Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional) | PAR 21 058

Opportunity Information: Apply for PAR 21 058

The NIH funding opportunity PAR-21-058, titled "Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional)," supports projects that move beyond biomarker discovery and focus instead on clinical validation. The main purpose is to rigorously validate a strong, already-identified candidate biomarker in the real clinical population where it is intended to be used, with the specific aim of establishing performance metrics such as positive predictive value (PPV) and negative predictive value (NPV) in a way that aligns with FDA expectations and guidance. In practical terms, the program is geared toward determining how well the biomarker distinguishes between clinically relevant states (for example, disease vs. no disease, subtype A vs. subtype B, progression vs. stability, or likely responder vs. non-responder) when applied to the target patient group under conditions that resemble intended clinical use.

This announcement is intentionally positioned late in the biomarker pipeline. Applicants are expected to come in with several key prerequisites already in place: first, a candidate biomarker that has already been identified and is supported by strong preliminary evidence; second, a detection or measurement method that has already been developed and analytically validated (meaning the assay or measurement approach has been tested for technical performance characteristics such as accuracy, precision, sensitivity, specificity, reproducibility, and robustness as appropriate); and third, a clear clinical or research need and a plausible "context of use" describing how the biomarker would be used in decision-making. The funding is therefore not aimed at inventing a new assay from scratch or conducting early discovery work, but at executing the clinical studies needed to establish whether the biomarker truly performs reliably and predictively in the intended population.

The activity uses a U01 cooperative agreement mechanism, which generally means the NIH anticipates substantial scientific and/or programmatic involvement during the project period compared to a typical research project grant. While clinical trials are described as optional under this announcement, the scope is still centered on clinical validation within patient populations, so applicants should expect to justify study design choices carefully and show that the clinical evaluation plan is rigorous and appropriate for generating clinically meaningful predictive values. A strong application would typically emphasize well-characterized cohorts, careful definition of clinical endpoints, appropriate reference standards or comparator measures, pre-specified statistical analysis plans, and steps to reduce bias and improve generalizability (for example, multi-site enrollment, standardized operating procedures, and attention to confounders that might affect biomarker readings).

Eligibility is broad and includes many types of U.S.-based organizations and governments, such as state, county, city or township governments; special district governments; independent school districts; public and state-controlled institutions of higher education; private institutions of higher education; nonprofit organizations (both with and without 501(c)(3) status); for-profit organizations (other than small businesses) as well as small businesses; public housing authorities/Indian housing authorities; and Native American tribal governments (federally recognized) and tribal organizations (other than federally recognized tribal governments). The announcement explicitly highlights 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, regional organizations, eligible federal agencies, and U.S. territories or possessions.

Foreign eligibility is limited. 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, meaning a U.S. applicant may include certain foreign collaborations or elements if they are justified and meet NIH policy requirements.

Administratively, the opportunity is listed as a discretionary funding program under the health category, administered by the National Institutes of Health, with CFDA number 93.853. The funding instrument is a cooperative agreement (U01). The original closing date provided in the source information is 2023-09-07, and the opportunity record creation date is 2020-11-19. The award ceiling and expected number of awards are not specified in the provided listing, so applicants would typically look to the full funding announcement and related NIH institute/center guidance for budget expectations, project period norms, and any institute-specific priorities.

Overall, the core idea of PAR-21-058 is to bridge the gap between promising biomarker candidates and real-world clinical usefulness in neurology and neuromuscular disease by funding the kind of careful, population-based validation work needed to support eventual regulatory qualification, clinical adoption, or use in therapeutic development and clinical research.

• The National Institutes of Health in the health sector is offering a public funding opportunity titled "Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.853.
• This funding opportunity was created on 2020-11-19.
• Applicants must submit their applications by 2023-09-07. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• 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 21 058

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