SESSION 1: OPPORTUNITIES TO IMPROVE CLINICAL TRIALS

Clay Johnston reiterated the point that traditional clinical trials are becoming more expensive, inefficient, and inaccessible over time. Given this dysfunction, he highlighted the need for the next generation of clinical trials, which, of course, should incorporate new technologies. Johnston then emphasized comments by workshop participants, such as the importance of using human-centered design and seeking input from patients early in the trial design process. He noted that several speakers stated that the main barriers to progress were a lack of will and creativity. If that is the case, he pointed out that education, reassurance, and demonstrating value would be key to changing the way clinical trials are run.

Linda Brady echoed some of Johnston's comments, adding that while there is reluctance across the different sectors for various reasons (e.g., concerns about the regulatory path forward, lack of experience engaging patients, or fear of the technology), the time to move forward is now. Brady said there seems to be opportunities for the National Institutes of Health (NIH) to do more in this space.

SESSION 2: EXPLORING CLINICAL TRIALS

Recapping the highlights of Session 2, Kelly Simcox said that remote digital tools are clearly having success in clinical care. What still needs to be defined, she said, is how to use these tools in a clinical trial beyond observational studies. Interventional clinical trials have made good use of virtual or direct-to-participant methodologies when digital tools are incorporated into the trial design from the beginning rather than when a traditional clinical trial, with all of its complexity, is later modified to incorporate digital tools. Another point she made regarding interventional trials was the importance of considering what participant access to data looks like in the virtual environment. Although returning data to participants in a virtual environment should be easier, the challenge is how to do this effectively and quickly, she said. Another challenge in this space is in regard to validating a participant's identity in a remote setting.

Simcox noted that there is still work to do regarding the use of electronic health record data given that some of these data may not be reliable or may be missing. She also pointed to the need to have more visibility regarding what clinical trials are occurring in the direct-to-participant space and the need to share the learnings gained from those trials so that direct-to-participant trials can move past doing pilots and become part of the mainstream of clinical trial methodology.

SESSION 3: ACCESS AND EQUITY

Kathy Hudson and Rebecca Pentz recapped some of the key points they heard. They emphasized the importance of relaxing exclusion criteria when they are not needed so that equitable participation in clinical trials can be increased. Another point they raised was the importance of continuous dialogue with patients throughout the entire trial process, from design to completion, to adequately capture their input. Hudson and Pentz also emphasized the importance of community engagement, noting that coordinating with community- and faith-based organizations, such as the African Methodist Episcopal Church in the African American community, can build trust in the trial process. Furthermore, returning data to community members in real time can empower participants and make them feel like partners in the clinical trials team. It will be important, they emphasized, to include partnering community organizations in the trial budget. If the goal of a study is to identify ethnically or disease severity-linked biomarkers, Hudson and Pentz noted that it will be important for investigators to think creatively about ways to engage diverse populations.

SESSION 4: POLICY CONSIDERATIONS

John Wilbanks said his key takeaway from the fourth session related to the disconnect between the technology needed for virtual trials and the policy environment. He also highlighted the importance of paying attention to the impact that policies may have on clinical trials, particularly given that most trials will be hybrids of clinic-based and decentralized trials in a way that does not necessarily align with the current policy infrastructure. He noted that industry's aversion to the risk of running afoul of regulations is something that policy can ameliorate through guidance.

One subject that did not come up, but that concerns Wilbanks, is the need to think seriously about the technical security associated with consumer-grade digital health technologies, which he said are “bleeding data out into the universe.” Along those same lines, he noted the importance of consent as an ongoing relationship, not a one-time transactional gate, when digital health technologies are passively collecting data.

Wilbanks suggested that the field look for places where federal and state regulations could be harmonized. He also suggested there could be ways to create “safe harbors” that would enable researchers to experiment and validate new approaches.

David McCallie added that he came to the workshop thinking that observational and interventional trials were two distinct types of trials, but he now sees more of a gray scale of gradation between those two. The regulatory environment changes along that continuum and can sometimes lead to confusing, overlapping, and conflicting policies. To address this problem, he emphasized the value of creating a taxonomy of the spectrum of methodological choices. Doing so, he suggested, could result in clever approaches for attacking the problems that research and clinical trials are trying to address without going through the formal clinical trial channel.

NEXT STEPS

Johnston discussed the value of establishing a regularly updated knowledge repository for sharing information and lessons learned based on ongoing and completed virtual trials (e.g., Health Insurance Portability and Accountability Act [HIPAA] considerations and state-based policies). Leanne Madre noted that as part of its mobile clinical trials programs, the Clinical Trials Transformation Initiative is creating an online resource of feasibility studies using mobile technologies. Initially, this resource will include published examples, but it might be possible to broaden the scope of this resource, said Madre. Brady remarked that the NIH Health Care Systems Research Collaboratory may also be able to host a resource portal. An unidentified workshop participant said some of the elements that might be included in such a knowledge repository may already be in place (e.g., TransCelerate resources), but it is also important to partner with the U.S. Food and Drug Administration and NIH to ensure that perspectives and contributions include stakeholders in addition to the pharmaceutical industry.