Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)

HHS-NIH11 PAR-25-450
Posted: Sep 22, 2025 12:00:00 AM EDT
Closes: 07/20/2028

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.

Synopsis

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.

Eligibility

Eligible Applicants:
State governments Public housing authorities/Indian housing authorities Others (see text field entitled "Additional Information on Eligibility" for clarification) Native American tribal governments (Federally recognized) City or township governments Small businesses Native American tribal organizations (other than Federally recognized tribal governments) Special district governments Private institutions of higher education Public and State controlled institutions of higher education For profit organizations other than small businesses County governments Independent school districts Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
Other Eligible Applicants include the following:
Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Eligible Agencies of the Federal Government; Faith-based or Community-based Organizations; Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Indian/Native American Tribal Governments (Other than Federally Recognized); Regional Organizations; Tribally Controlled Colleges and Universities (TCCUs) ; U.S. Territory or Possession; Non-domestic (non-U.S.) Entities (Foreign Organizations) are not eligible to apply.

Non-domestic (non-U.S.) components of U.S. Organizations are not eligible to apply.

Foreign components, as defined in the NIH Grants Policy Statement, are not allowed.

Funding Activity Categories

Health Income Security and Social Services

CFDA Numbers

  • 93.350 - National Center for Advancing Translational Sciences
  • 93.865 - Child Health and Human Development Extramural Research

Contact Information

Agency: National Institutes of Health
Contact: National Institutes of Health
Phone: 301-402-2541
NIH Grants Information
grantsinfo@nih.gov

Additional Information

Document Type: synopsis
Opportunity Category: Discretionary
Version: 1
Last Updated: Sep 23, 2025 10:00:04 AM EDT

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