Oct. 4, 2022

FDA Launches Pilot Program to Support Rare Disease Drug Development

FDA Launches Pilot Program to Support Rare Disease Drug Development

Under the Prescription Drug User Fee Act (PDUFA) VII, FDA has launched the 
Rare Disease Endpoint Advancement (RDEA) Pilot Program to support efficacy endpoint development for drugs that treat rare diseases, which are those that affect fewer than 200,000 people in the United States.  

This pilot program is designed for sponsors with an active investigational new drug (IND) or pre-IND for the rare disease, or sponsors who do not yet have an active development program but have, or are initiating, a natural history study where they intend to study the proposed endpoint (measurement of what happens to people in the clinical trial). 

RDEA provides an opportunity for increased engagement with FDA experts from the Center for Drug Evaluation and Research  or the Center for Biologics Evaluation and Research to discuss novel efficacy endpoints intended to establish substantial evidence of effectiveness for a rare disease treatment. An endpoint is considered novel if it has never been used to support drug approval or if it has been substantially modified from previous use to support drug approval.  

The FDA will give preference to proposals that have the potential to impact drug development in rare disease more broadly, or proposals that reflect a range of different types of endpoints. 

Under the pilot program, sponsors may begin submitting RDEA program proposal requests (RDEA proposals) in the fourth quarter of fiscal year 2023 (July 1, 2023, through Sept. 30, 2023), and FDA will accept a maximum of one RDEA proposal. For fiscal years 2024 through fiscal year 2027, FDA will accept up to one RDEA proposal per quarter with a maximum of three RDEA proposals per year.  For each proposal FDA admits into the pilot program, the agency will conduct an initial meeting and up to three follow-up meetings, after which the sponsor can participate in other traditional meeting pathways for development of their drug product. See the December 2017 draft FDA guidance Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products.   

For more information about RDEA, please visit the webpage. 

The Small Business and Industry Assistance (SBIA) program in the Center for Drug Evaluation and Research  provides guidance, education and updates for regulated industry.

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