Rare Disease Endpoint Advancement Pilot Program Grant

The Rare Disease Endpoint Advancement Pilot Program, initiated by the FDA, aims to enhance the development of novel efficacy endpoints for drugs addressing rare diseases. This initiative encourages collaboration between sponsors and the FDA throughout the endpoint development process. By fostering innovation through guidance and public workshops, the program seeks to equip FDA staff and improve evaluation methods for rare disease therapies. The pilot program is set to run until September 30, 2027, providing vital support for drug development in this area.

As announced in the Federal Register notice published on October 27, 2022, FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel endpoint efficacy development for drugs that treat rare diseases. The RDEA Pilot Program fulfills a commitment under PDUFA VII.
Goals of the RDEA Pilot Program
The RDEA Pilot Program is designed to:
Seek to advance rare disease drug development programs by providing a mechanism for sponsors to collaborate with FDA throughout the efficacy endpoint development process.Promote innovation and evolving science by sharing learnings on novel endpoint development through FDA presentations, guidance documents, public workshops, and a public-facing website.Develop FDA staff capacity to enable and facilitate the development and use of novel endpoints to evaluate the efficacy of rare disease therapies.
This program offers selected sponsors an opportunity for increased engagement with FDA experts from the Center for Drug Evaluation and Research and/or Center for Biologics Evaluation and Research to discuss novel efficacy endpoints intended to establish substantial evidence of effectiveness for a rare disease treatment. The pilot program period will run through September 30, 2027.

Eligibility

For the purposes of the RDEA Pilot Program, consistent with 21 CFR 312.3, a sponsor is a person who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. Therefore, various types of sponsors can apply to participate in the pilot. However, an RDEA proposal must meet the program eligibility criteria as stated on the RDEA Program webpage and in the October 27, 2022, Federal Register notice announcing the program.