Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R03 Clinical Trial Not Allowed) | PAR 23 160

Opportunity Information: Apply for PAR 23 160

Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (PAR-23-160) is an NIH discretionary grant opportunity that uses the R03 mechanism and is specifically limited to projects that improve readiness for future clinical trials, rather than running a clinical trial within the award. The focus is on practical, clinical-stage groundwork that helps rare disease programs move candidate therapeutics or diagnostics toward a well-designed trial more efficiently and with a higher chance of success. In plain terms, the FOA is aimed at closing the common gaps that stop rare disease trial programs from being feasible, interpretable, or fundable when they reach the trial stage.

The types of activities this FOA is trying to support center on building the evidence and tools needed to design strong upcoming trials. That includes developing, refining, and testing rigorous biomarkers (for example, measures that indicate disease status, progression, or response) and clinical outcome assessment measures (ways to measure how a patient feels, functions, or survives in a reliable and meaningful way). It also includes projects that more clearly define the clinical presentation and course of a rare disease, such as characterizing natural history and variability over time, so that future trials can set realistic eligibility criteria, choose appropriate endpoints, determine meaningful effect sizes, and plan timelines and visit schedules that match how the disease actually behaves. The overarching intent is to reduce uncertainty before a trial begins: what should be measured, how it should be measured, when it should be measured, and what a clinically meaningful change looks like in a specific rare condition.

This is a grant (FundingInstrumentType: Grant) in the Health, Income Security and Social Services activity category, associated with CFDA numbers 93.350 and 93.865, and administered by the National Institutes of Health. The listed award ceiling is $100,000. The original closing date provided in the source data is 2024-10-17. Because the mechanism is R03 and the title explicitly states "Clinical Trial Not Allowed," applicants should design proposals around preparatory clinical research and measurement development rather than interventional efficacy testing. In practice, competitive applications will usually have a clear line of sight to a plausible near-term trial, showing how the proposed readiness work directly enables trial design decisions (endpoint selection, biomarker qualification steps, outcome measure validation, cohort characterization, or similar trial-enabling deliverables).

Eligibility is broad and includes many U.S.-based public and private organizations. Eligible applicants listed include state, county, and city or township governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; federally recognized Native American tribal governments; tribal organizations other than federally recognized tribal governments; 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; small businesses; and other eligible entities. The FOA also calls out additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISI), 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, Indian/Native American Tribal Governments (other than federally recognized), and U.S. territories or possessions.

There are also important restrictions related to non-U.S. applicants and components. Non-domestic (non-U.S.) entities (foreign organizations) and non-domestic (non-U.S.) foreign institutions are stated as not eligible to apply. Non-domestic components of U.S. organizations are also not eligible to apply. At the same time, the FOA notes that "foreign components," as defined in the NIH Grants Policy Statement, are allowed, meaning a U.S. applicant organization may include certain well-justified foreign elements or collaborations that meet NIH definitions and requirements, even though foreign institutions cannot be the applicant organization.

Overall, PAR-23-160 is designed for teams working in rare diseases who are not yet ready to run a definitive trial, but who can make a strong case that targeted clinical readiness work will remove key barriers to a future trial. The most aligned projects are those that create trial-enabling infrastructure and evidence around endpoints, biomarkers, and disease course, so that subsequent interventional studies can be designed with clearer assumptions, better measurement quality, and a higher likelihood of producing interpretable results.

• 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 (R03 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 $100,000.00 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 23 160

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