Orphan Products Clinical Trials Grants Program

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases

Funds clinical research for rare disease products by supporting trial design and study execution.

The FDA Office of Orphan Products Development (OOPD) Clinical Trials Grants Program, established under the Orphan Drug Act of 1983, funds clinical trials of drugs, biologics, medical devices, and foods for medical purposes indicated for rare diseases or conditions affecting fewer than 200,000 people in the United States. Diagnostics and vaccines qualify only if the U.S. population to whom they will be administered is fewer than 200,000 people per year. The current multi-year Notice of Funding Opportunity (RFA-FD-25-020, R01 mechanism) covers annual receipt cycles through at least October 2027, with receipt dates of October 21, 2025; October 20, 2026; and October 19, 2027. Congress appropriates approximately $17 million per fiscal year for the overall program; of this, approximately $5 million funds new awards annually and approximately $12 million supports noncompeting continuation awards. OOPD typically issues 5 to 12 new grants each year, with 60 to 85 ongoing grant projects at any given time.

Grants are available to any domestic or foreign, public or private, for-profit or nonprofit entity, including state and local government units. Federal agencies are explicitly excluded from eligibility. Applications must target a clinical trial evaluating efficacy and/or safety in support of a new indication or a labeling change for an orphan product; purely observational or non-interventional studies do not qualify under this NOFO. The R01 mechanism applies, and submissions are routed through grants.gov under FOA RFA-FD-25-020, not through the FDA website directly.

FDA emphasises adaptive and seamless trial designs, modeling and simulation, and basket and umbrella trial approaches as preferred methodologies under this program. Collaborative use of shared infrastructure and engagement of patient organisations and caregivers are also valued in applications. Competitive proposals clearly articulate the unmet need in the rare disease target population, justify the clinical trial design against FDA-endorsed innovative methods, and present a realistic budget and timeline for completing the efficacy or safety evaluation within the grant period.