The FDA’s Office of Orphan Products Development (OOPD) is excited to announce a funding opportunity for fiscal year 2026 aimed at advancing natural history studies in rare diseases and conditions: “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” (https://grants.nih.gov/grants/guide/rfa-files/RFA-FD-25-020.html).
Receipt Date: October 21, 2025
FOA Number: RFA-FD-25-020
The purpose of this funding opportunity announcement 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. 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.
More information: https://www.fda.gov/industry/grant-programs-support-development-medical-products-rare-diseases/funding-opportunities-rare-disease-research