Program seeks Council approval for an initiative titled “Translational Research in Obstetric and Pediatric Pharmacology and Therapeutics”.
Therapeutics discovery and development for fetal, neonatal, pediatric, pregnant, and lactating patients continue to lag behind new and emerging therapies for adult and non-pregnant populations. Indeed, the need for safe and effective therapies for these populations is so great that federal legislation has encouraged or mandated research supporting pediatric and obstetric drug development, including the Pediatric Research Equity Act, the Best Pharmaceuticals for Children Act, and most recently the 21st Century Cures Act, which established the Task Force on Research Specific to Pregnant Women and Lactating Women (PRGLAC Task Force). Neonates, children, pregnant persons, and lactating persons undergo complex physiological changes that impact organ systems as well as drug pharmacokinetics and pharmacodynamics. Therapeutics taken by a pregnant or lactating woman may be passed to and adversely impact the fetus, neonate, or infant. Research on new and repurposed therapeutics as well as tools, biomarkers, models, and other technologies that elucidate the underlying mechanisms of drug action are necessary to drive innovation for the safe and effective treatment of pediatric, obstetric, and lactating patients.
In the last cycle of this initiative, we funded 17 awards (9 R01 and 8 R21 applications) spanning numerous scientific areas, from the discovery of new drugs to the repurposing of existing medications, development of disease and safety models, and identification of clinical and biological biomarkers of disease, among others. The awards shared the common goal of broadening our understanding of pediatric or obstetric pharmacology while addressing the critical need for new and safe therapeutics for these populations. Examples of awards funded during this cycle include a tool to detect unexpected adverse effects of new drugs given to pediatric patients using real-world data, a study on repurposed drugs for severe nausea and vomiting during pregnancy, and pharmacokinetic/pharmacodynamic models for a variety of treatments in infants.
The purpose of this initiative is to promote basic, translational, and clinical research to (1) advance the understanding of underlying mechanisms of drug action; (2) discover and develop novel therapeutics; (3) enhance the usage of existing drugs or drug repurposing for safer and more effective medications in children and pregnant and lactating persons. The overall goal is to improve safe and effective precision therapeutics for neonates, children, pregnant persons, and lactating persons.
This initiative aligns with the NICHD’s Strategic Plan 2020, particularly Theme 5, which promotes the goal of advancing safe and effective therapeutics and devices for children, pregnant and lactating persons, and people with disabilities. This initiative aligns with Theme 5’s scientific priorities, including the conduct of pharmacokinetic, pharmacodynamic, pharmacogenomic, dosing, and formulation studies; identification of specialized biomarkers and modeling approaches; and utilization of large-scale datasets to develop treatments that meet the need of children, pregnant or lactating persons, and people with disabilities. The initiative also aligns with numerous recommendations made by the PRGLAC Task Force.
Furthermore, this initiative aligns with the OPPTB’s research priorities to expand the knowledge in pharmacokinetics, pharmacodynamics, pharmacogenomics, biomarkers, new drug development, and drug repurposing for the treatment of pediatric and obstetric diseases.
Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)
Back to Concept Review by Council