Reissue of RFA-FD-22-001 - Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)
Last verified by NonDilute: 2026-04-29. Official notice and agency instructions control.
If you're running a longitudinal study on a rare disease with genuine FDA-relevant insights, this R01 funds exactly that—up to $600k per award with a 3-year window.
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What this is
This FDA funding mechanism supports investigators conducting natural history studies—longitudinal observations of disease progression and outcomes—in rare disease populations. The studies must produce interpretable, high-quality data that reduces barriers to product development and fills critical knowledge gaps. Successful projects will have broad impact on specific rare diseases or disease clusters with similar biology, and their data must support future clinical trials or FDA regulatory submissions for new treatments.
Who can apply
Eligible applicants include non-profit organizations, educational institutions, government agencies, and small businesses; for-profit companies are eligible under specified conditions. Solo founders and very small teams may face administrative burden. U.S. domestic organizations and some foreign entities are eligible; see full text for details on for-profit eligibility and cost-sharing requirements.
Eligible applicant types
- Others (see text field entitled "Additional Information on Eligibility" for clarification)
Full description — from the agency
The purpose of this notice of funding opportunity (NOFO) is to support efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs. Through the support of natural history studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.
Topics: rare disease · natural history studies · FDA funding · medical product development · unmet needs · clinical data
Public-source funding discovery only. This summary is generated from public agency data and may be incomplete or stale. NonDilute is not affiliated with, endorsed by, or acting on behalf of any government agency. Official notices and agency instructions control. NonDilute does not determine eligibility, provide grant-writing advice, or guarantee funding.