2022-23383: Rare Disease Endpoint Advancement Pilot Meeting Program

Summary

The seventh iteration of the Prescription Drug User Fee Amendments (PDUFA VII) included as part of the FDA User Fee Reauthorization Act of 2022 highlights the goal of advancing and facilitating the development and timely approval of drugs and biological products for rare diseases, including rare diseases in children. The Food and Drug Administration (FDA or Agency) is announcing the Rare Disease Endpoint Advancement Pilot Meeting Program (RDEA Pilot Program) established under the seventh iteration of PDUFA that affords sponsors who are admitted into the RDEA Pilot Program additional engagement opportunities with the Agency to discuss efficacy endpoint development in rare disease drug and biological product development programs. Meetings under the program will be conducted by FDA's Center for Drug Evaluation and Research (CDER) or Center for Biologics Evaluation and Research (CBER) during fiscal years (FYs) 2023 to 2027. For each sponsor whose RDEA program proposal (RDEA proposal or proposal) is admitted into the program, up to four meetings that will provide an opportunity for medical product developers to discuss rare disease endpoint development will be held between the sponsor and CDER or CBER. To promote innovation and evolving science, novel endpoints developed through the RDEA Pilot Program may be presented by FDA (e.g., in a guidance or public workshop or on a public-facing website) as case studies, including novel endpoints for drugs that have not yet been approved or biological products that have not yet been licensed by FDA for a given indication.

The agency’s own summary, as published.