Contributed: Trials and tribulations – the new world of decentralized trials

Asset Management Ventures’ Skip Fleshman outlines COVID-19’s impact on clinical trials and the resulting leap toward tech-enabled decentralized trials.

Clinical trials have become big news this year amid the search for COVID-19 vaccines and treatments. They are a critical component of helping the FDA validate that new drugs and diagnostics will be both safe and effective.

Pharmaceutical companies spend over $45B a year running clinical trials, which are, by design, very methodical and measured. Unfortunately, they are also slow, unpredictable and massively inefficient. Some estimates indicate that every day a phase 3 clinical trial slips costs the pharma company as much as $8M.

There are a lot of problems: Recruitment is often difficult, and the cost to find patients is expensive. Protocols, which can often change, may require patients to travel to specific locations or have specialists administer treatments or diagnostics.

Communication between clinical trial sites and patients is difficult. Communication between trial sites and site sponsors (pharma) is still typically done via phone or fax, and data collection is often done in Excel or on paper.

While digital health solutions have broadly improved healthcare with better therapeutic interventions, medical record tracking, billing and hospital operations, they have done little to transform the inefficient clinical trial landscape.

Furthermore, COVID-19 has made matters worse. During the first three months of lockdown, most clinical trials were effectively put on hold as patients were unable or unwilling to travel to trial sites.

During the COVID-19 pandemic, pharma companies have had to improvise on how they think about setting up, administering and monitoring trials. New digital health entrants are helping manage these changes, and a few new trends are emerging. The most prominent is the use of technology that enables trial participants to accomplish their regimen remotely from clinical trial sites.

The clinical trial landscape

First, here are some basics about how trials are administered. Clinical trials are prospective biomedical or behavioral research studies on participants, designed to answer specific questions about medical interventions. These include both new treatments as well as known interventions that warrant further study.

For decades, clinical trials have required participants to fit certain criteria, follow specific protocols and have treatments administered and measurements taken at clinical trial sites. These sites are generally administered by a physician at healthcare facilities or academic medical centers.

Initial trials (Phase 1) usually have a small number of subjects and are structured to determine if the treatment is safe. Phase 2 and Phase 3 studies assess efficacy, safety and side effects, and are larger in scale, often with hundreds or thousands of patients at dozens of research sites that may be international in scope.

The Emergence of decentralized trials

So where are we today? As mentioned earlier, COVID-19 has dramatically increased pharma’s desire to conduct trials away from traditional sites; the FDA refers to these as “decentralized clinical trials.” The FDA defines decentralized trials as those executed through telemedicine and mobile/local healthcare providers, using processes and technologies that differ from the traditional clinical trial model.

This decentralization eliminates the need for patients to come to a specific clinical trial site, resulting in many benefits. A primary advantage is that the pool of potential clinical trial participants is greatly expanded. Recruitment for trials is an expensive proposition (the average cost of recruiting a phase 3 patient is $36,000), and without the need to travel to specific sites, more people would be willing to participate, thereby lowering costs.

In addition, remote participation helps patients with mobility issues, as well as patients who would otherwise have to board an airplane or even relocate for specific trials.

The concept of decentralized trials has been around for a while. One startup company, Science 37, founded in 2014, started off by working on trials administered in patients’ homes. The company would send connected devices such as a scale or blood pressure cuff to the home and, if needed, order a phlebotomist to collect blood samples.

In the years since then, there had not been much progress in trials like this. However, due to constraints imposed by the COVID-19 pandemic, decentralized trials have again become a significant area of interest for pharma companies and investors. Indeed, from March to June 2020, most pharma companies did not initiate any new clinical trials as the industry was forced to rethink how trials are set up and administered.

How COVID-19 has affected trials

One approach would be to enable clinics not designated to run a specific trial to administer the proper protocols, including documentation and administration, to run the trials in more localities. For instance, a trial could be run at perhaps 30 primary sites, but also have hundreds of additional patients participate at their own physician’s office. This increases the pool of patients by adding those who won’t or can’t travel to a designated site.

Science 37 has modified their technology to facilitate this approach. Asset Management Ventures (AMV) portfolio company Freenome, for example, has a major CRO setting up clinical trial sites for them in their 14,000-person colorectal-screening trial, named PREEMPT CRC. To ensure diversity and broad access to much-needed cancer-screening procedures, the company is also setting up their own sites and using Science 37 to administer decentralized trials at a patient’s local clinic or hospital.

This multi-pronged approach has enabled this trial to be able to recruit participants from almost any zip code in the country, as opposed to the relatively few primary sites and zip codes covered by standard trials.

For their COVID-19 study, Eli Lilly has taken a different decentralized approach that is dramatically speeding their recruitment. They recently announced a partnership with Care Access Research and the use of mobile labs to rapidly enroll patients residing in skilled nursing facilities and nursing homes.

The moment a patient at a location tests positive for COVID-19, a mobile team rolls in with testing kits, a research coordinator, nurses, and physicians, and begins enrolling patients for their novel drug trial. The teams use the SaaS platform StudyTeam from Reify Health (an AMV portfolio company) to manage patient recruitment for the decentralized trial in one place and streamline communication between the teams on the ground and the pharma sponsor, Eli Lilly.

Evidation Health, which is also an AMV investment, focuses on recruiting patients at scale and collecting real-world evidence for phase 4 studies. Evidation has over four million users on their Achievement platform, where users consent to share their data (health records, genomic, surveys, labs, etc.) for studies that help pharma companies figure out how their drugs work in the real world. Companies often use Evidation study data for label extensions or to generate publications on how to use a drug or make it more effective.

COVID-19 has already catalyzed the telemedicine industry (Teladoc stock is up 2X since the pandemic) and the remote patient monitoring industry (Livongo sold for $18B), and it is driving strong adoption of technology in the clinical trial sector as well. The biggest impact has been pharmaceutical companies quickly modifying their approach to trials by using technology to implement decentralized trials. Given how much these technologies increase patients’ willingness to participate in trials, we expect the trend toward decentralized trials to continue to accelerate in the coming years.

Skip Fleshman (@skipfleshman) is a managing partner at Asset Management Ventures (AMV).  AMV is an investor in Freenome, Reify Health, and Evidation Health.

Original of this article is available on the Mobile Health News website.