Analytical and Clinical Validation of Biomarkers for Alzheimers Disease (AD) and AD-Related Dementias (ADRD) (U01 Clinical Trial Optional)

The National Institute on Aging (NIA) is accepting applications to support the analytical and clinical validation of biomarkers for Alzheimer’s disease (AD) and AD-related dementias (ADRD) under a U01 cooperative agreement. This initiative aims to accelerate the establishment of reliable biomarkers that are rigorously validated and align with FDA expectations, such as those outlined in the Biomarker Qualification Program (BQP). The program encourages applications that demonstrate the clinical utility of biomarkers to advance medical product development, improve patient care, and inform therapeutic interventions. The program is designed to support the validation of biomarkers within specific contexts of use (COUs), such as diagnostic, prognostic, predictive, pharmacodynamic, safety, or monitoring purposes. It targets the validation of biomarkers for AD, ADRD, and mixed pathologies, with a strong emphasis on underserved or heterogeneous populations. Applications must focus on candidate biomarkers that are already discovered, detailing analytical and/or clinical validation strategies to assess reliability and efficacy in predicting disease progression or therapeutic responses. Eligible applicants include institutions of higher education, nonprofit and for-profit organizations, local and federal government entities, and foreign organizations. Research should focus on human studies and must exclude early-stage biomarker discovery, non-human studies, or behavioral biomarker projects. Applicants may propose research budgets based on actual project needs, with project durations of up to five years for analytical and clinical validations combined or four years for analytical validation alone. Proposals must provide a detailed research strategy that includes rigorous design, data analysis, and validation metrics. Clinical validation efforts should address biomarker sensitivity, specificity, and predictive values tailored to the proposed COU. The program also emphasizes data management and sharing, with applicants expected to submit data to public repositories while adhering to ethical standards for confidentiality and informed consent. Applications will be reviewed based on the significance of the proposed research, the rigor of the validation methods, the expertise of the investigators, and the suitability of the environment. Proposals must include milestones and timelines with clear decision points, allowing for progress evaluations and potential adjustments. Successful applicants will collaborate with NIA staff to finalize project milestones post-award. Letters of intent are due by January 3, 2025, with the first application deadline on February 5, 2025. Applicants requesting $500,000 or more in direct costs for any year must consult NIA staff six weeks before submission. More details, including contacts for application assistance, are available in the funding announcement.