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: May 18, 2027 (Resubmission) | September 20, 2027 (Optional letter of intent) | October 19, 2027 (Application) | May 16, 2028 (Final resubmission)
Funding Amounts: Up to $650,000 annually for four years; up to $900,000 annually for qualifying innovative trials
Summary: Supports clinical trials of orphan products generating safety and/or efficacy data for new indications or labeling changes addressing rare-disease unmet needs.
Key Information: Applicable IND/IDE protocols must be submitted to FDA at least 30 days before the application deadline, with an active IND or approved IDE required for review.
Description
The FDA Office of Orphan Products Development (OOPD) supports phase 1, 2, and 3 clinical trials of orphan products addressing unmet medical needs in rare diseases or conditions. Eligible products include drugs, biologics, medical devices, and medical foods. Trials must evaluate safety and/or efficacy to support a new indication or a change in labeling, with the goal of increasing approved treatments and advancing rare-disease product development.
The program encourages shared infrastructure, established research networks, collaboration among industry, academia, and patient organizations, and early, ongoing patient and caregiver engagement. Additional funding is available for justified innovative and efficient approaches, including seamless, adaptive, basket, umbrella, and platform trials, or data modeling and simulations used to study product safety and efficacy.
