Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
This grant funds clinical trials to test the safety and effectiveness of treatments for rare diseases, aiming to address unmet needs and support new or updated drug approvals.
Funder: Food and Drug Administration
Due Dates: October 21, 2025 (Full application, new) | May 19, 2026 (Resubmission only) | September 21, 2026 (Letter of Intent, optional) | October 20, 2026 (Full application, new) | May 18, 2027 (Resubmission only) | September 20, 2027 (Letter of Intent, optional) | October 19, 2027 (Full application, new) | May 16, 2028 (Resubmission only / Final closing)
Funding Amounts: Up to $650,000/year (total costs) for 4 years; up to $900,000/year for innovative trial designs (with justification).
Summary: Supports clinical trials of orphan products to address unmet needs in rare diseases, aiming to increase approved treatments and impact rare disease drug development.
Description
This opportunity funds clinical trials evaluating the efficacy and/or safety of products intended to support a new indication or change in labeling for rare diseases or conditions with unmet medical needs. The program, administered by the FDA's Office of Orphan Products Development (OOPD), seeks to accelerate the development of new therapies for rare diseases through collaborative, efficient, and innovative clinical trial approaches. All phases of product development (phases 1, 2, and/or 3) are eligible, and applications proposing novel trial designs or data modeling methods may request additional funding. The ultimate goal is to increase the number of FDA-approved treatments for rare diseases and positively influence rare disease drug development.
