FDA Unveils Rare Disease Endpoint Advancement Pilot Program

The U.S. Food and Drug Administration said it is establishing a Rare Disease Endpoint Advancement Pilot Program to support novel efficacy endpoint development for drugs that treat rare diseases.

The RDEA Pilot Program fulfills a commitment under the seventh incarnation of the Prescription Drug User Fee Act, which President Joe Biden signed into law at the end of September.

The RDEA Pilot Program is designed to advance rare disease drug development programs by providing a mechanism for sponsors to collaborate with FDA throughout the efficacy endpoint development process.

It will promote innovation and evolving science by sharing learnings on novel endpoint development through FDA presentations, guidance documents, public workshops, and a public-facing website. The agency will also develop staff capacity to facilitate the development and use of novel endpoints to evaluate the efficacy of rare disease therapies.

The RDEA Pilot Program is a joint program of the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research.

Sponsors may begin submitting RDEA program proposal requests starting July 1, 2023, and FDA will accept only one RDEA proposal. For fiscal years 2024 through 2027, FDA will accept up to one RDEA proposal per quarter with a maximum of three RDEA proposals per year. For each RDEA proposal that FDA admits into the pilot program, the agency will conduct an initial meeting and up to three follow-up meetings.

Given that FDA expects to admit a limited number of RDEA proposals into the pilot program, the agency said it will give preference to proposals that have the potential to impact drug development more broadly, such as one that uses a novel approach to develop an efficacy endpoint or an endpoint that could potentially be relevant to other diseases.