Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
Open Posted
- Opportunity number
- RFA-FD-25-020
- Agency
-
Food and Drug Administration
Department of Health and Human Services
- Applications due
- Award size
- Up to $900,000
Opportunity facts
- Assistance listings
- Funding instrument
- Grant
- Categories
-
- Agriculture
- Consumer Protection
- Food and Nutrition
- Eligible applicants
-
- Other
- Points of contact
-
- Patrick Johnson patrick.johnson@fda.hhs.gov (301) 796-0154
Opportunity details come from Grants.gov. Updated .
About this opportunity
The Food and Drug Administration is soliciting applications for clinical trials of orphan products addressing unmet needs in rare diseases, with the next application deadline of October 21, 2025. Awards are capped at $650,000 per year for up to four years, with an optional $250,000 annual supplement available for innovative trial approaches, bringing the maximum to $900,000 annually.
Eligible applicants include higher education institutions, nonprofits, for-profit organizations, state and local governments, federal agencies, foreign organizations, U.S. territories, and faith-based or community-based organizations. The program requires a completed Investigational New Drug (IND) or Investigational Device Exemption (IDE) submission to FDA review divisions at least 30 days before the application deadline.
Clinical trials must evaluate efficacy and/or safety to support new indications or labeling changes for rare diseases with prevalence fewer than 200,000 people in the United States. All phases of clinical trials—phase 1, 2, and/or 3—are eligible. Applicants may propose collaborative, efficient, or innovative trial designs including adaptive designs, basket trials, umbrella trials, platform trials, or data modeling simulations. Applications must include the final protocol, informed consent forms, product availability letters, study site support letters, and evidence of patient engagement.
Awards provide one year of initial support with three additional years contingent upon annual appropriations and satisfactory performance. Two additional funding cycles occur October 20, 2026, and October 19, 2027. Optional letters of intent are due September 22, 2025 (and September 21, 2026; September 20, 2027 for subsequent cycles). Scientific merit review occurs February/March 2026, with earliest project start of July 2026 and program expiration of May 31, 2028. The opportunity is assigned to CFDA 93.103 (Food and Drug Administration Research).
Synopsis
The purpose of this Notice of Funding Opportunity (NOFO) is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.
Eligibility
Eligible OrganizationsHigher Education InstitutionsPublic/State Controlled Institutions of Higher EducationPrivate Institutions of Higher EducationNonprofits Other Than Institutions of Higher EducationNonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education)Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education)For-Profit OrganizationsSmall BusinessesFor-Profit Organizations (Other than Small Businesses)Local GovernmentsState GovernmentsCounty GovernmentsCity or Township GovernmentsSpecial District GovernmentsIndian/Native American Tribal Governments (Federally Recognized)Indian/Native American Tribal Governments (Other than Federally Recognized)Federal Governments U.S. Territory or PossessionOtherIndependent School DistrictsPublic Housing Authorities/Indian Housing AuthoritiesNative American Tribal Organizations (other than Federally recognized tribal governments)Faith-based or Community-based OrganizationsRegional OrganizationsNon-domestic (non-U.S.) Entities (Foreign Organizations)Foreign OrganizationsNon-domestic (non-U.S.) Entities (Foreign Organizations) are eligible to apply.Non-domestic (non-U.S.) components of U.S. Organizations are eligible to apply.Foreign components, as defined in the NIH Grants Policy Statement, are allowed.
Files
| File | Type | Posted |
|---|---|---|
| Foa_Content_of_RFA-FD-25-020.pdf | ||
| PKG00291183.pdf |
On GovTribe
Work this opportunity
- Track it in your pipeline
- Find teaming partners
- Similar grant opportunities
- Ask GovTribe AI about this opportunity