Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases
Funds foreign and domestic entities running rare-disease clinical trials through FDA orphan products research grants.
The Food and Drug Administration administers this grant through the Office of Orphan Products Development rare disease research grant programs. The R01 notice funds clinical trials of orphan products that evaluate efficacy or safety for a new indication or labeling change addressing unmet needs in rare diseases or conditions.
Foreign or domestic public or private for-profit and nonprofit entities…
Clinical trials evaluating efficacy or safety of medical products for new indications or labeling changes addressing unmet needs in rare diseases.
Sign up free to see the funding breakdown
Sign up free to see the industries in scope
Sign up free to see the full eligibility
Sign up free to see how to apply
Sign up free to see what you submit
Sign up free to see the timeline
Sign up free to see where teams trip up
Guides for this application
All guides →Grants for Biotech Startups: NIH, NSF, SBIR/STTR, and Non-Dilutive Funding
A practical guide to grants for biotech startups, including NIH SBIR/STTR, NSF, translational funding, evidence, regulatory readiness, and budgets.
DocumentsGrant Work Plan Template: Work Packages, Gantt, Milestones, and Risks
Use this grant work plan template to structure work packages, tasks, owners, deliverables, Gantt timing, dependencies, and risk registers.
Submit & reportSAM.gov Registration and UEI Guide for Federal Grant Applicants
Learn how SAM.gov registration, UEI numbers, Grants.gov accounts, organization profiles, workspace roles, and renewal timing fit together.
Find grantsGovernment Grants for Startups: What Deeptech Founders Should Know
A practical guide to government grants for startups, including Grants.gov, SBIR/STTR, state programs, eligibility, fit, timelines, and when to apply.
More from FDA
All grants from this funder →Minor Use Minor Species Development of Drugs
FDA · United States
Funds the safety and effectiveness testing costs of getting a designated new animal drug through FDA approval.
Laboratory Flexible Funding Model (LFFM)
🇺🇸FDA · United States
Funds state, territorial and tribal food- and animal-testing laboratories to run sample testing and lab capacity work for FDA's food safety system.
Reissue of RFA-FD-22-001 - Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases
FDA · United States
Funds natural history studies that track how a rare disease unfolds over time, to close data gaps blocking new treatments for it.
Animal and Veterinary Innovation Centers
🇺🇸FDA · United States
Funds academic institutions to become long-term FDA partners researching bird flu, animal genomics and unmet veterinary needs.
Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases
FDA · United States
Funds clinical trials testing whether a product works and is safe for a rare disease, aimed at a new indication or a labeling change.
Vet-LIRN Capacity-Building Project and Equipment Grants
🇺🇸FDA · United States
Funds equipment and capacity-building projects for veterinary diagnostic labs fighting animal-food hazards and drug resistance.
Similar grants
Browse the directory →
Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases
🇺🇸FDA OOPD · United States
Funds clinical research for rare disease products by supporting trial design and study execution.
Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases
🇺🇸FDA OOPD · United States
Funds natural-history studies that improve preparedness for rare disease trial pathways.
NCATS Preclinical Proof of Concept Studies for Rare Diseases
🇺🇸NCATS · United States
Funds U.S. researchers and companies conducting preclinical efficacy studies to advance therapeutic candidates for rare diseases toward IND applications.
NCATS Clinical Trial Readiness for Rare Diseases
🇺🇸NCATS · United States
Funds U.S. researchers and companies generating natural history data, biomarkers, and outcome measures needed to design clinical trials for rare diseases.
Clinical Trials Addressing Unmet Needs of Rare Neurodegenerative Diseases
🇺🇸FDA OOPD · United States
Funds clinical testing research for rare neurodegenerative disorders and therapeutic development.
Advanced Development and Validation of Emerging Technologies for Basic and Clinical Cancer Research
NIH · United States
Funds the development and hard technical validation that turns a working cancer research technology into one other laboratories will actually adopt.