PROs Have a Bigger Impact on FDA Decisions When Used as Primary Endpoints
(Thursday, July 1, 2021)
Patient Reported Outcomes (PROs) that capture patient experience data tend to play a central role in FDA decisions about an application when used as primary endpoints in pivotal clinical trials, says a report on FDA’s Patient Focused Drug Development (PFDD) program and its impact on regulatory decisions published last week. PROs are particularly important for rare diseases where the conditions associated with the disease are not well characterized. The PFDD program created under the 21st Century Cures Act mandates FDA to incorporate patient experience data into regulatory decisions whether to allow new endpoints in clinical trials or to make decisions regarding applications. FDA has conducted several public meetings under this program to understand patient perspectives, and made several recommendations to developers on how to use this information for product development. For many years, FDA has advocated for patient involvement in clinical trial design and data review since traditionally there was a disconnected between patient and developer perspectives. So far, the PFDD program also provided other useful information captured in the first report on this program. Patient experience data is now often used as a background and context of review, help design clinical trials, involving FDA’s internal Clinical Outcome Assessment (COA) experts, and sharing of findings with patient communities so they understand the context of FDA decisions. Going forward, FDA would increase the scope of PFDD by developing a list of recommendations tools to collect patient perspectives, list of endpoints, define criteria and expectations for consideration of patient experience data in application reviews. Patient advocacy has greatly impacted FDA’s crucial decisions in the last decade and its going to increase with time. Developers need to pay close attention as suggested in this report.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC