RDEA Pilot Program to support the development of novel efficacy endpoints for drugs that treat rare diseases is now accepting proposals!  

The goals of the RDEA program are to: 1) advance rare disease drug development through collaboration with FDA throughout the efficacy endpoint development process, 2) promote innovation and evolving science by sharing learnings on novel endpoint development through FDA presentations, guidance documents, public workshops, and a public-facing website, and 3) develop FDA staff capacity to facilitate development and use of novel endpoints.

If you have a novel efficacy endpoint that you would like to have considered under this program, you may submit an RDEA proposal from July 1, 2023, through September 30, 2023.  FDA will accept 1 RDEA proposal and 1 alternate in FY2023.  If accepted into the program, you would be eligible for an initial meeting and, if requested, up to three follow-up meetings with FDA to facilitate endpoint development.  If you are not ready to make a proposal submission this year, don’t fret!  FDA will accept 1 RDEA proposal per quarter up to a maximum of 3 RDEA proposals per year in FY2024 through FY2027.

Note:  FDA intends to give preference to proposals that have the potential to have a broad impact (multiple products/diseases) and reflect a range of different types of endpoints.