Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) is sponsored by National Institutes of Health (NIH) / Food and Drug Administration (FDA). This Notice of Funding Opportunity (NOFO) aims to fund clinical trials of products evaluating efficacy and/or safety to address unmet needs in rare diseases or conditions.

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gov Maintenance Calendar View similar opportunities Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required) Department of Health and Human Services Food and Drug Administration Document Type:Grants Notice Funding Opportunity Number:RFA-FD-25-020 Funding Opportunity Title:Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required) Opportunity Category:Discretionary Opportunity Category Explanation: Funding Instrument Type:Grant Category of Funding Activity:Agriculture Expected Number of Awards: Assistance Listings:93.

103 -- Food and Drug Administration Research Cost Sharing or Matching Requirement:No Last Updated Date:Apr 13, 2026 Original Closing Date for Applications:May 16, 2028 Current Closing Date for Applications:May 16, 2028 Archive Date:Jun 15, 2028 Estimated Total Program Funding: Eligible Applicants:Others (see text field entitled "Additional Information on Eligibility" for clarification) Additional Information on 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.

## Additional Information Agency Name:Food and Drug Administration Description: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.

Link to Additional Information:RFA-FD-25-020 Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required) Grantor Contact Information:If you have difficulty accessing the full announcement electronically, please contact: Grants Management Specialist Email: Patrick. Johnson@fda. hhs.

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