Funding Opportunities for Rare Disease Research

Clinical Trials 

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)
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Receipt Dates: October 21, 2025; October 20, 2026; October 19, 2027 (for new applications and resubmissions)

Resubmissions only Due Date: May 19, 2026; May 18, 2027; May 16, 2028

FOA Number: RFA-FD-25-020
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Funding Opportunity Purpose:
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.

Learn more about this program: Clinical Trials Grants Program.

Natural History Studies

Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases
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Receipt Dates: February 10, 2026; February 8, 2028 (for new applications and resubmissions)

FOA Number: RFA-FD-25-017
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Funding Opportunity Purpose:
The purpose of this notice of funding opportunity (NOFO) is to support efficient and innovative natural history studies that advance medical product development in rare diseases/conditions with unmet needs. Through the support of natural history studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.

Learn more about this program: Natural History Studies Grant Program. 

Rare Neurodegenerative Disease Grants Program Opportunities

Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases (R01) Clinical Trials Required
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Receipt Dates: October 21, 2025
FOA Number: RFA-FD-25-001
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Funding Opportunity Purpose:
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 neurodegenerative diseases for children and adults. Through the support of collaborative, efficient and innovative clinical trials, FDA hopes to exert a broad and positive impact by increasing the number of approved medical products for rare neurodegenerative diseases. 

See instructions and helpful hints (PDF) for applying for rare neurodegenerative disease grants. 

Learn more about this program: Rare Neurodegenerative Disease Grants Program.