Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R01 - Clinical Trial Optional)

The National Institutes of Health (NIH) seeks applications for R01 Research Project Grants under the program "Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns." Administered by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), this funding opportunity focuses on advancing technologies and therapeutic interventions for conditions included in newborn screening panels or that may benefit from early detection in the future. This initiative is critical for improving outcomes through early intervention and for expanding the scope of disorders that can be effectively screened and treated. Key objectives include the development of innovative screening methods, optimization of existing technologies, and creation of new treatments. Potential research topics span from improving screening test accuracy and reducing false positives to leveraging artificial intelligence for screening algorithm development and evaluating treatments for rare genetic conditions. Projects involving clinical trials or preclinical testing for novel therapies are also encouraged. The R01 mechanism supports long-term studies aiming to deepen knowledge and foster significant scientific advancements. This program provides an unrestricted budget that must align with the needs of the project. The maximum project period is five years. Applicants from a wide range of eligible organizations, including higher education institutions, nonprofits, for-profit organizations, and foreign entities, are invited to apply. Individuals from diverse backgrounds, including underrepresented groups, are strongly encouraged to participate. Applicants must adhere to NIH registration requirements, including SAM.gov and eRA Commons. Applications will be assessed on their scientific merit, innovation, rigor, feasibility, and alignment with the program’s objectives. Key evaluation criteria include the significance of the research, the proposed approach's robustness, and the investigators' expertise. Proposals must include a detailed data management and sharing plan, as required by NIH policies, and should emphasize strategies for recruiting diverse populations in clinical studies. Applications open on January 5, 2025, with due dates set three times annually: February 5, June 5, and October 5. Submissions must comply with NIH guidelines and be submitted via ASSIST, Grants.gov Workspace, or an institutional system. Proposals requesting over $500,000 in direct costs annually must seek prior approval at least six weeks before submission. Awardees will be subject to standard NIH reporting requirements, including annual Research Performance Progress Reports (RPPR) and adherence to policies on data sharing and ethical research practices. This funding opportunity offers a pivotal platform for researchers to drive innovation in newborn screening and improve healthcare outcomes for affected populations.