PAR-25-266
Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns (R21 - Clinical Trial Optional)
Summary
PAR-25-266: Innovative Screening Approaches and Therapies for Screenable Disorders in Newborns
Research Focus
This funding opportunity supports exploratory and developmental research to advance newborn screening and early intervention for genetic and congenital disorders. The program targets two main areas: improving screening approaches and therapeutic interventions for conditions already included in state newborn screening panels, and developing screening and treatment capabilities for potentially fatal or disabling genetic conditions not yet screened. Research should address critical gaps where effective treatments lag behind screening technologies, or where reliable screening tests are lacking despite available treatments.
Investigators may pursue basic, translational, behavioral, or clinical research including: development and validation of novel screening assays and technologies (especially those adoptable by state laboratories); higher-tier screening assays to reduce false positives; artificial intelligence and computational modeling for screening algorithms; optimization or development of new therapeutic interventions; comparative effectiveness studies; preclinical safety and efficacy testing; clinical trials with validated health outcome markers; genotype-phenotype analyses; and health systems research on access to care in underserved communities. The program also welcomes embedded ethical, legal, and social implications (ELSI) research on novel screening and therapeutic approaches. Priority conditions include metabolic disorders, lysosomal disorders, hemoglobinopathies, congenital immunodeficiency syndromes, neuromuscular disorders, congenital heart disease, congenital endocrine disorders, chromosomal aneuploidies, and intellectual disability syndromes.
At-a-Glance
- Who can apply: Not stated
- Funding & project length: Not stated
- Award mechanism: R21 (Exploratory/Developmental Research Grant); clinical trials optional
- Key dates: Applications due February 16, 2025 (earliest); subsequent rounds June 16, 2025 and October 16, 2025; expiration November 17, 2027
- Best fit for: Pediatric geneticists, metabolic disease specialists, clinical chemists, and translational researchers developing novel screening technologies, biomarkers, or early interventions for newborn-detectable genetic and congenital disorders
Key Facts
Deadline
Tue, November 16, 2027
Posted
Mon, December 2, 2024
Award / Year (direct costs)
$275,000
Max Total
$550,000
Max Duration
2 years
Keywords
Research Areas
Gotchas (3)
R21 mechanism is explicitly for exploratory/novel projects; companion R01 (PAR-25-265) is recommended for long-term projects or those in well-established areas. Applicants must choose the correct mech
95%
Source Text
“The R21 activity code supports projects that are exploratory or novel, break new ground, extend previous discoveries toward new directions or applications, or generate pilot data in preparation for a larger study. Long-term projects, or projects designed to increase knowledge in a well-established area are well-suited for the companion R01 Exploratory/Developmental Research Grant NOFO, PAR-25-265.”
Applicants with clinical trials or interventional studies in humans are strongly encouraged to contact NICHD Scientific/Research staff prior to submission. Failure to do so may result in misalignment
90%
Source Text
“Applicants are strongly encouraged to contact Scientific/Research staff prior to submitting an application that includes a clinical trial or interventional study in humans.”
Data Management and Sharing Plan (DMS Plan) must be included in all applications and will be reviewed and approved by NIH Program Staff prior to award; awardees must comply with approved plan
90%
Source Text
“NIH requires all applications submitted in response to this NOFO to include a Data Management and Sharing Plan (DMS Plan). The DMS Plan is expected to address the Elements as described in Supplemental Information to the NIH Policy for Data Management and Sharing: Elements of an NIH Data Management and Sharing Plan (NOT-OD-21-014). The DMS Plan will be reviewed and approved by NIH Program Staff prior to award. Awardees will be required to comply with their approved DMS Plan and any approved updat”