The "Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns" (R03 - Clinical Trial Optional) funding opportunity offered by the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) aims to support small-scale research projects on developing novel screening methods and therapeutic interventions for newborns. This program focuses on both conditions currently part of newborn screening and high-priority genetic conditions that may benefit from early detection in the future. The R03 Small Grant Program supports short-term, limited-funding projects that are appropriate for investigators seeking to explore preliminary ideas, pilot studies, or exploratory research that could lead to larger-scale projects. This R03 announcement is a companion to the R01 and R21 funding opportunities (PAR-21-353 and PAR-21-355).

The goal of this funding opportunity is to enhance the detection and management of potentially fatal or disabling conditions in newborns, leveraging NICHD's Hunter Kelly Newborn Screening Research Program, which was authorized through the Newborn Screening Saves Lives Act. This act enables funding for developing new technologies, screening tools, and therapeutic approaches that can help identify and manage newborn conditions early, ideally before symptoms appear. The small-scale R03 grants are particularly suited to projects that can be completed within two years and require a focused approach or pilot data generation rather than extensive development.

Eligible applicants for this opportunity include public and private higher education institutions, nonprofits, small businesses, government entities, and foreign institutions. Researchers may propose projects that are exploratory or innovative, focusing on novel screening methods, treatments, and validations for conditions either already included in newborn screening or those anticipated to benefit from presymptomatic detection. NICHD encourages researchers to use resources from the Newborn Screening Translational Research Network (NBSTRN), which offers tools for longitudinal data collection, a virtual repository of resources, and ethical guidance related to newborn screening.

Research areas of interest include developing new assays for existing Recommended Uniform Screening Panel (RUSP) conditions or for future additions, adapting technologies for low-resource settings, optimizing treatments, and exploring genetic or environmental factors affecting newborn conditions. Other potential topics include preclinical testing in animal models, clinical trials for new treatments, and studies on the ethical, legal, and social implications of these technologies. Applications focused on screening are encouraged to consider implications for scaling and state-level implementation, with an emphasis on multiplexed technology that can test for multiple conditions simultaneously.

Funding for each R03 project is limited to a maximum of $50,000 in direct costs per year for up to two years. Applications are evaluated based on the scientific significance of the research, the innovation and approach of the project, and the potential to contribute valuable preliminary data or new insights. Projects involving clinical trials may also be submitted under this announcement, although they are optional, and the proposed trial must address an unmet need or significant research question.

The application process requires adherence to NIH policies and guidelines, including data-sharing plans for large-scale human genetic data, which are expected to be shared through platforms like the NICHD Data and Specimen Hub (DASH). Key deadlines follow standard NIH dates, with the current opportunity open until November 17, 2024.