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 25,2022; October 24, 2023; October 22, 2024
Resubmissions only 
Due Date(s): June 6, 2023; June 4, 2024; June 3, 2025
FOA Number: RFA-FD-23-001
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Funding Opportunity Purpose:
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.  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. 

See instructions and helpful hints for applying for clinical trial grants.

Natural History Studies:
Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01)
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Receipt Dates: February 15, 2022 and February 13, 2024
FOA Number: RFA-FD-22-001
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Funding Opportunity Purpose:
The purpose of this funding opportunity announcement 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.

See instructions and helpful hints for applying for natural history study grants.