Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)
Forecasted Federal grant Posted
- Opportunity number
- FOR-FD-25-020
- Agency
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Food and Drug Administration
Department of Health and Human Services
- Applications expected due
- Not on record
- Award size
- Not on record
Opportunity facts
- Assistance listings
- Funding instrument
- Grant
- Categories
-
- Agriculture
- Consumer Protection
- Food and Nutrition
- Eligible applicants
-
- Other
- Points of contact
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- Kimberly Pendleton kimberly.pendleton@fda.hhs.gov (240) 402-7610
Opportunity details come from Grants.gov. Updated .
About this opportunity
The Food and Drug Administration is soliciting applications for clinical trials of orphan products addressing unmet needs in rare diseases and conditions, with a forecast posting date of July 11, 2025. This R01 opportunity funds efficacy and safety studies supporting new indications or labeling changes, with the stated goal of increasing approved treatments for rare diseases through collaborative, efficient, and innovative trial designs.
Eligible applicants include higher education institutions (public, private, and other), nonprofits with or without 501(c)(3) status, for-profit organizations including small businesses, state and local governments, Indian tribes (federally recognized and other), federal agencies, U.S. territories, faith-based and community-based organizations, regional organizations, and non-domestic entities. Non-domestic components of U.S. organizations are also eligible.
The opportunity operates under CFDA 93.103 (Food and Drug Administration Research). The parent program has distributed 90 to 167 awards per year over the past five years and maintains a portfolio heavily weighted toward cooperative agreements (58% of awards) and project grants (40%), with combined federal obligations totaling $4.17 billion across 3,008 grants as of the most recent program snapshot. Recent related awards in rare disease and pediatric oncology clinical trials have ranged from $6.8 million for Phase I therapeutic trials to smaller awards supporting regulatory engagement and workforce development.
No application deadline, cost-share requirement, award ceiling, floor, or expected number of awards appears in the source material provided.
Forecast synopsis
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.
Eligibility
Higher Education Institutions Public/State Controlled Institutions of Higher Education Private Institutions of Higher Education Nonprofits Other Than Institutions of Higher Education Nonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education) Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education) For-Profit Organizations Small Businesses For-Profit Organizations (Other than Small Businesses) Local Governments State Governments County Governments City or Township Governments Special District Governments Indian/Native American Tribal Governments (Federally Recognized) Indian/Native American Tribal Governments (Other than Federally Recognized) Federal Governments U.S. Territory or Possession Other Independent School Districts Public Housing Authorities/Indian Housing Authorities Native American Tribal Organizations (other than Federally recognized tribal governments) Faith-based or Community-based Organizations Regional Organizations Non-domestic (non-U.S.) Entities (Foreign Organizations) Foreign Organizations Non-domestic (non-U.S.) Entities (Foreign Organizations) are eligible to apply. Non-domestic (non-U.S.) components of U.S. Organizations are eligible to apply.
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