Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
Funds clinical trials of orphan products to evaluate efficacy and/or safety for a new indication or labeling change to address unmet needs in rare diseases or conditions.
⚑ Clinical trials required; R01 clinical-trials-required mechanism. · Funds product-focused efficacy/safety studies supporting FDA labeling or new indication; not general rare-disease research. · Foreign organizations and foreign components are allowed. · Award ceiling $900,000.
Unit fits — one characterization, each unit's own rules
| Physical Sciences & Engineering (demo) | 65 good | technical depth: minor; funds basic research |
| IPPRA | 58 good | peripheral portfolio topic: public_health; social/behavioral work is minor; funds basic research; biomedical core — IPPRA health lane is communication/crisis/policy (capped); clinical-trial/biomedical core — IPPRA angle is policy/community (capped) |
| Tom Love Innovation Hub | 15 none | deep-tech content; no commercialization signal |
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.
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.
Apply
View on Grants.gov → CONTACT: Stephanie Bogan Grantor <Patrick.Johnson@fda.hhs.gov>
Proposal brief SEE AN EXAMPLE →
A one-page internal memo: fit assessment, submission requirements, document scaffold, and next steps dated back from the deadline — tailored to your project idea if you add one.
Proposal shell · HHS services agencies (SAMHSA / HRSA / CDC / ACF) conventions SEE AN HHS EXAMPLE →
Funder-faithful document skeletons — HHS services agencies (SAMHSA / HRSA / CDC / ACF)'s document set with section headings, page limits, reviewer guidance, and writing prompts; add a project idea to get [DRAFT] starter bullets. Download as .md for Word or Overleaf.