Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R03 Clinical Trial Not Allowed) | PAR 18 952

Opportunity Information: Apply for PAR 18 952

The NIH funding opportunity titled "Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R03 Clinical Trial Not Allowed)" (Funding Opportunity Number PAR-18-952) is a discretionary grant program designed to support small, focused clinical projects that help rare disease programs get ready for future clinical trials without actually running a clinical trial under this award. The main purpose is to close practical gaps that often block or slow rare disease therapeutic or diagnostic development, especially where the next logical step is a trial but key foundational elements are missing. The FOA emphasizes work that makes the path into later-stage clinical testing more efficient and more likely to succeed by strengthening the evidence base, measurement tools, and disease understanding needed for well-designed trials.

Projects supported under this announcement are expected to focus on clinical trial readiness activities such as developing and testing biomarkers, creating or validating clinical outcome assessment measures, and improving the rigor of endpoints that could be used in future trials. Another major track involves defining the clinical presentation and natural course of a rare disease, disorder, or syndrome. That can include characterizing how symptoms emerge and change over time, identifying meaningful clinical milestones, mapping variability across patients, and generating the types of data that allow investigators to select appropriate participants, calculate sample sizes realistically, choose endpoints that capture true clinical benefit, and set feasible trial durations. In short, the program is aimed at helping investigators move candidate therapeutics or diagnostics toward trials by ensuring the clinical and measurement groundwork is solid.

The award uses the NIH R03 mechanism, which typically supports limited-scope, discrete projects rather than large multi-year programs. In the source data provided, the listed award ceiling is $50,000. The activity category is Health, Income Security and Social Services, and the CFDA numbers associated with the opportunity are 93.350 and 93.865. The sponsoring agency is the National Institutes of Health. While the FOA title clearly states "Clinical Trial Not Allowed," it is geared toward preparatory clinical research that directly supports the design and execution of later clinical trials funded through other mechanisms.

Eligibility is broad and includes many types of U.S.-based organizations and governmental entities. 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; Native American tribal organizations other than federally recognized tribal governments; public housing authorities and Indian housing authorities; nonprofits with and without 501(c)(3) status (excluding institutions of higher education where applicable); for-profit organizations other than small businesses; and small businesses. The FOA also explicitly calls out additional eligible applicant types 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 agencies of the federal government, regional organizations, Indian/Native American tribal governments other than federally recognized ones, and U.S. territories or possessions.

At the same time, there are important limits on non-U.S. participation. Non-domestic (non-U.S.) entities (foreign organizations) and non-domestic (non-U.S.) entities (foreign institutions) are stated as not eligible to apply, and non-domestic components of U.S. organizations are also not eligible to apply. However, "foreign components" are allowed as defined by the NIH Grants Policy Statement, meaning a U.S. applicant can include certain project elements performed outside the U.S. when permitted under NIH rules and properly justified, even though a foreign institution cannot be the applicant organization.

Key administrative details from the source data include an original closing date of 2021-10-25 and a creation date of 2018-10-04. Overall, the opportunity is best understood as targeted NIH support for the hard, enabling clinical science that rare disease communities need before a therapeutic or diagnostic program is truly ready to enter a well-powered, interpretable clinical trial.

• 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 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 $50,000.00 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 18 952

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