Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed) | PAR 23 159

Opportunity Information: Apply for PAR 23 159

Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (PAR-23-159) is a National Institutes of Health (NIH) funding opportunity that supports short, focused clinical research projects aimed at getting rare disease programs ready for future clinical trials. It uses the R21 grant mechanism and explicitly does not allow clinical trials under this announcement, meaning the supported work should be preparatory rather than testing an intervention for efficacy in humans. The main purpose is to remove common bottlenecks that slow rare disease translation by strengthening the evidence, tools, and study infrastructure needed to design high-quality trials later on.

The projects NIH is looking for are those that make it easier to move promising therapeutics or diagnostics toward the clinic by improving the foundations of trial design. A major emphasis is on developing and testing robust biomarkers and clinical outcome assessment measures, because rare diseases often lack validated endpoints, sensitive measures of change, or reliable markers that track disease activity. Another key focus is building a clearer picture of the disease itself, such as carefully defining the clinical presentation, natural history, and progression patterns. This kind of work can directly inform practical trial elements like inclusion and exclusion criteria, stratification approaches, visit schedules, sample size assumptions, endpoint selection, and the time window in which change can realistically be detected.

The announcement falls under the broad federal activity category of Health, Income Security and Social Services, and it is associated with CFDA numbers 93.350 and 93.865. As an NIH discretionary grant program, it is intended to fund targeted, high-value clinical readiness studies rather than large multi-year trial programs. The listed award ceiling is $275,000, indicating a relatively capped budget consistent with the R21 mechanism and the expectation of well-defined, milestone-oriented preparatory aims.

Eligibility is intentionally broad across sectors to encourage participation from the full rare disease ecosystem. Eligible applicants include state, county, and local governments; special districts; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized tribal governments and other tribal organizations; public housing authorities/Indian housing authorities; nonprofits with or without 501(c)(3) status (other than institutions of higher education); for-profit organizations (other than small businesses); and small businesses. The FOA also calls out additional eligible applicant types, including Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISIs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, eligible federal agencies, regional organizations, and U.S. territories or possessions. This breadth is designed to make it feasible for academic centers, patient-focused groups, clinical networks, and industry partners to contribute to readiness work where they have unique strengths.

There are important restrictions related to foreign involvement. Non-domestic (non-U.S.) entities and foreign institutions are not eligible to apply as the applicant organization, and non-domestic components of U.S. organizations are also not eligible to apply. However, foreign components are allowed as defined in the NIH Grants Policy Statement, which typically means a U.S.-based applicant can include certain international collaborations or activities when they are well-justified and compliant with NIH policy.

Key administrative details include the funding opportunity title and number (Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes; PAR-23-159), the agency (National Institutes of Health), and an original closing date of 2024-10-17. Overall, the opportunity is best suited for teams that can demonstrate a clear path from the proposed readiness activities to a more feasible, better powered, and higher probability-of-success clinical trial in the future, particularly in rare conditions where validated biomarkers, outcomes, or natural history data are currently limiting progress.

• The National Institutes of Health in the health, income security and social services sector is offering a public funding opportunity titled "Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.350, 93.865.
• This funding opportunity was created on 2023-04-13.
• Applicants must submit their applications by 2024-10-17. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Each selected applicant is eligible to receive up to $275,000.00 in funding.
• Eligible applicants include: State governments, County governments, City or township governments, Special district governments, Independent school districts, Public and State controlled institutions of higher education, Native American tribal governments (Federally recognized), Public housing authorities/Indian housing authorities, Native American tribal organizations (other than Federally recognized tribal governments), 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, Private institutions of higher education, For-profit organizations other than small businesses, Small businesses, Others.

Apply for PAR 23 159

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