Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional) | PAR 24 097

Opportunity Information: Apply for PAR 24 097

Clinical Validation of a Candidate Biomarker for Neurological or Neuromuscular Disorders (U01 Clinical Trial Optional), Funding Opportunity Number PAR-24-097, is an NIH cooperative agreement designed to move promising neurological or neuromuscular biomarkers from the "strong candidate" stage into true clinical validation. The core aim is not discovery or early assay development, but rigorous testing of a biomarker in the real clinical population where it would be used, with study designs capable of producing reliable estimates of performance, especially positive predictive value (PPV) and negative predictive value (NPV). The opportunity explicitly frames validation in a way that aligns with FDA expectations for biomarker evidence, meaning applicants should be prepared to justify how their design, analysis plan, and patient selection will support clinically meaningful and regulator-relevant conclusions about how well the biomarker classifies or predicts the condition or outcome of interest.

This announcement assumes several key pieces are already in place before a project begins. First, the biomarker itself has already been identified as a strong candidate based on prior evidence. Second, the method for detecting or measuring the biomarker (the technology and assay) has already been developed and analytically validated, so the project is not meant to troubleshoot basic assay performance or invent a new measurement platform. Third, the clinical or research need is already articulated, along with the biomarker's potential context of use, such as diagnosis, prognosis, patient stratification, disease monitoring, pharmacodynamic response, or another defined role. In other words, this PAR is meant to fund the expensive, careful stage where you prove that the biomarker measurement holds up in the intended patient population and setting, producing clinically interpretable predictive values rather than just statistical associations.

The mechanism is a U01 cooperative agreement, which generally indicates substantial NIH programmatic involvement compared with a standard investigator-initiated grant. Practically, that often means awardees should expect active coordination with NIH staff on milestones, study conduct, data quality expectations, and possibly harmonization with broader NIH biomarker efforts. The "Clinical Trial Optional" label means applicants may propose a clinical trial if it is appropriate for validation, but they are not required to do so; strong observational or prospective cohort designs may also be suitable, depending on the biomarker's intended use and what evidence is needed to establish performance.

Eligibility is broad across government, academic, nonprofit, and industry organizations. Eligible applicants include state, county, city, 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; tribal organizations that are not federally recognized; public housing authorities/Indian housing authorities; nonprofits with or without 501(c)(3) status; for-profit organizations (other than small businesses); and small businesses. The announcement also calls out additional eligible applicant types, including Alaska Native and Native Hawaiian Serving Institutions, AANAPISISs, faith-based or community-based organizations, Hispanic-serving institutions, HBCUs, tribally controlled colleges and universities, regional organizations, eligible federal agencies, and U.S. territories or possessions. At the same time, it draws clear lines around foreign involvement: non-domestic (non-U.S.) entities are not eligible to apply, and non-domestic components of U.S. organizations are not eligible to apply. However, foreign components as defined by the NIH Grants Policy Statement are allowed, meaning certain international collaborations may be permissible when they meet NIH definitions and are justified within the project.

The opportunity is categorized as discretionary funding under the health activity area and is associated with CFDA number 93.853. The agency is the National Institutes of Health. It was created on 2024-01-22 and is described as a reissue of PAR-21-058, signaling continuity with an earlier program but updated as PAR-24-097. The listed original closing date is 2026-06-22. While an award ceiling is not specified in the provided source text and the number of expected awards is not shown, applicants should treat this as a competitive, milestone-driven biomarker validation program where budgets, timelines, and enrollment plans will likely be scrutinized for feasibility and for the ability to deliver definitive clinical validation evidence.

Overall, this PAR is best suited for teams that already have a well-supported biomarker candidate and a validated assay, and who are ready to run a carefully designed clinical validation study that can credibly establish predictive values in the target patient population. The emphasis is on rigor, real-world clinical relevance, and producing results that can support a clear context of use consistent with FDA-oriented biomarker evidentiary standards.

• 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 2024-01-22.
• Applicants must submit their applications by 2026-06-22. (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 24 097

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