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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
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
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