Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
Last verified by NonDilute: 2026-04-29. Official notice and agency instructions control.
If you have a drug candidate for a rare disease and need $500K–$900K to run a pivotal or supportive clinical trial, FDA will fund it—but you must have a clear regulatory pathway and strong trial design.
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What this is
This FDA funding opportunity supports clinical trials evaluating the efficacy and safety of pharmaceutical products in rare disease indications—either for new uses or label modifications. The agency prioritizes collaborative, efficient, and innovative trial designs to accelerate rare disease drug development and approvals. Awards range up to $900,000 and run through May 2028. This is ideal for biotech firms, academic medical centers, and sponsors with orphan drug candidates seeking trial funding to support regulatory submissions.
Who can apply
Eligible applicants include domestic for-profit and non-profit organizations, academic institutions, and government agencies with capacity to conduct clinical trials of orphan products. Foreign organizations may be eligible in limited circumstances; consult the full NOFO eligibility section. Applicants must have a product in development targeting a rare disease with unmet need and a credible plan to support a new indication or label change.
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 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.
Topics: orphan drug · rare disease clinical trial · FDA funding · drug development · unmet medical need · clinical efficacy · R01 clinical trial
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.