Reissue of RFA-FD-22-001 - Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)
This grant funds innovative natural history studies to advance medical product development and address unmet needs in rare diseases by generating high-quality, interpretable data.
Funder: Food and Drug Administration (FDA)
Due Dates: Letter of Intent: January 9, 2026 | Application: February 10, 2026 | Letter of Intent: January 7, 2028 | Application: February 8, 2028
Funding Amounts: Prospective studies: up to $400,000/year (max 4 years); Retrospective: up to $150,000/year (max 2 years); up to $600,000/year for innovative designs.
Summary: Supports efficient and innovative natural history studies to advance medical product development for rare diseases with unmet needs.
Key Information: Clinical trials are optional; non-U.S. and foreign organizations are eligible.
Description
This funding opportunity supports the development of efficient and innovative natural history studies that address unmet needs in rare diseases or conditions. The goal is to generate high-quality, interpretable data that will advance medical product development, fill critical knowledge gaps, and remove significant barriers to progress in the field. Both prospective and retrospective observational studies are eligible, with an emphasis on collaborative, standardized, and innovative approaches—including the integration of biomarkers, clinical outcome assessments, and advanced analytics such as machine learning. The program encourages the use of existing infrastructure and stakeholder collaboration among academia, industry, and patient organizations.
