Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01 Clinical Trials Optional)
🏛 Food and Drug Administration (HHS-FDA)
✓ Free, no account · Source: Grants.gov · Last verified Jul 16, 2026
Can you apply?
This grant is for researchers conducting natural history studies of rare diseases. Eligible applicants include academic medical centers, research institutions, and nonprofit organizations with research capacity. Domestic and foreign institutions may apply, though domestic priority is typical. Studies must address an unmet need in rare disease understanding and advance FDA's ability to evaluate future treatments.
The grant supports observational research, patient registries, and longitudinal studies. Clinical trials are optional but not the primary focus. Research must contribute to the scientific knowledge base for rare diseases without being a traditional clinical trial.
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Program description
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.
Who can apply
Eligible applicants
How to apply
Application links
Required documents
- SF-424 (R&R)
- Project Narrative
- Specific Aims
- Research Strategy
- Budget and Justification
- Biographical Sketches
- Letters of Support
- Data Management Plan
Program contact
- 👤 Kimberly Pendleton
- 📧 Kimberly.Pendleton@fda.hhs.gov
- 📞 240-402-7610
Funding track record
Recent awards under CFDA 93.103 from the last 3 years — real organizations that won funding through this same program.
Top 10 Largest Recent Awards
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$121,795,918
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$76,105,626
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$50,217,964
-
$47,940,304
-
$36,000,000
-
$35,573,997
-
$35,391,995
-
$30,732,300
-
$23,332,999
-
$21,347,288
Top States by Funding
- AZ 3 awards $131.4M
- MD 7 awards $108.7M
- CA 9 awards $106.5M
- VA 5 awards $96.6M
- PA 10 awards $77.4M
Source: USAspending.gov — federal spending transparency. Data covers last 3 years.
Funding history
Annual funding for this program — Federal obligations (CFDA 93.103). How funding has trended year over year.
| 2016 | $170,482,435 | |
| 2017 est. | $208,900,832 | |
| 2018 | $173,077,408 | |
| 2019 | $198,507,896 | |
| 2020 | $212,448,590 | |
| 2021 | $218,918,739 | |
| 2022 est. | $255,910,458 | |
| 2023 est. | $246,894,600 |
FAQ
Who can apply for this grant?
Academic institutions, research hospitals, nonprofits with research capacity, and established investigators can apply. Domestic institutions receive priority.
What types of studies does this fund?
Natural history studies, patient registries, observational cohorts, and longitudinal research on rare diseases. Clinical trials are optional.
What is "unmet need" in this context?
Research addressing gaps in disease understanding, patient characterization, or outcomes data that FDA currently lacks.
How competitive is this funding?
Highly competitive. Strong preliminary data, clear scientific rationale, and experienced teams are essential.
What is the typical funding range?
R01 grants typically range $250,000-$500,000 per year for 3-5 years, but varies by disease and scope.
💡 Tips for applicants
- Clearly identify the specific unmet need your study addresses and why it matters to FDA's decision-making.
- Include detailed recruitment and retention plans, especially for rare disease cohorts with small populations.
- Provide preliminary data showing feasibility and team expertise in rare disease research.
- Align your study design with FDA's current knowledge gaps for your target disease.
- Build strong collaborations with patient advocacy groups and clinicians treating the disease.
⚠️ Common mistakes
Vague justification of unmet need or lack of FDA relevance. Unrealistic recruitment projections for small patient populations. Weak team qualifications or no prior rare disease research experience.
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