Reissue of RFA-FD-23-001- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
Can you apply?
This grant is for researchers and organizations developing drugs, biologics, or devices for rare diseases with unmet medical needs. Eligible applicants include research institutions, universities, nonprofit organizations, for-profit companies, and individual researchers. Principal Investigators must have appropriate academic credentials and institutional affiliation. Applicants must propose clinical trials testing orphan products that address conditions affecting small patient populations. The grant supports clinical research activities only, not basic research or product development alone.
This grant is for researchers and organizations developing drugs, biologics, or devices for rare diseases with unmet medical needs. Eligible applicants include research institutions, universities, nonprofit organizations, for-profit companies, and individual researchers. Principal Investigators must have appropriate academic credentials and institutional affiliation. Applicants must propose clinical trials testing orphan products that address conditions affecting small patient populations. The grant supports clinical research activities only, not basic research or product development alone.
Program description
The purpose of this Notice of Funding Opportunity (NOFO) 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.
Who can apply
Eligible applicants
Details
This grant is for researchers and organizations developing drugs, biologics, or devices for rare diseases with unmet medical needs. Eligible applicants include research institutions, universities, nonprofit organizations, for-profit companies, and individual researchers. Principal Investigators must have appropriate academic credentials and institutional affiliation. Applicants must propose clinical trials testing orphan products that address conditions affecting small patient populations. The grant supports clinical research activities only, not basic research or product development alone.
How to apply
Application links
Required documents
- SF-424 (R&R) - Application for Federal Assistance
- Project Narrative and Specific Aims
- Research Plan (Background, Significance, Approach)
- Biographical Sketches of Key Personnel
- Budget Narrative and Detailed Budget
- Letters of Support from Patient Groups or Clinical Sites
- Protection of Human Subjects Documentation
- Preliminary Data and Supporting Publications
Program contact
- 👤 Stephanie Bogan Grantor
- 📧 Patrick.Johnson@fda.hhs.gov
- 📞 3018276802
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
-
$121,795,918
-
$76,105,626
-
$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 $108.5M
- 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.
FAQ
Who can apply?
Research institutions, universities, nonprofits, companies, and independent researchers with appropriate credentials and institutional support can apply. Applicants must have capacity to conduct clinical trials.
What is the deadline?
The submission deadline is May 16, 2028. Applications open July 11, 2025. Check the NIH grants.gov portal for specific receipt dates and submission windows.
What activities does this fund?
This grant funds human clinical trials of orphan products for rare diseases. Studies must evaluate safety and efficacy in patient populations with unmet medical needs.
How competitive is this grant?
This is very competitive. Reviewers prioritize strong scientific rationale, experienced research teams, feasible study designs, and clear demonstration of unmet medical need.
What is the typical funding range?
Typical R01 awards range from $250,000 to $750,000 per year for 3-5 years, depending on research scope and complexity. Actual amounts vary by proposal.
💡 Tips for applicants
- Establish clinical feasibility early. Show you can recruit and retain patient participants for a rare disease trial.
- Secure letters of support from patient advocacy groups and recruitment sites. This demonstrates access to study populations.
- Address regulatory pathway clearly. Explain how trial results support FDA approval or orphan drug development.
- Use strong preliminary data. Published studies or pilot work on the orphan product strengthen credibility significantly.
- Build a multidisciplinary team. Include clinicians, biostatisticians, and rare disease experts on your research team.
⚠️ Common mistakes
Underestimating patient recruitment challenges for rare diseases. Lack of clear regulatory strategy or FDA engagement plan. Weak preliminary data or insufficient evidence of unmet medical need.
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