FDA Rare Disease Endpoint Advancement pilot program

The Rare Disease Endpoint Advancement (RDEA) pilot is a joint CDER and CBER programme in which FDA works with a small number of sponsors on novel efficacy endpoints for rare disease drugs, set up to meet a PDUFA VII commitment and announced in October 2022. It gives admitted sponsors focused meetings, not a qualified endpoint or any assurance of approval. Proposals are accepted on a quarterly cycle through 30 June 2027. FDA states a preference for surrogate endpoint proposals that collect more clinical data before approval to help validate the endpoint. None of the opened pages mentions prion disease, so any prion fit is an inference by the atlas, not a statement by FDA. Information only, not regulatory advice.

Key facts

  • RDEA is a joint pilot of CDER and CBER that fulfils a PDUFA VII commitment; it was announced in a Federal Register notice of 27 October 2022.
  • Its target is a novel efficacy endpoint meant to support substantial evidence of effectiveness; novel means never used to support an approval, or substantially modified from such use.
  • A sponsor normally needs an active pre-IND or IND for a rare disease. Exceptions are a sponsor with a natural history study in which the endpoint will be studied, and a common-disease programme with innovative endpoint elements that FDA judges could apply to a rare disease.
  • Any entity that takes responsibility for a clinical investigation can apply; the proposal is filed in eCTD module 1.6.1 against one IND and is limited to 12 pages excluding front matter and references.
  • Proposal deadlines fall on 31 March, 30 June, 30 September and 31 December, with rolling submission through 30 June 2027, and sponsors hear about 60 days after a deadline whether they proceed to disclosure discussions.
  • Admitted proposals get an initial meeting plus up to three follow-ups on request, each scheduled within 45 days of a complete request.
  • FDA may admit one proposal in the first window, then up to one per quarter and no more than three per year in fiscal 2024 to 2027; the submission page wording on these caps can be read more than one way.
  • FDA prefers proposals with broad impact and, for surrogate endpoints, proposals that use novel ways to gather extra clinical data before approval; sponsors proposing a surrogate are encouraged to first hold a Type C Surrogate Endpoint meeting.
  • RDEA does not qualify an endpoint and is separate from Drug Development Tool qualification, which is the route for a qualified biomarker or clinical outcome assessment; its advice is not a special protocol assessment.
  • FDA does not announce admitted sponsors; a sponsor may disclose its own admission. Disclosure terms are agreed before admission and the proposal is dropped if no agreement is reached. FDA says it will publish the total numbers received and selected.
  • One sponsor-announced admission is Abeona Therapeutics' ABO-503 gene therapy for X-linked retinoschisis, reported in October 2025 by trade press (a company claim, not on an FDA page).

Timeline

  1. 2022Federal Register notice announces the pilot.
  2. 2023Public workshop "Novel Endpoints for Rare Disease Drug Development" begins (two days).
  3. 2023First proposal window opens (Q4 of fiscal 2023, to 30 September 2023).
  4. 2025Abeona announces ABO-503 selection, as reported by trade press.
  5. 2026Next quarterly proposal deadline in this atlas' view; later ones 2027-03-31 and 2027-06-30.
  6. 2027Last submission deadline under PDUFA VII.
  7. 2027Stated end of the pilot.

Last checked 1 November 2026

Sources for this page