Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed) | PAR 18 953

Opportunity Information: Apply for PAR 18 953

The National Institutes of Health (NIH) is soliciting applications under the funding opportunity titled "Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)" (Funding Opportunity Number: PAR 18 953). This is a discretionary grant program in the health arena (CFDA 93.350 and 93.865) aimed at strengthening the groundwork needed before a rare disease therapy or diagnostic can move into a clinical trial. The core idea is to fund clinical research projects that make future trials more feasible, better designed, and more likely to succeed, especially in areas where rare disease development programs often stall because basic clinical trial infrastructure, outcome measures, or disease characterization are not yet mature.

The FOA focuses on "clinical trial readiness" rather than running a clinical trial. In practice, that means studies and activities that help a field reach the point where a well-justified, well-powered, and appropriately measured trial can be launched. The announcement highlights several priority types of work. One major area is the development and testing of biomarkers that are rigorous enough to support later interventional studies, including efforts to establish reliability, validity, and practical utility in real-world clinical settings. Another major area is the development, refinement, and testing of clinical outcome assessment measures (for example, endpoints, rating scales, functional measures, or patient-focused outcome tools) that can detect meaningful change and can be deployed consistently across sites. The FOA also supports projects that define the presentation and course of a rare disease, such as natural history style work or deep phenotyping efforts, when that information is needed to design an upcoming clinical trial (for example, to understand progression rates, variability, subtypes, and the best windows for intervention).

The award mechanism is an NIH R21, which is typically used for exploratory or early-stage projects that can produce high-value foundational data within a limited scope and budget. The listed award ceiling is $200,000. While the opportunity is intended to accelerate the path toward trials, it explicitly does not allow clinical trials under this FOA, so applicants must propose pre-trial clinical studies rather than interventional efficacy or safety trials. The emphasis is on enabling later trials by improving measurement tools, establishing disease course knowledge, and strengthening the evidence package needed to move a candidate therapeutic or diagnostic forward efficiently.

Eligibility is broad and includes many types of U.S.-based organizations and governments. Eligible applicants include state, county, city or township, and 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; other than federally recognized tribal organizations; public housing authorities/Indian housing authorities; nonprofits with or without 501(c)(3) status (excluding institutions of higher education in those categories); for-profit organizations other than small businesses; small businesses; and other organizations as permitted. The FOA also explicitly calls out additional eligible applicant categories such as Alaska Native and Native Hawaiian Serving Institutions, Asian American Native American Pacific Islander Serving Institutions (AANAPISISs), Hispanic-serving Institutions, Historically Black Colleges and Universities (HBCUs), Tribally Controlled Colleges and Universities (TCCUs), faith-based or community-based organizations, regional organizations, eligible federal agencies, and U.S. territories or possessions.

Foreign eligibility is limited. Non-domestic (non-U.S.) entities and non-domestic components of U.S. organizations are not eligible to apply as the applicant organization. However, "foreign components" are allowed as defined by the NIH Grants Policy Statement, meaning a U.S. applicant may include certain international elements in the project when justified and compliant with NIH policy.

Key administrative details in the posted record include the original closing date of October 25, 2021, and a creation date of October 4, 2018. Overall, the opportunity is best understood as NIH support for the practical, measurable building blocks that make rare disease clinical trials possible and credible: validated biomarkers, dependable and meaningful outcome measures, and clear characterization of how a rare disease presents and progresses so future trials can be designed with the right endpoints, timelines, and target populations.

• 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 2018-10-04.
• Applicants must submit their applications by 2021-10-25. (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 $200,000.00 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 18 953

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