to follow if pharma is to adopt these electronic measures in clinical trials knowing the FDA will require a certain level of rigor. The ISPOR task force indicates when moderate changes are made to a PRO, equivalency testing is required which subsequently turns into a full-fledged psychometric testing protocol when substantial changes are implemented. In a nutshell, equivalency is measured by comparing the psychometric properties of the data collected from the device to that from the paper, and under the psychometric testing. It is assumed that the substantial changes may render the ePRO as a new measure and thus should be subjected to the same original process of development and validation before this new ePRO can be utilized in clinical trials.

In all modification levels usability testing is required, which is completed in the target patient population where it examines the ability of the participants to use the device and software and complete the digital assessment as intended.

Factors to consider when optimizing and implementing ePROs are covered in a paper by Fleming and colleagues in the journal Therapeutic Innovation & Regulatory Science (2015). Among them is the modality and device type on which the ePRO is presented, that includes audio, computer, tablet, smartphone, touchscreen, keyboard and mouse. Although a meta-analysis of over 40 ePRO studies found that device type did not impact equivalency, it is still important to keep in mind user experience as a factor in compliance, accuracy and longitudinal data capture.

While site-based ePRO compliance is ensured by the clinical research team, capturing remote ePRO compliance is more challenging as it depends on the patient's engagement and adherence to the ePRO study protocol reporting schedule and requires continuous monitoring by the site. This adds a burden to an already overwhelmed research and clinical staff.

Alternative mechanisms to monitor patient engagement are needed. One possible solution is real time analysis of data and utilization of notification applications embedded in the ePRO delivery platform to help nudge and encourage action by patients.

When designed and developed appropriately with thoughtful implementation for engagement, patient-centered and clinically meaningful ePROs can make a significant difference in the approval of a new medical product and treatment.

References and Resources

Coons SJ, Gwaltney CJ, Hays RD, et al. Recommendations on Evidence Needed to Support Measurement Equivalence between Electronic and Paper-Based Patient-Reported Outcome (PRO) Measures: ISPOR ePRO Good Research Practices Task Force Report. 2009. Value in Health 12(4):419-429

Doward LC and McKenna SP. Defining Patient Reported Outcomes. 2004. Value in Health 7(1):S4-S8

Fleming S, Barsdorf AI, Howry C, et al. Optimizing electronic capture of clinical outcome assessment data in clinical trials: the case of patient-reported endpoints. 2015. Therapeutic Innovation & Regulatory Science 49:797–804

Food and Drug Administration. Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2009.

Powers JH, Patrick DL, Walton MK, et al. Clinician-Reported Outcome Assessments of Treatment Benefit: Report of the ISPOR Clinical Outcome Assessment Emerging Good Practices Task Force. 2017. Value in Health 20(1):2-14

Walton MK, Powers JH, Hobart J, et al. Clinical Outcome Assessments: Conceptual Foundation—Report of the ISPOR Clinical Outcomes Assessment – Emerging Good Practices for Outcomes Research Task Force. 2015. Value in Health 18(6):741-752

Weldring T and Smith SMS. Patient-Reported Outcomes (pROs) and patient-Reported Outcome Measures (pROMs). 2013. Health Services Insights 6:61–68

Topics: decentralized trials medable DCT eCOA ePRO
2 min read

Illuminating NMO: The Power of the Patient Voice

By Sumaira Ahmed on Feb 24, 2021 10:08:37 AM

In a new podcast, Sumaira Ahmed, Founder of the Sumaira Foundation shares her story around Neuromyelitis optica and how she started a foundation that's mission is to amplify NMO patient voices and raise money for researching a cure.

 


The Sumaira FoundationThe Sumaira Foundation for NMO is a 501(c)(3) organization dedicated to generating global awareness of neuromyelitis optica spectrum disorder (NMOSD), fundraising to help find a cure, and creating a community of support for patients + their caregivers.
 

"Neuromyelitis optica (NMO) is a central nervous system disorder that primarily affects the eye nerves (optic neuritis) and the spinal cord (myelitis). NMO is also known as neuromyelitis optica spectrum disorder or Devic's disease. It occurs when your body's immune system reacts against its own cells in the central nervous system, mainly in the optic nerves and spinal cord, but sometimes in the brain.

 

The cause of neuromyelitis optica is usually unknown, although it sometimes appears after an infection, or it can be associated with another autoimmune condition. Neuromyelitis optica is often misdiagnosed as multiple sclerosis (MS) or perceived as a type of MS, but NMO is a distinct condition.

Neuromyelitis optica can cause blindness in one or both eyes, weakness or paralysis in the legs or arms, painful spasms, loss of sensation, uncontrollable vomiting and hiccups, and bladder or bowel dysfunction from spinal cord damage. Children can have confusion, seizures or coma with NMO. Neuromyelitis optica flare-ups might be reversible, but they can be severe enough to cause permanent visual loss and problems with walking."

 
9 min read

Getting Her Say: The Immortal Legacy of Henrietta Lacks

By Allison Kalloo, MPH on Feb 18, 2021 9:32:45 AM

What follows is a collaborative blog that commemorates the life and legacy of Henrietta Lacks written by two women of color— both members of the Medable Patient Advocacy Council (PAC). We offer our observations here in two parts.

It seems unfair that so many are unaware of her - a woman responsible for saving countless lives and counting. However, in celebration of Black History Month, I feel privileged to educate our readers about the unforgettable story of a young black woman whose remains transcend mortality and quantifiable impact.

 She was only 31 years old when she passed away, after failing multiple treatments for cervical cancer. She left behind five devastated children, her husband, and parents. Her name was Henrietta Lacks.

 Unbeknownst to her, a sample of Henrietta’s cancer cells had been biopsied by Dr. George Gey, the Head of Tissue Culture Research at Johns Hopkins Medicine. The hospital lab frequently obtained specimens from cervical cancer patients but found that average human cells had a brief shelf-life which resulted in virtually ineffective samples, the vast majority of which only survived a few days outside their human host. In an ironic twist of fate, given her tragic end, the cells biopsied from Henrietta Lacks were found to be freakishly durable. Gey was able to consistently propagate these cells in tissue culture. Paying homage to Henrietta Lacks’ monumental contribution to science, Gey did the decent thing and named the cell line for her.

 As it turned out, HeLa cells were unlike any other, both replicating and mutating under direction at a rate that provided ample cell supply for future, groundbreaking discoveries in a broad array of research conducted by scientists all over the world. In fact, growing HeLa cells quickly became a very profitable business that struggled to keep up with demand.

The beauty of it all? By utilizing the HeLa cell line, scientists have made significant advancements in medicine without having to resort to tying their discoveries in live human beings as their first step. Scientists could now pursue all kinds of novel treatments by testing in HeLa cells first.

 The uses for HeLa cells quickly expanded beyond cancer research. Most notably, the cell line played a significant role in developing polio vaccine and subsequently eradicating the disease. Additionally, Henrietta’s special cells have had an impact on a long list of scientific breakthroughs across a wide spectrum of therapeutic areas and applications including but not limited to virology, genetics, and even in ‘space microbiology.’

 The infinite contribution of Henrietta Lacks to science inspires me as a woman, a person of color, a patient, and an advocate. While it’s upsetting to think that she’ll never know the magnitude of her contributions, I feel privileged to share that out of Henrietta’s tragic story has come so much good. Her posthumous impact on the entire human species is one of the most fascinating stories I have ever heard and will forever be etched in my mind and heart.

 — Sumaira Ahmed, a member of the Medable PAC

 

As Sumaira outlined, the unusually robust HeLa cell line utterly revolutionized medical research. As a matter of fact, famed scientist Jonas Salk used the HeLa cell line to develop the polio vaccine, which sparked mass interest in these cells that lead to massive and commercial efforts to clone Henrietta’s cells starting in 1955. Since that time, registered patents involving HeLa cells have exceeded 10,000 (ten thousand). Researchers have consistently benefitted by using Henrietta Lacks’ cells to study disease en masse, and millions of living people (three generations to date) have benefitted significantly, mostly unbeknownst to them. It’s accurate to say that literally millions of human lives have been impacted by the knowledge derived from testing the safety and sensitivity of new products and substances on HeLa cells.

But the story of Henrietta Lacks is an object lesson in how betraying public trust impacts the entire scientific community. Adding more insult to more injury, Henrietta Lacks’ family were entirely clueless about her scientific legacy and the existence of HeLa cells until 1973. At that time, her living relatives were contacted not to finally extend an apology, an explanation, or compensation for what Henrietta had provided the world, but to further exploit her relatives in pursuit of extending their library of genetic samples. The family represented the potential to gather more medical insights from the people who carried the same unique genes.

Exposure snowballed when the BBC screened an award-winning documentary on Lacks and HeLa, which prompted Rebecca Skloot to author her very successful book titled The Immortal Life of Henrietta Lacks. The rest has become history, albeit a conflicting blend of medical miracle, systemic racism and patient abuse.

You see, the story of Henrietta Lacks is also a profile highlighting the lasting harm of medical mistrust based on historical abuses, unresolved accountability, and modern-day urgency to address disparities in the very patient community from which she came. All of these issues swirl around, but for people who look like Henrietta and my mother, the conflicting issues persist.

At the heart of this story lies the issue of trust— or lack thereof. As a community, we experienced the Tuskegee Syphilis Experiment through the lens of our grandparents who depicted the medical establishment as stone cold, self-serving and paternalistic. We then witnessed our parents’ palpable and righteous outrage at Henrietta’s demise. And for millennials who have now witnessed a barrage of injustices in real-time— sometimes up close— pursuing medical solutions to persistent health disparities against the backdrop of police brutality and other overt forms of toxic racism keeps eager participation at bay. Not surprisingly, simmering anger is the more likely response, and it is sometimes not even beneath the surface. Thus, is it any wonder that concerns about exploitation. on repeat,have become multigenerational in this environment.

 The exploitation of Henrietta Lacks took place in a void of medical ethics that has since been addressed in an official and regulatory capacity. But the reality is twofold: This story is sadly emblematic of a bigger history of immorality fueled by ongoing systemic racism, but is alive in our collective memory of how that feels and these issues cannot easily be teased apart. And yes, it is ultimately ironic that our distrust can itself be a barrier to better outcomes and is outright harmful to the very people most likely to benefit from medical progress. You will observe that “betwixt and between” results in a schizophrenic relationship with health and healthcare, yet we sit here at this intersection.

 Given the dark legacy of medical research conducted by virtue of sanctioned racial discrimination, it is entirely unsurprising that African Americans continue to report greater mistrust of physicians and medical researchers than any other groups. A 2006 study found that 44 percent of African Americans reported low trust in health care providers compared to one-third of white patients. When consistent, positive, or consistently positive interactions with physicians are scant, so too is the trust.

Don’t ask why Black people would not move swiftly toward providers made more accessible through Obamacare or toward interventions that would appear to suit our own best interest, or to participate in clinical trials. Instead, ask how minorities— and Black people in particular—would not be leery of medicine and all things related to it.

 It’s an undeniable fact that minority groups have been touched by racism and discrimination in the health care system. However, so is the harm that comes from withholding our trust in the medical sphere. Medical distrust (not “mistrust” because in this case, lack of trust is based on actual observations or experiences) has dangerous, even lethal consequences for those withholding their confidence in the context of health. Numerous studies found an association with lower health care engagement, compromised medication compliance, and (surprise!) less participation in research meant to address disparities and with the intention and potential to enhance health outcomes.

 As contentious relationships within the clinical care environment persist, it cannot help but feed stereotypes of Black patients being "difficult" or disagreeable, which can ultimately result in some providers justifying the suboptimal care still documented by some Black patients. Not surprisingly, this feeds a vicious cycle of unsatisfactory clinical encounters and patients who respond with more distrust and a reluctance to engage in practices that can ostensibly protect against the need for more aggressive medical interventions. And so, it goes.

 Reluctance on the part of African Americans and other minority groups to participate in medical research risks significant harm to the health of our communities. When medical treatments like cardiovascular disease, diabetes or cancer drugs are derived from exclusively white research participants, they've been found to be less effective for minority groups. Or, at the very least, adjustment of dosages was required in order to be more effective in some cases, and less toxic in others.

While the most basic common sense says that equal inclusion in research trials for people of all races and ethnicities is essential to finding the best treatments for all patients, it doesn’t address the issues of trust.

 Racism, paternalism, deception, exploitation and injustice permeated both clinical care and clinical research during Henrietta’s lifetime. It is true that the world is not quite the same as it was 70 years ago, ethically or medically. But, it must also be said that just coming to grips with medicine's past is not enough. We have to acknowledge the many ways those antiquated attitudes have been stoked, reignited, and reinforced under the deplorable guidance of recent administrations. Progress has experienced a setback. Much must be addressed again as if for the first time. However, we cannot let the grip of the past and the psychosis of white supremacist ideology be a barrier to delivering on the promise of medical ethics, health equity and doing what pragmatically serves everyone's health today.

 Henrietta Lacks was singularly responsible for ushering in better screening tools, diagnostics, and treatments across the spectrum and beyond the speculum, if you will. The advent of pap smears and the HPV cervical cancer vaccine are but one area of impact by HeLa cells. Henrietta’s legacy is, at once, invisible and undeniable, both microscopic and telescopic. Her epic cells have reduced pain and suffering and extended life of millions of human beings. The irony that she would never know this always catches me in the throat.

 I don’t have all the answers, but I do have perspective. As someone who is also a Black woman, who is a scientist by training and who was raised by a Black woman scientist, who is now an entrepreneur who has carved out a niche in patient recruitment and inclusion, and who also remains a cautiously enthusiastic participant in clinical trials, I will tell you that the answers must be bathed in a solution of transparency and allowed to germinate in a basic acknowledgement of the larger social construct in which we operate. The answers probably require that you continue to endure more venting about these things on the patient and community side. There will be no avoiding that. The catharsis demands it, actually. Just pledge to be better listeners moving forward, in both structured and casual settings. It should also go without saying that the solutions we seek should include regulatory policy that actually possesses ‘teeth’ and consequences for our industry’s lack of compliance diversity standards that are still lacking in many areas. Enough is enough.

 And, as someone who very easily sees her own mother in the profile of Henrietta Lacks, her story sits a little differently in my spirit. The obligation to address this chasm is definitely personal, so you’ll have to forgive my impatience.

— Allison Kalloo, MPH, a member of the Medable PAC

 

Source:

https://www.hopkinsmedicine.org/henriettalacks/

Topics: D&I Diversity Henrietta Lacks, HeLa Cells
2 min read

Why I Joined Medable: Our Mission Matters

By Christopher Chung on Feb 9, 2021 1:16:29 PM

Helping people and finding ways to directly impact patients has always been an objective of mine, but in addition, to find a great company to share that passion with was another. I heard about Medable from a good friend who helped spark my excitement about the great colleagues that he was working with, and the amazing potential opportunities/ growth path the company was heading towards. I could not resist wanting to be part of the solution and mission.

Since joining Medable a year ago, my experience here has only opened my eyes to see how much growth it has accomplished in so many areas: the number of employees from diverse backgrounds, the flexibility of our technology and innovation of our platform, the number of new sponsor clients, and the services we are able to offer to them. We serve as a one-stop shop with our two largest services being our Decentralized Clinical Trial and Televisit solutions that helped change the clinical trial industry greatly! More so now than ever with the current pandemic of COVID-19, the need for these technical solutions to support existing and new clinical trials to become flexible and remote-based is pressing and motivating.

I can not express enough how happy and blessed I am to have made the decision to join Medable. The company is comprised of really intelligent, collaborative, and innovative members with the same mission of getting “effective therapies to patients by transforming clinical drug development with disruptive technologies” led by our strong senior leadership team. Despite the challenges for most of us working remotely from all over the world, it really introduces a lot of diversity of perspectives and thoughts, which I feel is a strong competitive-edge compared to other previous companies I have worked at. In addition, our human resources team does a wonderful job to create a nurturing, close-knit culture to create the feel of “being in the office,” such as our company power hour challenge where employees posted how they used their “power hour” such as taking walks with scenic views in their local areas, cooking amazing dishes, and picking up new hobbies. 

Medable is only continuing to exponentially grow in hiring more employees who are also interested in contributing to the company mission and be part of the fast-paced startup vibe/culture. We are looking for potential members who are solution-oriented, outside of the box-thinking, team player-based, and willing to put their 110% forward. If you feel these qualities describe you, I strongly encourage you to visit our careers page to join the Medable team. Together as one, we will be able to “radically accelerate cures for diseases” across the world!

Visit our careers page here: https://www.medable.com/company/careers

Topics: decentralized trials medable careers Inclusion
5 min read

January 2021 Pulse Check on Decentralized Clinical Trials: Looking back on 2020 and at What Lies Ahead in 2021

By David Swanger on Feb 5, 2021 11:10:12 AM

Throughout 2021 we’ll take a look at the latest developments in the realm of Decentralized Clinical Trials. Read this monthly roundup to discover the latest trends, insights, and modalities of decentralized clinical trials.

2020 was a pivotal year for those of us who work in clinical development. Covid-19 threw the ultimate curve ball to patients, investigators, CRA’s, and just about everyone else who contributes to ensuring that clinical trials are reliably executed. Suddenly the status quo of asking patients to visit a clinical site for the lion’s share of scheduled assessments was no longer tenable. And as a result our industry turned to Decentralized Clinical Trial (DCT) methodologies for both the continuity and rescue of existing studies as well as the design of new ones.

As we entered 2021 we’ve observed a subtle and important shift in the thinking of many of the stakeholders we interact with on a daily basis. Rather than asking “why” a DCT makes sense, individuals have moved beyond this, now asking “how” to implement one. 

Medable started the month by sharing lessons learned in how we successfully deployed DCT’s in 2020. We also started to look forward to how clinical trials could be reimagined with DCT’s serving as a new foundation. We covered these topics in depth at JPM’s Annual Healthcare conference by  leading five different sessions at the conference. You can view each of the sessions here:

The Digitalization of Clinical Trials

Michelle Longmire Founder and CEO,  Medable,  Maria Fotiu Executive VP of Decentralized Solutions, Syneos Health, Cathy Gao – VP, Sapphire Ventures, Juliet Moritz – COO, Illingworth Research - 

Hindsight is 2020: Lessons learned from DCTs and 2021 predictions

Alison Holland Head of Decentralized Trials, Medable and Craig Lipset Advisor & Founder, Clinical Innovation Partners 

Interview with Rasmus Hogreffe

VP Decentralized Clinical Trial Innovation at Medable 

 Rerouting: How Real-Time Patient Data Will Drive the Future of Healthcare Michelle Longmire with guests: Andy Coravos, CEO, Elektra Labs, Maria Fotiu, Executive VP of Decentralized Solutions, Syneos, Thomas Switzer, Digital Health Scientist, Genentech, Priyanka Agarwal, Director and Head of Digital Health, Myokardi

And finally, you can read VP of Decentralized Clinical Trial Innovation, Rasmus Hogreffe’s overall thoughts and reactions on this year’s JPM’s conference here, on his blog. 

In January of 2021 we also welcomed two important leaders to Team Medable:

Andrea Valente joined Medable as our new Chief Operating Officer to scale the global delivery of Medable Decentralized Clinical Trials. As research and patient care move from the controlled environment of the clinic to the home, focus on operational excellence is paramount. Valente is a proven expert in operationalizing patient technologies in clinical research. She played an early role driving adoption and scale of electronic Clinical Outcome Assessments (eCOAs), one of the first technology categories to interact directly with patients outside of clinical settings. She most recently led development and launch of Oracle’s public health platform to address COVID-19 pandemic response and vaccine trials in the U.S., UK and several developing nations. You can read more about Andrea joining Medable here. 

Parag Vaish joined Medable as our new Chief Product Officer to transform the clinical trial experience for patients and clinicians. As Medable continues to expand its product portfolio, Vaish brings valuable expertise creating products that are intuitive and enjoyable, removing friction from the user experience. During his tenure as head of digital product at Tesla, Vaish led a team of 43, dramatically changing how people buy the Tesla Model 3 through advance deposits, paperless transactions, and personalized home delivery. His team also built a payment system for in-car upgrades. At StubHub, Vaish and team led development of the award-winning StubHub mobile app, featuring a personalized, seamless experience that enabled users to buy tickets in just three taps. At Epocrates, Vaish evolved its mobile app from a 1.0 forms-based product to a modern 2.0 experience to help doctors minimize drug-to-drug interaction dangers. You can read more about Parag joining Medable here.

January 2021 also marked Martin Luther King day. And with this special day in mind, we partnered with Allison Kalloo of the Medable Patient Advisory council to explore the very real disparities in diversity and inclusion in clinical trials. 

Allison was kind enough to share her visceral experiences of growing up and coming of age in the healthcare field dating back to her mom’s experience as a participant in the Civil Rights Movement and her subsequent career as a virologist focused on infectious diseases. This shaped Allison’s experience and led to her becoming one of the foremost thinkers in how to improve Diversity and Inclusion in clinical development. 

You can read Allison’s posts and watch her video interview with Dr. Michelle Longmire here:

 

You Believe in Diversity, Equity and Inclusion? Okay. Show Us.

Blog from Medable PAC member Allison Kalloo

Fit for a King: Bending the Arc of Legacy Toward Action

Blog from Medable PAC member Allison Kalloo

Q&A With Medable PAC's Allison Kalloo On MLK Day 

Video interview with Michelle Longmire, Founder and CEO of Medable, and Medable PAC member Allison Kalloo

In the coming months Medable will be focusing on how to get started with a decentralized trial approach from both a strategic and operational perspective and then turn to looking at the benefits of launching a DCT platform that standardizes how systems, processes, people and data can come together in a predictable way to drive even greater efficiencies in this exciting new realm. 

Stay tuned!

Topics: decentralized trials remote trials PAC medable MLK eCOA
2 min read

Wither the Caregiver. Pharma and Healthcare’s new Best Friend.

By Paul Kidwell on Feb 4, 2021 3:57:41 PM

You will never recognize them by sight or outward appearances. They come in all shapes, sizes, genders, ethnicities, and ages. Spotting them in a crowd is also challenging, as they are typically more comfortable on the periphery of activities, preferring to speak “only when spoken to” or when something is worth sharing. 

They are equal parts loving partner, master planner, keeper of schedules, proactive listener, quick study on all things medical, thorn in the side of the biopharmaceutical industry, sometimes loudest voice in the room, and madly in love with the person they support. Which, above all else, is why they are comfortable in this role, the role of a caregiver. When asked why an individual becomes a caregiver to a spouse, child, partner or loved one, they will tell you simply; “it wasn’t my choice, but it starts with love.”

Every caregiver is born in a different way. When I was thrust into a caregiver role nearly 15 years ago, I was your typical “babe” in the woods. Nobody expects to get sick until they do. For caregivers, this sentiment is exactly the same. The role and its inherent demands are never part of a person’s thinking. As in, “well, when I become a caregiver I will know exactly what to do because I have been preparing for it all my life.” One day you’re not a caregiver and the next day you are. More importantly, you're often a caregiver to those who are most important to you. In my case, I was a caregiver for my wife, on whose face the sun rises and falls. Perhaps more frighteningly, I was a caregiver facing tasks I had no experience with. 

This is particularly true when someone like myself accompanies my care partner to a physician appointment, or a meeting with a team to discuss potential involvement in a clinical trial. While doctors meet with patients on a regular basis, it becomes quite clear during initial engagements that pharma and the supporting healthcare ecosystem have minimal exposure to caregivers. To their credit, the industry has done yeoman's work during the last decade in ensuring that patients are at the heart of any drug development and/or clinical process. However, missing in this equation – and I really mean totally absent – is the lack of consideration to or involvement with caregivers.  If it is a patient perspective in which these professionals are interested, they would do well to look at the other person in the room and tap into this huge reservoir of information. Real-world data that’s gathered within close proximity of the person for whom they care, and includes medical, personal, and all-important anecdotal information. Information that can be used when developing drugs, trial protocols, clinical wearables, marketing campaigns and the like.

Caregivers and biopharma share the same goal. The optimum health and improved outcomes of the patient. And yet, there is little evidence that they ever talk to one another. Furthermore, when information is presented, usually by the biopharma professional, it comes from a lofty peak of complex medical data and rarely understood by the non-scientist caregiver. When I am immersed in this type of conversation I usually quote a line from one of my favorite movies, Margin Call, whose main character says; “Explain it to me as you would to a small child or Golden Retriever.”

So, what’s the main takeaway here? It’s simple. Caregivers are interested in partnering with medical professionals across the continuum. They also recognize that the information they have to share with biopharma researchers may just be the missing secret weapon from R&D arsenals. Imagine incorporating data on how medicines are used, and their effectiveness (or lack thereof) during all phases of drug development and approval. But that's not all, we also provide value with trial recruitment, patient engagement and retention, and any number of ancillary pieces of data that will help improve the patient journey.

How do you gain access to all of this?

Well, all they need to do is ask.

2 min read

The language of eCOAs

By Dr Ingrid Oakley-Girvan on Jan 28, 2021 2:41:06 PM

What do patient’s care about most and what do clinicians consider essential to assess regarding their patient’s health and wellbeing while participating in clinical trials? These are commonly referred to as clinical outcome assessments (COAs). 

The four categories of COA

In patient-centric clinical trials, COAs are essential to understand the impacts of a drug on certain endpoints, for example, whether it is improving or diminishing quality of life and everyday activities that matter to patients. The FDA, signaling the importance of COAs and the need to develop additional ones, has even created specific guidance and pathways for their development and validation.  

Fortunately, one of the lessons from COVID-19 is that we can be flexible and efficient and conduct a multitude of COAs remotely, or what we call eCOAs. No longer do patients need to come into the clinic for every assessment:

  • ClinROs can be measured over a televisit
  • PROs can be measured through patient directed surveys on a mobile app
  • ObsROs can be measured on mobile apps with care partners assistance at home
  • And device data can be used to generate PerfOs

Not only can we collect these at standard times as if patients were “in clinic” but we can collect them even more frequently adding critical new data to better assess health related quality of life impacts that patients often highly value. If the most important thing to a patient is being able to remain mobile, feel less foggy or fatigued, we can incorporate additional time points to better understand the effects of a trial medication or concomitant meds on outcomes and endpoints.

Furthermore, in many cases longitudinal data collection may be analyzed to monitor participants for simmering adverse events and provide early warning of declining health with which clinical teams can act quickly.

Technology enables this to now be done at scale in a modular fashion which not only helps protect patients but also prevents trial drop out and can encourage retention as patients may feel they are receiving better care.

There is a freedom created by these discoveries and solidification of approach. Patients are no longer tethered to a clinical site, this means patient travel burdens are reduced and distance is of diminishing importance when making a decision to participate or remain in a clinical trial. At the same time, in a decentralized clinical trial model, facilitating remote data capture that occurs at greater frequency in an easily scalable approach will enable statistical assessments that are reliable and allow for new COAs to be developed that can support labeling claims and improved patient health. In sum, the language of eCOAs is a win-win for pharma and patient health.

Learn more here: https://www.medable.com/products/ecoa-epro

Topics: decentralized trials Hybrid Trials TeleCOA clinical trials eCOA
5 min read

At Medable’s PAC, Patient Advocacy is Personal

By Paul Kidwell on Jan 26, 2021 10:34:17 AM

The Patient Advisory Council (PAC) at Medable consists of a small group of experienced professionals who work within the life science industry in various capacities. This includes biomedical research, communications, caregiving, patient advocacy and patient engagement. This growing group of nearly 10 individuals, Richie Kahn, Jennifer McNary, Gaurav Dave, Sumaira Ahmed, Allison Kalloo, Joan Venticinque, Paul Kidwell and Jena Daniels help Medable bring a patient perspective to the many technology-based programs in which the company is involved as they strive to decentralize the clinical trial experience for patients.   

 The high percentage of available patients who are not participating in ongoing clinical trials is staggering and continues to grow exponentially despite the ongoing efforts of an extremely interested biopharmaceutical industry who is willing to present an array of experimental therapeutics across all disease areas with the potential to address serious unmet medical needs. It’s Medable’s goal to create technical applications and platforms for patients that will allow and encourage them to more readily engage in clinical studies and gain access to these novel medicines by making the process easier and less daunting. 

Many times, people who engage professionally within the realm of patient advocacy have a personal backstory or a professional connection as part of their motivation for involvement. Whether a patient advocate, caregiver, researcher or an affiliated role within the life science industry, it is not unusual for these individuals to bring their particular story to this crucial role. This is true with members of Medable’s PAC, whose diverse professional backgrounds add to the integrated capabilities they offer individually. To have an industry perspective as a scientific researcher or industry communications or patient engagement professional provides Medable with an impressive array of talents and knowledge to benefit patients and company collaborators. 

Chief among them is Richie Kahn; a PAC member and medical researcher who taps into his professional repertoire when asked to offer his unique take on a Medable project. Researchers are at the heart of any biopharmaceutical company and the data they are able to provide and the ability to view the patient experience through a lens that was borne out of years of time and experience spent in a research lab cannot not be underestimated. Medable clearly understands this notion, ensuring that company leaders and/or employees who have scientific research are a central part of who is hired. This also extends to the PAC where research is represented in the person of Richie Kahn.

“I bring my experience as a clinical researcher to bear in each PAC engagement and each engagement outside of the PAC,” said Kahn. Where I am working to engage the patient perspective. I have a pretty good understanding of how studies are designed; how industry recruits patients and some of the methodologies used that don't bear a lot of fruit. Even receiving pushback that is likely to be received when we suggest to them reviewing patient-friendly methodologies.``  

As important as research is to understanding the patient journey, there is no substitute for the experience of an actual patient or the input from a patient’s caregiver. This first-hand, real-world exposure presents the most unique viewpoint imaginable, and any information provided by individuals who occupy these categories has added immeasurable value to biopharma companies developing therapeutics. Particularly when considering issues impacting clinical trials where patient involvement is anything but robust and there is a concerted effort to attract, recruit and retain qualified individuals across the entire disease landscape. A fact underscored by two-time cancer patient and survivor – and PAC member – Joan Venticinque.

“One of the biggest barriers to trials is access. You have to take them to where the patients are, to their doctors; their trusted communities. Not only to academic institutions where access is limited. You have to ask patients what is important to them; whether the potential outcome of the trial is relevant to them. You have to start in the beginning and get patients involved on the study team. They cannot be an afterthought.”

When a patient goes through their disease journey which often does include engaging in a clinical trial an active and involved caregiver is with them every step of the way. Besides a patient’s medial team, the care partner is the most important person involved. They don’t experience the disease physically, but a caregiver lives as the patient does and feels the emotional ups and downs of an illness, plus is equal parts counselor, homemaker, chauffer, advisor, and quasi-expert on all issues impacting their loved one. Medable’s PAC has the good fortune of Jenn McNary’s involvement. Jenn brings her experience as a life science industry and rare disease patient advocacy consultant to PAC. More importantly, is her lifelong involvement as a Mother of two sons who have Duchenne Muscular Dystrophy and another son who lives with Primary Immune Deficiency. Jenn lives and breathes the patient experience and brings a unique understanding to her advisory role.

“I know what a burden clinical trials and healthcare can be to families. So, I think my biggest contribution to the group – and Medable clients – is to make everything that we do a bit more friendly to the patient.  When working on the Access app we knew we wanted to attract as many people as possible to participate in the study. In order to accomplish that, we knew that we had to make it easy and understandable; and to design a website that really appealed to patients. And so we were able to create tag lines that helped reach patients. I think it’s important to remember that there are a lot of people pulling patients in a lot of different directions, and especially when you are talking about non-interventional, observational studies where compliance is a real issue. We have to make the process less burdensome and worthwhile, and if patients get frustrated, they will end up not participating.”

Supporting Jenn’s contribution as a caregiver is her Boston colleague and fellow caregiver, Paul Kidwell, who brings over 13 years of caregiving experience and two decades worth of involvement as a biopharmaceutical industry communications professional and story creator to the PAC.

 “The caregiver is the silent voice of the life science industry. Much like in companies who took the time to learn from patients and what they could offer, and the information they could provide to help with clinical trials, the same is now true for interaction with care partners. We are the closest person there is to the patient and the same information and experiences the patients are living through; we also live through.” 

Within the context of all groups there is the need to have someone act as the “connective tissue” between members, the company and their clients. For the PAC, the “straw that stirs the drink” is Jena Daniels, a trained research scientist currently who holds the position of Medable’s Director of Research and serves as company Advisor to PAC. In her role she directs the administration, client/patient engagement, content development and all ancillary PAC programs and forges the link between Medable and PAC. All with the intent of “amplifying the patient voice.”

“For patients, understanding that only having a seat at the table, is no longer enough. In a past position, I served as Clinical Research Manager for kiddos with autism and some of my favorite moments were being that connective tissue and advocating for the families while they are participating in our trials, and then sharing that information with our trial team. I recognized how excited I was within that advocacy component and working for a company that believes in patient centricity and the importance of having that patient voice and bringing in that perspective of the DNA of what we do.” 

The role of patent advocacy within the biopharma industry has taken on increasing importance in the past decade with the majority of established and emerging companies. Engaging with and acting on behalf of patients is at the core of this business sector, and company leadership is relying on these professionals to help triage the challenges this role typically confronts; including disease awareness, clinical trial recruitment, drug development and regulatory involvement to name a few. This function is becoming a common necessity in all corners of the industry sector and companies like Medable recognize that when patients speak the entire industry listens. 

 

 

1 min read

Why I Joined Medable: A Unique Opportunity To Change Lives

By John Walton on Jan 21, 2021 7:33:32 AM

When we see a pharmaceutical company’s drug pipeline, we are viewing a roadmap to a better quality of life for patients; however, the main obstacle we encounter is the time it takes to bring these drugs to market. In the world of disease progression, the average 10-12 year timeline to approve new therapies can mean the loss of hope for many.

IMG_1113

When I discovered Medable and their mission to bring effective therapies to patients faster by reducing clinical trial timelines by 50%, it was a statement that hit very close to home. Growing up with Cystic Fibrosis, I have been the beneficiary of medical breakthroughs that have advanced me from a life of predetermined struggle to enabling me to participate in collegiate athletics at the NCAA Division I level. What was once innovation, now necessity. Medable’s decentralized cloud-based platform enables high-risk patients, like me, simple and safe access to study enrollment while eliminating the risk of clinical on-site exposure. On the operations end, I cherish the challenge of providing a high-quality decentralized data capturing platform to
Sponsors and CROs to efficiently drive their studies to conclusion. This motivation comes knowing first-hand what it’s like to receive a next-gen therapy and the life changing benefits that go with it.

At Medable, we offer the once-in-a-lifetime opportunity to change the way clinical drug trials are conducted. Here you are not just another number in the system, but rather, an integral member of the project team. The teammates I have the privilege to collaborate with make it exciting to wake up every day and work towards that next therapeutic breakthrough.

While the Customer Success team operates under a remote setting, we have still found a way to develop a tight bond within the division. One of my first takeaways was the tight-knit family environment with a work-hard play-hard mindset. To any highly motivated individuals with a self-starter mentality, I encourage you to visit our career opportunities page and help our dream of bringing new treatments to market in half the time, become a reality.

Visit our careers page here: https://www.medable.com/company/careers 

Topics: decentralized trials medable careers life sciences clinical trials
4 min read

For Medable’s PAC Members, Blood Donor Awareness Month is Personal

By Joan Venticinque, PAC member on Jan 19, 2021 9:14:55 AM

Every 2 seconds, someone in the United States needs blood. Individuals will be in car accidents, need emergency organ transplants, give birth to babies in critical condition, or have conditions which require regular transfusions and need chemotherapy. Whether they receive whole blood, or red cells, platelets or plasma, when you donate you are making a generous life-saving gift. Richie Kahn, optic atrophy patient advocate and Medable Patient Advisory Council (PAC) member said it best.

 

"Though I only committed to regularly donating blood in the last few years, I can confidently say that it is a simple and incredibly impactful way to make a positive change in the community. By giving blood, you save up to three lives. I can't think of many other opportunities where I can make such a big difference. I just wish I had started donating sooner." –Richie Kahn

Whether donating blood or receiving it, this gift of life is of personal importance to many members of Medable’s PAC.

Jenn McNary, Rare Patient Advocate and PAC member is a mom of a son with an immune deficiency disorder. She counts on the many people who donate so her son can receive the blood plasma he regularly needs.

 

James receives weekly blood plasma infusions. This has made it possible for him to live a fairly normal life without the fear of major infections. James does not have a working immune system, he has primary humeral immune deficiency. Basically, this means that he never develops the immunity to most viruses and infectious conditions to which the general population is generally immune. Before he started getting his infusions, James, by age 10 was hospitalized multiple times with massive infections affecting his ear and skull cavity, body and throat. One such infection caused him to be hospitalized,  intubated, and sedated for almost a week because he lost his airway due to swelling. I am grateful to the people that take the time to donate blood and blood plasma because to families like mine, it makes a huge difference in quality of life. James doesn’t have his own immunity, so he borrows it from the hundreds of thousands of people who donate plasma.” –Jenn McNary

Sumaira Ahmed, is the Founder of The Sumaira Foundation for NMO, and Medable PAC member. She lives with Neuromylelitis Optica.

"Plasma has saved my life not once, but twice. Thanks to a procedure called plasmapheresis / plasma exchange [PLEX], my care team was able to restore some of my vision and all of my mobility that had been severely affected from relapses due to neuromyelitis optica. I am eternally grateful for those who donate plasma; it is because of them that I am living the quality of life I live today." – Sumaira Ahmed

Both James and Sumaira need plasma donations. Plasma is the liquid portion of blood that suspends the red and white cells, and platelets. Plasma helps to induce clotting and control bleeding. Plasma is important depending on the patient’s underlying disease. Allison Kalloo, founder of Clinical Ambassador and iParticipate Inc. and PAC member, is a regular plasma donor.

“After always being denied the opportunity to donate blood at blood drives (apparently, no amount of spinach consumption was ever enough for me not to be labeled "too anemic" for the Red Cross), I was surprised and delighted to discover that my iron levels are within range for plasma donations. I have been a twice-weekly plasma donor for two and a half years because I think my aversion to needles is a small price to pay to make a life-saving contribution.”

 –Allison Kalloo

For me blood donation became personal after suffering a hemorrhage after my third cancer surgery. I remember the doctor calling for pints of blood as I was being rolled into the OR for emergency surgery. I was never so thankful for the generous people who made sure I had the blood I needed when I needed it the most.

“I don’t donate blood, I give it and within that nuance consider it as a gift to another person who is in need. It’s not a gift in the traditional sense, but more important it truly is the gift of life. I think the decision to give blood becomes easy – maybe even heroic - when a person considers the impact on another’s life. It might even be that one offering that saves or extends a life.” –Paul Kidwell, Parkinson Caregiver and Medable PAC member

My mother instilled in me the importance of blood donation. I was always impressed with her many “10 Gallon Donor” pins dangling from her keychain. She never missed her regular blood donation appointments. As a child I couldn’t wait until I hit that magic age and weight where I could start to donate blood.

After numerous attempts at donating, I discovered I have a blood clotting disorder which makes it difficult to ‘fill a pint’ in the required time. There are many reasons a person may not be able to donate blood, but there are still things you can do to help. I am still able to donate blood for medical research, and as with Allison, she donates plasma. You can host a blood drive at your place of business, community organization or school, help spread the word about the importance of donating on your social media and visit your local blood donation center and volunteer to help.

In the time it took you to read this, approximately 622 people needed blood in the United States. Jenn, Sumaira and I cannot thank Richie, Paul, Allison and blood donors enough for their life-saving gift. I hope during Blood Donor Awareness Month you consider donating. Here are some organizations that can help you find a local blood donation center:

AABB

America’s Blood Centers

American Red Cross

Armed Services Blood Program

Blood Centers of America

 

 

 

9 min read

You Believe in Diversity, Equity and Inclusion? Okay. Show Us.

By Allison Kalloo, MPH on Jan 18, 2021 12:10:30 PM

We are excited to announce the addition of Allison Kalloo to Medable’s Patient Advisory Council. She is the founder of Clinical Ambassador and iParticipate, and is a passionate advocate for diversity in clinical research.

It is my hope that you will read this over and over again. 

The fact that minorities (and African Americans in particular) are over-indexed in measures of poor health outcomes is nothing new. The coronavirus crisis has simply highlighted persistent disparities in both disease prevalence and the underrepresentation of minority populations in clinical trials. The fierce urgency to find real solutions to combat this deadly virus means that longstanding inequity may finally be getting the exposure it has needed. Hooray for silver linings and tipping points. This surreal period in human history confirms for me that there is no better time to dream than in the darkness. 

Imagining the possibilities translates loosely into dreaming of dynamic public awareness campaigns, diversity-tailored programs, patient-centric campaigns, and more culturally sensitive protocols overall, and maybe even new interventions based on cutting-edge precision medicine initiatives that would allow medicine to take better care of its most vulnerable populations. Imagining the possibilities in the age of COVID also means being bombarded by news coverage from all corners discussing this need for diversity and the scientific community’s newfound dedication to achieving it. But if my hope and excitement are tinged with irony and incredulity, there’s a litany of historical reasons for that.

Industry stakeholders are appropriately acknowledging the need to be deliberate about recruiting participants of color. Attention here is long overdue. But sponsors are also more than familiar with the challenges they have faced with doing so. Failing to recognize pharmacogenetic and drug metabolism differences aside, being Black in America and being made to deal with systemic racism has proven to be its own risk factor. There is zero doubt that America’s unseemly past has managed to make African Americans fearful of stepping up to take advantage of participating in the very research that has the promise to save lives. Ironically, too few people know that the heinous Tuskegee Experiment had its own silver lining in being singularly responsible for the advent of informed consent. 

COVID-19’s growing toll on all Americans, but the wide disparities by race reveals that the heaviest losses among Black and Indigenous Americans nationwide—more than twice as high as the rate for Whites and Asians. Attention is now on getting Black and Brown people to participate in clinical trials in numbers that reflect disproportional disease burden.

The industry needs help communicating about what vested interest in research really means, and the pandemic is poised to drive the point home. There has to be a reconciliation of these facts with all stakeholders— that when studies do not reflect the diversity of the real world, not only will modern medicine continue to be complicit in perpetuating disparities, but those minorities who categorically refrain from participating in research will be complicit in less than favorable health outcomes and our own demise. This is no time to sugarcoat the facts. I tell people as often as possible that we simply cannot afford to sit this out. Frankly, we should consider inclusion in clinical research as much a social justice issue as protesting against police brutality. 

While I am a staunch advocate for diversity in clinical trials and stepping up to participate (and I have, in more than a dozen studies over the years), there has been an elephant in my headspace taking up room when I sat down to coalesce my thoughts on the matter. Regardless of our tone of voice, body language, or facial expression and regardless of whether we might have been justifiably angry enough to “burn [edifices] down” there is a collective acknowledgement that we have to be able to estimate the blowback to speaking truth to power. These mental gymnastics have themselves caused trepidation and exist as a source of justifiable anger. These communication challenges inherent to issues of diversity, inclusion, equity, social determinants, historical injustices and so forth — whether real or imagined— bring about additional obstacles downstream when trust and collaboration must be negotiated. But here is where we must start:

Whether it’s a matter of hiring Black staff, recruiting Black executives, asking Black community members to join your community advisory boards, contracting with Black-owned companies, or asking a member of your patient advocacy group to lend some perspective to publications calling for more diversity in clinical trials, I can guarantee that we all do some form of this calculus in our heads:

Are you really ready for the truth? Can you handle it? And if you can, will there be tangible evidence of the changes we recommended? And what will we be the price for making the establishment uncomfortable or disrupting the status quo?

We— Black and Brown people— are not inspired to believe you when you say that diversity is important to you if you don't put skin in the game. Making public statements that include the phrase “Black Lives Matter” will fall flat without adequate follow through. What will your ethos matter if you don’t actually DO something? It is no longer acceptable to say that this is not acceptable. We have no use for your anger. We need your action. 

Epic, concrete and systemic changes are needed and will be the only context under which real clinical trial diversity will transpire. We call bullshit on any minority recruitment endeavors that coexist alongside any of the following. Wherever the shoe fits, consider the cognitive dissonance you bring about when you allow these scenarios (and in no particular order):

  • Whenever you treat Black and Brown communities as monolithic groups who have the same life experiences, backgrounds, income, education, language, customs, interests, attitudes, and opinions in common. We don’t. 
  • Whenever you pile people of color into presumptive stereotypes and shallow narratives, not the least of which is that Black people can only be reached through the church. Wrong.
  • Each time your approach to minority inclusion is to treat unwillingness as cliche and to presume that distrust among Black people is tied only to Tuskegee, Henrietta Lacks and the infamous atrocities of the past. Today’s racism is more relevant to reluctance.
  • Every time you fail to factor in relevant disease prevalence data— or to acknowledge patterns of participation disparities in those areas— when designing protocols or planning recruitment initiatives.
  • Whenever privilege allows you to ignore the NIH mandate for inclusion and you complete enrollment without adequate minority representation; AND, whenever the NIH allows you to continue to receive federal funding in spite of your noncompliance.
  • Every single time you do “just enough” to comply with the FDA for approval of your NDA, and when you have spent more time crafting the explanation for your shortfalls than you have investing in a real plan to prevent them.
  • Each time you use mere familiarity or convenience as your site selection criteria when these sites possess no actual history with— or cultural connection to— the communities of color you seek to enroll.
  • Whenever you presume a team approach or collaborative mentality and rely heavily on physician referrals to fill your study’s patient rosters.
  • Whenever physicians are neither provided accessible education about clinical trials, challenged to embrace medical research as a viable treatment option, nor expected to broach the topic in consultations with their patients.
  • Whenever your study goals and timeline so blind you to the humanity of your target patients that you fail to properly introduce yourself first, or invest in establishing a relationship and a foundation of trust.
  • Whenever you implement the strictest (and least realistic) inclusion/exclusion criteria for participating in your clinical trials, creating de facto barriers to diverse, equitable and inclusive participation at the study gate.
  • Each time you slash a patient recruitment budget, minimize its importance, fail to make patient recruitment a priority, or allow it to be an afterthought.
  • Whenever you complain that diversity is “too expensive” or decide in advance that minority recruitment is too complicated. 
  • Whenever the IRB or any regulatory body alters the language that consultants have suggested related to anything patient-facing, including marketing, education or outreach— as if authority alone is synonymous with transparency or drives engagement.
  • Whenever you don’t make space, set aside resources or allow time for an external task force to review and assess cultural sensitivity and patient-centricity of study protocols and to identify biases and barriers.
  • Every time you default to doing business with majority-owned (read: White) marketing firms to resolve minority representation simply because they seem “safe” and familiar.
  • Whenever fear of change forces you to default to using status quo recruitment and retention activities simply because they seem “safe” and familiar.
  • Whenever you fail to establish quantifiable recruitment expectations for recruitment brokers based on demographics that reflect known data about the therapeutic area.
  • Every time you second-guess the pricing, professionalism, scalability and ability to deliver the goods and services from a small minority-owned business when you would be less likely to treat a larger business of privilege with comparable scrutiny.
  • Whenever you solicit feedback from consultants (whether professionals, study participants, patients, or members of the community) and fail to implement their recommendations— or fail to give them credit and compensation for their time.
  • Whenever your perception of expertise is cloaked in whiteness first.
  • Whenever you rely on study participants to take risks while you remain risk-averse in every possible way.
  • Whenever you set high conference fees that are functionally exorbitant to attendees of color [often classified as historically underutilized businesses (HUB) and/or certified minority- and women-owned businesses], thereby making their attendance cost-prohibitive and effectively shutting out competent and competitive collaborators and doubling down on disparities.
  • At the points where you compensate keynote speakers to address diversity, equity and inclusion, and that person is not Black or Brown; conversely, whenever you expect minority experts to provide that expertise pro bono.
  • Whenever your website’s homepage lacks a “DIVERSITY, EQUITY & INCLUSION” section— or it is difficult to find readily— it belies corporate DEI policies and the stated priorities of your organization. 
  • When your public overtures regarding dedication to diversity, equity and inclusion (DEI) — whether at the corporate, HR or patient recruitment level — are not matched with adequate resources or a timeline of changes for which you’re also willing to be publicly accountable.

While the system has long been broken— and addressing it will require more than  publicity statements and grandiose gestures— I recognize the value in framing these issues in such a way that you will know that solutions are within reach. 

Let’s discuss this in an open manner that benefits the greater good. Let’s take a deeper dive and embrace the powerfully positive approaches we can cue up that will make the most difference. 

In the blog series that will stem from this, I plan to explore dynamic recommendations from multiple sources (yes, more than just my own), and to showcase a tantalizing variety (read: diverse array) of people, organizations, policies and clinical interventions poised to change the landscape for the better over time. I hope you will stick with me for the ride. 

But in the meantime, know that It will take much more than throwing a fistful of money at a rescue rather than planning better from the beginning. 

Start by taking a hard look at the invitation list you inadvertently created with your protocol design. Who are you inviting to your party?

If you are sincere about filling the studies of tomorrow with a broader spectrum of individuals, implementing the tools of transformation will require the guts to be honest with yourself

One can’t expect to tease apart attitudes about participating in a clinical trial from how a person feels the system treats them. And this absolutely cuts across socioeconomic class. Even African American professionals and other high income earners openly express concerns about participating, and others of us harbor misgivings that still linger in the back of our minds. 

Reconciling these issues is fundamentally a matter of perception. Getting to a better place in DEI work will demand significant outreach going forward— both broad and deep. Lamenting lack of diversity in studies while not doing the work to get out of your own way is disingenuous at best.

As an industry that uniformly proclaims how dedicated it is to diversity, you will want to pay attention to this pivot point in time. This is your opportunity to actually rise to this occasion to DO something. Make it profound. 

Now is not the time to hold back.  

Industry will need to address head-on the hesitancy of many who came by theirs honestly. But building trust is not rocket science. It's about building relationships and employing trusted messengers outside your direct influence. 

Reaching out and across effectively is hinged on those in charge of clinical trials being more trustworthy. In other words, you first. Show us what you’re working with.

As for me, nobody is more interested to see how this goes.

—Allison Kalloo, MPH



Topics: D&I Diversity Inclusion Equality
5 min read

Fit for a King: Bending the Arc of Legacy Toward Action

By Allison Kalloo, MPH on Jan 18, 2021 12:07:19 PM

We are excited to announce the addition of Allison Kalloo to Medable’s Patient Advisory Council. She is the founder of Clinical Ambassador and iParticipate, and is a passionate advocate for diversity in clinical research.

More than ever, I am convinced that anyone who does anything has got to hold a deep and abiding belief that one person can actually make a difference in the world. Dr. Martin Luther King, Jr. clearly believed that. My mother believed that. I believe that also. I had little choice.

My mother was a spitfire who radiated warmth and grace, and she very deliberately instilled in me that I alone am enough to have an impact. That my unique talents and passion are actually supposed to be of service to the world. 

Both Dr. King and my mother had an abiding commitment to civil and human rights that stemmed from their fundamental belief that we are all called to take action…each of us. That being “created equal” also confers an equitable obligation—no matter who we are, where we’re from, or “what” we’re from—to use our talents for the greater good. 

My mother grew up in the racially hostile south, coming of age during the 50’s and 60’s where not having access to equitable health care easily set into motion poor health outcomes. My mother was class Valedictorian and the first to go to college in her family. She’d been an avid reader from early childhood and became enthralled by science. GIven her chronic health struggles exacerbated by discrimination and lack of access to health care, the fact that she did go to college was remarkable. 

I was recently granted a copy of her college admissions essay, which she wrote longhand in 1959 at the age of seventeen. My mother’s essay ends with the following passage: 

“In light of the above facts, I feel that North Carolina College is the institution which will prepare me adequately to adjust myself to the various situations of life as well as equip me in such a manner that I might make a definite contribution to society.”

—Marian Suitt

Among hundreds of other young, gifted and Black students who attended HBCUs and who remained somewhat insulated from the outside world, they were fully cognizant of navigating the segregated south. She and her classmates felt duty-bound to participate in the lunch counter sit-ins later credited with being catalysts of the civil rights movement. She would also attend the March on Washington in 1963, the summer she graduated. 

The civil rights struggle was not just an abstract captured in grainy black & white news footage. It was a vivid first-person experience that my mother lived. When she retold the stories, it was in riveting detail. For me, the earliest and most compelling images of who Dr. King was and what he represented came straight from my mother’s mouth. 

Armed early on with the prospects of not having a lot of time, my mother possessed a sense of urgency and decisiveness about everything. She would become a lead scientist for the District of Columbia Bureau of Laboratories as an expert on swine flu, rabies, and smallpox. My mother was a trailblazer and scientist and the context for my first superhero being depicted in a white coat.

In the wake of the coronovirus pandemic, not only have I thought of my mother for the scientist she was, but ironically, I’m reminded of her every time Dr. Anthony Fauci is mentioned or interviewed. You see, Dr. Fauci is my mother’s peer and was a colleague. My mother was a virologist, too. They attended the same scientific conferences and Dr. Fauci’s name was often the topic of dinner table conversation. 

When her health began declining further, my mother made the bold decision to participate in a clinical trial. She signed up for a trial at the National Institutes of Health— led by Dr. Fauci. As she shared with the family, she knew participating would not save her life. Her participation would help somebody else, and that’s what mattered to her. And she was uniquely equipped to recognize how important representation was— even in the long shadow of the Tuskegee Experiment. 

It’s impossible to look around and not be taken aback by how far we’ve come in the last thirty years since my mother’s enrollment in that clinical trial, but also how far we still must go if we are to make good on Dr. King’s dream of equality. 

Despite profound medical and technological advances that have occurred even recently, racial and ethnic minorities have failed to achieve equal health outcomes. By virtually every measure, minorities experience higher morbidity and mortality rates than the general population— even when income, education, and other socioeconomic variables are controlled for. One doesn’t have to wonder what Martin Luther King would have thought about the disproportionate burden of disease among certain groups. He actually said it: “Of all the forms of inequality, injustice in health care is the most shocking and inhumane.”

Exactly a year ago on Dr. King’s birthday, I was leaving a study appointment for a trial I was participating in. As I walked through, I locked eyes with an elderly Black woman seated in reception. We both did ‘double-takes.’ I smiled at her. She smiled at me. (We could see each other smiling because it was pre-COVID and neither of us were wearing masks). Turns out, she wanted to accompany her niece to her study screening. She told me that while she had been a bit leery, they decided to show up anyway. I remember telling her that I thought minority participation in clinical trials is about  health equity and social justice. That on a personal level, I think of participating as an issue of awareness, empowerment and exercising options which include access to cutting-edge healthcare. I’ll never forget her response. She said, “You know, you're right. I hadn’t thought of it that way. It is important that we participate. I'm glad you took the time to speak with me.”

I felt emotional walking back to my car. It hit me that my encounter was infused with Dr. King’s spirit. I had also experienced validation that in our increasingly digital, tech-driven world, face-to-face connections (and virtual ones for these times) remain the holy grail of connecting with people and we need to make sure we give this brand of magic its due.

It’s hard to imagine that any of our high-tech modern conveniences could possibly have enhanced Martin Luther King’s profound impact on the world any more than he did. His movement was simply about connecting people. 

My mother and Dr. King both lived by an edict that we are all beholden to do something significant. That we all carry an obligation to do more than just dream. 

I’ve come to realize that the concept of legacy is not only something that remains of you after you have gone. Legacy is actually what you create daily. It’s what you’re actively building, and all of what you do pays homage to those who’ve invested in you. 

So, may it be for us today and always, that the best way to show respect for Martin Luther King’s principles—and that of our ancestors—is to DO SOMETHING of service to humanity.  Do something that MATTERS. Pay it FORWARD.

“Now, therefore,” wrote Barrows Dunham, “since the struggle deepens, since evil abides and the good does not yet prosper, let us gather what strength we have, what confidence and valor, that our small victories may end in triumph, and the world awaited be a world attained.”

Happy Martin Luther King Day 2021!  

Allison Kalloo, MPH



Topics: life sciences D&I Diversity Inclusion MLK
1 min read

Q&A With Medable PAC's Allison Kalloo On MLK Day

By Allison Kalloo, MPH on Jan 18, 2021 12:06:10 PM

We are excited to announce the addition of Allison Kalloo to Medable’s Patient Advisory Council. She is the founder of Clinical Ambassador and iParticipate, and is a passionate advocate for diversity in clinical research.

Each year, on the birthday of Dr. Martin Luther King Jr., the nation takes a moment to pause, reflect and think back on the life and impact of his work. 

Today, our own Patient Advisory Council member Allison Kalloo sat down with Medable CEO and co-founder Michelle Longmire to incite a moment of pause and reflection, by discussing the very real and continued issues and challenges that African American communities face in our own industry.

 

Allison has had a remarkable life shaped by the work of Dr. King, her mother, and trials and healthcare in general, and she presents the industry with a number of challenging thoughts and questions that should make us all stop, pause, and reflect on the work that we do everyday, as well as the lives we lead outside of Medable.

Topics: life sciences D&I Diversity Inclusion MLK
2 min read

Decentralized Shouldn't Mean Disorganized

By Mary Costello on Jan 15, 2021 9:30:06 AM

Decentralized clinical studies are referred to by a variety of names - virtual, digital,  - the list goes on. Yet, whatever you may call it matters not; instead it’s the core tenets of this idea that matter most. Here, the inherent patient-centricity of decentralized studies is both the goal and the reward.

For those who may not know, decentralized trials are executed through a combination of TeleHealth and traditoinal in-office settings. Instead of office visits, DCTs often include visits that may be conducted remotely, with patients at home for some or all of the trial. 

However, simply arming potential participants with access to or actual apps and devices is not a recipe for success. A clinical trial is, at minimum, an exercise in detailed planning and should always be subjected to a “what if” modeling to plan and to identify  what can be prevented or mitigated.

Thus, it comes as no surprise to veterans of clinical trial logistics that the unprecedented challenge of vaccinating waves of Americans with an ultra-cold product was going to be difficult. The decision on how to deploy was left up to states, with seemingly little planning for a central repository to pre-register or track patients nationally.

The goal was to have 20 million vaccinated by December 31st, 2020. The end result, according to Reuters? A much lower 2.8 million. 

The reason? According to multiple reports across Reuters, Newsweek, CNBC and others was simple - a failure in planning and communication.

While the Federal government was responsible for, and relatively successful in providing the allocated number of doses to States, progress halted there. A number of key oversights, such as leaving States to plan their own distribution to residents, as well as developing last minute contracts with retail vendors like Walgreens to administer vaccinations have slowed the pace.

As such, Americans have found themselves left in the dark, with little to no information on how to register for the vaccine, where to go, and most importantly, the timeline to vaccination?

 So, what’s the lesson to be learned here? That decentralized does not have to mean disorganized. 

Clinical trial leaders need to employ the same planning principles that have underwritten successful trial conduct for decades. Proper planning, communication, and room for flexibility are key for all trials - decentralized or traditionally managed. Thankfully, there are several examples in the clinical trial world that sponsors and CROs can look to in order to plan a proper decentralized trial.

New tools in the industry afford us new hope and new access for communities that have historically been denied a chance to participate in research. However, the imperative should be in making all aspects of the study as accessible as possible including thinking through how to communicate, register and prepare participants. 

Here in Texas, we are struggling to move into 1B. Houston, one of the largest and hardest hit areas of the US, has been overwhelmed with too few locations offering the vaccine. In fact, many rural Texas counties have not even received a shipment of product. Imagine if we had used the time we had from February until now to create an online registry for people to register, learn about the vaccines, identify accessible locations, and share confidential health information that would help pre-qualify and stratify vaccine recipients. We would be in a much better position today.

Never shortchange up front planning or underestimate the value of communication. It will be the foundation for success.

 

2 min read

J.P. Morgan Week Trends, Observations, and More With Rasmus Hogreffe

By Rasmus Hogreffe on Jan 14, 2021 1:06:50 PM

With a few days of this year’s J.P. Morgan (JPM) already behind us, we took the time to speak with  Rasmus Hogreffe,VP, Decentralized Clinical Trial Innovation, to summarize his key observations, thoughts, and feelings around what he’s seen during this year’s conference.

 

Interest in DCT is Growing, and Key Players are Learning the Language

“This is perhaps the most I’ve ever seen DCT mentioned during JPM before. It’s great to see that DCT has become not only the buzz word of the conference, but rather, much more understood than it was in previous years. 

One of the more underscored elements of DCT, is that it operates within its own framework, or language within the clinical trial space. Now, with COVID-19 forcing multiple pharma companies to adapt, we’re having much more in-depth conversations with other organizations than ever before, allowing us to really explore the space with an ease not available to us in previous years.”

 

Clinical Trials Seem Unlikely to Go “Back to Normal”

“At JPM, everyone seems to be understanding that what we’re doing today is the “new norm.” Many of the major players are now understanding what it takes to be decentralized, and realizing that they don’t have to abandon site networks, that they don’t have to give up old standards, nor do they have to “throw everything they’ve learned out” in order to decentralize. 

What this brings is a new sense of wonder, and a new fervor in how we do business. With many seeing the benefits that DCT brings, it almost feels like the new era is here, and here to stay.”

 

A Worrying Trend - Less Focus on Patient-Centricity

“This year, we’ve seen more of a rise and focus on remote approaches, decentralized solutions, and new buzz words and business models that can help organizations transition themselves.

However, I’ve unfortunately noticed a dropoff in the amount of content geared towards patient understanding and centricity. As many will note, the ultimate goal of decentralization is to benefit the patient, making trials easier for everyone to participate. 

While I’m not surprised to see more focus on remote settings, decentralization, and more. I do hope that the industry realizes what decentralization means to the patient. Or rather, that they must build their remote offerings with the patient first.”

 

About Rasmus

Rasmus Hogreffe, MSc.Med., MBA is considered as a Virtual Clinical Trials expert. His main goal is to improve treatment for patients by making trials better and faster than ever before, using digital and remote innovations.

He holds an Executive MBA and a Master in Medical Science (MSc. Medicine) and has, since 2010, been working in the healthcare industry in various positions within clinical research & development.

Rasmus has extensive experience in the pharmaceutical industry and has been involved in more than 60 clinical trials, giving him a broad knowledge regarding every aspect of clinical research.

Furthermore, Rasmus is a serial entrepreneur and has a successful record of starting and selling companies within his broad scope of interests: Digitalization of clinical trials, Patient recruitment, Real World Data, and Health innovation. 

In addition, as an external lecturer and supervisor at Aalborg University, Rasmus has developed and given courses and workshops on management of clinical studies on topics such as the development of recruitment and retention strategies, site-optimization, site-selection and patient engagement to sponsors, investigators and study teams at leading medical centers.

His innovative approach within the field of digital health, Patient Recruitment, and Virtual Clinical Trials make him a frequently used chairman and speaker.

3 min read

Medable Hires Andrea Valente as Chief Operating Officer to Scale Global Delivery of Decentralized Clinical Trials

By Big Valley, on behalf of Medable on Jan 13, 2021 4:22:38 PM

PALO ALTO, Calif. — Jan. 13, 2021 — Medable Inc, the leading cloud platform for patient-centered drug development, today announced the hiring of Andrea Valente as chief operating officer. Valente is a veteran life sciences technology leader who brings deep experience delivering clinical trial technologies to patients at global scale. She has managed operations, product and service delivery for Oracle, IBM, ERT, Siemens and Phase Forward.

 As research and patient care move from the controlled environment of the clinic to the home, focus on operational excellence is paramount. Valente is a proven expert in operationalizing patient technologies in clinical research. She played an early role driving adoption and scale of electronic Clinical Outcome Assessments (eCOAs), one of the first technology categories to interact directly with patients outside of clinical settings. She most recently led development and launch of Oracle’s public health platform to address COVID-19 pandemic response and vaccine trials in the U.S., UK and several developing nations.

 “Andrea is not only an accomplished leader but she has industry-leading experience delivering technologies directly to patients, all over the world. This is very different than delivering solutions into the confines of a clinic,” said Dr. Michelle Longmire, CEO and co-founder of Medable. “Andrea knows what it takes to achieve operational excellence at global scale. She will be an invaluable addition to our leadership team as we continue to execute on our mission to build a highly impactful life sciences technology company.

 Medable experienced record growth in 2020 as the COVID-19 pandemic drove demand for remote clinical trial technologies. By minimizing the need for in-person site visits, Medable customers have achieved unprecedented results – including 3X faster enrollment and over 90 percent retention rates. Medable launched five new products and onboarded more than 50 new clients during the year, growing revenues by more than 400 percent – and fueling $118 million in new funding to accelerate digital and decentralized clinical trial adoption.

 “I’m thrilled to join a leadership team that looks at this space with fresh eyes, while learning from the past – and not necessarily repeating it,” said Valente. “Medable is truly transforming clinical research with a relentless focus on the patient experience and end-to-end process efficiency. We are scaling globally with consulting and implementation services that are grounded in science, clinical research and data analysis – across multiple therapeutic areas.”

 Medable executives are participating in several events at this week’s J.P. Morgan Healthcare Conference, sharing insights, advice and lessons learned from the front lines of decentralized trial adoption. Please click here for more information about Medable’s presence at JPM 2021, or to schedule a meeting with Medable’s executives.

 For ongoing insight about decentralized trial adoption and patient-centered research, follow Medable via our blog, LinkedIn, Twitter and Facebook channels.

About Medable

Medable is on a mission to get effective therapies to patients faster by transforming clinical drug development with disruptive technologies. The company’s digital platform streamlines design, recruitment, retention and data quality for decentralized trials, replacing siloed systems with integrated digital tools, data and interfaces to accelerate trial execution. Medable connects patients, sites and clinical trial teams to improve patient access, experience, and outcomes. Medable is a privately held, venture-backed company headquartered in Palo Alto, California.

# # #

Media Contacts:

Lisa Barbadora, Big Valley Marketing for Medable, +1 (610) 420-3413, media@medable.com

Topics: clinical trials Decentralized Andrea Valente
2 min read

Glaucoma Awareness Month: Educate Yourself on the Often Symptom Free Disease

By Richie Kahn on Jan 12, 2021 2:03:49 PM

January is Glaucoma Awareness Month which means it’s the perfect time to learn more about this often symptom-free cause of vision loss. As a patient with optic atrophy, one of the hallmarks of glaucoma, I’m intensely passionate about building awareness of the importance of routine exam eyes. Read on to learn more. 

For those who may not know, glaucoma is the leading cause of permanent blindness worldwide with over 76 million patients impacted. 

Technically speaking, glaucoma includes a number of diseases that result in damage to the optic nerve which connects the eyes to the brain. Damage is caused by increased pressure in the front of the eye which results from an increase in fluid build up. When left untreated, this can eventually damage the optic nerves and result in irreversible vision loss. 

Glaucoma is a chronic condition which means it stays with you for life. When properly treated, vision loss can often be prevented or slowed. Glaucoma often displays no signs or symptoms until the condition has significantly progressed. 

In order to accurately diagnose glaucoma, your eye doctor will perform a number of tests: 

  • Ophthalmoscopy: The shape and color of the optic nerve is reviewed for damage. After this exam, you’re eyes will be sensitive so you’ll want to wear sunglasses for a few hours
  • Tonometry: This test measures eye pressure. The average range is 12-22 mm Hg though it is possible to develop glaucoma when pressures fall in this range
  • Perimetry: This is sort of like a video game where you’re handed a clicker and asked to stare straight ahead. When a spot of light appears, you click
  • Gonioscopy: Here, your doctor will use a purpose-built lens to visualize the angle between your iris and cornea
  • Pachymetry: This test measures corneal thickness

 

If a diagnosis of glaucoma is made, your doctor will work with you to tailor the most appropriate course of treatment which may include eye drops; a variety of laser surgeries; and implantable products designed to reduce pressure in the eye and ultimately slow or potentially halt disease progression. 

Since there are relatively few outward signs and symptoms of vision loss, glaucoma is often called the Silent Thief of Sight. That's why it's so important to build awareness. Here are a few things you can do to help your friends and neighbors preserve their eyesight:

  • Talk, talk, talk! Both about glaucoma itself and the importance of getting screened for symptom-free vision loss
  • If your eyes are healthy, be sure to keep up on your routine eye exams. These typically happen every two years. They’re a great excuse for a new pair of glasses if that’s your thing
  • If you’re having trouble with your vision, be sure to tell your eye care professional as you may require specialized testing or treatment

 

For more information, be sure to visit the Glaucoma Research Foundation's website at www.glaucoma.org. If you'd like to read about optic atrophy from the patient’s perspective, please visit http://richiekahn.wordpress.com

Topics: life sciences glaucoma
3 min read

Medable Sets Stage for Broad Decentralized Clinical Trial Adoption in 2021

By Big Valley on Jan 11, 2021 1:36:48 PM

Shares Business Impact, Insights and Predictions for Patient-Centered Drug Development at JPM 2021

PALO ALTO, Calif. — Jan. 11, 2021 — Medable Inc, the leading cloud platform for patient-centered drug development, experienced record growth in 2020 as the COVID-19 pandemic drove demand for remote clinical trial technologies. By minimizing the need for in-person site visits, Medable customers have achieved unprecedented results – including 3X faster enrollment and over 90 percent retention rates.

Medable onboarded more than 50 new clients in 2020, as pharmaceutical, biotech, and clinical research organizations used Medable’s software to enable patients to participate in trials remotely — reducing their dependence on physical sites due to quarantines and social distancing. Decentralized trial adoption grew Medable revenues by more than 400 percent in 2020. Additionally, Medable launched five new products to support patient-centered research, and secured $118 million in total funding to accelerate digital and decentralized clinical trial adoption.  

“COVID-19 accelerated patient-centered research by five years,” said Dr. Michelle Longmire, CEO and co-founder of Medable. “2021 will be marked by the democratization of access to research. Leveraging Medable, partners can provide remote access to new investigational medicines to patients worldwide. We are driving radical transformation in clinical trials through patient-centered technologies that remove traditional bottlenecks and transform key processes. In 2021, Medable will continue to drive the emergence of a global ecosystem that truly enables research anytime, anywhere and for every person.”

Digital and mobile technologies played a pivotal role for clinical trials in 2020, enabling the continuation of in-flight research efforts via remote care while also accelerating development of vaccines and therapeutics for COVID-19. Building on that experience, Medable executives will share lessons learned from 2020 and predictions for 2021, via five sessions at this week’s Fierce JPM Week 2021 and Digital Medicine & MedTech Showcase events:

Rerouting: How Real-Time Patient Data Will Drive the Future of Healthcare

Moderator: Dr. Michelle Longmire, CEO, Medable

Panelists: Andy Coravos, CEO, Elektra Labs | Maria Fotiu, Executive VP of Decentralized Solutions, Syneos Health | Thomas Switzer, Digital Health Scientist, Genentech | Dr. Priyanka Agarwal, Director and Head of Digital Health, Myokardia

Patient-Driven Therapeutics: Charting the Future of Healthcare

Presented by: Dr. Michelle Longmire, CEO, Medable

Interviewed by: Sari Kaganoff, General Manager, Rock Health

The Digitalization of Clinical Trials

Moderator: Maria Fotiu, Executive VP of Decentralized Solutions, Syneos Health

Panelists: Dr. Michelle Longmire, CEO, Medable | Cathy Gao, VP, Sapphire Ventures | Juliet Moritz, COO, Illingworth Research 

Hindsight is 2020: Lessons Learned from DCTs and 2021 Predictions

Presented by: Allison Holland, Head, Decentralized Clinical Trials, Medable | Craig Lipset, Industry Founder and Advisor

User Experience: From the Patient, Site and Sponsor View

Presented by: Rasmus Hogreffe, VP of DCT Innovation, Medable

Interviewed by: Karina Marocco, EBD Group

Please click here for more information about Medable’s presence at JPM 2021, or to schedule a meeting with Medable’s executives. For ongoing insight about decentralized trial adoption, advice and best practices, and patient-centered research, follow Medable via our blog, LinkedIn, Twitter and Facebook channels.

About Medable

Medable is on a mission to get effective therapies to patients faster by transforming clinical drug development with disruptive technologies. The company’s digital platform streamlines design, recruitment, retention and data quality for decentralized trials, replacing siloed systems with integrated digital tools, data and interfaces to accelerate trial execution. Medable connects patients, sites and clinical trial teams to improve patient access, experience, and outcomes. Medable is a privately held, venture-backed company headquartered in Palo Alto, California.

# # #

Media Contacts:

Lisa Barbadora, Big Valley Marketing for Medable, +1 (610) 420-3413, media@medable.com

Topics: decentralized trials remote trials medable Hybrid Trials DCT life sciences
9 min read

Mental health coping mechanisms for caregivers with Dr. Lindsey Matt

By Jennifer McNary on Dec 23, 2020 1:12:43 PM

Creative Hands Co (1)

The following text is a truncated version of Medable Patient Advisory Council Chair Jenn McNary’s interview with psychologist and Patient Champion Member Dr. Lindsey Matt. Jenn and Lindsey were kind enough to film parts of their conversation for inclusion with this blog. Thus, we're pleased to present and recommend viewing the remaining portion of their interview using the videos placed throughout the article. 

 

2020 has been a tough year for us all, with many spending time away from their families and feeling isolated this holiday season. This is especially true for caregivers of chronically ill children. In fact, research indicates that 40% to 70% of caregivers have had clinically significant symptoms of depression with nearly a quarter to half meeting the diagnostic criteria for major depression. Additionally, 35% of parents with chronically ill children meet criteria for clinical depression with 57% meeting criteria for what is considered anxious symptoms. .

With that in mind, Medable Patient Advisory Council Chair Jenn McNary (JM) spoke with Patient Champion Network member and licensed clinical psychologist, Dr. Lindsey Matt (LM), to discuss coping mechanisms and considerations as a caregiver for chronically ill children that can benefit many of us and our relationships with others this holiday season. 

LM: I would love if you could start by introducing yourself and telling folks a little bit about you.

JM: Sure. I’m Jenn McNary. First and foremost, always in my introduction is that I’m a mom because I became a mom at 18. That is what I always introduce myself as. I have three sons that live with rare diseases of different kinds and I also have a healthy nine year old daughter. 

LM: Thank you! Speaking to that piece, when you think about becoming a caregiver, how was that for you or can you speak a little bit more to that process or transitioning to mom and to caregiver? 

JM: Yeah, so, it’s interesting because I’ve always been a really responsible person. I was a nanny; I was a babysitter, and, so, the transition to mom was pretty easy for me surprisingly...even at 18. I found that my mental health status of sort of dealing with a lot of anxiety and dealing with depression and things like that actually made me a better and more understanding parent. I understood when my kids were throwing tantrums. There were no pre-existing expectations for behavior. So, when I had a colic-y infant, I could really empathize with him. I found, even for me transitioning as a caregiver to sons with a rare disease...my sons were diagnosed when I was 21. Austin was three years old and Max was three months old. I didn’t have any preconceived notions of what it was going to be like to raise children so I just became the person who was raising children who were fatally ill.

LM: Speaking to that piece, just coming into motherhood and then learning it sounds about the illnesses that were going on with your children, what does the road of a caregiver look like for you from diagnosis and then over the years? Being in their lives and through that process?

JM: It’s interesting because, at first, it didn’t look any different than just being a mom. Very quickly it becomes being a protector. Being an advocate. There are doctors that don’t necessarily mean well or maybe they mean well but they’re just not having your child’s best interests in their mind. I had a lot of doctors say that there’s nothing you can do; just take them home and love them. All the way to doctors saying you may not want to get attached. You’re looking at a newborn baby and they’re saying, “That child is going to die. Don’t get attached.” So, I became an advocate for them to find the best care and a lot of it was external. In my mind, you know, just standing there blocking bad things that were gonna happen and then also moving into schools and being protective about what was gonna happen to them physically; whether they were being treated kindly; whether they were being picked on...and, so, it was a lot of defense. That was really our early years. As they’ve gotten older, it’s been raising them to be decent human beings so that other people will want to be around them because it’s harder to make friends; and have friends; and be in relationships when you’re disabled. When you need somebody to take care of you and it’s not your mom, that person doesn’t have to take care of you. And, so, teaching them really how to behave towards caregivers and how to be respectful. Sometimes, it’s tough love and really teaching them to be good citizens first and I think that that’s rare. I think that, a lot of times, these folks that depend on care can be really difficult to care for and I didn’t want that because I’m also a human and so, even though I have the best of intentions, I get irritated if I get woken up in the middle of the night over and over. 

LM: And you had mentioned, I think, something so important. At some level, having some anxiety, things like that, coming into motherhood, coming into parenthood, can actually make you well suited for the job. I’m curious, for you, as you added on this caregiver role, what were some of the, I suppose, advantages and disadvantages of that? You mentioned the irritability. Sometimes it can be a little bit much. You’re a human being. I wonder, for you, some of the ways this impacted your mental health over the course of time.

JM: A lot of times I have said in the past, and I maintain, that having the boys really saved my life. For some people, it can be a pet; it can be a plant that you have to water at least once a week. Just knowing that I really don’t have the option of just losing it, especially as a single mom. Even on the days where, and certainly with bipolar disorder, I have days where I’m super mom and I’m super functional and our house looks amazing and immaculate and I’m playing boardgames and I’m really, really good. Then, there are days where I’m like, “I don’t even want to leave my bedroom.” I don’t have that option. So, in a way, being forced into reality has been good for me and I think that also having realistic expectations about moods has made me a better parent sometimes. We yell. We’re a family of yellers. I’m a yeller. My kids are yellers. We all swear but we’re all pretty good at apologizing. Being somebody with a mental illness, you become pretty good at apologizing. You apologize for forgetting something; you apologize for not showing up; and you apologize for being irritable. So, all of my kids know that forgiveness is an option and they know that people are human. I think sometimes that takes the pressure off of the people in your life.

 

 

LM: You mentioned your boundaries, things you do to look after yourself and I think that’s a very, very important part about this conversation about what it means to be a caregiver. I wonder are there resources you have found helpful for supporting your mental health or areas where it’s been hard to have the support you’re looking for?

JM: I guess the resources--Facebook and support group, please where people get it--and that doesn’t mean that everybody with a child with a rare disease gets it--and so I have an ongoing chat group with about six women that have children with Duchenne of varying ages and we’re in constant contact every day. We have Zoom calls. We live across the country but that’s where I go when I want to vent so, making sure you find your people. Also making sure you find your people that don’t have anything to do with rare disease, totally don’t get it, don’t understand it, don’t want to understand it, and so you don’t have to talk about it. I have a couple of those friends and, in fact, I have friends without children. Those are my favorite friends because we can be really selfish and self-centered and they don’t even know that it’s happening. Those are the resources. Also, of course, advocacy organizations and things like that are great if you’re struggling. For me, the biggest thing I’ve done for myself is keep myself strong since I have to do a lot of lifting. My kids are heavy. So, I’ve invested in exercise. I don’t love exercise but yoga; Peloton; treadmill; you name it is here and it’s in my face staring at me in my room and in my office. Making sure that I’m taking care of my health has been incredibly important.

 

LM: Thank you so much for speaking to all these different areas. Is there anything else that you would want to add or other things that we haven’t talked about already that are really important in this area?

JM: I think that it’s just important for everybody to realize, again, if I were speaking to caregivers or to people who both struggle with their own mental illness and then also are trying to take care of another human being, I’d say what I just said to a mom who wrote in a chat group about completely losing it on her kid in the middle of the night. Like, completely losing it. We’re talking screaming; crying; she may have even punched a hole in the wall. I remember that the comments were so unkind. “Get help. Get help. Get help. You’re a psycho.” Even from our community. I remember commenting and saying, “If these people are saying that they’ve never felt that way, they’re lying to you.” I really want to remove the stigma of struggling and that martyrdom, you know? None of us is built to care for people exclusively forever. That is not the way that we are made. We’re not those kinds of people. We’re not superhumans. I really want to work to removing that barrier, you know, to feeling what you feel.  

LM: That’s an excellent point. Thank you so much.

JM: Thank you!

 

Resources

  • If you or a loved one is receiving medical treatment, start by asking your hospital, center, clinic, or provider if they are aware of mental health resources for patients and/or caregivers. 
  • If cost or provider availability is a concern:
    • Search “university counseling center” to locate low cost clinics in your area where therapy is provided by graduate students. These students are supervised by licensed professionals and often provide evidence-based care supported by current research.
    • Search “sliding scale therapy near me” to locate therapists whose session cost is negotiable based on income. Many therapists are also currently practicing via telehealth, so consider searching outside of your town or city on sites like Psychology Today or Good Therapy to access a greater number of available providers.
  • If therapy feels out of reach or you prefer the support of a group, robust communities for patients and caregivers alike exist within organizations and  across Facebook, Meetup.com, and Instagram. These can be found by searching for “support group” or “caregiver support group” along with the name if you or or your loved one’s condition. Examples include:
    • Family Caregiver Alliance - A list of support groups for adult and young adult caregivers of those with chronic health conditions
    • The Cancer Patient - An Instagram community that takes a satirical look at life as a cancer patient or survivor 
    • The Dinner Party - A peer community for those in their 20s or 30s who have lost someone close to them 
Topics: life sciences caregivers mental health
2 min read

The First Step is Education: Medable’s Jennifer McNary nominated by Reuters for Her Work in Rare Disease Advocacy

By Dan Horsey on Dec 21, 2020 10:31:17 AM

“The reason the Master Class is happening is because advocates like myself were not given a roadmap when our children were diagnosed with rare diseases,” states Jennifer McNary, Rare Disease Advocate. She’s flanked virtually, by her peers and fellow patient advocates for Reuter’s Events 2020 Pharma awards.

Jennifer, alongside three other nominees, is being honored for her work in furthering patient advocacy. Jennifer supports the Patients Rising Master Class she mentions as a faculty member, which is facilitated by fellow Reuters nominee, Lilly Stairs, and is simply a part of her everyday work in furthering the education that exists for patients who suddenly find themselves at the tail end of a rare disease diagnosis.

For Jennifer, and many advocates like her, proper education is the first step in creating a roadmap towards treatment. “Really, the key is that when you have success, instead of keeping that to yourself, share it.”

Thankfully, Jennifer has been instrumental in creating roadmaps for rare diseases for years, most notably with Duchenne Muscular Dystrophy (DMD).  In fact, her work as the Director of Outreach and Advocacy at a Massachusetts based non-profit foundation is the reason for what was the largest FDA advisory committee hearing in history for DMD.

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Jennifer’s end goal? Better access to treatment and greater speed to cures.

“Approved doesn’t mean accessible,” Jennifer says. “There are so many disparities around who can participate in a trial. When my son was in a trial we were flying back and forth across the country every week for blood work.”

Thankfully, Jennifer acknowledges that those days are starting to change. Companies across the globe, such as Medable, where Jennifer chairs the Patient Advisory Council for 2020, are helping to bridge the gap between patients and trials. Home measurements, remote assessments and other technologies greatly increase access to trials that were previously only available to a select few.

Recently, advancements in DMD treatment have given advocates like her hope.  Yet, she acknowledges there is still much work to be done, not only in DMD, but in rare diseases with little to no treatment plans.  

“My hope for all the rare disease community is that we continue enhance flexibility at companies like ours, enhance education, and be prepared to provide access and reimbursement for these kinds of therapies,” Jennifer notes, “So that when someone is born with a rare disease in five years there’s a pre-established treatment plan to help them.”

To learn more about the Master Class, please visit the Patients Rising Now website here

 

About Jenn:

Jenn McNary is a trusted voice in the rare disease community, as a mother, public speaker and fierce advocate. Her work in the rare disease space as a thought leader earned her the Ryan’s Quest Ryan’s Hero award in 2013, a nomination for the Global Genes Champion of Hope award in 2014, and the prestigious 2017 Meyer- Whalley instrument of change award. Formerly as the director of outreach and advocacy at a Massachusetts based non-profit foundation, she was responsible for the organization of the largest FDA advisory committee hearing in history, with over 1000 Duchenne Muscular Dystrophy (DMD) advocates, families, clinicians and researchers in attendance.

Topics: life sciences DMD rare diseases
2 min read

Medable named “Life Sciences Innovation Champion” in the 2020 Accenture HealthTech Innovation Challenge

By Dan Horsey on Dec 17, 2020 12:48:07 PM

Medable is proud to announce we have been named “Life Sciences Innovation Champion” in the 2020 Accenture HealthTech Innovation Challenge. Accenture has recognized Innovation Champions since 2016, by inviting healthtech startups across the globe to demonstrate how they can bridge the gap between startups and life science companies to put innovation at the heart of their business and patents. This year, they invited life science startups to showcase their businesses and answer:

“How can we transform the patient experience prior, during or after care (at-home, outside of clinics)?”

Accenture selected Medable as Life Sciences Innovation Champion for the 2020 Accenture HealthTech Innovation Challenge for providing patients with an integrated digital and physical experience during clinical trials, allowing them to access clinical care sites and clinical trial teams from their mobile phones.

At Medable, we believe that healthcare works best when it’s designed to improve the lives of patients everywhere. Thus, we’re excited, humbled, and grateful to be recognized as a leading company that’s working to simplify clinical trials through decentralization, while providing greater access to patients across the globe. 

Our own Tyler Pugsley, VP of Life Science at Medable expresses our commitment to improving the clinical trial experience:

“We are extremely honored to receive this recognition from the Accenture HealthTech Innovation Challenge. Clinical innovation is only possible through the great contributions of patients. This recognition from Accenture allows us to further improve the patient experience so that any person, anywhere, can participate in a clinical trial.”

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The Accenture HealthTech Innovation Challenge also showcases the breadth of advances happening every day across the life science ecosystem and recognizes more than just the hard work of Medable. It also honors the work of every startup in life sciences and the greater healthcare industry who has brought and inventive patient solutions to our market. This is especially true in 2020, as the industry works tirelessly to react and adapt to a world faced with new challenges and adversity as a direct result of the COVID-19 pandemic.

We’re also proud that so many of our peers have taken new and novel approaches towards solving the issues that continue to afflict our industry. So, if you have a moment, please take a second to recognize the following companies!

  • Cardiolyse, a cloud electrocardiogram (ECG) & heart rate variability (HRV) analytics platform that enables real-time remote heart health monitoring, providing plain-language personalized reports, detection and up to two months data-based prognosis on dangerous heart events;
  • Donisi, a global company at the forefront of contact-free health monitoring that works to change lives without changing lifestyles;
  • Particle Health, which is helping healthcare companies by offering simple, secure access to vital medical data by breaking down data silos that stunt innovation; and
  • Sidekick Health, which operates a patient-centric digital care platform driven by gamification and is scalable across a wide range of chronic illnesses.
Topics: decentralized trials DCT life sciences Innovation
3 min read

What Does Healthy Skin Mean To You?

By Sumaira Ahmed on Dec 14, 2020 10:30:53 AM

Did you know that Medable's co-founder & CEO, Dr. Michelle Longmire, is a practicing Physician specialising in Dermatology? In the spirit of learning, how about an interesting skin fact? Did you know that skin is the largest organ in the human body? It’s true! Factoids aside, what does healthy skin mean to you? 

Keeping my skin moisturized and itchiness-free” - eczema patient

Pimple-free skin! I focus my routine on keeping my skin clear and I feel my skin is the healthiest when I don’t have to wear any makeup” - acne patient

Feeling comfortable in my own skin” - psoriasis patient

We connected with a healthy skin expert and dermatologist, Dr. Georgina Ferzli, to get some tips on how to keep your skin healthy while you winter in quarantine:

  • Limit shower time to under 5 minutes and keep water lukewarm
  • Invest in a good humidifier
  • Use antioxidants to help your skin looking fresh
  • Use creams (in jars) instead of lotions (with pumps) as moisturizers
  • Avoid harsh acidic toners or washes
  • Limit exfoliation to once per week with a glycolic acid wash
  • Stay hydrated!
  • Incorporate hyaluronic acid into your routine 
  • Consider switching from retinal to bakuchiol

According to Dr. Ferzli, there are a variety of factors that impact our skin including gut health, mental health, stress levels, habits, and the way we feel about ourselves. Understanding the relationship between our skin and other variables is a huge step towards healthier skin. Some variables to consider when working towards improving your skin health are:

    • Sleep - are you sleeping enough? Also, did you know that sleeping on the same side of your face too much may result in an uneven distribution of wrinkles and creases in your skin?
  • Diet - as the saying goes, “you are what you eat!” Limited / avoiding oily, fried foods and abundance of alcoholic beverages is a good start!
    • Water intake - stay hydrated! Water does a body good.
    • Air quality / pollution - poor air quality and high exposure to pollution can lead to skin conditions including but not limited to dermatitis, eczema, psoriasis, and acne
    • Exposure to the sun (don’t forget to apply your SPF moisturizer even when it’s not sunny!)
    • Smoking - did you know that smoking is one of the fastest catalysts to aging your skin?
    • Cell Phones - there is an alarming amount of bacteria that can be detected on our phones. Be sure to clean your phone with antibacterial wipes. 
  • Makeup - remove your make up completely before going to bed! 
  • Products - with an abundance of skincare and makeup products available to consumers, it’s important that you’re using the products that work for YOU! Using the wrong products may result in adverse effects on your skin. 

About Georgina Ferzli, MD

Doctor

Dr. Georgina Ferzli is a cosmetic and laser dermatologist in New York City. As a former acne patient herself, Dr. Ferzli chose dermatology as her specialty in order to help others who suffer from skin-related issues. Dr. Ferzli spends her time curating personal skin care routines for her diverse patient community. 

Resources: 

Get in touch with Dr. Ferzli via email at georginaferzlimd@gmail.com or send her a direct message on Instagram @dermdocny

Topics: PAC PatientCare Skin Care Health Care
2 min read

Why I Joined Medable: An Inspiring Mission to Improve Patients' Lives!

By Michelle Paulin on Dec 9, 2020 3:37:13 PM

Impacting something bigger than myself has always been part of my philosophy. So, the day I met Michelle Longmire and she talked about Medable’s mission, I knew I wanted to be part of the team. Imagining that my job contributions could change the lives of patients waiting for a cure fit my idea of making my work life meaningful. Little could I have imagined how relevant and tangible Medable’s impact into the lives of the world population could be. At the time, the era of COVID-19 was not on the horizon and the race to achieve our company BHAG (to cut trials timelines in half with the use of technology) seemed far away. Fast forward to 2020, just two years after I joined, and I witnessed that in a matter of three weeks Medable was able to develop and deploy a TeleVisit solution. Our solution kept clinical trials running while patients remained safe and socially distanced in their houses as the world went into lockdown.

Now more than ever, we understand that making patients the center of everything is top priority. At Medable we work relentlessly to leverage technology, and provide engaging, high-quality data capture systems that enable sponsors, patients, and sites to feel confident about adopting Decentralized Clinical Trials (DCTs). In fact, this is how we materialized our mission of “enabling effective therapies to reach patients faster”. Thankfully, market adoption has made us leaders in the DCT space. With this leadership comes the commitment from all of us who work at Medable, to work as a unified team and empower one another to reach new heights every day.

Joining Medable has been a fascinating life experience for me. It has shown me how the passion of an engaged group of individuals can go beyond frontiers and make a remote team feel as driven and connected as if we were all collocated. It has taught me to stop at nothing and find a way to make it happen. It has made me feel like an integral part of a company where we work hard, while still making the time to connect as a team to celebrate victories and promote wellness. 

Medable is a dynamic place where I can see the impact and value of my work on a daily basis, not only within the “company walls” but in real life. It’s wonderful knowing that thanks to my work someone is finding a cure, and little by little we are improving the quality of life for millions while making the world a healthier place. If you are looking for a compelling reason to wake up everyday feeling energized about your work, please consider joining us at Medable!

Topics: decentralized trials medable careers DCT life sciences
4 min read

Ready for Remote: eCOA Adapts to a Hybrid World

By Big Valley on Dec 8, 2020 4:46:38 AM

COVID-19, for all of the pain and suffering it has wrought, has driven some positive change. In the life sciences industry, for example, necessity has been the mother of adoption—spurring a notoriously risk-averse industry to shift from in-patient visits to remote interactions by embracing mobile and digital technologies. In a matter of months, researchers started to decentralize clinical trials by adopting remote consents and clinical outcome assessments (COAs) through a variety of digital applications. 

One such tool is the new Medable TeleCOA application, which allows patients and researchers to harmonize outcome assessments captured remotely in the clinic and home. Medable TeleCOA works with Medable’s TeleVisit and TeleConsent applications to improve the trial experience for patients, sites, and sponsors. 

The company recently hosted a webinar to talk about the shift to remote collection of outcomes, hosted by CEO and co-founder Dr. Michelle Longmire, along with Dr. Robert Bissonnette (CEO of Innovaderm, a specialist in dermatology clinical research), and Richie Kahn (a patient advocate and member of Medable’s Patient Advisory Council).

While decentralization is a major industry trend, Dr. Longmire and the panelists reinforced that it doesn’t necessarily mean all trials will be 100% remote. Biotech and pharma sponsors need flexible and modular solutions to enable the unique needs of a given protocol. That requires additional digital technologies to connect with a patient as early as possible to engage in the trial, from recruitment to study close.

Dr. Bissonnette noted that some clinical outcome assessments—specifically patient reported outcomes (PROs)—have often been captured remotely and electronically in many clinical trials. Before COVID-19, though, most of the other assessments were almost exclusively done in a physician’s office. Bissonnette believes many more assessments can be done remotely, and “we’ll see more and more assessments being done remotely” over time, even after the pandemic.

Factors that Determine Feasibility for Remote eCOA

The feasibility of eCOAs depends on many factors. Dr. Longmire said understanding the disease and the condition is first and foremost, followed by evaluating which COAs are needed for the protocol—and then determining which COAs are suitable for remote.

Of the four kinds of outcome assessments—patient reported outcomes (PROs), performance outcomes (PerfO), clinician-reported outcomes (ClinRO), and observer-reported outcomes (ObsRO)—PerfOs and ClinROs have traditionally been most dependent on onsite clinics.

Dr. Longmire shared that more PerfOs and ClinROs are transitioning to eCOA, but many factors determine feasibility: therapeutic area, disease, disease stage, disease prevalence, patient preference, access to technology, privacy, safety, and so on.

Screenshot 2020-12-07 at 20.09.37

The panel discussed specific factors that determine remote activity, including:

Therapeutic area: Different areas of medicine require different levels of onsite care. PerfO assessments in dermatology, according to Dr. Bissonnette, can use telemedicine to a greater extent than neurology.

“Some research in dermatology should ideally be done 100 percent remote. For example, non-drug studies looking at quality of life or resource utilization or impact on work. At the other end of the spectrum, you have Phase I studies with an intravenous medication that obviously will need to be done in the research setting or environment. In between, we have many options.”

Kahn knows this firsthand from participating in research for his optic atrophy. “There are ways to collect information on visual fields to actually perform remote visual field exams at home. But every now and then you’re going to have something that cannot be done electronically.”

The need for physical contact doesn’t automatically require an onsite visit. There’s also the possibility of local nursing agencies stepping in to do things like blood work. And wearables can track a patient’s vitals even more comprehensively than less frequent onsite visits.

Disease prevalence: Finding patients with rare diseases may require more remote activity because of the difficulty recruiting. On the other hand, common conditions can often be researched onsite due to the broader pool of potential participants. For example, acne is so common that it’s easy to find patients near a research site.

Said Dr. Bissonnette: “If you do a study on a rare genetic disease, most of your patients will have to travel hours. That’s where one needs to try to do everything that is possible to avoid site visits and replace them with telemedicine visits.”

Disease stage: A patient’s condition can also determine the feasibility of remote care. “Typically in phase II-III studies, patients in the past had to come to the office for every visit,” said Dr. Bissonnette. “I would favor a hybrid model, where some visits are done at the site, some are done remotely, specifically for primary and endpoint visits where it’s a subjective evaluation.”

Customizing to Meet the Needs of Patients and Protocols

Regardless of modality, an important requirement of eCOAs is that they provide valid, reliable and meaningful endpoints equivalent to onsite COAs. This is happening more and more, while research on equivalence is still ongoing. Dr. Longmire reinforced the importance of a hybrid model that adapts to both patient needs and protocol needs. “Medable TeleCOA can connect with a patient as early as possible, on any device, to engage and enable participation in the clinical trial, from recruitment forward.”

Dr. Bissonnette noted that patients “want more and more telemedicine.” They would rather stay home than go to the physician’s office because it’s easier for them. “In the future, given the choice between two similar studies, if a study has 10 visits at the site versus another one with three visits at the site and all the other visits remotely, patients will probably favor the second study.”

Whatever COA strategy is used by a trial, focusing on patient needs should be a central design philosophy to ensure successful and sustainable studies. Said Kahn: “We are finally at a point where there’s all this momentum to decentralize trials and bring them to patients where they are and how they want to participate. … There’s going to be some stuff we get right and some stuff we get wrong. When in doubt, always incorporate the input of the patients early and often.”

# # #

For more information about capturing outcomes in a decentralized world, check out the full Medable TeleCOA webinar on demand (here) or reach out to our team of experts to schedule a meeting (here).

Topics: decentralized trials remote trials DCT TeleCOA life sciences
2 min read

Why I Joined Medable

By Dr Ingrid Oakley-Girvan on Dec 2, 2020 2:04:50 PM

Can you imagine waiting around in the kitchen for the phone to ring so you could do just one thing – talk to someone? Some of us remember when we had no choice because it was attached to the wall and we didn’t imagine all that a phone could be but then, that all changed. 

Smartphones untethered us and opened up incredible freedom with 3-D global video calls, digital maps and spoken directions, social and shopping apps, an embedded personal assistant, instant food delivery to our doors, endless internet searches no matter where we were, tagging and sharing of photos and information, calendars and health data at our fingertips, and so much more. 

This type of revolution is why I joined Medable.  When interviewing with the CEO Dr. Michelle Longmire and her co-founder Tim Smith, it was clear – we shared the goal of doing the same for clinical trials. Decentralizing operations, opening up freedom for patients and sites, creating digital therapies that could be delivered anywhere the patient chooses, enhancing robust remote data collection, cutting trial times in half, doing things so differently while maintaining scientific rigor – we were clearly aligned. In retrospect, I think when I joined many of my colleagues thought “wow, that is crazy, truly BHAGs, these guys are a bit nuts!” But now, as we watch vaccine development take place at an incredible pace and necessity forced regulations to facilitate Televisits and therapy development, our BHAGs hatched almost six years ago in the courtyard of a lively watering hole off Sand Hill Road don’t seem that nuts. In fact, they seem entirely achievable. 

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Our early recognition by NIH with two Small Business Innovation Research awards that I lead, work with multiple academic organizations and commercial customers to support their creative efforts to accelerate and provide better health care and trial experiences for patients and sites, and the belief in our goals and approach as documented by our recent $91 million in Series C funding bringing our total capital raised to over $136 million has been incredibly rewarding and validating.

As we work to continue to achieve our BHAGs this includes exquisite longitudinal ePRO, TeleCOA and digital data to help improve patient lives and deliver therapies in half the time, reduced paper and data burdens for sites, rigorous eSource and SaMD regulatory frameworks and metrics, delivery of interim endpoints and digital therapeutics, monitoring for early warning of simmering symptoms that warrant rapid clinical response and so much more all in a space where we re-envision the clinical trial experience. 

No two days in my life at Medable are the same with the exception of always being exciting and building on the team expertise and innovative mindset. I thrive off of being challenged to do more for patients and customers, creatively push harder, help solve disease disparities, and deliver validated solutions all while following our team ethos of Power Hour for self-care and Focused Fridays to thoroughly wrap up each week feeling I’ve made a difference and that my work is incredibly satisfying. The team spirit and energy is infectious and dynamic; we are empowered to have fun and move fast. If you are an expert in your area, hate being bored and share our BHAGs to do more for patients and help our clients deliver treatments sooner, I invite you to view our career opportunities.

Topics: decentralized trials remote trials careers life sciences
8 min read

Diabetes: It’s a Family Affair

By Paul Kidwell on Dec 1, 2020 12:33:11 PM

The world changes for people diagnosed with diabetes. Moreover, their families, loved ones, and friends are also impacted; and not merely as spectators.  We realize, however, that as is the case of most diseases or medical conditions, health issues not only impact the patient, but those close family members, friends and loved ones as well.

Diabetes affects everyone in some way. It not only affects the individual with the disease, it also affects those who live with the person or somehow involved in the life of the person affected by the disease. There are over 34 million people in the United States living with diabetes, with 1.5 million new cases presenting each year. A small, but significant group of that larger contingent consists of people under 20 who have contracted the illness. In fact, over 208,000 young boys and girls have Type 1 or Type 2 diabetes and for them their illness is truly a family affair. 

How well families cope with these changes can mean the difference between rapidly worsening disease and a relatively healthy life. It's an opportunity for families to strengthen their relationships with each other and to improve every member's overall health. But it is not without a serious commitment to patient health and well-being. That was certainly the case with the Shumsky family of Ann Arbor, Michigan; and sister/daughter Erica, who was diagnosed with Type 1 Diabetes at the age of 9. 

Her diagnosis came as a complete shock to her mother, Jen, as she saw this otherwise healthy young child  diagnosed with  this disease and tried to figure out what Erica’s illness would mean for her as the rest of the family which included Jen’s husband and elder son. That was six years ago, and recently Jen and Erica spent time with me discussing the influence and challenges of diabetes on the Shumsky family.

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Medable: Briefly please share Erica’s story.

Jen: I had been an RN for 19 years when Erica was diagnosed with Type 1 Diabetes (T1D) in 2014.  We also have a son with a rare disease, Duchenne Muscular Dystrophy, so my husband and I were no strangers to taking on additional duties when it came to additional medical needs in our children.  Erica was having some nighttime incontinence that originally appeared to be connected with a growth spurt as she had grown a bit taller and was looking thinner.  However, after a couple weeks it was not getting better.  The kids had spring break and were home for a week and we watched her drink and drink and drink apple juice.  My husband and I were both familiar with the signs of diabetes, but really couldn’t believe it would affect our family.  We had a glucometer at home that we kept for occasional checks of our son and if family members needed to check.  We decided to check her fasting blood sugar and it was high (238).  

Medable: Was the diagnosis immediate? 

Jen: We called the pediatrician and they sent us to the ER.  They confirmed the diagnosis in the ER with blood and urine testing.  Her A1C (a blood measurement to look at how blood sugars have been running) was 8.9, which is high, but not as high as many will have when they are first diagnosed.  We were told that we caught her T1D incredibly early.  

Medable: What resources were you offered by your medical team to find out more about Diabetes 1?

Jen: In addition to a very supportive pediatric endocrine clinic with nurse educators that were regularly accessible, we were connected with the Juvenile Diabetes Research Foundation (JDRF).  We were also very lucky to have a couple close friends that had T1D that we could lean on for support and advice.  

Erica: I remember I got a bag from the JDRF (called the bag of hope) that had a stuffed bear named Rufus that I really liked.  Rufus has areas on his body where you can practice giving insulin injections.  

Medable: What is the difference between Type 1 and Type 2 Diabetes:

Jen: Type 1 diabetes (T1D, insulin-dependent or juvenile) can occur at any age, but most commonly is diagnosed from infancy to the late 30s. With T1D, a person’s pancreas produces little or no insulin. Although the causes are not entirely known, scientists believe the body’s own defense system (the immune system) attacks and destroys the insulin-producing cells in the pancreas.  People with T1D must inject insulin several times every day or continually infuse insulin through a pump. While its causes are not yet entirely understood, scientists believe that both genetic factors and environmental triggers are involved. Its onset has nothing to do with diet or lifestyle. There is nothing you can do to prevent T1D, and — at present — nothing you can do to get rid of it.

Type 2 diabetes is much more common than Type 1.  Type 2 diabetes typically develops after age 40, but can appear earlier, and has recently begun to appear with more frequency in children. In this form of diabetes, the pancreas still produces insulin, but the body does not produce enough or is not able to use it effectively. Treatment includes diet control, exercise, self-monitoring of blood glucose and, in some cases, oral drugs or insulin.

Erica: It frustrates me at times that people do not understand that there are different types of diabetes. There is a lack of understanding that type 1 is something that just happens to people.   I do sometimes get frustrated  by all the diabetes memes and jokes out there about getting diabetes from eating junk food and lifestyle choices.  Many times people will see me eat something they deem unhealthy and ask if I should be eating that.  It doesn’t matter what I eat with my type 1, I will always have to take insulin for it.  I eat a fairly healthy diet and participate in school sports including field hockey and lacrosse,  which is just the opposite of what many people may think when they learn I have Type 1. Plus, because of this disease I will always be insulin dependent because my body does not make it naturally.

Medable: What was this new life this disease was presenting to you? 

Jen: Suddenly I had to do algebra every single day to figure out how much insulin to give based on how many carbs were in the food she was eating.  Never realized I really would need that much math  in my adult life.  We had to check her blood sugars every 3 hours at night at a minimum.  In those early days, my husband and I would do this together to learn the process.  We progressed to taking turns.  

Erica: I am now 15, but at the time of the diagnosis, I was very scared of these words, I really did not know how my life was going to change in the beginning. When I learned more about what I would have to do to keep myself healthy, such as daily injections or counting the carbs that I eat.

Medable: What was the emotional impact of this diagnosis? 

Jen: As a parent it is just devastating when your child is given any type of challenge in their life.  You want their life to be as easy as possible.  A serious medical diagnosis is hard, especially when it is going to be an ongoing lifelong condition, that is impacted by stress, hormones, food, exercise, alcohol, and countless other things.  Your child loses a bit of childhood when encountered with so much responsibility.  For my husband and I, we had already had one horrible diagnosis day when our son was diagnosed, so in perspective, this T1D diagnosis was so very hard, but knowing that if we worked really hard we could control some aspects of this disease was so encouraging.  

Erica: For me, I was very scared when the doctor came in and delivered the diagnosis. I remember crying and wondering why this was happening to me. I think the emotional impact for some is bigger than others. From my personal experience with this disease, I have learned that it is ok to have multiple mixed feelings, there is no one right way to feel. 

Medable: What has been the impact of the past few years living with the illness?

Jen: Technology has come so far in the 6 years since Erica has been diagnosed.  She started on a CGM (Continuous Glucose Monitor) about 4 months after her diagnosis as she had (and still has) hypo unawareness (she does not feel low blood sugars).  When she started with a CGM she carried a small receiver with her that would read her sugars, now her phone reads her blood sugars and sends those readings to our phones, so we always know how she is trending.  She also has been on an insulin pump for about 5 years.  The technology in pumps is constantly improving.  She now has a Tslim X2 pump that works with her CGM in what is called Control IQ.  This Control IQ system works by giving her extra insulin if her blood sugar is high and reducing her insulin if she is trending low.  Erica has come so far in learning how to manage her condition also.  As she is about to turn 16 next month, we need her to be responsible as she takes on driving and other activities.   

Erica: These past few years have been easier than the first to second years of living with type-one. Everything has gotten a little bit easier, for example, counting carbs has gotten way easier for me because I have been doing it for so long. Another thing that has been easier is knowing how to correct my blood sugar and how to keep it in a manageable range. I also have a pump as well. The pump makes life so much easier to manage. The pump I have is the Tandem X2, this is a great pump that has definitely changed my life for the better. 

Medable: Jen, how do you separate your role as a mother with that of a caregiver?

Jen: Well actually, I think all parents are caregivers.  What level of care you have to give your child is different in each household. I chose to become a nurse many years ago because I am a caregiver at heart, it brings me joy.  My husband and I decided early on that we were not going to parent our kids any differently than we planned because they have medical issues.  They have chores and expectations, they are punished and rewarded the same as their peers.  

Medable: Erica, how do you separate your “lives” as a teen-ager, student, patient?

Erica: I think that I separate my “lives” in different ways, but ultimately, they are all connected to each other in some way. As a teen, I have to make sure that my sugars are controlled if I'm going somewhere. If my sugars are too high or too low, it is very difficult to concentrate on schoolwork, If I am taking a test or a quiz, I must try to make sure to keep my sugar in the normal range. Lastly, as a patient, I like to think that I am a pretty decent one. If I see someone else in public that is a fellow “diabuddy” especially if they are younger than me, I will try to smile at them and I will show them that even though we have this disease, we can still be happy, healthy and amazing people. 

Medable: Erica, how supportive have your friends/classmates been?

Erica: My friends have been very supportive of me, from the time that I was diagnosed, there were friends that came to visit me in the hospital. Whenever I have told my classmates that I have T1D, they have also been very supportive as well. Some of my closest friends are like built-in reminders to check my sugars to make sure I’m alright. I love my friends to death, and I am definitely very glad to have them and that they love me too and look out for me.

Erica 2 - Friends

Diabetes Patient Resources:
Association of Diabetes Care & Education Specialists
Diabetes Foundation
Juvenile Diabetes Research Foundation

Topics: PAC PatientCare Diabetes DiabetesAwareness
8 min read

Profiles in Caring

By Paul Kidwell on Nov 30, 2020 1:35:45 PM

Former First Lady, Rosalyn Carter, is attributed to a quote that perfectly sums up the impact and the deep reach of caregiving in all of our lives.

"There are only four kinds of people in the world: those who have been caregivers, those who are currently caregivers, those who will be caregivers, and those who will need caregivers."

There are over 50 million people in the United States who care for adults and children afflicted by some type of medical condition, with illness preventing these individuals from caring for themselves. Whether caring for an elderly parent, a seriously ill spouse or child, or some other special person in need, those individuals who are on the front lines of caregiving put themselves second as they immerse themselves in a life of personal sacrifice of time, energy, and income. 

In this Medable blog we celebrate November as National Family Caregivers Month and highlight the lives of four individuals who are caregivers.  Legions of people call themselves caregivers and do so in quiet strength and without expecting anything in return for their selflessness. It is an isolating existence; one that often produces a sense of frustration and despair. We live in a culture where a growing contingent of Americans are thrust into this role - we call upon everyone to support them during this month and always. 

Daniel Zahn
Project Coordinator, Medable

My Son Ethan was born with CHARGE Syndrome in 2017, so my wife and I have had to take on the caregiver role as well as being new Parents.  Caregiver duties include drawing up daily medications, giving meds, g tube feedings, trach care, suctioning, and transportation to numerous Doctor visits per month at Children’s Hospital. 

Q: How do you balance your two lives; caregiver and non-caregiver?

My Son Ethan has a Physical Therapist, Occupational Therapist, Speech Therapist, Vision Therapist, and Hearing Therapist that all come to our home during the week.  During the pandemic, the home visits have stopped, but the therapy sessions continue via zoom meetings. This is not ideal, but my wife and I have become a ‘jack of all trades’ to help continue Ethan’s progress. Juggling work and therapy sessions has been challenging and tiring.  We do what we have to as parents and caregivers to make it work the best we can.

Jennifer McNary
Consultant. Public Speaker. Advocate. Non-Profit Leader
Manager, McNary Consulting

I was 18 when my oldest son Austin was born. He was a normal healthy baby but by age 3, following my second son Max’s bith, it was apparent something was wrong. Austin and Max were diagnosed with Duchenne about 3 months later. My now 13-year-old youngest son was diagnosed three years ago with primary immune deficiency at 10. So, I am a mom and caregiver to three sons with rare diseases.

I have always tried to separate being a mom from day to day caregiving by having daily help with Austin and Max's physical needs. At 21 and 18 they need total care, lifting, bathing, grooming, everything. I have had to work and have additional clients to earn enough money to supplement the personal care budget afforded to us by state medicaid. It's important to me that Austin and Max have care from professionals- I don't like the dynamic of being the only caregiver. That said, I don't have 24/7 care for them- so much of it still falls on me. James needs weekly infusions, which I have to provide him. That can be a battle- what 13 yr old wants his mom pushing needles into him every week. It's hard to even get them to shower at that age and take a multi-vitamin. Caring for the boys’ healthcare alone can be a full time job - from juggling specialists visits, appealing denied medications and durable medical equipment, to making sure they have all of their specialty meds. So much paperwork. It's mentally draining and there aren't enough hours in the day. I often joke that I need an admin, just to deal with that piece. 

Q: How has the pandemic impacted the emotional strain that already existed as a caregiver?

Worrying about bringing the help we need, PCAs, nurses, etc into the home has weighed on me. On one hand, I can't do this alone- it's been almost 9 months.  For the first month, I didn't have anyone come into the house. It was way too much work for me to do alone while working full time and juggling my 9-year-old daughter's remote schooling and care. On the other hand, my sons likely wouldn't survive COVID. There would be so much guilt on my side if someone I brought in gave the virus to one of the kids. Their immune systems are all compromised- I have seen James be intubated when he caught paraflu at age 9. I think I have a fair amount of secondary medical trauma so we live in fear of people. I'm also totally alone with the kids. As a single mom, I used my work travel and time away as a time to recharge - it’s going to be a long time before that can happen again. 

I've also had to give a lot of thought to what we would "do" if one of us got COVID. What would the kids do if I was sick and couldn't care for them? What if I didn't survive? It's crazy, maybe, but it took this pandemic for me to buy life insurance, and to start on a will. I realized they really only have me. These terrifying thoughts are what keeps us isolating as long as entirely possible. 

Q: How do you make sure that you have “me time?” Describe those private moments.

I am a huge believer in maintaining a sense of self even with kids, especially with kids with health conditions. I take what some might call excessive amounts of time to myself. I set reminders on my calendar to exercise on my spin bike or do yoga daily- I walk the dogs, I take time to read and write when I feel the urge. My kids are as independent as they can be, and I encourage them to find ways to do things themselves if possible. I also have regular help, as I mentioned. I don't believe in being a martyr - I don't think it helps your loved ones to believe you're the only one who can care for them either. With any luck, they will outlive me and need to learn how to have others do things for them as well. I used to add a few days or sometimes just hours to every work trip - just to explore. I love to travel - I love to try new foods and see new sights. The kids and I travel a fair amount together, but that's usually exhausting. Where traveling alone or with a colleague is energizing. I hope we can get back to that life soon!

Rasmus Hogreffe
VP of DCT Innovation, Medable

When my dad was diagnosed with Prostate cancer eight years ago. It was a shock for the whole family, and due to my medical background, I was the obvious choice to support him where and whenever needed. He passed away three years after his diagnosis and my role as caregiver expanded during that time as the disease progressed. The most challenging part of being a caregiver is realizing that you are about to lose a significant person in your life. In the last days of his life, you live in constant fear of the next call, is it now, is he dead, is he dying? This phase took out all my energy, making it difficult to cope. Unfortunately, I didn’t cope as well as I should because I kept all my feelings of sadness and impending grief inside without sharing with others. The experience made me think that I needed to be more open with my feelings, something I hope to improve upon in a future and similar situation. 

Q: We talk often about how our healthcare system needs to account for indirect costs of being sick, such as lost productivity and the emotional toll for both the patient and their caregiver. Can you expand on what these indirect costs mean to you? What are you doing to help offset these costs?

I believe that you only have 100% mental capacity. When you are a caregiver, you allocate a big amount of this mental capacity in taking care of your loved ones. This directly effect productivity, which can and will result in increased frustration and loss of focus. We are all unique and we all have different ways to measure the value of a treatment. There is definitely room for improvement in this area. I do not believe that we will be able to measure this 100 percent accurately. But by utilizing technologies where the patients and caregivers are, we would be able to collect precious data that could help us calculate a more precise value of a treatment. As the shadow of the patient, a caregiver will rarely be recognized, but the importance of this role is a fact and will never disappear. The value, however, comes mostly from the patient and not from others outside that experience. The industry must recognize the value of caregiving and that behind a patient, there is nearly always a caregiver/caregivers involved. It has taken far too long to put action behind the words “patient centricity”, let’s not make the same mistake with the patient caregivers. 

Ching Tian
SVP, Strategy and Solutions
Medable


In February 2019, my dad was diagnosed with late-stage gastric cancer. A month later, my mom found out that she has breast cancer. My brother and I barely had time to absorb the shocking news before we had to spring into action. My brother lived in northern California, near my parents, while I lived across the country on the east coast. We immediately started looking into treatment options and reaching out to doctors for second opinions. We had to make difficult decisions to try to balance their quality of life while not giving up the fight. For almost a year, my dad and mom not only went through chemo, surgery, and radiation therapy but also dealt with multiple emergency room visits and hospitalizations in between. In January, my dad lost his battle with cancer and passed away. My mom has now been cancer-free for a year but is coping with the progressive symptoms of Parkinson's disease.

Q: The term “patient-centricity” is often used to describe how biopharma companies involve patients in each element of the drug discovery/development process. How would you define “Caregiver-centricity?”

Over the past few years, I have been working on patient-centric clinical trial solutions with the key objectives of expanding patients' access to clinical research and reducing patients' burden of participation. The focus has been almost exclusively on the patient, and the caregiver was only brought up occasionally. Through undergoing the intensive caregiving period for my parents, I realized the importance of the caregiver role. For some patient populations and diseases, many patient care activities are handled by the caregivers. Some caregivers participate in making treatment decisions. The burden on the caregivers can be immense, and sometimes a patient has to rely on multiple caregivers working together as a team. Their ability to support the trial participation, in person or sometimes from a remote location like in my situation, can determine whether the patient can participate in the trial. Therefore, we have to incorporate caregiver-centricity in our solutions. Making caregivers aware of research opportunities and providing tools for convenient study information access (e.g. managing visits, medications, reporting events, and communicating with the site team) can ultimately help the industry achieve patient-centricity. 

Topics: PAC PatientCare caregivers caregiving awareness
4 min read

A Visual Connection to drive Health Insights

By Dr. Reem Yunis on Nov 18, 2020 12:22:47 PM

The future is here.

For all its terrible consequences on humanity, COVID-19 has spurred a notoriously risk-averse life sciences industry to embrace a new clinical trial model along with the remote technologies that patients and clinicians have wanted for years. Today, researchers are suddenly conducting clinical outcome assessments (COAs) remotely through applications like Medable TeleCOA. Remote eCOA solutions and other technologies that allow a decentralized clinical trial model offer the potential to upend the current research paradigm.

According to a new PPD survey, nearly half (45%) of respondents cited eCOAs as having the most positive on trial performance. Also, remote eConsent (42%), televisits (41%), home healthcare (37%), direct-to- and direct-from-patient services (35%).

Of central importance is the potential for decentralized clinical trial (DCT) models and supporting solutions to enable a level of patient care and trial access that has never been possible before. Their adoption could close the clinical trial inequality and improve diversity. Telemedicine enabled eCOA solutions can open trial participation by enabling recruitment of patients who are no longer limited by proximity to the clinical site, an obstacle for recruiting minorities and people in lower socioeconomic environments.

Ironically, it’s a simple idea – by conducting patient assessments remotely, fewer in-person visits are needed. As a result, more rural patients, busy single-parent patients, and patients with less access to health care may be more willing to participate in trials that allow for more remote interactions with site teams.

TeleCOA can also eliminate healthcare burdens such as the stress of taking time off from work and lost wages, and long commutes to and from the site. A Harvard School of Medicine study, in fact, showed that an in-person doctor visit in the U.S. takes about 121 minutes – and only 20 minutes of that time is spent face-to-face with a doctor. This amounts to 1.1 billion wasted hours each year and $25 billion in lost productivity from employed adults!

Utilizing TeleCOA in clinical trials also has the potential to better capture the natural history of disease, and enhance recruitment and retention of patients with rare diseases by offering remote access to specialized medical care. For instance, a recent study was designed to test the utilization of Televisits and TeleCOA for patients with narcolepsy, a rare underdiagnosed disorder that requires a multidisciplinary approach for its diagnosis, monitoring, and management, allowing more patients to participate in the study. Remotely managing patients at their home eliminates the need to travel to seek consultation at the scarce number of sleep centers and allows a longer monitoring period compared to the limited one conducted at sleep center visits. It also provided a better understanding of the disorder within the patients’ natural environments for collection of richer real-world data.

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TeleCOA provides an extraordinary opportunity, too, to capture longitudinal data that allows for more precise and rapid assessment to changes in health. With this data, trial teams know more about how patients are doing in real time because visits are less likely to be missed (due to travel or work issues) and assessments can be done with greater frequency. More frequent assessments also provide better insight into interim outcomes and provide patients with that gold level of care.

Take, for example, cancer patients in the COVID-19 pandemic: concerned about their safety, many avoided office visits, impacting their performance assessments and, subsequently, their treatment plans. When clinics adopted televisit models, clinicians were able to conduct TeleCOA by remotely observing their patients and asking them to complete specific physical activities live and on-screen, allowing in-home clinical assessment of patient’s physical capability and better informing clinical decisions and treatment plans.

Having their clinic visit from the comfort of their home, patients are likely to be more relaxed and, as behavioral health literature suggests, more accurate data may be captured for some measures versus in-office visits when many patients experience high blood pressure (BP) due to the anxiety from the “white-coat” effect. White coat hypertension increases the risk for cardiovascular diseases and overall mortality as was found in a metanalysis by Cohen et al (2019).

Monitoring and recording BP at home between clinic visits is also becoming an integral part of a patient’s care management plan for many chronic diseases, and it must be correctly captured. In another example of better data capture and care, if there is a concern that an Alzheimer's drug may increase or decrease BP, a patient could easily be provided with a remote BP cuff and participate in more frequent assessments than would normally be done through office visits. In a TeleCOA visit, visual assessment of the BP measurement process could be observed and if the patient is improperly conducting the measurement such as incorrectly placing the BP cuff, standing up, or crossing their legs, this could be instantly addressed and fixed. Moreover, TeleCOAs might drive early insight into previously unexpected symptoms such as dehydration prompting doctors to offer rapid advice – improving patient care and preventing patients from dropping out of a trial unnecessarily.

Additionally, it is commonly understood that patients may provide a rosier picture of their health when meeting doctors in person and are reluctant to provide more sensitive data. TeleCOAs could reduce these effects and instead allow patients to feel more comfortable providing a more realistic picture of health concerns. For example, if early data suggests an oral chemotherapy may cause sexual dysfunction, patients might provide a true accounting of this information over a televisit but not in person. Regular longitudinal critical data could also reveal these early health concerns during a time period in which they can be easily addressed rather than when the situation is either acute or entrenched and then requires significant intervention or causes the patient to drop out of the trial.

It is impossible to know what the future still holds, but one thing is for certain – remote clinical trial technologies like TeleCOA have permanently changed how research is conducted. Now, regular clinical outcome assessments can be easily done from the comfort of the patient's home, which improves the quality of data captured to expand trial participation and indications. It enables a wholesale improvement on the patient experience, real potential for increased diversity in enrollment, increased trial efficiency, fewer trial dropouts, and ultimately shorter trial timelines. It’s a win, win, win, win.

Topics: remote trials RareDisease DCT TeleCOA
2 min read

Decentralized Clinical Trials Are Already Helping to Increase Patient Access: Strategies to Right-Size Your Next Study with Flexible DCT Operating Models

By David Swanger on Nov 12, 2020 10:24:35 AM

Clinical trials are continuing their march towards greater decentralization after the industry rebounds from COVID-19. A recent study completed by Greenphire Data indicates that this rebound has been faster than expected:

As the Greenphire Data Report summarizes:

While there was a lull in new participant enrollment in the second quarter, new data from Greenphire indicates that new patient enrollment in trials that it provides services for globally has rebounded - up 85% since the trough in April, and has surpassed pre-COVID-19 participant enrollment trends (up 3% since January 2020). This trend reaches across geographic regions, with Europe experiencing a surge in new patient enrollment (up 6% since January 2020) and North America not far behind (up 5% since January 2020).

At Medable, we have seen first-hand that much of this growth in patient enrollments spurred the adoption of Decentralized Clinical Trial (DCT) methodologies. Greenphire found that of those surveyed in the report that:

 “84% of sponsors and CRO’s are actively seeking to increase the use of technology to better support decentralized trials.”

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Greenphire’s Chief Product Officer Kyle Cunningham sums it up well:

The concept of using technology to help create value in a decentralized or hybrid trial environment isn't new per se, but COVID-19 has certainly encouraged wider adoption by forcing everyone to find ways to bring greater connectivity between clinics and patients. 

At Medable, we know that to unlock the value of a decentralized clinical trial approach fully, each study team must assess their unique protocol and analyze which elements of the study schedule---whether consents, outcome assessments, or site visits---can be decentralized. Essentially it boils down to taking a flexible and modular approach.

As an industry, we need to offer greater choice, and decentralization -- in any form -- gives us the tools to do so successfully. Patients benefit from greater choice about where, when, and how they participate in a trial. Sites benefit with ways of managing trials and treating patients. Sponsors and CROs enjoy better data quality with more data capture mechanisms in real life.

Medable’s Mary Costello recently authored an article in Applied Clinical Trials, that provides tips to help research sites implement decentralized trials. It charts the course towards determining the right level of decentralization for a given indication or study, and includes these five tips:

    1. Change management: Know what to expect
    2. Technology: Know what to look for in partners and solutions
    3. Charting the course: Know what to plan and train for
    4. Guiding principle: Know that patients drive process
    5. Cycle of improvement: Know what to measure and adjust

Medable believes that a modular approach to right-sizing a DCT for each unique study will ensure a smoother transition than a one-size-fits-all approach. We start every engagement working with customers to complete oour DCT Readiness Framework to determine the right level of decentralization:

While Covid-19 fundamentally disrupted clinical trials in 2020, there is a silver lining. The groundwork has now been laid for an incremental approach to decentralization. Medable’s readiness framework and modular, integrated platform fosters a flexible approach to each study, promising brighter days ahead for investigators, sponsors, and -- most crucially -- patients.

 

 

 

3 min read

Introducing Medable TeleCOA: A New Path Forward for Patients, Sites, and Sponsors

By David Swanger on Nov 3, 2020 12:57:25 PM

Yesterday we announced the latest addition to our growing portfolio of Digital & DCT offerings: Medable TeleCOA. For full details on the announcement, you can read the press release here. 

At Medable, we’re thrilled about this latest advancement to our flexible and modular platform. Medable TeleCOA combines electronic Clinical Outcome Assessments (eCOAs) with TeleVisits on web and mobile, to enable critical clinical trial outcome capture from the comfort of a patient’s home, anywhere in the world. 

We believe in choice at Medable

That’s why our platform is designed to offer consumer level experiences for patients to engage with their Study team on site or remotely, within the framework enabled for the clinical trial. We offer our apps through multiple modalities, accessible via web browsers. Core to the Medable design philosophy is that patients and site staff should use the same devices that they engage in other daily activities such as shopping and social connections.  

In practical terms this means that a multi-country BYOD approach for the decentralized elements of a trial is now possible. For example, a patient in India could use her personal mobile phone (IOS or Android) and connect directly to the Medable Patient app. When connecting to the app she will initiate a TeleVisit with a Healthcare Professional (HCP) hundreds of miles away. During the TeleVisit, the patient and HCP can complete consents and outcome assessments side by side within the TeleVisit in the same application. Medable TeleCOA is designed with patients and healthcare professionals at clinical sites in mind 

Just as a patient can access Medable TeleCOA on virtually any device with a web browser, so too can a HCP. The means HCP’s can more flexibly incorporate Medabe TeleCOA into the existing collection of systems, workflows, tablets and computers that are currently in use at their site - or even with their own mobile or desktop devices away from the site.

For clinical trials to continue their evolution towards greater digitization and decentralization after the industry rebounds from COVID-19, they must not only be well integrated with the systems that sites and sponsors use regularly but they must also be built with flexibility and modularity in mind. 

The Medable enterprise cloud platform is architected to enable study data to be securely collected, integrated, and harmonized from a variety of devices and sources

Whilst many very well designed point systems exist today, being able to sequence modules together into a seamless and flexible workflow is transformative to the ease and consistency of the user experience. This is really key as it is imperative that digitization reduces and eliminates administrative burden, streamlining our working practices and doesn't just push it down the line to the sites and patients.

Similarly, in this age of data security and privacy awareness, the robust platform enables all the user interface systems to be controlled and managed to collective standards. It’s what allows our partners to securely deploy TeleVisit, TeleConsent, and TeleCOA across a variety of devices in the knowledge that the entire platform conforms to the highest levels of global data privacy and security practices. And in practical terms it means that we’ll work hard to evolve our platform so that it will continue to assist sites and sponsors to better serve the patients that are so critical to the continuing the life-saving advancements of our industry. 

A Site and CRO Perspective

Dr. Robert Bissonnette, MD, FRCPC, founder and CEO of Innovaderm, a leading dermatology CRO, eloquently sums up the potential of the Medable platform to power a variety of digital and decentralized interactions:

“For more than 20 years we have dedicated our efforts at Innovaderm to advancing dermatology research. As the leader of a specialty CRO and as an investigator who has focused exclusively on dermatology, I have seen firsthand how patients and site staff can benefit from decentralized clinical trial methodologies. Medable’s flexible and modular platform means that we can deploy their platform to shift a number of scheduled visits from onsite to remote while still maintaining the patient/investigator relationship that is core to every clinical study.”

Medable is hosting a panel webcast that includes Dr. Robert Bissonnette, Patient Advocate Richie Kahn, and Dr. Michelle Longmire on Wednesday November 18 that will explore how Medable TeleCOA was designed in conjunction and for the benefit of patients, sites, CRO’s, and sponsors: “eCOA in a Decentralized World: Enable Remote Collection of Outcomes to Improve Study Access and Engagement.”


Register for the webcast here: https://www.medable.com/telecoa-webinar

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Topics: DCT TeleCOA
2 min read

Why I joined Medable: Dark arts and shining lights!

By Gillian Livock on Oct 20, 2020 6:11:17 PM

Way back in the day, I remember a world when only paper CRFs existed and the transformation to EDC rapidly changed the way we managed trials.  Fast forward over 20 years and we are now experiencing another paradigm shift, moving from focusing on the site to putting the patient at the center of everything we do and leveraging digital technologies to remove the burden of participating in research.

Being part of a team, culture and organisation that drives innovation, bringing flexibility and choices to our industry, makes the weeks go fast! Never have I been so excited to explore each new day and see how we can help our sponsors and partners make a difference to their digital strategies. Bringing research to patients and the chance to deliver effective therapies faster to patients is a passion that everyone at Medable lives by.

Now the next exciting opportunity is how do we take these advancements in technology and acquisition of data to identify digital therapeutics. The interoperability of the Medable platform means that outside data streams can enrich our existing modules to bring more insights. We don't know what we don't know. However, applying data science and machine learning to these data insights can identify trends and outliers which can help us look at patient populations differently and in more depth. 

Imagine a day when patients can be digitally assessed to match the best treatment that they will respond to based on how their individual physiology is impacted by a disease.  We have made exciting advances in treating patients and finding new ways to tackle life threatening illness, however in many cases patients are still treated based on the overall response of how others have progressed and responded. I am excited to be part of a company who can contribute significantly to this next wave of advancement in finding digital therapeutics for patients. 

Certainly for me, technology is part of how I function in my everyday life. My parents are now experts in online shopping and accessing their grandchildren through technology….arguably the pandemic has provided the biggest learning opportunity of our lifetime!  Where technology may once have been seen as a “dark art” by certain generations, it is now their way of life and opportunity to connect to a world which has become smaller. Medable is helping patients, sites and study teams stay connected in times of darkness and shining a light to deliver a new way of conducting research.

Medable is rapidly growing and we’re looking for passionate teammates to join our mission across every part of our company. If you’re interested in joining us, please visit www.medable.com/careers.

Topics: decentralized trials careers DCT
3 min read

Luck Fupus: Life with lupus is not a cakewalk

By Jennifer McNary on Oct 19, 2020 7:04:42 PM

One of the most exciting parts about being a patient advocate is the chance to connect with fellow advocates. MarlaJan Wexler, a well-known lupus advocate and superhero, was kind enough to virtually sit down with us to share her story.

In the beginning….

Though lupus is often called an invisible disease, 39 year old pediatric cardiology nurse MarlaJan Wexler is an incredibly visible Lupus advocate. 

She was diagnosed with Lupus in 2008 at the age of 27 though she suspects her symptoms began much earlier. She went to her PCP for occasional joint pain, rashes and fatigue, but chalked them up to being a hard-charging nursing student who also worked full-time and partied a bit too much (hello, early twenties!); didn’t eat healthy; or sleep enough. 

It wasn’t until after MarlaJan’s honeymoon that it became apparent too much sun and fun had kicked off what turned out to be her first Lupus flare. UV light can trigger disease activity, and she became very sick mere weeks after returning from her honeymoon.

“My body literally rejected my marriage,” MarlaJan laughs as she describes her divorce foreshadowing the future. Unfortunately, after this initial flare and eventually being diagnosed, she realized Lupus was a real disease and she needed to take it seriously. 

Becoming an advocate 

The first time MarlaJan felt a sense of connection to another patient with Lupus was when a woman read her blog online and reached out from across the ocean. She had decided to put herself out there in hopes people would read her story. 

“Everything I was putting out there was how she felt and she just thanked me. Even if that was just one person, it made a difference and made her feel like she wasn’t the only person facing this.“ MarlaJan says after this, everything snowballed. She created various social media pages and founded her advocacy organization, Luck Fupus to make sure there was reputable data from reliable sources for those with Lupus

In 2014, MarlaJan won the WEGO Health Hilarious Health Advocate award, ultimately finding her place on stage sharing her story and in the broader patient community. “I felt completely isolated my whole life…and then (through this award process) I’ve connected with so many people with a smorgasbord of various diseases.” She went on to say, "As much as being sick sucks, it took me down this path that I never knew existed and that I never would have thought for myself."

 

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MarlaJan’s Mission

Now that MarlaJan is firmly entrenched in the advocacy space, she uses her platform to connect with other patients. She’s especially passionate about the need for consideration of women's reproductive choices before starting on a therapy that may limit the ability to have children safely in the future. When a patient is first diagnosed, she recommends all patients prescribed immunosuppressive therapy talk with their care team about their fertility risks and options. Similarly, MarlaJan is passionate about ensuring that all patients have access to relevant and reputable sources of medical information so they can make better informed choices about their care. 

MarlaJan’s Call to Action

In today's climate where several Lupus medications are being viewed as potential therapies for COVID, MarlaJan knows all too well that some patients are dealing with an additional, unforeseen complication: limited access to the lifesaving medications they so often depend upon. “Regardless of the fact that hydroxychloroquine has been found to not be beneficial in the treatment of COVID, many [Lupus patients] people are still having so much trouble getting their medications.” 

Access issues aside, MarlaJan also wants the broader community to know that more voices are needed in the Lupus community. “There are 1.5 million Americans living with it [Lupus] and over 5 million people in the world living with it. It’s not a rare disease. We need more voices.” 

At this point in the interview, one of MarlaJan’s two cats, Professor Puddin’ Pop, made an unexpected cameo. We would be remiss if we neglected to mention the earlier cameo by MarlaJan’s other cat, Dunkin Nonuts, a charming and spirited tabby. 

If, like us, you can’t get enough of MarlaJan’s story and message, check out her blog, https://www.luckfupus.com/ 

For more information about Lupus and resources for those living with Lupus, check out these links;

Topics: LupusAwareness
3 min read

Why I Joined Medable: a New Generation of Leaders

By Mary Costello on Oct 15, 2020 11:54:08 AM

Like many of you, I was saddened to learn of Ruth Bader Ginsberg’s passing a couple weeks ago. She served as an icon and inspiration to many. I think women who benefited from her work and her decisions, the loss felt almost personal. For me, spending most of my career as a single mother working in a very fast paced industry, I often felt as though in order to succeed professionally, I needed to minimize the impact of balancing motherhood with the demands of work.   

Times are changing and traditional roles are evolving. Now many fathers are sharing the responsibilities of children. But when I was coming up through the ranks of the workforce, that evolution had not yet taken place.

In my 30 plus years of working, every single leader of every single company for which I worked were men. I’ve had the good fortune to work with inspirational company heads and to learn from their styles and their leadership and vision.  

Today I find myself at Medable, and it’s the first time I have worked for a female CEO. So, is there a difference?  

My experience has been similar to those of many others. A recent article in Newsweek, focusing on the difference in responses between female led countries versus male led countries amidst COVID noted that "world leaders who are women have been more likely to use language focusing on compassion and social cohesion”. Forbes published an article last year examining the different ways genders are socialized and found that “women excel in leadership positions because they have developed soft skills necessary for effective leadership...traits like empathy, communication and listening". In 2019, HBR  evaluated 360 degree reviews and found that "women in leadership positions" were perceived just as - if not more - competent as their male counterparts". Yet the percentage of female CEOs of public companies still does not reflect the population.  

There is some evidence of progress. A woman just assumed the CEO position for the BNSF - the first female to hold the leadership position for a railroad. GlaxoSmithKline, one of the largest pharmaceutical companies in the world, is led by a woman.  

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So what's been the difference for me, a woman in her late 50s, arguably closer to the final years of my career than I am to its beginning? Medable's culture is shaped by our leader, Michelle Longmire, a physician and founder who believes in the power of passion and drive.  But she also believes strongly in the power of people and connection and works to connect with her teams and collaborators in a very personal way.  

When I was contemplating leaving my former position to join Medable, I asked one of my colleagues for her thoughts on the best thing about Medable and her instant reply was "Michelle. She's inspirational". Despite the stereotype, women want to see one another succeed and it is inspiring to see how our CEO has taken an industry problem as a challenge and an opportunity and built a company around solving those problems. Her skills as a listener mean that the team is drawn to her and willing to share ideas; her openness to multiple perspectives feels different than what I've experienced at other organizations.

We mourn the passing of Justice Ginsberg – a petite, soft voiced judge who will be remembered partly for her belief that "you can disagree without being disagreeable."  Justice Ginsberg showed us that leaning into our traditionally female qualities wasn't limiting and may in fact be liberating.  I appreciate all she did for our country and, in particular, the women in this country.  Equally today I appreciate the opportunity to experience a new generation of female leadership.  

Thanks Michelle and the Medable team – it’s been a great journey so far!

References:

3 min read

Why I Joined Medable: Putting Patients (and Clinicians) at the Center of the Trial Experience

By David Swanger on Oct 9, 2020 1:36:26 PM

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Clinical Development rapidly advanced this past decade. New endpoints and measures covering genomics and devices emerged. New design methodologies like adaptive trials and basket trials gained acceptance. And new advances in clinical operations in areas like risk based monitoring improved safety and efficacy.

In spite of substantial progress, several challenges remain. Today patient access and patient retention persist as the costliest and most time consuming challenges our industry faces. We’re all familiar with the statistics and persistent challenges they underscore. A relatively recent analysis of ClinicalTrials.gov data, “Terminated Trials in the ClinicalTrials.gov Results Database: Evaluation of Availability of Primary Outcome Data and Reasons for Termination” underscores the challenges surrounding patient participation in clinical trials:

The analysis finds that of the studies that were terminated in the ClinicalTrials.gov database, 68% were done so due to reasons other than data accrual, and of that cohort, 57% were terminated due to an insufficient rate of accrual.

Patient enrollment and retention continue to challenge our industry, and that was prior to our global pandemic. And with precision medicine leading to ever narrower treatments and patient populations, these twin challenges will likely persist.

Improving on these challenges will require a rethink across the clinical development continuum that prioritizes patient experience at every step in the process. Ultimately the sponsors and CRO's that prioritize the patient's experience throughout the clinical development lifecycle will benefit from faster FPI to LPO cycles and a higher proportion of studies with sufficient data accrual to complete the study.

What will putting the patient at the center of the clindev experience look like?

If you ask me, it looks a lot like Medable's Patient, Site, and Sponsor apps.

Each has been developed in close partnership with some of the leading patient advocates, doctors, researchers, and clinicians across clinical development. And with every standardized offering or workflow, Medable has stopped to ask the question, "Does this enhance or hinder the patient (and clinician) experience?"

The result is a fundamentally new approach to clinical development, one that utilizes best practices from Medable’s deep learnings in hybrid and decentralized clinical trials (DCT's). These learnings are then complemented by a commitment to delivering the same consumer-grade experience that delights patients and clinicians when they use their favorite apps like Netflix or AirBnB.

In practice this means that we strive to allow sponsors and CRO’s to right-size their journey to DCT’s by offering a Readiness Framework that clearly shows the progression from light-weight TeleVisit and TeleConsent capabilities to capturing digital endpoints via COA’s, PRO’s, and other forms of digital source data.

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At Medable we recognize that each study is unique. Each covers a different treatment and operates in a unique combination of phase, therapeutic area, regulatory environment, and much more. That means that every Decentralized Clinical Trial (DCT) must fit the unique needs of the patients, investigators, clinical, and regulatory professionals and ensure clinical trials continue to deliver the quality our patients deserve and regulators require.

That’s why I’m so excited to be part of a team of diverse clinical leaders who have the experience, perspective, and passion to help the clinical development process to evolve in a meaningfully positive way. And it starts with our CEO Michelle Longmire, a true visionary who leads by example and is steadfastly committed to ensuring we succeed as a team.

Getting the chance to shape a fundamentally new approach to something as important as drug discovery and commercialization is quite rare. And for that I am grateful to be part of an amazing company that is laser focused on solving the twin challenges of patient access and recruitment. It’s why I joined Medable.

If you'd like to join Medable and help shape the future of the growing DCT movement, we invite you to join us. We are hiring (or will be soon) in just about every department and every geography. I’m looking for strong marketers in product marketing, demand gen/growth, brand/creative, and more. Feel free to apply at www.medable.com/careers.

Topics: careers
6 min read

No One Should Walk a Cancer Journey Alone/Lessons from My Father

By Joan Venticinque, PAC member on Oct 7, 2020 4:39:35 PM

In 2020, over 276,000 American women and 2,600 men will be newly diagnosed with breast cancer. For these individuals, Breast Cancer Awareness month is especially poignant. Those hearing  the words ‘you have cancer’ quickly realize the importance of having a support network as they move through diagnosis, treatment, and survivorship.

PAC Breast Cancer Awareness blogI had the pleasure of interviewing Dolores Moorehead who has supported cancer patients for over 30 years. Dolores Moorehead, BS, MS, APCC, is the Lead Client Navigator/Multicultural Client Support Clinician, at the Women’s Cancer Resource Center (WCRC) in Berkeley, CA. The WCRC offers free services to people diagnosed with cancer, with a focus on those who are low-income, people of color, and members of the queer/trans community. Among her many roles, Dolores facilitates the Sister to Sister support group for African American/Black women diagnosed with cancer. Prior to joining WCRC in 2002, Dolores worked as the Patient Services Manager at the American Cancer Society for more than thirteen years. 

 

     Dolores, what drove you to work in cancer support?

     Dolores:  I can say that one of the things that really drove me to work in this field was the         fact that my father was diagnosed with Hodgkin's lymphoma when I was 14, and culturally       my younger sister and I were not allowed to tell anyone. Also, as a teenager I was afraid         that my father would die and I did not have a place to talk about it. When I started working       in cancer support, I realized how important it was for patients to never walk a cancer                 journey alone and that they needed to have someone with whom to share their story. I am      dedicated to do my best to provide that attentive ear with the resources, help and support        that patients need.

 

     How do you approach supporting the women in your community?

     Dolores: When I chose to work in mental health, I realized it was important to look at the           cultural relationships of my clients because relationships are how many people of color             build trust. It is also important that I look like them and let them know that mental health is       important. To truly support someone, I look at all aspects of someone’s identity, not just           race or gender. It is their religious or spiritual beliefs, their educational background,                  disabilities, visible or not, and what has impacted them in life. I strive to see that person for      who they really are. 

 

    “I think it's important to admit that they are the experts of their life,        you aren’t, so asking rather than assuming, and also                              acknowledging if they say something and you don't really                      understand what it means, be big enough to say, ‘I haven’t heard          that before, can you explain it to me’. This is a way to build trust”

 

     In October we seem inundated with pink ribbons. How do women in your community                 who’ve had an experience with breast cancer feel about Breast Cancer Awareness Month?

     Dolores: From my experience, African American survivors have really connected with pink       ribbons and breast cancer awareness month and that’s good. For me, I consider how I             utilize that month to educate people about breast cancer. I remind them that breast cancer       occurs throughout the whole year and that while it gets highlighted in October, it's                     important to take the message of awareness and continue it year-round. 

 

     The Covid-19 pandemic has exposed existing racial disparities in health care. With your           experience in navigation and supporting patients, what steps do you see that need to be         taken in order to increase participation of people of color in clinical trials?

     Dolores:  While there is more awareness now about the importance of getting individuals         of color to participate in clinical trials, recruitment really hasn’t changed. One of the things       that I've really thought about is how do you bring individuals that you want from the                   community on board? How do you make them feel comfortable? How do you let them               know their words and thoughts are important? You can do this by mentoring community           members, tell them you understand this is new to them, yet, YOU make them feel                     important because they are. That will make an impact. 

     These community members can go out to their faith-based communities, sororities, and           other community venues and teach others about cancer research. People learn about             research and clinical trials from a trusted  individual who is a part of their group and looks         like the people they are serving. It all comes down to building trust.

 

    “I think it's important for researchers to know their community and        then to pull the gatekeepers from that community so people will            realize, oh, she is like me and I can trust what she's saying.” 

 

      You have to do the groundwork first. It’s important for researchers to be willing to come to        the community and be willing to hear those hard ‘Whys’. Why are you here? Why do you          want to do a study on us? It is really empowering to an individual to be able to ask the              questions. For communities of color who have not felt empowered, it’s important to let              them know they have the right to ask questions and receive answers. Then when the                study is over, the most important step for researchers is to return and share the                        information, and what is going to happen next. 

 

    “I really think that it is important for the researcher to acknowledge         the history of clinical trials in people of color, for example, the              Tuskegee Airmen study and Henrietta Lacks, and explain that this        will not happen again, and this is how they are different.” 

 

     One thing that’s true whether I am supporting women with breast cancer or talking to folks       who may be clinical trial candidates, is that we have to meet them where they are and we         have to be quiet, sit back, have humility and listen even when it becomes uncomfortable. It       is only then we can understand the community and build trust.

 

I want to thank Dolores for taking her time for this interview and for her tireless work in supporting women with breast cancer. Breast Cancer Awareness month is more than just cancer awareness especially for people of color. As Dolores has stated, it is understanding people’s cultures, beliefs, relationships and history. It is listening to their stories, answering their questions and above all empowering them to be partners not only in their healthcare but as important contributors to clinical trials.

 

This interview has been edited and condensed for clarity.

Topics: Breast cancer awareness
2 min read

Decentralized Clinical Trials (aka Hybrid Trials) Are Here to Stay

By David Swanger on Oct 1, 2020 12:33:44 PM

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Yesterday Clinical Research News published a noteworthy article titled: ‘Hybrid’ Trials Will Be Post-Pandemic Norm. The piece provides a solid overview of the state of the Decentralized Clinical Trials (DCT’s) in the “new normal” clinical development leaders find themselves in. Not surprisingly, a recent survey by GlobalData confirms what Medable has observed internally, with 67% of survey participants indicating that they plan to use decentralized clinical trials in the future.

At Medable we’ve seen this shift first hand. In the article, Medable’s Ali Holland confirms that:

          “The shift toward decentralization has already begun, with protocols more often                          designed from the get-go to give participants the option of starting at home for many                of their required study visits rather than traveling to a trial site.”

A key distinction of DCT’s relative to Virtual trials is that DCT’s operate on the hybrid-trial continuum. Whereas fully virtual trials can at times disinter-mediate investigators from patients, DCT’s aim to provide existing patients and investigators with the technologies, workflows, and processes that provide a flexible approach for every sponsor or CRO’s unique study needs. Essential to the success of a DCT or hybrid trial strategy is right-sizing the approach for each study’s unique mix of patients, investigators, lab and clinical practitioners.

Medable allows sponsors and CRO’s to right-size their journey to DCT’s by offering a Readiness Framework that clearly shows the progression from light-weight TeleVisit and TeleConsent capabilities to capturing digital endpoints via COA’s, PRO’s, and other forms of digital source data.

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As Ali Holland puts it in the article:

        “Different studies need different capabilities and Medable’s technology flexibility means             we can meet the unique needs of each study and enable the flow of data consistently               between [any] ecosystem of partners.”

At Medable, our ecosystem does not only include CRO’s, patient recruiting services, device makers, lab networks, and more, but it also includes site and patients. Medable’s Patient Advisory Council ensures that every experience embedded within the Medable DCT platform is assessed and improved by patient advocates. As the article summarizes and Medable’s Rasmus Hogreffe confirms:

“Independent patient advocates on the PAC, meanwhile, will be reviewing design   features of Medable’s technology and proposed study workflows for opportunities to   relieve participation burdens and, as Rasmus puts it, “minimize the gap between theory   and practise.” Incorporating the patient perspective in this way has accelerated patient   recruitment and resulted in “extremely high” patient satisfaction for recent studies.”

If you’d like to learn more about Medable and it’s new TeleConsent offering that makes fully remote informed consents (and reconsents) a reality, sign up for our webcast on Oct 6

 

Topics: decentralized trials Hybrid Trials
6 min read

Lessons from a Pandemic: Quarantine is a Gift and Medical Testing is Love

By Jennifer McNary on Sep 29, 2020 5:21:37 PM

SMA Awareness

Ask just about anyone across the globe what was the worst thing to happen to them in 2020 and they will concede it was Covid-19. Medable Patient Advisory Council member Khrystal Davis doesn’t see things the way most of us do. For her and her family, the Covid-forced quarantine means more time together with her husband and five children. 

Khrystal learned, startlingly, the value of time with family in 2011 when her youngest baby was diagnosed with Type 1 spinal muscular atrophy (SMA), a genetic neuromuscular disease characterized by weakness of the skeletal and respiratory muscles. SMA is a rare disorder occurring approximately one in 11,000 births, and about eight million Americans are genetic carriers. It robs children of strength by affecting the motor nerve cells in the spinal cord, and impacting their ability to walk, eat, and breathe. SMA is the leading genetic cause of death for babies.

At the time, there were no approved treatments for SMA. 

“We were shocked,” said Khrystal. “We went home with our eight-week-old son and cried all night. We let it all out, and grieved. But then we decided we were going to make the most of our time with Hunter so we took him to the zoo the very next day.”

The Davis’s did not fall apart (at least, not for long) and did not accept defeat. “The doctors told us that Hunter would lose his ability to move, swallow, and eventually, breathe. It was gut-wrenching but we decided to pack as much into his remaining time with us as we could,” added Khrystal.

Khrystal and her husband, Curtis, chose hope rather than despair and – incredibly – got to work.

“Curtis connected with an SMA researcher who had a compound that had gone through preclinical studies but it was shelved,” explained Khrystal. “We acquired the chemistry for that compound and hired a company to manufacture it. Next, we went to Mexico because Hunter didn’t have time for the regulatory rigor of the FDA. His disease was breaking down motor neurons every day.”

Eight weeks after Hunter’s diagnosis, he received his first treatment and became the first SMA patient in the world to ever be treated. 

At the time, a similar treatment was in clinical trials in the U.S. but it was unavailable to Hunter. Thanks to the 21st Century Cures Act and the FDA’s increasing regulatory flexibility with n of 1 trials, it’s becoming increasingly easier to treat patients like Hunter fast. This wasn’t possible nine years ago. 

The Davis’ flew from St. Louis to Mexico every six weeks for five years until Hunter crossed over onto an expanded access program (EAP) for a similar treatment in the U.S. shortly before its approval. Hunter responded extremely well to his treatments in Mexico and grew strong, crawling on his belly, sitting up unassisted, and was just about to start therapy for walking in a harness for the first time. Suddenly, Hunter came down with pneumonia and that would prove to be a lasting setback.

Hunter survived but the damage caused was too severe for his motor neurons to ever fully recover. 

“He was two years old and we had such hope but we would never realize the same gains that we had from the treatment as we did those first 24 months,” Khrystal said. “This is why I am now such a passionate advocate of pre-symptomatic diagnosis and treatment. If not treated right away, patients are unable to rebuild the motor neurons as they age. Once they are gone, they are gone forever.”

Today, at the age of nine, Khrystal says that Hunter is the happiest of her five children. And, as promised when he was first diagnosed, the Davis’s have packed as much living as possible into Hunter’s life, taking him all over the world including China, London, Canada, Mexico, Spain, and Portugal. He still loves to see the animals at the zoo, and – of course – is a big fan of Disney. 

Hunter is wheelchair-bound and cannot talk very easily but communicates using a device he controls by looking at a screen that ‘talks’ for him. “It was life-changing, though it has taken some time to learn how to use it,” smiled Khrystal. “Even so, he has outpaced his mother in mastering the device!”

Khrystal is now one of Texas’s leading advocates for SMA specifically and rare disease more generally. “What we learned from Hunter is that treatment approvals and research was not progressing fast enough so I reached out to a grassroots organization called Families for the Acceleration of SMA Treatments and we secured a landmark meeting with the FDA in Washington DC about the only SMA treatment in clinical trials in the U.S. called SPINRAZA. We had four requests.”

The FDA responded with overwhelming support and took fast, decisive action. By July 2016, the FDA announced that the current treatment met the primary endpoint so the placebo trial for Type 1 babies stopped and all babies crossed over on to the effective treatment. In August, the FDA and Biogen worked to launch an EAP for the treatment for Type 1 SMA patients – consisting of more than 65% of all SMA patients – under age 18 across all 12 trial sites.

On December 23, 2016, Khrystal and all SMA patients received the greatest Christmas gift: FDA approval of SPINRAZA. It was the fastest drug approval in the history of the FDA.

“Patients and their caregivers finally had new hope, so we wanted to get the information out quickly,” said Khrystal. “Various organizations worked together to educate top neurologists and spread the word through educational campaigns, social media, and various patient groups and communities.”

They thought their work was done when the FDA approved SPINRAZA but that was not the case. “We quickly learned that FDA approval doesn’t guarantee access.” FDA approval means that patients can take an approved treatment, but it doesn’t mean payers will cover it. 

Khrystal and the rest of the group have also challenged payers’ to cover SPINRAZA for SMA patients pursuant to the broad FDA label. Collaboration with regulators, researchers, doctors, biotech, and advocacy groups, she said, is crucial.  “We have witnessed the research industry and FDA come together, and the overwhelming collaborative effort that it takes to get a treatment to patients. It’s the only way we will move forward on any of our goals,” Khrystal explained.

Newborn Screening Crucial for Rare Pediatric Disease Treatment

August was SMA awareness month, and September is newborn screening awareness month. “It’s perfect that newborn screening month follows SMA awareness month. SMA is the leading genetic cause of infant mortality when left untreated. It’s a great example for the need of newborn screening.” 

Khrystal, founder and president of the Texas Rare Alliance, now works tirelessly to advocate for newborn screenings for rare diseases. “We’re revamping Texas’s newborn screen program, not just for SMA but for all rare conditions as they are included on the Recommended Uniform Screening Panel (RUSP). This is crucial to affording access to pre-symptomatic treatment and to ensure the least permanent damage is done by any of these diseases,” she added.

Through her tenacity and endless persistence, Khrystal and the Texas Rare Alliance helped secure legislation that now provides the necessary funding for newborn screening of all new conditions in Texas so doctors can automatically include a condition that raises the cost of the newborn screening and still test for it. 

“Texas is nearly RUSP-compliant, which is so vital to helping prevent the onslaught of symptoms for rare diseases on babies. It needs to be done nationwide. The rare disease community needs to come together and make all states recognize how this small step can mean all the difference,” Khrystal said. “We’re not done yet. We want to make sure Texas is RUSP-compliant, and educate our Senators, Representatives, and their health policy staff on the need for an implementation requirement. Max Bronstein helped secure an implementation requirement in California in 2016, making it the first RUSP-compliant state.” 

Khrystal now serves on the Texas Newborn Screening Advisory Committee. She also regularly speaks and advocates on the importance of strengthening newborn screening programs. Her ultimate goal is to work toward securing newborn whole genome sequencing which she believes affords an opportunity to democratize the diagnosis and eliminate the diagnostic odyssey for most rare diseases. “An important step is to provide whole genome sequencing to critically-ill NICU and PICU patients with unknown etiologies.”

Today, thanks to the relentless work and courage of so many SMA patients, advocates, doctors, and researchers, we have three FDA-approved treatments for SMA. “We had nothing less than a decade ago,” said Khrystal, “now there are three options. That’s what I want for the entire rare disease community where only 5% has even a single approved treatment.”

Khrystal is focusing her advocacy on newborn screening and the pursuit for newborn whole genome sequencing. “I believe newborn screening is crucial to treating rare pediatric conditions. Babies can be tested shortly after birth and is the key for many early-onset conditions.” 

As the Davis family learned from this gut-wrenching journey, nothing good comes from anger. They continue to struggle with physical, mental, and spiritual challenges that most cannot imagine but they haven’t stopped living as full of a life with Hunter as possible. They push forward. They choose hope. It’s not surprising, then, that as the rest of the world bemoans the temporary inconveniences thrust upon us by the pandemic, the Davis’ see good.

 

# # #

Hunter Davis is one of many patients living with rare diseases, many of which have no known cure or treatments. Medable will continue to highlight stories of patients and caregivers like the Davis family, as we encourage the industry to invest resources into rare disease research. Please follow our Patient Advisory Council via blog and LinkedIn for regular updates.

Topics: SMAawareness
3 min read

Insights on Decentralized Trial Execution: An interview with CEO Michelle Longmire in Applied Clinical Trials

By David Swanger on Sep 25, 2020 3:17:21 PM

Last week our CEO Dr. Michelle Longmire sat down with Moe Alsumidaie from Applied Clinical Trials for an interview where they discussed how decentralized clinical trial (DCT) methodologies will transform how clinical trials are executed for years to come. You can read the full interview here on Applied Clinical Trials.

Michelle Longmire
 
 

In the interview Mr. Alsumidaie and Dr. Longmire discuss several important insights and considerations including:

  • An overview of the current DCT landscape
  • How COVID-19 has impacted DCT’s
  • Keys to ensure a DCT is successful
  • Results from DCT implementations
  • How clinical trials will evolve in the future

During the interview, Dr. Longmire highlighted how DCT’s tackle four core challenges that clinical trials have historically faced:

    “The first is patient access. Virtual screening and remote eConsent can dramatically improve access to clinical trials. We’re seeing that these decentralized processes consistently enhance patient recruitment, and we’re developing a baseline dataset for comparison.”

    “The second challenge DCTs help overcome is patient retention. Compared to traditional methods, we see significant improvements when we’re able to reduce site visits that are more focused on basics such as screening, enrollment, consent, and non-invasive evidence and data capture.”

     “DCTs also help overcome issues with data quality, as you mentioned. At a minimum, we want to be meeting the standard for data quality. And in many settings and across various therapeutic areas, we can do that remotely. At Medable, we are starting to think about digital endpoints as a complement to traditional scales and measures. DCTs help with the development of new digital endpoints that are of the same, or better, quality than conventional endpoints. Digital endpoints also provide the opportunity to dial into more disease-based measures for diagnosis and severity.”

    “The fourth main challenge DCTs tackle is efficiency. Traditional clinical trials take much time and come with very discrete processes, as different teams generally do recruitment and consent and evidence generation with diverse strategies and varying technologies. In contrast, we provide a process solution versus a point solution with one interface that drives streamlined workflows and provides seamless decentralized capabilities. This improves overall trial execution efficiency.”

At Medable we are continuing to develop new offerings to address these challenges in a modular and flexible way. Our Decentralized Trial Readiness Framework makes it easy to right size your onramp to a DCT for either an in-flight study or a new one.

During the interview, Dr. Longmire also touched on the importance of shifting physical consents and re-consents to a decentralized approach utilizing technologies to enable the Investigator & patient relationship to be replicated remotely :

     “Consent is another ideal opportunity for decentralization, which also improves the patient experience. There’s a lot that can be managed remotely, and some actions that are better handled in person – like an invasive procedure. There is a push for some of those activities to be conducted in the home, but as a physician myself, I still see great benefit to having the full suite of access that a hospital affords.”

This week we introduced Medable TeleConsent, a new offering that makes fully remote informed consents and re-consents a reality for both patients and investigators. Doing so will potentially save thousands of hours of investigators and patients meeting physically and reducing their exposure risk during the COVID-19 pandemic. To learn more about Medable TeleConsent, sign up for our DCT Panel Webcast featuring Medable’s Ali Holland and Rasmus Hogreff along with Craig Lipset from Clinical Innovation Partners on Oct 6.

Topics: decentralized trials Teleconsent
7 min read

TeleConsent: A new chapter in the decentralized clinical trial approach

By Allison Holland- Head of Decentralized and Remote Trials on Sep 23, 2020 2:11:53 PM

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognizing that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy, TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

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Executive Summary

Informed consent is an essential process in clinical trial conduct that enables a qualified health professional to impart balanced information to potential participants about the benefits, risks and expectations of a clinical trial. eConsent, the electronic acquisition of a clinical trial participant’s signature that confirms the understanding and agreement to the information shared, offers a way to streamline that process and allows for better tracking, particularly if protocol changes should occur during the trial period. eConsent has the potential to reduce the bottlenecks, effort burden, and the expense of travel that in-person clinic visits place on future participants. Current restrictions issued by governing bodies in European  nations restrict wholesale adoption of eConsent, but there is broad support to provide trial participants with more user-friendly and better information before, during and after consenting. TeleConsent takes the process a step further by allowing the participant and clinical study staff to be located in different geographies while participating in and obtaining informed consent. This method effectively removes the burden of manual paperwork and facilitates the further decentralization of clinical trials.

The ideal clinical trial generates a minimal amount of credible, replicable, and evaluable data needed to answer meaningful questions with the least time and cost burden on participants,” said Donna Cryer, president and CEO of the Global Liver Institute. It is imperative that we can provide potential trial participants with clear information to outline the trial risks, benefits, schedule and data framework to enable them to make an informed choice about their healthcare decisions. As clinical trials have become more complex and more information is needed to be shared with participants, it is not unusual today for an Informed Consent Form (ICF) to be over 60 pages long, and this alone presents challenges for ensuring knowledge transfer and comprehension for participants. One of the further benefits of a digital consenting solution is the ability to present the approved trial information (ICF) in more digestible formats, with chapters, supporting information in hyperlinks, video files, animations and other media options that offer greater ability for a participant to review, digest, and comprehend the entire information shared. Having this information available to consider from the comfort of home, with time to discuss with family members/caregivers can significantly benefit a participant’s understanding and motivation. An important component of the traditional face-to-face consenting process is the ability of the participant to actively question and discuss the ICF information with a qualified professional, and it is vital that this opportunity is also available in a digital environment too. Enabling active discussion of the ICF via teleconference is a valuable and important step in ensuring a fully informed process is achieved.

As the informed consenting process increases in complexity for global, multi-site studies, requiring localization of language for patient facing materials and accommodating multiple version controls with local country and site approved document versions, the reliance on manual tracking becomes even more burdensome. The facilitation of centralized overview tracking through a digital platform offers immediate efficiencies and quality benefits, as well as providing options for automation of workflow management to improve compliance and visibility. Aspects like tracking, maintaining, and understanding where patients/sites are in the consenting/reconsenting process are challenging to monitor and can increase the risk of non-compliance if not accurately monitored. Ultimately, it can cause a significant delay in the trial and jeopardize regulatory compliance and data integrity; if a participant’s ICF is not captured in a compliant manner, the data may be removed from the study, which can result in an underpowered study. 

As the industry progresses towards remote and hybrid trials, sponsors need to capture evidence and data of the patient performing fully informed consent outside of the traditional site environment, while standardizing processes to generate the evidence for submission. We have an opportunity to improve the participant experience of learning and understanding about the clinical trial and setting patients up for a high value, high satisfaction overall study experience. 

The idea of conducting “site-less,” virtual, hybrid or decentralized clinical trials has been around for some time. TeleConsent can, and will, reduce barriers and enhance the clinical trial experience for patients and sites while improving participants’ convenience, resulting in increased patient motivation and engagement. Like any innovative approach, the rethinking of clinical trial execution has often been met with skepticism or reluctance; however, due to the recent COVID-19 pandemic, the pharmaceutical industry has begun to recognize and accelerate the various possibilities and benefits of a decentralized approach.

 

About eConsent 

The primary intent of Informed eConsent is to protect the patient’s safety; therefore, safety, data quality, and the need for scientific rigor should be clearly articulated. In an article from Clinical Leader, experts at Otsuka Pharmaceuticals commented that “A better patient experience leads to a more informed subject and engaged subject – a more adherent subject that may help improve retention rates for patients to complete the study. This could lead to faster trial timelines.” The full article can be found here

Complex language and lengthy explanatory information make it increasingly difficult for participants to understand what they are consenting to, reducing compliance, and increasing the likelihood of withdrawals. The development and adoption of new technologies make it possible to communicate, educate, and ensure that the participant has been provided sufficient and accurate study information. These new advances also allow for the acquisition of participants’ signatures electronically – a process referred to as eConsent. The use of eConsent currently has some restrictions in certain jurisdictions, such as Europe, specifically with the signature capture component. However, there is broad support for the use of an interactive, multimedia approach to providing patients with more appropriate study information. 

The potential benefits of eConsent are numerous and will impact multiple stakeholders and sponsors. eConsent increases patient comprehension compared to a lengthy paper document and allows study participants the option to choose their preferred method (electronic or paper). Well-informed participants are more likely to make a knowledgeable decision about their voluntary participation in a clinical trial, allowing them to enter with realistic expectations and become more active partners in the study process. Study sites are able to reduce paperwork, lowering the burden on staff. Electronic management of updates to the informed consent document due to protocol changes also reduces staff burden and enables better document management and regulatory reporting. Even with these positive benefits, sponsors may have difficulty changing to a new process as current SOPs (standard operating procedures) and workflows are built for the paper and onsite process. Change management and stakeholder education with sponsors and the site will be important to gain traction and adoption. 

As decentralized clinical trials have gained significant adoption in response to the global COVID-19 pandemic, there is a growing need for platforms that allow for TeleConsent to connect patients worldwide virtually with clinical sites. This allows clinical trial sponsors to screen, enroll, and consent participants in an environment where many patients are being told to shelter in place.


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About TeleConsent 

Traditionally, consent has been obtained using a paper process where the clinician and the patient are located in the same facility. Both parties sign the paper document to confirm it has been reviewed and understood. eConsent allows this process to be digitized and enables electronic signatures to be obtained at the site with both patient and clinician. This new process removes manual paper workflows and enables increased quality and compliance while checking real-time patient comprehension throughout the process. TeleConsent, on the other hand, means that a patient can be located geographically anywhere, distant from the site, and follow the eConsent process. Patients can be in their own homes and connect virtually to the site by TeleVisit. This process can also be used in a “re-consent” for any future changes in a clinical trial. Like eConsent, it is crucial with TeleConsent that the system is in line with the parameters outlined by the FDA in their December 2016 guidance and 21 CFR Part 11, and ICH GCP E6 R2. In terms of security and privacy, the eConsent system should be aligned to HIPAA and GDPR. 

On Tuesday September 22nd, we announced general availability of Medable TeleConsent™, a new product that enables fully remote informed consent and re-consent for clinical trials. Unlike traditional eConsent products that require both patient and investigator to be physically present together in the clinic, Medable TeleConsent allows patients, doctors, nurses, and clinical trial staff all to connect and sign remotely from any location. 

Medable TeleConsent solves one of the most complex aspects of clinical trials for patients, sites and sponsors and defines the first experience for patients of trial participation. By eliminating the need for multiple round-trip visits to clinical sites, TeleConsent dramatically improves patient accessibility to studies from their home location connecting them directly with their PI’s and site teams resulting in faster enrollment and greater participant diversity for trial sponsors—and better retention over the course of a study as patients have more options to enhance understanding of trial expectations. TeleConsent also improves patient knowledge and comprehension  by providing medical information in visual and multimedia formats, which patients can review in depth together with family members and caregivers—and then engage visually with their physician to sign consent forms digitally from the comfort of their home or local clinic.

Medable TeleConsent is especially critical in the COVID-19 environment, where many patients are staying home to avoid social interaction and minimize exposure. Sites and sponsors can now screen, enroll and consent study participants without meeting in person, taking advantage of Medable’s TeleVisit application to conduct personalized interactions that improve patient understanding. Sites and sponsors benefit from streamlined workflows and enhanced data quality and compliance. Sponsors also get increased transparency with real-time reporting and insight into study progress. TeleConsent can also be used for re-consenting patients for any future changes that may happen in a clinical trial.

“Medable TeleConsent is a critical step in the evolution of decentralized research, as consent is the gateway to trial participation,” said Dr. Michelle Longmire, CEO and co-founder of Medable. “This has the potential to enable global patient access and improved knowledge, leading to greater participant diversity, retention, and ultimately research quality and speed. We’re excited to break down these barriers to benefit patients, sites and sponsors alike.”

To learn more about our TeleConsent visit us online or attend our webcast, “TeleConsent in Decentralized Clinical Trials Patient preference complemented by digital innovations-delivering on a long overdue promise.” on October 6th, 2020 register here

 

Topics: decentralized trials Teleconsent
2 min read

Introducing Medable TeleConsent

By Allison Holland- Head of Decentralized and Remote Trials on Sep 22, 2020 1:31:03 PM

Today we’re excited to announce Medable TeleConsent™ here.

Medable TeleConsent has been designed as part of our flexible and modular Decentralized Clinical Trial (DCT) platform. This is a big step forward in addressing the monumental challenge of rethinking physical informed consent and re-consent processes in a pandemic world.

eConsent Old vs New Graphic 06

Medable TeleConsent solves one of the most complex aspects of clinical trials for sites and sponsors—and transforms the initial experience for patients. By eliminating the need for multiple round-trip visits to clinical sites, TeleConsent dramatically improves patient access to studies, connecting them directly with trial investigators and site teams from their home location. This results in faster enrollment, greater participant diversity and better retention for trial sponsors.

TeleConsent also improves patient knowledge and comprehension by providing medical information in visual and multimedia formats, which patients can review in depth together with family members and caregivers. They can then engage visually with their physician to sign consent forms digitally from the comfort of their home or local clinic.

“Medable TeleConsent is a critical step in the evolution of decentralized research, as consent is the gateway to trial participation,” said Dr. Michelle Longmire, CEO and co-founder of Medable. “This has the potential to enable global patient access and improved knowledge, leading to greater participant diversity, retention, and ultimately research quality and speed. We’re excited to break down these barriers to benefit patients, sites and sponsors alike.”

Dr. Remo Moomiaie-Qajar, president and CEO of Cytonus Therapeutics, agrees with this approach. “At Cytonus, our diverse portfolio includes a COVID-19 therapeutic. One major problem we have had is the constantly changing regulatory requirements in various regions and countries. This has added multiple layers of complexity when it comes to consenting and necessitates re-consenting patients. Given that most therapeutics to treat viruses like COVID-19 are time-sensitive for the health of the patient, without a solution like Medable TeleConsent we would have significant delays enrolling patients that are extremely costly. Medable TeleConsent eases the administrative burden of thousands of hours safely spent remotely consenting for both investigator and patient.”

Please click here to register for a live webinar October 6 on using TeleConsent to reduce patient burden in decentralized trials.

Topics: decentralized trials Teleconsent
2 min read

Why Medable: CEO Manifesto

By Michelle Longmire on Sep 21, 2020 1:54:09 PM

 

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Mission matters

Throughout my career, I have had the opportunity to work as a scientist, physician, and CEO. I have enjoyed every job I have ever had, whether it was researching epigenetics of disease or caring for patients with skin disease. Yet, prior to Medable, I had not felt that “I was born to do this.” 

Founding and building Medable is something that I truly feel like I was born to do. I say this not because it is easy or that I do not have many areas of growth as a CEO, I say this because of the impact of our mission - enabling effective therapies to reach patients faster through better clinical trials means less suffering and better health, worldwide.

Our team deeply believes that as we execute on our mission, we will enable better treatment of the more than 7,000 uncured and sub optimally treated diseases. At Medable, our team lives to deliver on our mission and we strive everyday to execute towards this goal. 

Medable team

Team matters

Among the many things I have learned in building a company, is that the team is everything. At Medable, our motto is to be the strongest teammate and fiercest opponent. Rasmus Hogreffe, VP of Clinical Trial Innovation recently said to me, “Michelle, while other companies can replicate software, no one can replicate people, our team is what makes us Medable.”  At Medable, we empower one another to be the best on the field in our respective positions. It is through this team philosophy that we achieve synergy and outcomes beyond the capability of any single individual and often surprise even ourselves in what we can accomplish.

Impact matters

The fascinating thing about building a company, is that you are truly building something. Every person impacts the current and future form of the company and the smaller the company, the bigger the impact. From project managers to sales people and engineers, together we are all building Medable. This means that every single person is shaping and influencing our current and future state. It is incredibly exciting to see Medable get stronger as we develop and grow and how each team member truly shapes the future of the company with the unique skills, perspective, experiences, and passion that they bring to our team. 

medable team

Join us if ...

If you love learning, are humble but confident, empathetic yet unrelenting in the pursuit of goals, are inspired to be the best teammate and fiercest opponent - and, most importantly - are dedicated to the mission of getting effective therapies to patients faster, Medable wants you.

Visit our careers page. 

Topics: medable careers
2 min read

How Hybrid Trials Will Help CRO Market to Grow to $64B by 2024

By David Swanger on Sep 18, 2020 1:24:03 PM

Analyzing Frost & Sullivan report “Hybridization of Clinical Trial Designs Reviving Global CRO Market Post-pandemic; 2019 - 2024”

Frost & Sullivan, the research & consulting firm, recently released an analysis of the CRO market projecting the overall market would grow on a compound annualized basis of 8.2% to $63.83 billion by 2024. This would represent a significant increase from the current market size of $43.03 billion in 2019.

Frost & Sullivan summarizes their findings from the analysis below:

“Due to the impact of the coronavirus, many large pharmaceutical companies have placed new trials on hold and numerous small, mid-size, and large enterprises have suspended ongoing ones, thereby demonstrating the magnitude of the disruption,” said Unmesh Lal, healthcare industry principal at Frost & Sullivan.

“Going forward, to minimize the adverse effect of the pandemic, organizations such as site management organizations (SMOs) and patient recruitment organizations (PROs), that operate traditionally, will have to explore collaboration opportunities with emerging virtual trial platform vendors, eRecruitment providers, and remote monitoring solutions providers to expand the capability to support distributed/hybrid CT models.”

These findings are consistent with what we have been seeing at Medable. Since the pandemic struck, we have helped an array of sponsors and CRO’s from all over the world restart their studies with the use of decentralized study modules ranging from utilizing remote electronic informed consents (and reconsents) to televisits, ePRO’s, and connected devices.

To help study managers right size their approach to hybrid/virtual/decentralized studies we developed a readiness framework that makes it relatively easy to determine the right tradeoff between decentralized trial modules and implementation time:

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Figure 1: Readiness framework for transforming to decentralized trials

As you can see from the readiness framework, just about any study can benefit from lightweight accelerated virtualization whereby electronic consents (and reconsents) as well as televisits are introduced to an in-flight study. Doing so provides HIPAA-compliant options to reduce the number of visits patients and site staff need to make to a clinic. In our post-pandemic world, many sponsors and CRO’s have found that shifting some visits on the study schedule has enabled studies to continue that might otherwise not.

To learn more about Medable’s modular decentralized clinical trial platform, feel free to schedule a demo.

 

6 min read

Three Recommendations to Improve Trial Experience for People of Color

By Nina Baltierra on Sep 17, 2020 2:24:28 PM


Today, I’m comin’ atcha with something a little different: my experience randomizing into a COVID vaccine trial. I want to share this to remove some of the mystery of the process and, in doing so, hopefully encourage more people to participate, as well as point out some serious shortcomings.

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When the NIH Revitalization Act was passed in 1993[1], one of the expressed priorities was to increase participation of people of color (POC) in clinical trials. In 2016, Black Americans were roughly 13% of the population but only 5% of research participants.[2] Latinx folks comprised over 17% of the US population but less than 7% of research patients.[3] Whites? 2/3 of the population and nearly 83% of research participants.[4] Keeping this in mind, and as a “white-passing” POC, I was curious to see if, in the midst of a global pandemic, considerations were being made for participants of color in a COVID vaccine trial. This is my take.

Despite, or perhaps due to, all the chaos and disorganization of the pandemic, researchers have designed over 1,200 clinical trials to evaluate myriad COVID-19 treatment and prevention strategies. I’m participating in a trial for one vaccine candidate that is now entering a Phase II/III study that will compare the results of the people who receive the investigational vaccine with those who receive a placebo (which is a saline solution).

Some quick facts about this trial:

  • Approximately 30,000 healthy people could take part in each phase
  • Participants are randomly assigned to receive the investigational vaccine or placebo
  • Participants receive two injections in their upper arm (where you get your flu shot), approximately three weeks apart
  • Additional follow-up time points are one month, six months, 12 months, and 24 months after the second shot

But what’s it really like? I randomized into the study in mid-August, so I can share a bit about my experience: what to expect, and where I felt the patient experience fell short.

After answering some questions in a quick phone call, my enrollment appointment was scheduled. The actual appointment lasted approximately three hours. It went a little something like this:

First, I did all the paperwork. Forms to review and sign included:

  • Patient Information Sheet: Contains your basic information like name, address, phone number, emergency contact info, known allergies, etc.
  • Informed Consent Form: Contains a summary of the study, details on the structure of the study, discussion of side effects and reactions, and frequently-asked questions and answers. Basically, this is where you agree to participate in the study.
  • HIPAA Authorization: Here, you agree to let your protected health information be accessed and shared for scientific purposes. There are also details about how they keep your information private and secure.
  • Authorization for Use and Disclosure of Protected Health Information: Gives the study permission to use protected health information for specified purposes (in this case, research).
  • Confidentiality Statement: Describes how your medical information may be used and disclosed, and how you can get access to it.

I was left in the exam room to review everything on my own and then the research coordinator came in to answer any questions I had and watch me sign. I used to work in research and conducting similar enrollment appointments was one of my responsibilities. During my time in that role, I would be sure to review each document with each participant--reading it aloud and pausing for understanding and answering questions throughout.

Comparing my recent study experience with my history as a researcher, I was a little surprised. I think it might have had something to do with the urgent nature of the race to develop a COVID vaccine. For example, when I asked about whether working on this study was taking priority over other studies at the site, they responded with a resounding yes. In fact, the study coordinator I talked with was brought in from another site to better respond to the onslaught of patients enrolling at the site where I enrolled.

My personal experience makes me curious as to whether study participants with distrust of clinical trials (and medical providers in general) and/or low levels of medical literacy (or literacy in general) were treated similarly and decided not to give their consent to participate in this trial because it seemed too mysterious. Or, was an assumption made about my level of trust and literacy when I walked in, and that’s why I was left alone?

Once my paperwork was done, it was time to see if I was eligible for the study. This includes:

  • Answering questions about my health:
  • Assessing my current risk for COVID;
  • Having the study doctor complete a short physical examination;
  • Getting a COVID test with a nasal swab (it’s like a sharp feather and it is not painful but is very uncomfortable); and
  • Taking a urine pregnancy test

Once all that was settled, I was in. Then, the study staff took a small blood sample and gave me my shot! Participants are then required to stay for 30 minutes for observation.

I was instructed to download an app that serves as an electronic diary to track my temperature (the study provides the thermometer), symptoms (from a list), and measure any redness or swelling of the injection site (the study provides a device). The app is very easy to use, and I was pleased to learn that if you don’t have a smartphone (or are uncomfortable with apps), the study will provide a device on which to record your diary.

Something that struck me was how difficult it was to measure the redness of the injection site. I had spent some time in the sun on the day after my appointment, and couldn’t tell if I had any redness! I eventually found some lighting that made a little circle of irritation visible, but if my skin had been any darker, I wouldn’t have been able to see it at all.

While my day in the sun covered up any irritation, it exposed a blind spot in the study design: If my skin were darker like many other POC, would I have received different instructions for how to measure redness at the injection site? One hopes so.

Based on my personal experience as a “white-passing” POC with high medical literacy, below are three recommendations to improve the patient experience for people of color:

  1. Use inclusive recruitment strategies
  2. Ensure the informed consent process is accessible to all potential patients, regardless of their level of medical literacy
  3. Evaluate data points of interest to ensure they can be accurately captured for a diverse array of patients

Ensure that the study incorporates inclusive recruitment strategies in communities with a variety of demographic backgrounds. This starts with including advocates from communities of color in the development of the study. Expanding recruitment into these areas may require direct-to-patient recruitment methodologies that seek to identify and recruit patients first, and then match them with a site and/or investigator.

Design an informed consent process that takes into account different levels of medical literacy and trust. It is critical that patients fully understand what they’re consenting to in the study. This process should be the same for all patients so that there isn’t any variation based on assumptions.

Test the feasibility of collecting the data points within a diverse cohort of patients to ensure that they will be useful and accurate for the study (e.g., how skin irritation presents and is measured on a variety of skin tones).

If consideration isn’t given to people who distrust clinical trials specifically or medical providers in general, have different levels of literacy, are not tech savvy, or have darker skin, they may not feel welcome or safe participating in a COVID trial. If patients aren't made to feel like valued members of the study team, 30% drop out before study completion.[5] The result is a homogenous participant population that does not reflect the population the vaccine is designed to protect.

 

The good news is that there are a few tangible steps study managers can take to improve the patient experience for POC. Doing so promises to improve safety and efficacy for a broader array of the patient population.

[1] https://orwh.od.nih.gov/sites/orwh/files/docs/NIH-Revitalization-Act-1993.pdf

[2] https://www.nature.com/articles/s41591-018-0303-4

[3] https://khn.org/news/latinos-left-out-of-clinical-trials-and-possible-cures/

[4] https://www.linkedin.com/pulse/patient-recruitment-clinical-researchs-white-whale-jason/

[5] https://www.centerwatch.com/articles/15008

2 min read

Apple Watch and Research Kit May Accelerate Digital Endpoint Adoption

By David Swanger on Sep 16, 2020 4:49:14 PM

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Yesterday, the Washington Post recapped Apple’s fall product launch event:

Apple’s New Watch Draws Attention To Its Health-Care Play: Apple Watch takes the spotlight usually reserved for new iPhones at the September event

The article summarizes Apple’s shift toward services with a focus on the healthcare vertical, especially clinical research through Apple’s Research Kit:

“The Apple Watch is the physical manifestation of the company’s health-care play, and perhaps the most important but least understood prong of the company’s new business model anchored around services rather than gadget sales. The announcement also comes during the peak of a global pandemic in which the conversation about health and technology has accelerated.”

“Apple’s ResearchKit has been used for clinical trials, some of which it was announced to great fanfare. Last year, the company announced three major studies on women’s health, heart health and hearing. In terms of the massive amount of research constantly being conducted by drug companies and in academia, Apple isn’t much of a player.”

“In addition, ResearchKit isn’t a full-service offering. Companies that aim to use ResearchKit for trials still must have the resources to create customized software, and companies such as Thread Research and Medable have sprung up to meet that need. Large drug companies tend to opt for bespoke software with actual medical devices for traditional trials, industry experts say, rather than rely on ResearchKit.”

Medable views the collection of measurable data, direct from patients, in real-time as a key enabler of decentralized clinical trials and effective remote patient oversight. Being able to integrate data, such as ResearchKit, with other contextual data such as Patient Reported Outcomes, enables Medable to offer sites, patients and sponsors a modular suite of data in real time for actionable decisions and patient care management. Use of this data powers patient care initiatives that are now a part of the Medable Decentralized Trial Platform. These patient care initiatives range from enabling HIPAA-compliant workflows, interactions, and televisits between patients, doctors, and study managers using Apple, Android, and just about any mobile or desktop browser.

At Medable we are also excited to see Apple’s continued investment in utilizing the iWatch and iPhone to generate digital endpoints that perform as well or better as devices (like pulse oximeters) that are traditionally used at physician sites or medical labs. While the article notes that there is work to be done in ensuring that the veracity and reliability of these digital biomarker measurements meet or exceed traditional devices and measures, we at Medable are excited about the progress being made across the industry with digitally-enabled devices and measures.

The future is bright for bringing the clinical trial experience closer to a patient’s home and in the meantime helping to bring effective therapies to patients faster in our post pandemic world. If you’d like to learn more about Medable’s Decentralized Trial Platform, feel free to sign up for a demo.

 

Request Demo

 

Topics: decentralized trials remote trials
3 min read

Prostate Cancer Awareness Month: Navigating an Increasingly Complex Disease

By Joan Venticinque, PAC member on Sep 14, 2020 10:56:38 AM

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During September, Prostate Cancer Awareness month draws attention to prostate cancer screening and those living with the disease. Over the years, my grandfather, father, and four close friends have received a prostate cancer diagnosis. Their ages range from 40s to their 90s and their diagnoses varied from early stage through metastatic disease. For all of them, their diagnosis was both unexpected and distressing. They were fearful and overwhelmed at the prospect of being asked to take a more active role in their treatment decision-making, often without understanding the diagnosis, treatment plan, or prognosis.

–“I have seen three different doctors, and all recommend a different treatment plan. How do I know what to do?” –Jeff

There is so much confusion about screening and treatment for prostate cancer. “I’ve heard measuring PSA is not an accurate test.” “It’s slow growing, so why worry?” “They say all men will have it [prostate cancer] in their lifetime, so why go to the doctor?” “How will the different treatments effect my sex life?” The array of available treatments varies widely from surgery to radiation, anti-hormonal medication, immunotherapy, gene therapy, and watchful waiting. What do patients believe and how do they decide what to do? These questions demonstrate the need for clinical trials that provide the answer of the ‘right’ screening and treatment for the ‘right’ patient.

–“I did as much research as I could and had two second opinions. I found a clinical trial for a new type of radiation treatment. I was glad I did. It was important that I found the right treatment for myself.”–Jack

New Treatment Options as a Result of New Screening Modalities & Therapies-graphic

 

Clinical trials play a vital role in moving new screening modalities and treatments to patients. Recent screening trials have included combining magnetic resonance imaging (MRI) with ultrasound for more accurate prostate biopsies. This method can increase the detection of high-grade prostate cancers while decreasing detection of low-grade cancers that would not progress. New imaging techniques also include using a PET scan that looks for a specific protein called prostate-specific membrane antigen (PSMA) found on prostate cancer cells.  The ability to detect very small amounts of metastatic prostate cancer could help doctors and patients make better-informed treatment decisions.

Targeted Therapies for a Complex Disease in the Age of Precision Medicine

Targeted therapies based on PSMA, the same protein that is being tested for imaging prostate cancer, are being studied for radiation treatment.  The molecule that targets PSMA is chemically linked to a radioactive compound. The new compound can potentially find, bind to, and kill prostate cancer cells throughout the body.

 Over the last few years, several new approaches to hormone therapy for advanced or metastatic prostate cancer have been approved for clinical use. Many prostate cancers become resistant to standard hormonal treatment over time. After successful trials, three recently approved drugs have been shown to extend survival in men with hormone-resistant prostate cancer.

Current trials are using immunotherapies that work with the immune system to fight cancer. These therapies can either help the immune system attack the cancer directly or stimulate the immune system in a more general way. Currently, vaccines and checkpoint inhibitors, two types of immunotherapy, are being tested in patients with prostate cancer. 

–“I relied on my doctors for my treatment plan, but it wasn’t until I joined a support group and realized everyone was different with different choices, including clinical trials, that I felt more comfortable with my decisions.” –Larry

 

right_decisions+treatments_patients_medable_dct

 

My family members and friends eventually all made their treatment decisions. Given their age differences and goals for their therapies, each one had a different treatment plan. Although my grandfather is gone, others live with side-effects. Still, others are on treatment for the rest of their lives without much in the way of side-effects. All benefited from patients who participated in clinical trials. Without clinical trials we may never understand what the ‘right’ screening and treatment will be for the ‘right’ patient.

 

Topics: decentralized trials PAC RareDisease prostate cancer cancer awareness
6 min read

One in 75 Million: Parents Seek Cure for Ultra-Rare Genetic Condition

By Jena Daniels- Director of Research on Aug 20, 2020 4:57:46 PM

Raghav Sanath is celebrating his second birthday this month.

Something so simple, one of many cherished moments for parents of young children. And yet for Raghav’s parents, Sanath and Ramya, it’s one of many days that is truly a gift.

The reason: Raghav was born with an ultra-rare genetic disorder caused by mutation in a gene called GPX4. At the time of Raghav’s birth, only two other babies were reported to have it. Both passed away a few weeks after their birth.

Raghav’s father Sanath says they knew something was wrong two or three hours after Raghav’s birth. He was not moving his hands and legs like a normal newborn, and he could not suckle on the bottle. “That night the doctor came to us and told us there is something significant happening with him, and I’m not sure what it is.”

Four or five days later, Raghav was moved to Seattle Children’s Hospital, where specialists started looking at his bones and his brain and his GI system for clues. Geneticists ran several panels of tests. After 20 days or so, Raghav went home with his parents, knowing only that there was some undetermined genetic issue.

Raghav then underwent exome sequencing to analyze large portions of his DNA to determine a cause for his disease. The genetic clinic tested for a variety of indications that all came back negative. Finally, on Raghav’s first birthday, right before cutting the cake, his parents received a call from the genetics clinic that Raghav has a rare mutation in his GPX4 gene.

The doctor shared that Raghav was “doing better” than others with the disease, though his future was described as wheelchair-bound, non-verbal and prone to premature death.

As Sanath wrote in a blog post last September: “This is a future no parent wants for their child! Raghav is over one year old, but cannot sit up without support. He doesn’t have the muscle strength to hold a toy in his hand. He doesn’t have the oral strength to swallow food. He cannot hear normally. His bones are not developing as expected. We really don’t know what his future looks like.”

Once they received the diagnosis, Sanath says, “It helped us take care of him better because we now know the severity of the condition. It also gave a name to the problem, which previously was hiding in plain sight. At that point, we could start to find a solution.”

Sanath and Ramya immediately shifted into problem-solving mode, starting their own personal search for a cure. After reading another parent and fellow computer scientist’s journey online, Sanath published a blog post sharing Raghav’s story with an appeal for help. Sanath shared the post with family and friends, and it “sort of went viral” – resulting in $100,000 in donations in two or three days.

      "That transformed our perspective from being not hopeful to being incredibly hopeful,                 because so many people believed in us enough to actually give us money – and give us           a lot more than we asked. And they all sent us messages and phone calls saying, hey,             you can do this.”

Sanath, Ramya and others set up Facebook groups and WhatsApp groups to find researchers and other patients and any resources that could help their son.

They found a researcher who is working on a paper about GPX4 gene mutation, through which they learned about eight other cases around the world. The first reported condition was in 1980, and then there was a long pause before the next significant paper in 2014. Some of the gene mutations have been lethal, while some have been compatible with life.

They focused initially on drug repurposing – using available drugs to treat new conditions. Working with researchers, they were able to identify 36 relevant drugs to repurpose. They tested 3-4 supplements that were deemed highly safe, one of which showed significant improvements in the short term but plateaued later.

     “The initial few months were fueled by the interest and also the hope to solve a problem,”          reports Sanath, who works as a senior software engineer at Amazon. “It was exciting to            solve one of the most challenging problems that exist in humanity. It’s like going to Mars,          but this is by far even more challenging because you’re dealing with life.”

That excitement, though, was “followed almost immediately with deep sorrow” as Sanath and Ramya had to help Raghav navigate everyday life and deal with his condition. “Even the most mindless things are serious. So even like now when he gets a little rash, for example, we take that extremely seriously. And every time he gets a cough or a fever, it’s an existential crisis for us. We have to get to the bottom of it and try to fix it right away. We just don’t wait.”

Sanath and Ramya eventually identified an experimental drug that aligns very closely with the gene’s mechanism of action, so they filed an application with the FDA to secure approval for a “compassionate use” single trial. The paperwork was finished last Thanksgiving, and they just recently received FDA approval – and the first shipment of the drug.

While Sanath is hopeful, he doesn’t want to bank on the experimental drug, so they are also exploring gene therapy as another treatment option. Like clinical drugs and trials, gene therapy is extremely expensive – millions of dollars per patient. So Sanath and Ramya are raising new funds to pursue that path. While that may be an option for Raghav, Sanath is concerned about what happens to other kids with limited resources.

      “We need to make gene therapy accessible to ultra-rare diseases, because right now for           conditions with so few patients, there’s no commercial interest.”

Given Sanath’s software engineering background, he is also exploring how behavioral therapies might work for his son, tapping into the latest computer science research for eye tracking, body language tracking and other techniques. (Sanath recently transitioned to Amazon’s health care division, which better aligns his role at work with his parental mission.)

Sanath reinforces that he and Ramya are not alone in this journey, and they benefit so much from other parents in the rare disease community. “We’ve learned from so many other parents and researchers in the industry people along the way. Because this is a community that just shares, no questions asked. The first time you meet someone who’s in the community, you feel as if you’ve known them for 10 years – and we share such an immediate bond that you don’t really share with others.”

Ultimately, though, this journey is about Raghav and the love and aspiration that parents have for their child:

     “To me, I want a high quality of life for my son. I want him to be independent. I want him to        get a job. I want him to go to school and learn and just lead an independent life like you            and I do. And that means, you know, until we are confident that he’s there, we still have            work to do.”

 

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Raghav is one of many patients living with rare diseases, many of which have no known cure or treatments. Medable will continue to highlight stories of patients and caregivers like Raghav, Sanath and Ramya, as we encourage the industry to invest resources into rare disease research.Please follow Raghav’s story via his family’s bi-weekly podcast. And please follow our Patient Advisory Council via blog and LinkedIn for additional stories.

Topics: PAC RareDisease
3 min read

Zookeeper Living with Rare Disease Advocates for Change

By Medable Patient Advisory Council on Jul 23, 2020 10:54:11 AM

Being a zookeeper comes with a lot of surprises – late nights waiting for a rare birth, small animals displaying unexpected feats of strength, and watching a warm-weather animal loving the snow. However, there are some surprises that aren’t quite as fun, like learning the occupational hazard is acquiring a rare disease.

Michael Illig, now age 66 and living in an idyllic Oregon community, discovered he had sarcoidosis a few years ago. Sarcoidosis is a rare inflammatory disease that affects multiple organs in the body, but mostly the lungs and lymph glands. Abnormal masses or nodules (called granulomas) of inflamed tissue can form in certain organs of the body. One of the most common side effects is difficulty breathing – a symptom that would dramatically change Michael’s life as an outdoorsman who hikes nearly 200 miles a month in the lush green mountains near his home.

Like so many rare diseases, there is no cure. Some people walk around with sarcoidosis for years without ever becoming symptomatic while others experience massive organ failure. Michael’s own doctors call it a ‘mysterious’ disease. It typically manifests itself with shortness of breath, wheezing, chest pain, and persistent cough. Treatment often starts with corticosteroids to reduce inflammation followed by other medications such as methotrexate (Trexall) and azathioprine (Azasan, Imuran) that work by suppressing the immune system. Additional medications are used to treat specific symptoms.

Michael first experienced symptoms in his mid-40s when he was admitted to the hospitals with kidney stones but didn’t think much of it. It wasn’t until years later while hiking with his sister that he experienced severe chest discomfort and difficulty breathing. He was sick for weeks and finally went to the doctor when a CT scan found multiple nodules on his lungs. He was immediately prescribed the steroid prednisone, which helped, and then prescribed two inhaler treatments – a routine he continues today to relieve the heaviness in his chest.

Sarcoidosis, untreated, can lead to permanent scarring of the lungs, inflammation of the eyes, kidney stones and reduced kidney function, granulomas on the heart, and even nervous system problems.

Michael attributes the disease to environmental factors after decades of breathing in animal dander, feather particles, zoo cleaning aerosols, and other irritants but he is not bitter in the slightest. He reminisces fondly of his years working with numerous species of animals. Michael worked at various zoos, but spent the bulk of his career at the Oregon Zoo where he cared for hundreds of animals and worked from sun-up to sun down. He now advocates for improved safety measures for zookeepers today. “You used to have to wear a mask only when working with primates because they are biologically so similar to humans, but since COVID-19, zookeepers will need to start wearing protective masks when working with all animals,” said Michael. “That is long overdue.”

There are an estimated 150,000-200,000 people in the U.S. living with sarcoidosis, and while some don’t experience symptoms, others require medical treatment like Michael. Even so, Michael insists that nothing can stop him – and he may be right! Where Michael used to huff and puff walking up just slight hills, he now flies up 25-degree inclines with a 30-pound pack on his back, thanks to the dual-inhalant treatment regimen.

Michael is one of the ‘lucky’ ones living with a rare disease. Unfortunately, 300 to 400 million patients suffer from nearly 7,000 different rare diseases and 95% do not have a single FDA-approved drug treatment, leaving more than 30 million people in the U.S. with few to no options. There are many reasons for the lack of rare disease research – small patient populations, heterogeneous disease characteristics, lack of scientific knowledge, and absence of historical data – but one of the biggest hurdles is the prevailing clinical trial model that makes it difficult to recruit enough patients to generate statistically meaningful results.

The good news is that the technologies needed to improve patient access and trial experiences are here like Medable’s new mobile application called Trial-Fit TeleVisit which allows patients to connect virtually with research sites. These technologies, including wearables and site-facing apps, can overcome barriers to trial execution and improve data sharing and process efficiency across organizations conducting rare disease research. 

Another benefit of virtual or decentralized trials is that they provide more and higher quality data. Trial participants that track progress using a diary or recounting specific events that are pertinent to the trial at their next office visit create retrospective bias and inaccurate data capture. Decentralized trials enable patient reporting in real time for increased accuracy. For example, if the patient had an adverse event like a headache, she can capture the event at that specific moment while experiencing it. Patients can also capture a broader set of contextual data, such as their location when they had the episode or whether they were sleep deprived, hungry, or light-headed with digital tools. Researchers can use the contextual data to better understand the triggers around the adverse event. 

Decentralized trials allow researchers to continuously collect data about a treatment over time – this is particularly important with rare diseases since so little is known about their etiology, like with Michael’s sarcoidosis. Remote technologies including wearable devices, in conjunction with mobile health applications, can track symptoms automatically and without pause. 

Every six months, Michael takes a pulmonary function test that shows his oxygen exchange rate diminishing by just a point or two each time but he is feeling great. In fact, he says that he lost 30 pounds, is eating healthy, and happier than ever. We wish Michael – all those living with rare disease – good health and continued happiness. Thanks to decentralized clinical trial technologies, the future looks bright to discover life-altering new treatments for rare diseases.

 

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Topics: decentralized trials remote trials
3 min read

Are UV Staying Safe in the Sun?

By T.J. Sharpe on Jul 16, 2020 1:56:58 PM

July is UV (Ultraviolet) Safety Awareness Month.  As summer gets into full swing, it is the perfect time for a reminder on how to best protect yourself and your family from harmful UV rays during this long, hot, sunny season.

As a two-time melanoma survivor, I have lived with the risks of skin cancer for nearly 20 years. I was fortunate that the first small mole on my clavicle was removed and biopsied almost as an afterthought; who thought one little spot would cause so much havoc?  Twelve years later, cancer that was under that melanoma mole had spread into my lungs, liver, and spleen, a stage IV diagnosis with a ~10%  5-year survival rate. All this from one mark on my chest that admittedly saw too much sun and not enough screen.

UV safety begins with sun protection. Sunscreen, tanning lotion, sunblock – whatever your term for it, learn what you’re putting on. The SPF number doesn’t specify a set time you can be in the sun; it is merely a relative measure of the amount of protection a sunscreen offers.  Most sunscreens, no matter how high their SPF, lose effectiveness after about two hours.  Higher numbered SPFs increase the protection you get over those two hours.  The Mayo clinic has an understanding sunscreen guide that more fully explains the differences between types and the need for a “broad spectrum” sunblock that protects against multiple types of ultraviolet  rays.

A quick primer on two main types of UV rays:  UVA waves are what cause the skin to tan, and also to age.  UVB is what makes skin burn, but also produce vitamin D.  A person in typical weather – especially during the summer - only needs a few minutes a day of sun to generate enough vitamin D.  While there’s no harm in overdosing on vitamin D, there is an elevated risk of melanoma if a day in the sun turns into a night of sunburn.

The most harmful thing you can do to your skin is get sunburnt, but it is not the only UV concern.  Repeated exposure to intense UV rays can cause melanoma and using a tanning bed exposes the skin to high doses of UVA light. Numerous studies slice this data into very understandable bites of information. Want to increase your melanoma risk by 75%?  Hop into a tanning bed before 35.  Perhaps the most enlightening tidbit from the above link is, “More people develop skin cancer because of indoor tanning than develop lung cancer because of smoking.

A skin cancer patient’s journey post-cancer is a constant reminder of their prior illness and susceptibility to future episodes.  Skin cancer, and particularly melanoma, is known to return from either continued UV exposure (a “new” case) or from cancer cells that lie dormant in your body (a recurrence).  Both paths can be deadly, and it is essential for anyone with a history of skin cancer or risk factors (like significant sun exposure, childhood sunburns, or a history of going to tanning salons) to be conscious of sun safety and proactively check themselves for atypical moles.

Being a lifelong Jersey Shore fan (the destination, not the show) that now lives in South Florida, I’m reminded of the risk of UV exposure every time my shiny bald head exits the Fort Lauderdale air conditioning, or I get my kids ready for a day at the NJ beaches. Being safe in the sun begins with awareness and preparation. When you know you are going to be out, be ready with sunblock, hats, and shirts. See when the sun will be at its strongest – usually between 11a-3p every day – and plan activities to give relief from the sun’s intensity.  

The Sharpe family still enjoys time on the beach and in the ocean.  We all wear sun shirts, and sunscreen is reapplied as much as possible.  There are pop up tents for the beach and the soccer field, and hats and long sleeves for the boat and the bikes. Respecting the power of the sun’s rays does not need to be a complete compromise of outdoor activities. Still, for our family, it is the combination of preparation and diligence that will minimize the chances any of us hear “You have skin cancer.”  

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13 min read

Cures 2.0 PAC Views

By Medable Patient Advisory Council on Jul 1, 2020 9:56:18 AM

The 21st Century Cures Act, passed at the end of 2016, has laid the groundwork for faster innovation of therapies and products to patients, while modernizing numerous parts of the drug/device development life cycle, including clinical trial design, real-world evidence use, and patient engagement.

The half-billion-dollar program extends through 2025, looking to streamline the process of clinical research, shorten the drug development lifecycle, and get therapies to patients faster. While it has achieved some successes, many initiatives touted as bringing transformative results have yet to come to fruition. The high-level 21st Century Cures Act goals are summarized below:

  1. Preventing and curing serious illness
  2. Accelerate drug/device development
  3. Address opioid crisis
  4. Improve mental health service delivery
  5. Interoperability between EHRs
  6. Adoption & Interoperability of EHRs

Near the end of 2019, the authors of the original Act, Reps. Diana DeGette (D-Colo.) and Fred Upton (R-Mich.), brought forward a proposed second iteration of healthcare reform they called "Cures 2.0", seeking to build on the substantial bi-partisan support of their first Act. This encore presented new, progressive, and more specific ideas than the original bill, touching the topics of digital health, healthcare coverage, patient data empowerment (including real-world evidence), and addressing the need to support caregivers and the families of patients. The window for public input is closed, but many of the more influential organizations have released their wish list for Cures 2.0. With that as the backdrop, the Patient Advisory Council (PAC) at Medable gathered our thoughts on the progress made by the original Act and where Cures 2.0 could take healthcare in the United States.

Building on Cures 1.0

Initially, the PAC focused on what was available to build on from the first Act. However, several ideas came from the dissecting the original Act and brainstorming what stronger federal involvement could mean for patients. The PAC saw several opportunities to build more expansive legislative efforts to address where the original Act has fallen short. Some fantastic results were achieved at the Oncology Center of Excellence, such as the first drug approval (for Keytruda) based on a tumor's unique biomarkers; and in-vitro diagnostics identifying patients who benefit from a multitude of approved treatment. However, other areas of the 21st Century Cures Act saw dramatically less progress made.

Though the NIH was tasked with including more women and minorities in research, clinical trials are still not as diverse and equitable as they need to be. Certainly, part of this is an access issue, and part comes down to awareness of clinical trials or lack thereof.

· Richie Kahn, Medable PAC member

Initiatives that are tangible and measurable, such as diversity or EHR interoperability, need more specific and more forward-looking regulatory guidance. These are the places that legislation can bring industry innovators into a common sandbox so that valuable resources are spent iterating the next big leaps forward in the ability for a patient to accumulate and distribute their own health records seamlessly. The success of the passage of the 21st Century Cures Act gave bipartisan support to a meaningful initiative in a turbulent political environment. How that translates into passable, actionable, impactful legislation in an expedited fashion remains to be seen.

There is opportunity to build Cures 2.0 to swiftly put wind in the sails of full interoperability of EHRs by making patient data access a key driver of change in placing into the hands of every patient, and their health care providers, the full data of their medical records easily, completely, and understandably.

· T.J. Sharpe, Medable PAC member

As part of the Act, the FDA was required to develop a patient-focused drug development (PFDD) guidance, which helped to increase the visibility of rare diseases at the agency. Through both internal and external PFDD meetings, many industry guidance documents were created for rare conditions.

With 2.0, we should see a focus on pushing these guidance documents out, and finding ways to create metrics to assure the patient voice is included in the regulatory process.

· Jenn McNary, Medable PAC member

Dr. Stephen Hahn, the newly-appointed head of the FDA, discussed three areas of focus at the organization's first all-hands meeting that roughly align with Cures 2.0 priorities: unleashing the power of data, empowering patients and consumers, and supporting innovation and competition. With the FDA seemingly on board with the concepts being presented, there is alignment between Congress and the regulatory agencies on top priorities, which should lead to a more significant influence on the industry converging on commonalities.

1. Digital Health Technologies

As advisors to a technology company within digital health, it is part of our role to be familiar with the areas of weaknesses, pain points, and the opportunities for advancing technologies within the healthcare world - specifically where they are impactful to patients and caregivers. The evolution of technologies will open doors to greater knowledge, greater access, greater support, and, likely, greater outcomes.

There are countless ways an individual could utilize ingrained digital technologies in their health care. Monitoring with mobile devices and services. Personalized information access across digital channels, giving a better picture of overall health. Telemedicine, allowing better access to experts or specialists (particularly in hard to reach populations) while reducing the impact on the quality of life for patients, caregivers, and the Healthcare Professionals (HCPs) who serve them.

We would like to see thinking outside the status quo. Provide all private and Medicare insurance patients with specific reimbursements, linked to their diagnoses, to spend for technology-delivered care they want to access when they want to access it.

· Ingrid Oakley-Girvan, Medable SVP of Research and Strategy

 

Until technology-based solutions such as internet-based video conferencing, mobile apps, and health/fitness trackers are covered by payers, it will be difficult to increase access and show the effectiveness of technology to address health disparities.

· Joni Venticinque, Medable PAC Member

With the development of new therapies for rare and fatal disease, patients need an earlier diagnosis to lead to better outcomes. We know that one-time curative approaches that are now being studied in patients will need to be applied early in the disease process to make the most significant impact. There currently is a distinct gap in care when it comes to diagnostic testing access. Only a handful of known fatal diseases are tested for at birth, leaving families to struggle, sometimes for years, searching for an accurate diagnosis. Insurance often covers more invasive and less accurate tests when full genetic testing would lead to a much faster and accurate diagnosis.

I would like there to be a focus on better access: to testing, better diagnostics, and earlier whole genome sequencing (expanding the newborn screening tests).

· Jenn McNary, Medable PAC member

Digital health is positioned to transform the conduct of clinical trials and the experience of trial participants by removing many of the barriers to trial participation. Logistic issues are a primary driver of under-enrollment in clinical studies for the medically underserved; the physical location of the study, the number of study visits required, and the time commitment required by the study. The tools and technologies now exist to bring clinical trials options to patients unable to make it to a research site in a scaleable, reliable manner. Remote participation can be facilitated via mobile technologies and better mobile trial design, resulting in broader participation, more representative population results, and a faster timeline to make treatments available.

Ideally, these advances in clinical research access can serve to build awareness of clinical research as a care option, particularly for those segments of the population - the homebound; those with limited transportation options; patients unable to make traditional appointments - for whom clinical research participation has been exclusionary. Perhaps the Digital Health Innovation Action Plan can help guide the development of new technologies that can more easily reach historically under-enrolled and underserved patient populations.

· Richie Kahn, Medable PAC member

According to recent surveys, rates of smartphone ownership are 71% for both low-income populations and rural populations. By interacting with the study and researchers via mobile devices, patients can take part in research without having to visit a hospital or site. This technology can increase inclusiveness and diversity, and represent much-needed improvements that have the potential to substantially increase our understanding of disease, how potential therapies work for all patient groups, and capture real-world data to drive new research.

Recruiting study participants is one of the most challenging tasks in the clinical trial process, and arguably one of the biggest barriers to success in clinical research. Enrollment of minority racial/ethnic groups and the medically underserved are particularly unrepresented in clinical trials considering their populations' incidence rate of cancer.

· Joni Venticinque, Medable PAC Member

2. Coverage Reform

Coverage reform encompasses a broad array of topics, including government-funded coverage that currently exists in Medicare and Medicaid. In the digital space, this reform can accompany digital health initiatives already discussed to rapidly implement and adopt meaningful, patient-impactful programs to achieve better healthcare outcomes with less burden. However, without billing codes or other mechanisms to generate revenue, technology companies are unlikely to innovate in this space. Coverage billing codes are essential for technology-based solutions to reach patients when and where they want.

We must do a better job providing rural, mobility-limited, and geriatric patients with rapid access to care solutions. Technology can provide that access through telemedicine and mobile apps for precision education and clinical trial pre-screening.

· Ingrid Oakley-Girvan, Medable SVP of Research and Strategy

Faster approval for rare disease treatments, through the accelerated approval pathway, has highlighted a need for better oversight in the access and reimbursement area. State Medicaid programs are a particularly bad actor, so enforcement of coverage of innovative therapies would be a fantastic achievement for Cures 2.0.

Threatening access to newly approved therapies are entities like ICER, which seek to de-value and result in non-coverage of these novel and often expensive treatments. Cures 2.0 should seek to ban the use of such valuations, which do not serve patients in any way and stifles innovation.

· Jenn McNary, Medable PAC member

Another possible area for significant impact on patients is the ability to shop for the payers who offer digital health as part of their plans. Insurance companies could be rated on their adoption of technology solutions considered in-network, and a score maintained so patients who prefer - or need - technology-based care could shop for providers that reimburse for the types of healthcare solutions they want. The model already exists - ask anyone who has eaten at a New York City restaurant.

There are many levers in the health provision ecosystem. Creating an environment that provides clarity around the benefits of newer innovations, such as digital health and personalized medicine, is a stepping stone for getting these enhancements paid. Providing the ability to pull levers to motivate both the development of solutions and their adoption is critical to innovation, implementation, and wide-scale adoption.

3. Data Empowerment

Two major provisions in the 21st Century cures act addressed the use of patient information: promoting patient access to their medical records and penalizing HCPs and Health IT systems for "information blocking." These innovations are still struggling to take hold, but Cures 2.0 should build on these first steps to further break down barriers to data access. The results of interoperability have yet to trickle down into everyday medicine, and patients still have a difficult time piecing together the entire picture of their medical history.

More troubling, physicians also have a difficult time accessing patient health information outside of their network and integrating that information to provide a holistic view of a patient's health status. Support for innovative platforms is necessary, but equally important are guidelines on integrating data access in real-world settings. Shiny, new tools and technologies will not positively affect the patient experience, and the patient's health outcomes, if they are cumbersome to integrate or significant barriers (both real and perceived) exist. That paper forms are still an integral part of many physician visits is telling about the pace of adoption.

Patients should be empowered to specify how and when their data are shared; not only could this contribute to countless health research initiatives, but it could also offer the patient more information into their own disease journey, enable earlier and better insights on how treatments are affecting them, and stratify results and likely outcomes in a more specific, personalized way to include the advancement of digital biomarkers and phenotypic mapping. Gaining data access to similar patients, with similar conditions and health profiles, is only the first step. Enabling like vs. like comparisons, instead of bigger population ones, now gives data meaning at a more granular, actionable level, improving therapies by enriching populations and having higher efficacy due to patient similarities.

 

Two areas of data empowerment that can have an immediate impact with relatively low integration efforts are Real World Data/Real World Evidence (RWD/RWE), and clinical trial results return. In the RWD/RWE realm, this means reaching beyond the existing eCOA/ePRO solutions and embracing the rapidly changing, and rapidly expanding, suite of technologies to capture, measure, and extrapolate data for both more personalized healthcare and better population health information. The needed collaboration between regulatory, industry, and healthcare institutions is possible - ASCO's CancerLinQ being a prime example - but initiation, momentum, and funding across the healthcare landscape for widespread RWE capture and utilization would benefit exponentially from legislative support.

For clinical trials, there is significant opportunity to both publish ALL trial results - a simple, basic step mandated by law - and returning specific trial results to participants. Getting results back to clinicaltrials.gov is simply a matter of enforcing legislation already in place. Failure to report the results of a clinical trial diminishes the critical contribution of study participants, keeps valuable evidence from being used in clinical practice, and results in wasted resources spent on medical research. That this is still an issue (that the NIH and FDA continually turn a blind eye to) is frustrating and derogatory to optimal patient outcomes. Even "clinical trial shaming" has not significantly moved the needle for basic trial results reporting; one of every three trials do not report results, and the FDAAAA of 2007 allows for fines of $10,000/day for non-compliance. There is a staggering $7.5 BILLION in fines that could have been imposed and wasn't, money that could fund numerous 21st Century Cures and Cures 2.0 initiatives while, theoretically, addressing one of the things the Acts are trying to fix.

Related, it is absurdly simple to broadcast updates on clinical trials, on an opt-in basis at the trial or biologic level. Creating a common, standard, vetted method of allowing patients, caregivers, family, and any other interested party to opt-in would satisfy the need for patients to understand what the outcome was of their contribution to medicinal progress.

That trial results, and their impact on a group of patients, are still being withheld is a travesty. Patients sacrifice their time, their biology, and their privacy to enable treatments to get to other patients - and allow pharmaceutical companies to develop, patent, market, and profit off of drugs based on those sacrifices. The ease of information distribution is only trumped by the industry's consistent reluctance to make results return a priority instead of a nuisance and compliance cop-out. Clear, ENFORCED regulation would be a significant win for patients at negligible costs for trial sponsors.

· T.J. Sharpe, PAC member

 

While the publication of clinical trial data are a must, it is merely the first step in overhauling the information sharing process. Patients are still impacted by their Electronic Medical Records systems holding data hostage, as opposed to sharing a 360-degree view of that patient's health with ALL of their healthcare providers, including their study trial team. Creating seamless, secure, and comprehensive medical records will impact treatment efficacy and, more importantly, patient safety. Concomitant medications and medical history cannot be inadvertently forgotten if this data are linked to a patient electronically. Deterministic data matching is a must to help improve outcomes.

4. Healthcare Support

The topic of healthcare support touches every patient, and caretaker, on the Medable PAC. Increased support - through literacy, caregiver support, treatment options, and financial support - are known to better clinical outcomes, improve patient satisfaction, and assist with coping. Support of some type exists for nearly every patient, but too often, they are siloed, poorly funded, and are unable to be easily accessed by the patient/caregiver at the exact time of need. It is a need that has been articulated by patient communities across all demographics.

I am particularly heartened by the desire to improve the ability of families and caregivers to support their loved ones. I love the focus on health literacy and the recognition and focus on the caregiver as well.

· Richie Kahn, Medable PAC member

At the 2019 American Society of Hematology annual meeting, evidence was presented that disparities in cancer care for the socioeconomically disadvantaged may disappear with broader support mechanisms in place. Although this was one study of one disease at one institution, the implications are widespread across the entire healthcare ecosystem. If patient navigation was a billed/covered expense, then its effectiveness regarding patient outcomes and costs could be measured.

Patient navigation and health literacy are paramount for positive healthcare outcomes. Patient navigation services have been rapidly expanding but with no consensus as to what is actually done. It is known that navigation services can help coordinate patient care so that nothing "falls through the crack," connects patients and caregivers with needed social and financial support, and helps patients understand their diagnosis and their treating healthcare system.

· Joni Venticinque, Medable PAC Member

To help patients get assistance, metrics for incorporating the patient voice in healthcare are needed; as a Council, we have encouraged Medable to develop a strategy for measuring the effectiveness of PAC to refine our impact to the company and to the patient groups they serve - and we represent. Quantifying protocol co-creation is one step towards requiring patients with a disease to be equally represented during the design and revision process.

The California Breast Cancer Research Program (CBCRP) describes the benefits of patients being integral to research:

A growing body of evidence shows that health and medical research benefits from being directly informed by the experiences and knowledge of those affected: those who have or had the disease, those who care for people with the disease or those who represent a specific community impacted by the disease. These people can become advocates for their community by helping scientists identify the needs of patients, families and communities and offering a practical perspective on research topics, methods and results.

 

As educated patients and caregivers, the members of the Medable PAC applaud the inclusion of more significant healthcare support as a top priority of Cures 2.0.

Looking Ahead

Since the 21st Century Cures Act was passed in late 2016, progress has been made towards several of the Act's stated goals. The second iteration, Cures 2.0, looks to build on the momentum generated by the original legislation while also better incorporating the needs of patients, caregivers, and their families.

The three industry/technology areas of focus - digital health technologies, coverage reform, and data empowerment - all have similar themes flowing through them. Get the right information into the right hands at the right time to make the right decision for every medical situation. That can mean standardizing and utilizing the digital innovations already in existence to positively disrupt patient access to personalized diagnoses and medicines, including clinical trials. It can mean revamping the coverage ecosystem to empower patients as the driver of their health decisions, instead of the payer. It means capturing the right data, being able to use it, and then ensuring that it is available to use for every person at every provider, every time.

The topic that hits especially close to home for many, the Medable PAC included, though, is healthcare support. Higher-quality and better-tailored support can provide improved access and information, leading to a higher quality of life, fewer burdens, and, hopefully, better outcomes - all which impact the patient and their support system.

It takes the voice and the will of the people to transform how healthcare is delivered. As a collective patient voice, the Medable Patient Advisory Council members are committed to representing that voice as a guiding force towards building a better healthcare experience for all as we support the objectives of Cures 2.0.

 

Topics: PAC
7 min read

Foresight is 20/20 - Drug Development's Future

By Jennifer McNary on May 12, 2020 11:32:02 AM

COVID-19 has taken us all by storm, and as a result, much of the world has come to a grinding halt.. As you can imagine, this includes the clinical trial universe, as well. Healthcare resources are being diverted, rightfully so, to combat the pandemic. Suddenly, the world is clamoring for what many in clinical research have aspired to for years - expedited clinical trials leading to treatments to patients faster. Perhaps the coronavirus is gifting us a glimpse into the future of drug development.

Existing Trials: Adapting in Real Time

Globally, there is a pandemic unfolding, rippling across countries and targeting everyone in its path. No one - not Hollywood, athletes, politicians (or their spouses), royalty - is safe, but the eldery and the immunocompromised seem to be particularly vulnerable to the virus causing serious or life-threatening problems. Hospital systems are stressed domestically and on the verge of collapse in other parts of the world. Pharmaceutical companies are beginning to ration their resources, delaying start-up or enrollment to lessen the burden on existing healthcare sites.

For clinical trials that are able to move forward, study teams are scrambling to minimize disruption, both to get existing patients treated and maintaining the trial’s integrity in the face of an unparalleled disruption. Veer too far off course, and current and future patients suffer costly, potentially life-threatening delays. To prevent this, adjustments to standard operating procedures have to be made, whether the slow-to-adopt-change world of clinical research wants to or not.

The FDA has, very quickly, issued guidance on conducting clinical trials during this pandemic. It reads as a sort of wish-list of potential operational advances in clinical research:

  • “Since trial participants may not be able to come to the investigational site... sponsors should evaluate whether alternative methods for safety assessments (e.g., phone contact, virtual visit, alternative location for assessment, including local labs or imaging centers) could be implemented”
  • “The need to put new processes in place or to modify existing processes will vary by the protocol and local situation.”
  • “Sponsors and clinical investigators are encouraged to engage with IRBs/IEC as early as possible when urgent or emergent changes to the protocol or informed consent are anticipated…FDA recommends consultation with the appropriate review division regarding protocol modifications for the collection of efficacy endpoints, such as use of virtual assessments, delays in assessments, and alternative collection of research-specific specimens, if feasible.”
  • “The implementation of alternative processes should be consistent with the protocol to the extent possible, and sponsors and clinical investigators should document the reason for any contingency measures implemented.”
  • “Changes to policy and procedures could address, but not be limited to, impact on the informed consent process, study visits and procedures, data collection, study monitoring, adverse event reporting…”

 

Think of ways to bring the study to the patient.

Consider how to be flexible.

Engage early with stakeholders.

Accept variance will happen; proactively address, and then record why.

The notoriously reserved clinical research industry is now faced with a decision: adjust your strategy or drop out of the race altogether. Instead of resisting change, adapt and adjust. This is what the future of clinical research will look like - more agile, less rigid; more adaptable, less authoritarian; more personalization, less one-size-fits-all. Most importantly, more human.

COVID-19 Trials: The Future is Now

While existing trials struggle to find their way through this uncertain time, the barriers to clinical research have come down for COVID-19 studies. As of March 23, 2020, there were already five completed studies for COVID-19, and by the time you read this, there’s likely to be more. Notably, there are 113 studies somewhere between “Not yet recruiting” and “Active, not recruiting”, with 62 in recruiting/enrolling/active status. Considering that the World Health Organization wasn’t notified until the very end of 2019 about this new virus, and the average study start-up time is north of 7 months, the swift response to this global pandemic has been unprecedented.

Why has the COVID-19 reaction been so drastically different from “normal”? Was it the life sciences industry changing their standard operating procedures to adjust for the surge in demand for health care? Was it capitalism - and consumers - pushing for a product that a market was literally dying for? Or was it the world collectively realizing that the typical barriers to innovation were not nearly as important as getting treatments or vaccines to patients as quickly and safely as possible? And, importantly, what are we learning about the pace at which clinical research can be done?

First, the caveats - COVID-19 is unlike anything in the modern medical world, so every stakeholder in the drug development process is likely willing to bend on strict adherence to dogmatic Standard Operating Procedures. Many of the trials are for either existing compounds (eliminating the need for a Phase I study) or for diagnostics, both of which accelerate the startup timeline. Physicians and governments are desperate for ANYTHING that could help.

That being said, this is the same clinical research world that regularly regurgitates the following median numbers - seven years, $750 million+, and a success rate of 1 in 5,000, all to get one drug to market. In Duchenne Muscular Dystrophy, it took 8+ years to get exon skipping from mouse models to the approval of Exondys51, the first treatment approved in the United States for DMD. Approval for new sunscreen ingredients languishes, some for the better part of two decades. Even Merck’s hyper-accelerated timeline for Keytruda approval - less than 5 years, an unheard-of number in oncology treatment - was still glacially, frustratingly slow to those who could not access the drug.

 

The COVID 19 trials were enrolled and complete stunningly quick because:

  • Populations of patients were easily identified - this illness could affect ANY and EVERYONE, and potential cases were being brought forward on a daily basis,
  • Time was THE most critical factor; the normal back-and-forth during study start-up was plowed over with compromise,
  • Existing compounds already had safety data, thereby allowing researchers to skip time-consuming early-phase trials, and
  • Pressure was applied universally - from governments, health care facilities, and most importantly, potential patients - to get treatment.

 

The catchphrase “we are all patients” has never been more applicable, and every stakeholder in this specific development process worked collaboratively to make these trials happen, and happen quickly. Technologies already exist that facilitate all of the above FDA considerations (and a slew of new ones are appearing to specifically address COVID-19), yet adoption prior to 2020 was slow and deliberate. Now, the leadership team at Medable has been actively promoting and incorporating decentralization of trials for COVID-19, getting a trial mobile app live in Italy in under two weeks. Drive through testing has shown mobile healthcare is deployable on a large scale. Virtual trials are on the horizon. Clearly, we can do better than we were - we are now.

Key Takeaways From COVID-19

There are numerous positive things to take from the response to the current pandemic and push forward as our world slowly comes out of its hibernation.

 

The US FDA can have flexibility in standards. There remains a shortage of diagnostic tests for the detection of COVID-19. In response, the FDA allowed some labs to begin using validated tests before the regulators had finished their review. We will (hopefully) find out soon if the same acceleration will occur if and when a treatment is found; the FDA continues to work with the public and private entities researching those treatments. It is important to balance the rigorous standards for efficacy and safety with the demands for improved and accelerated healthcare.

 

Guidance that is well-crafted and useful to both patients and sponsors was able to be published in less than 30 days. Too often, this guidance is delayed. The rare disease community has been waiting over 18 months for the new rare disease drug development guidance. The FDA needs more resources to accelerate the rate of product development.

 

Embracing digital medicine and digital health tools will continue to drive healthcare modernization and mobilization, while simultaneously working to remove man-made barriers to effective and efficient clinical research practices. The world is now pilot-testing-by-necessity many of the technologies that sound great in presentations, but have been largely kept on the sidelines by risk-averse trial sponsors and sites.

 

Trial design will continue to evolve, and the use of non-traditional trials - virtual, decentralized, direct-to-patient, whatever they are called - should become more commonplace, as sponsors realize they can reach broader patient populations in more effective ways. Trials will be brought to patients, instead of the other-way-around.

 

Deadly illness is deadly illness, whether a pandemic or a fatal disease. The urgency with which we develop therapies needs to mimic the response and flexibility shown with COVID-19. Let’s not let this crisis - and the accelerated pace of research it forced - go to waste.

Topics: telemedicine decentralized trials
3 min read

Mobilize and Decentralize

By Allison Holland- Head of Decentralized and Remote Trials on May 12, 2020 11:29:18 AM

Taking Action to Ensure Clinical Trial Progress During COVID-19

Why does it take so long to get new vaccines and medicines to market?

 

It is not a new question. But as governments, communities and medical professionals around the world focus their energy and resources on containing the COVID-19 pandemic, it’s being asked with increasing urgency. It deserves – in fact, it requires – our collective attention and effort.

The good news amidst so many troubling headlines is that the technologies we need to improve patient access and experience are here now. The smart application of those technologies can overcome barriers to trial execution and improve data sharing and process efficiency across organizations. Most urgently, those technologies can be used immediately to ensure progress for thousands of clinical trials in an environment where patients are expected to stay at home.

 

Decentralized and hybrid trials – the time has come

At a recent congressional hearing, NIAID director Anthony Fauci gave a frank assessment about the shortcomings of the U.S. system for coronavirus testing: It’s “not really geared to what we need right now.”

The same can be said for our traditional clinical trial model. Limiting trials to a handful or two of physical sites inherently limits patient access, while limiting interaction to in-person visits is not only grossly inefficient in many cases, but it also limits data frequency and quality. And during a crisis like the COVID-19 pandemic, it’s simply infeasible.

By contrast, decentralized clinical trials look more compelling than ever. The Clinical Trials Transformation Initiative (CTTI) defines decentralized trials as those trials executed through telemedicine and mobile/local healthcare providers, using procedures that vary from the traditional clinical trial model. In layperson terms, the trial is conducted remotely with the participant remaining at home.

The industry has been looking to decentralize trials for years. Now, as health authorities worldwide struggle to contain the COVID-19 outbreak, there is a renewed push to rapidly implement remote healthcare delivery capabilities.

There are currently more than 55,000 interventional clinical trials actively enrolling and providing care for participants worldwide. In light of the current outbreak, it is critical that we continue to deliver high-quality healthcare to research participants, while also continuing to advance clinical drug development programs.

Decentralized trials are largely “geared” for exactly this type of situation. Of the 55,000 trials in flight, some are good candidates for a fully decentralized model — while many others can be managed in a hybrid model. Patients can be recruited and consented remotely. Physician “visits” can be conducted remotely via telemedicine. Data can be captured remotely (and frequently) via medical devices and mobile technology.

All of this expands our ability to conduct research by “untethering” it from physical sites — critical when people around the world are being told to “stay home” due to the COVID-19 pandemic. It reduces the risk of pathogen exposure to research participants, while potentially accelerating drug and vaccine development.

Shifting to decentralized or hybrid trials often requires changes to study design or regulatory approvals. While that’s no small task, it’s an urgent one for many clinical trial leaders right now. We need to take collective action as an industry — including regulators — if we want to ensure productive client trial progress and avoid significant loss of patient participation.

 

Let’s mobilize

With all of this in mind, there is an immediate opportunity for decentralized and hybrid trials to help us through these challenging times, using digital and mobile technologies to improve patient access, experience and outcomes. Let's work together as a community to drive forward faster, whether it’s streamlining trials for COVID-19 vaccines, reducing timelines for other therapies, or initiating new trials to address the 7,000 rare diseases that have no therapies on the market.

 

The COVID-19 outbreak has made it crystal clear: Decentralized trials are no longer a nice-to-have. Reducing trial timelines has to be a long-term industry imperative, and digital technology can help valuable research continue to move forward while keeping participants safe. Let’s start now, and let’s move faster together.

 

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If you want to join us in this effort, please contact Covid19@medable.com or reach out to anyone on the Medable team at www.medable.com. We’re working closely with regulators, pharma sponsors, biotech sponsors, clinical research organizations and other tech companies worldwide in an effort to mobilize and accelerate decentralized trial adoption. We welcome everyone’s participation and feedback, and we look forward to working with you.

 

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Alison Holland is the head of decentralized and remote trials at Medable. Ali has more than 30 years of clinical trial experience, most recently as Global VP & General Manager for General Medicine at Covance, a leading global clinical research organization. Ali has managed more than 300 clinical trials, working successfully with biotech organizations as well as global pharma on some of their most critical initiatives.

 

Topics: telemedicine decentralized trials remote trials
8 min read

How Decentralized Trials Accelerates Rare Disease Research

By Jena Daniels- Director of Research on May 12, 2020 11:28:48 AM

David Fajgenbaum’s life changed forever one summer day in 2010. After experiencing fatigue, his liver, kidneys, bone marrow, heart, and lungs suddenly began to shut down. He was admitted to the intensive care unit where he had a retinal hemorrhage that left him blind in his left eye. David drifted in and out of consciousness, was put on a feeding tube, and received many blood transfusions. In weeks, the 26-year-old medical student sharply deteriorated and was given last rites.


David was diagnosed with a multi-centric form of Castleman disease (CD), a rare disorder where the immune system attacks and shuts down the body’s vital organs. Like most rare diseases, CD does not discriminate and can occur in people of all ages, genders, races and ethnicities. After many months of hospitalization, David miraculously recovered and returned to medical school. He was put on an experimental drug called Cetuximab, the first drug to ever undergo a randomized controlled trial for CD – and the only drug in development for a disease that was identified nearly 60 years prior. His prognosis, though, remained unclear.

 

In the U.S., between 6,500 and 7,500 new CD cases are diagnosed each year compared with 220,000 new cases of lung cancer. Consequently, little research has been done for CD. At the time of David’s first episode, there were no diagnostic criteria, treatment guidelines, or FDA-approved treatments. Worse, there was no research infrastructure in place to find a cure: no registries or biobanks, no federal funding, and no collaborative research network to advance understanding.

 

This is the same story for most rare diseases, even today with scientific advancements that cure cancer. Collectively, 300 to 400 million patients suffer from nearly 7,000 different rare diseases. Currently, 95% of rare diseases do not have a single FDA-approved drug treatment, which means, in the U.S., more than 30 million people living with rare diseases have few to no options. There are many reasons for the lack of rare disease research – small patient populations, heterogeneous disease characteristics, lack of scientific knowledge, and absence of historical data – but one of the biggest hurdles is today’s clinical trial model that makes it difficult to recruit enough patients to generate statistically meaningful results.

 

“In rare disease, it’s difficult to reach a representative sample of patients simply because trials are few and far between…literally. One of the trials that responded to me was based in Belgium, which was simply impractical,” said Alycia James, a healthcare consultant based in Wilmington, NC, living with Myasthenia Gravatis, a rare neuromuscular disease. “It we could use digital tools to connect to the site without physical visits, more patients could participate in more trials. And the technology is here.”

Indeed, the technologies needed to improve patient access and experiences are here now. The smart application of those technologies can overcome barriers to trial execution and improve data sharing and process efficiency across organizations conducting rare disease research. And, most urgently, those technologies can also be used immediately to ensure progress for thousands of clinical trials during the current COVID-19 pandemic when patients are expected to stay at home.

 

The Need is Grave, The Technology is Here

At a recent congressional hearing, NIAID Director Anthony Fauci gave a frank assessment about the shortcomings of the U.S. system for coronavirus testing: It’s “not really geared to what we need right now.”

The same can be said for our traditional clinical trial model. Limiting trials to a handful or two of physical sites inherently limits patient access especially patients with rare diseases – more than 50% of whom are children, dependent on their caretakers. Further, limiting interaction to in-person visits is not only grossly inefficient, but it also limits data frequency and quality. And, during a crisis like the COVID-19 pandemic, it’s simply infeasible.

By contrast, decentralized clinical trials are more compelling than ever – and ideal for exactly this type of emergency situation. While some trials are good candidates for a fully decentralized model, others can be managed as a hybrid. Patients can be recruited and consented remotely. Likewise, physician visits can be conducted remotely via telemedicine and data can be captured remotely (and frequently) via medical devices and mobile technology.

The Clinical Trials Transformation Initiative (CTTI) defines decentralized trials as those trials executed through telemedicine and mobile/local healthcare providers, using procedures that vary from the traditional clinical trial model. In simplest terms, the trial is conducted remotely so the patient remains at home. These studies often require wearable sensors and mobile health applications that allow the collection of important physiologic data such as respiratory rate, heart rate, temperature, and blood pressure.

For example, a new mobile application called Trial-Fit TeleVisit allows patients worldwide to connect virtually with clinical trial sites. The application enables the following:

● Patient consent for the use of telemedicine in their native language;

● Real-time video capabilities on a single platform that is 21 CFR, Part 11 compliant; and,

● Virtual connections for patients with their site coordinators, investigators or other care professionals from anywhere in the world.

 

The industry has been looking to decentralized trials for years. Now, as health authorities worldwide struggle to contain the COVID-19 outbreak, there is a renewed push to rapidly implement remote healthcare delivery capabilities. It’s critical that the industry continues to deliver high-quality healthcare to research participants, while also continuing to advance clinical drug development programs to find treatments for rare and other diseases.

 

Decentralized Benefits to Rare Disease Research

There are more than 55,000 interventional clinical trials actively enrolling and providing care for participants worldwide, but only approximately 3% of potentially eligible patients enroll. For many, low enrollment is due to a lack of awareness – this is especially true for rare disease trials. For others, low enrollment is caused by lack of access. Decentralized trials open the door for participation to a much broader audience so that a trial being conducted in Belgium now becomes feasible for rare disease patients like Alycia James living far from that country. Decentralized and hybrid trials increase enrollment and enable greater patient representation that is missing in many rare disease studies.

 

For instance, direct-to-patient decentralized trial technology allowed one mid-sized pharmaceutical company to not only conduct reverse feasibility for site selection, reducing the projected number of sites from 100 to 25, but also saved the company over $20M in study-related costs. The study needed to pre-screen more than 11,000 patients with a rare genetic variant that only affects roughly 2% of the population. By utilizing decentralized solutions, patient enrollment increased two-fold in half the time and reached underrepresented populations, improved patient data capture, decreased patient and site burden, and enabled real-time data capture.

 

Another benefit of decentralized trials is that they provide more and higher quality data. When you have a participant filling a diary or recounting specific events that are pertinent to the trial, you have an element of retrospective bias or inaccurate data capture. Decentralized trials enable patient reporting at the moment in which an event occurred for increased accuracy. For example, if the patient had an adverse event like a headache, they can capture the event at that specific moment while they are experiencing it. Patients can also capture a broader set of contextual data, such as their location when they had the episode or whether they were sleep deprived, hungry, etc. Researchers can use the contextual data to better inform about triggers around the adverse event.

 

An additional advantage for rare disease research is the ability to continuously collect data about a treatment over a long period of time – this is particularly important with rare disease research since is so little known about its various forms, like David’s CD. Remote technologies like wearable devices in conjunction with mHealth applications can track symptoms without pause.

 

“Many rare disease patients have good days and bad days, and it’s often unpredictable. Many of the treatments, too, take months to work,” explained James. “Digital technologies can automatically track and measure the impact of an experimental treatment over time since patients may not realize results right away. For me, I have to actively think what my physical abilities were two months ago to realize whether I’m seeing any benefit from a new medication.”

 

Untether from the Past for a Brighter Future

Decentralized trials expand and accelerate the industry’s ability to conduct research by untethering it from physical clinical trial sites. This is certainly critical today as people around the world are being told to stay home due to the COVID-19 pandemic while trials need to continue without the risk of pathogen exposure. Yet, moving closer to perfecting this new model just as critical for the future to enable rare disease research that involve small patient populations far-flung around the globe while enabling research continuity in the inevitable event of future pandemics.

 

Shifting to decentralized trials will require changes to study design or regulatory approvals and there will be other important considerations regarding data collection -- to be clear, there’s no “one size fits all” solution. However, if there is a silver lining to the recent pandemic is the opening of our collective eyes to the great potential for better clinical trial execution with decentralized trial technologies. While that’s no small task, it’s an urgent one for many clinical trial leaders right now.

 

With all of this in mind, there is an immediate opportunity for decentralized and hybrid trials to help us through these challenging times, using digital and mobile technologies to improve patient access, experience and outcomes. Let's work together as a community to drive forward faster, whether it’s streamlining trials for COVID-19 vaccines, reducing timelines for other therapies, or initiating new trials to address rare diseases.

Fortunately, there are a large number of new development programs for drugs targeting a wide range of rare diseases. According to a 2019 Tufts University study, rare disease drug development is one of the fastest-growing areas in research and development, accounting for as much as one-third of products in the pipeline. BIO also reports almost every pharmaceutical company has, or is opening, a rare disease division. The path to overcoming the anticipated research hurdles runs through new remote and digital technologies.

David Fajgenbaum, tenuously stable, understands this better than most. After graduating from medical school, he embarked on a mission to find a cure – knowing that his symptoms could return at a moment’s notice. He established the Castleman Disease Collaborative Network (CDCN) and his volunteer army has taken back momentum in the war against the disease. The CDCN is using technology and other resources to crowdsource the most promising research questions and recruit world-class researchers to tackle them.

Decentralized trials are no longer a nice-to-have. Reducing trial timelines has to be a long-term industry imperative, and digital technology can help valuable research continue to move forward while keeping participants safe. Let’s start now, and let’s move faster together.

 

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If you want to join this effort, please contact Covid19@medable.com or reach out to anyone on the Medable team at www.medable.com. We’re working closely with regulators, pharma sponsors, biotech sponsors, clinical research organizations and other tech companies worldwide in an effort to mobilize and accelerate decentralized trial adoption.

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About the Author

As Director of Research at Medable and part of the Digital Science Team, Jena Daniels addresses persistent problems, disconnects in care, and disparities in various therapeutic areas through collaborative projects that use innovative methods to connect patients, clinical teams, patients, and caregivers. In 2019, Daniels co-established Medable's Patient Advisory Council, which engages and empowers patient advocates to assist product development and research teams to develop meaningful, actionable, and patient-focused solutions. Additionally, Daniels is responsible for completing milestones for Medable's NIH NCI SBIR awards and assists in developing strategic partnerships.

 

Prior to joining Medable, Daniels was a clinical research manager at Harvard Medical School and Stanford University School of Medicine. She has published more than 20 peer-reviewed publications that focus on how to leverage machine-learning, artificial intelligence, and remote patient monitoring to triage, diagnose, and provide early intervention for children with autism spectrum disorder. Daniels can be reached at jena@medable.com.

 

 

Topics: decentralized trials