Announcement of Grant Funding Opportunity for Rare Disease Research:

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce a new funding opportunity for the Orphan Products Grants Program to support clinical trials for rare diseases and conditions.  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. 

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)

Receipt Date: October 20, 2026

FOA Number: RFA-FD-25-020

•    Purpose: 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. 

•    Focus: Efficiency, innovation, impact, collaboration, leveraging patient input, infrastructure and financial resources

•    Efficiency: Encourage efficient and innovative clinical trials in all phases of product development through adaptive and seamless trial designs, basket, umbrella and platform trials studying multiple rare diseases/products and the use of data modeling and simulations. Allow additional total costs up to $250,000 per year for studies using efficient and innovative designs.

•    Contact: Katherine Needleman, Director, Orphan Products Grants Program

                     E-mail: katherine.needleman@fda.hhs.gov

•    Additional Information: OOPD Website

Please forward this announcement to any parties who may have need and interest in this grant program and please post the information on social media to alert interested parties.  

Thank you for your help in bringing awareness to this important opportunity.

Bridget Nugent, Ph.D.

Health Scientist, Office of Orphan Products Development

Office of the Commissioner

U.S. Food and Drug Administration

U.S. Department of Health and Human Services

Tel: 301-796-9443