Clinical Trials 

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)
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Receipt Dates: 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. 

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Natural History Studies:
Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01)
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Receipt Dates: 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.

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Rare Neurodegenerative Disease Grants Program Opportunities: 

Systematic Review of Clinical Outcome Assessments (COAs) for Communication Brain-Computer Interface Devices (cBCIs)  in Amyotrophic Lateral Sclerosis (ALS) (UH2/UH3)
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Receipt Dates: May 24, 2023
FOA Number: RFA-FD-23-030
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Funding Opportunity Purpose:
The purpose of this funding opportunity is to solicit applications for UH2/UH3 cooperative agreements to support and inform the future development and use of COAs for cBCIs in patients with amyotrophic lateral sclerosis (ALS). The UH2/UH3 cooperative agreement involves two milestone driven phases: 1) the UH2 Phase will include a systematic landscape analysis of the available literature, relevant data sources, and interviews with key opinion leaders (KOLs) to document COAs for cBCIs used clinically and identify gaps between current COAs used in cBCI studies and other outcome measures that could demonstrate functional benefits for ALS patients with severe communication limitations; 2) the UH3 phase will consist of patient and caregiver focus groups to collect information about symptoms, functional status, and perceived benefits/risks of cBCIs.

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