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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
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
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.

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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

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.

 

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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

 

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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
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
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