FDA Orphan Products Grants Program

The FDA Orphan Products Grants Program is a set of Congressionally mandated grant programs run by the FDA Office of Orphan Products Development. They pay for clinical trials, natural history studies and, since 2021, rare neurodegenerative disease work, all aimed at getting medical products for rare diseases approved. The Clinical Trials Grants Program has operated since 1983 and FDA credits it with helping more than 85 products toward approval. Any foreign or domestic organization, public or private, for profit or not, can apply, if the disease affects fewer than 200,000 people in the US. For prion disease it is a plausible but unproven route: no award on the recent-awards page mentions prion disease or CJD, and the full database was not searched. Information only, not funding advice.

Key facts

  • The Clinical Trials Grants Program has run since 1983 and the Natural History Studies Grants Program since 2016.
  • Eligible applicants include universities, nonprofits, small and other for-profit businesses, governments and foreign organizations; federal agencies are excluded.
  • Products must target a disease with US prevalence under 200,000 (or annual incidence under 200,000 for acute disease); diagnostics and vaccines qualify only if fewer than 200,000 people a year would receive them.
  • The current clinical trials call, RFA-FD-25-020, covers phase 1, 2 and 3 trials supporting a new indication or labeling change, with a ceiling of 650,000 USD total costs a year for up to four years, plus up to 250,000 USD more a year for innovative or efficient designs (maximum 900,000 USD a year).
  • RFA-FD-25-020 application due dates are 2025-10-21, 2026-10-20 and 2027-10-19, each with an optional letter of intent about a month earlier; resubmissions only on 2027-05-18 and 2028-05-16.
  • FDA estimates about 17 million USD a year for the clinical trials program (about 12 million continuations, 5 million new awards), with 60 to 85 ongoing projects and about 5 to 12 new grants a year.
  • FY2026 clinical trials round: 66 applications, 6 new awards, about 16.5 million USD over four years. Natural history round: 90 applications, 7 new grants, about 13.9 million USD over four years.
  • Natural history grants are awarded every two years and fund protocol-driven studies that support trial design, outcome measures and biomarkers. A search summary puts the FY2024 prospective study cap at 400,000 USD a year for up to four years.
  • No award on the recent-awards page mentions prion disease or CJD, and none of the 13 publications listed on the grantee publications page is about prion disease; the complete database was not queried.
  • A secondary report gives FY2024 as 17.2 million USD to seven trials from 51 applications (unchecked against FDA).

Timeline

  1. 1983Clinical Trials Grants Program begins.
  2. 2016Natural History Studies Grants Program begins, introduced by a May 2016 Federal Register notice.
  3. 2021The rare neurodegenerative stream is added through the ACT for ALS, administered by OOPD.
  4. 2025RFA-FD-25-020 posted; opens 2025-08-22.
  5. 2025First application date under RFA-FD-25-020.
  6. 2026Second application date under RFA-FD-25-020; passed as of 2026-11-01.
  7. 2027Third and last application date under RFA-FD-25-020.

Last checked 1 November 2026

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