Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Can you apply?
This grant is for researchers developing clinical trial readiness for rare diseases, disorders, and syndromes. Eligible applicants include academic institutions, medical centers, research organizations, and individual researchers with doctoral degrees. Awards support preliminary research, feasibility studies, and proof-of-concept work—not full clinical trials. Projects must focus on rare disease patient populations and advance knowledge toward future trial readiness.
Applicants must have institutional affiliations or be supported by eligible institutions. International institutions may apply if supported by U.S. partners. Projects should demonstrate scientific merit and innovation in rare disease research methodology.
The program prioritizes high-risk, high-reward exploratory research. Applicants should have demonstrated capability in rare disease research but not necessarily extensive prior funding in this specific area.
Program description
This Notice of Funding Opportunity (NOFO) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare diseases research by enabling efficient and effective movement of candidate therapeutics or diagnostics toward clinical trials, and to increase their likelihood of success. This could be through the development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials.
Who can apply
Eligible applicants
How to apply
Application links
Required documents
- SF-424 (R&R)
- Project Narrative
- Budget and Budget Justification
- Biographical Sketches (senior key personnel)
- Specific Aims page
- Research Strategy
- Letters of Support (if applicable)
Program contact
- 👤 National Institutes of Health
- 📧 grantsinfo@nih.gov
- 📞 301-402-2541
Funding track record
Recent awards under CFDA 93.350 from the last 3 years — real organizations that won funding through this same program.
Top 10 Largest Recent Awards
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$139,753,636
-
$118,592,298
-
$118,098,707
-
$112,138,469
-
$102,422,256
-
$100,095,982
-
$99,978,264
-
$98,955,036
-
$98,090,822
-
$97,784,066
Top States by Funding
- CA 13 awards $652.0M
- NY 11 awards $496.8M
- MA 6 awards $321.4M
- NC 5 awards $313.7M
- OH 5 awards $162.0M
Source: USAspending.gov — federal spending transparency. Data covers last 3 years.
Funding history
Annual funding for this program — Federal obligations (CFDA 93.350). How funding has trended year over year.
| 2024 | $595,597,945 | |
| 2025 | $626,227,752 |
FAQ
Who is eligible to apply for this R21 grant?
Individual researchers, academic institutions, research hospitals, and nonprofit research organizations are eligible. Your institution must have an active SAM.gov registration and DUNS number.
Can my project include a clinical trial?
No. This mechanism explicitly prohibits clinical trials. Focus on readiness activities like patient registry development, biomarker validation, or protocol optimization instead.
What is the typical funding range?
R21 mechanisms typically provide $275,000-$500,000 total costs for two years. Specific funding depends on project scope and reviewer assessment.
What makes an application competitive?
Innovation in rare disease research approaches, clear feasibility plans, strong scientific rationale, and evidence of preliminary work strengthen competitiveness. Demonstrate how your project bridges gaps to future trials.
When is the next deadline?
The deadline is July 20, 2028. Applications open September 22, 2025. Plan to submit at least two weeks before the deadline.
💡 Tips for applicants
- Focus on readiness activities, not clinical outcomes. Describe how your research prepares the field for future trials.
- Include preliminary data or strong published evidence to show feasibility. Reviewers expect some proof of concept.
- Clearly identify your rare disease population. Explain why this group is underserved and why your approach matters.
- Build a realistic timeline. R21s fund two-year exploratory work—plan achievable milestones.
- Identify a mentor or advisory team if you lack prior rare disease research experience. This strengthens perceived feasibility.
⚠️ Common mistakes
Proposing a clinical trial design will result in desk rejection. Overestimating preliminary data or overstating significance weakens applications. Failing to clearly define the rare disease population and explain its clinical relevance causes reviewer confusion.
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