Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
🏛 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 seeking to conduct clinical studies of orphan products (drugs, biologics, or devices) to address unmet medical needs in rare diseases. Eligible applicants typically include academic institutions, research hospitals, nonprofit organizations, small businesses, and commercial pharmaceutical or biotech companies that have received orphan drug designation from the FDA. The grant supports U.S.-based clinical research addressing rare diseases with limited treatment options. Applicants must demonstrate scientific merit, feasibility of the clinical trial design, and a clear path to FDA approval or regulatory authorization. Activities supported include Phase I, II, and III clinical trials; post-market surveillance studies; and comparative effectiveness research on orphan products. Preference is generally given to projects addressing significant unmet medical needs where commercial incentives may be insufficient.
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Program description
The purpose of this opportunity 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
How to apply
Application links
Required documents
- SF-424 (R&R) application form
- Project narrative (specific aims, background, research design, regulatory strategy)
- Detailed budget and budget justification
- Biosketches of key personnel
- Institutional review board (IRB) approval or letter of IRB commitment
- FDA orphan drug designation letter
- Letters of institutional support and trial site commitments
- Clinical trial protocol or protocol synopsis
- Evidence of regulatory authority approval (IND, IDE, or equivalent)
- References and supporting publications
- Data safety monitoring 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
-
$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 $108.7M
- 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 is eligible to apply for this grant?
Academic research institutions, hospitals, biotech companies, pharmaceutical firms, nonprofit organizations, small businesses, and other entities with capacity to conduct FDA-regulated clinical trials. Principal investigators typically need relevant research credentials and experience.
What types of activities does this grant support?
Controlled clinical trials (Phase I-III) of orphan products, post-market surveillance studies, comparative effectiveness research, and related clinical investigations needed to advance FDA regulatory approval for rare disease treatments.
How competitive is this funding?
Highly competitive. Review panels assess scientific quality, clinical trial design rigor, experienced research team, and likelihood of advancing a product toward FDA approval. Projects addressing significant unmet needs with strong preliminary data score higher.
What is the typical funding range?
Award amounts vary widely depending on study scope, duration, and required infrastructure; typically ranging from $100,000 to several million annually for multi-year clinical trials.
Are there any geographic or institutional restrictions?
There are no geographic restrictions, but trials must comply with U.S. FDA regulations. Applicants may be based anywhere but must have institutional capacity to conduct compliant clinical research.
💡 Tips for applicants
- Clearly document the orphan drug or product designation from FDA and explain the unmet medical need your study addresses. Reviewers prioritize projects with significant therapeutic gaps.
- Design a rigorous clinical trial protocol aligned with FDA guidance documents. Include detailed patient population definition, endpoints, sample size justification, and safety monitoring plans.
- Demonstrate your research team's expertise in rare disease clinical research and FDA regulatory pathways. Include letters of collaboration from trial sites and institutional support commitments.
- Provide strong preliminary data or published evidence supporting the product's potential benefit and your team's capacity to execute the trial successfully.
- Budget realistically for trial-specific costs including regulatory compliance, patient recruitment in rare disease populations, specialized monitoring, and regulatory submissions to FDA.
⚠️ Common mistakes
Applications are frequently rejected when the clinical trial design lacks sufficient rigor or doesn't align with current FDA guidance for the specific rare disease indication. Many proposals fail to adequately justify sample size for rare populations or provide insufficient preliminary data demonstrating product efficacy. Weak or inexperienced research teams, inadequate budget justification, and failure to address regulatory pathway challenges are also common reasons for rejection.
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