FDA Seeks Patient Input on Strategies to Increase Trial Enrollment & Improve Study Design

The first meeting of FDA’s Patient Engagement Advisory Committee (PEAC) sought to improve clinical trials and product development through better study design and greater patient participation. The panel discussed how requirements for informed consent, randomization, and study duration affect enrollment and participant retention in clinical trials, and what methods might be most effective for recruiting diverse patient populations.

Established by the Center for Devices and Radiological Health (CDRH), the PEAC is FDA’s first advisory committee composed entirely of patients and patient representatives. CDRH seeks to bring patient perspectives into the process of defining risks and benefits of research and designing clinical trials to incorporate real-world evidence (RWE) and patient-reported outcomes (PROs). PEAC deliberations will support agency-wide steps, including the possible creation of a new Office of Patient Affairs to support and coordinate agency-wide patient engagement activities.

CDRH director Jeffrey Shuren opened the meeting by noting a rise in product development programs that incorporate patient views. Over 50% of premarket applications for devices now include PROs, he said, and bringing patient views into study design may lead to more robust research. Owen Faris, CDRH clinical trials program director, pointed to difficulties in patient enrollment as the “biggest cog” in the clinical research process. He said he hopes that the panel will help FDA devise strategies for enrolling patients in clinical trials who are most likely to use the test product and are willing to adhere to study’s procedures.

Ken Getz, chair of the Center for Information & Study of Clinical Research Participation, noted that the challenges facing studies for medical devices are the same as for drugs, which are experiencing record high failure rates that drive up research costs. One reason for unsuccessful research is that study protocols are increasingly complex, with more primary endpoints and demands for data on cost effectiveness and economic benefits as well as product safety and efficacy. The more complex the study, Getz said, the greater the number of study participants and the need to enlist more sites across more countries.

Patient engagement can help address these problems, said Getz, particularly by advising on the design of more targeted studies with meaningful endpoints that are relevant to the patient community. Simplified protocol design can help drive success rates and make clinical trial participation a positive experience. Some sponsors are establishing patient advisory boards, Getz noted, but many are holding back on patient engagement due to uncertainty about changing traditional practices. Regulatory clarity is a major need, he said, particularly on tricky issues such as sharing study results with volunteers. Members of PEAC advised sponsors to develop outreach strategies to recruit patients and to reduce the burden of participation in a trial by providing reimbursement, transportation, lodging, and other support.