Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R21 - Clinical Trial Optional)

The "Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns" funding opportunity, offered by the National Institutes of Health’s (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), supports research on new screening methods and treatments for severe, potentially fatal conditions detectable through newborn screening. This initiative, classified as an R21 Exploratory/Developmental Research Grant (PAR-21-355), aims to encourage novel approaches for both currently screened and high-priority conditions—those with the potential for future newborn screening inclusion, where early detection would significantly improve outcomes. The R21 grant mechanism is suited for early-stage, exploratory research or pilot studies that can feasibly be completed within two years and are designed to generate preliminary data or innovative techniques for larger studies. This funding opportunity is open to diverse research areas, including translational, basic, and clinical research focusing on conditions in existing newborn screening programs or those that could benefit from presymptomatic detection. Examples of eligible research include developing novel screening tools, improving existing screening and treatment for newborns, and understanding the genetic and environmental factors affecting disease onset and progression. Eligible applicants include a wide range of organizations, including higher education institutions, nonprofits, small businesses, local governments, and other public entities. Foreign institutions and non-domestic components of U.S.-based organizations are also eligible. Applications should detail how the proposed project will integrate with existing infrastructures, such as the Newborn Screening Translational Research Network (NBSTRN), which provides resources to support newborn screening research, including longitudinal pediatric disease data and common data elements (CDEs). Funding for these projects is capped at $275,000 in direct costs for the two-year project duration, with no more than $200,000 allocated in any single year. Applicants may propose projects that include clinical trials, but it is optional. Applications are encouraged to employ innovative strategies to address major gaps in screening technology or treatment. Proposals focused on therapeutic development should define the primary and secondary endpoints for measuring success, while screening-focused proposals should emphasize scalability and sensitivity across diverse populations. Applications undergo a rigorous review process, emphasizing significance, innovation, approach, and investigator expertise. Proposals must align with the objectives of advancing screening and treatment for newborn conditions, with clear relevance to clinical or public health needs. Additional evaluation criteria for clinical trials include study design, management, and analysis plans. Awardees must follow NIH’s data-sharing policies, contributing data to recognized repositories like the Genotypes and Phenotypes database (dbGaP) or the NICHD Data and Specimen Hub (DASH). Application deadlines occur three times a year on standard NIH submission dates, with the next deadline on February 16, 2024. This funding opportunity will expire on November 17, 2024.