Program seeks Council approval for an initiative titled “Translational Research in Maternal 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 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 women, and lactating women 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. Advances in therapeutic effectiveness and safety research will also require the translation and implementation of evidence-based interventions (EBI) into clinical care.
In the last cycle of this initiative, we funded 31 awards (22 R01 and 9 R21 applications) spanning numerous scientific areas, from pregnancy and lactation pharmacology, maternal-fetal and neonatal therapeutics, pharmacokinetics/pharmacodynamics and PBPK modeling, drug safety and toxicology, and biologics and novel drug delivery, 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 the scientific areas funded include the development of disease and safety models, dosing algorithms, novel small molecule therapeutics, PKPD models, therapeutic biomarkers, repurposed therapeutics, and novel biological therapeutics.
The purpose of this initiative is to promote translational and clinical research in pregnant women, lactating women, infants, children, and adolescents to: (1) advance precision medicine through the development, testing, and implementation of novel tools, models, and other technologies; (2) discover and develop novel therapeutics or enhance the usage of existing drugs for safer and more effective medications; (3) enhance patient-provider communications about drug effectiveness and safety risks; and, (4) optimize the dissemination, implementation, sustainment, and scale-up of evidence-based interventions to these patient populations. The overall goal is to improve safe and effective precision therapeutics during pregnancy, lactation, and across childhood.
This initiative aligns with the NICHD’s Strategic Plan 2025, particularly Theme 5, which promotes the goal of advancing safe and effective therapeutics and devices for children, pregnant and lactating women, 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 and validation of biomarkers and modeling approaches; the support of clinical research and dissemination and implementation research to evaluate scalable, accessible therapeutics, diagnostics, and medical devices for these populations; and utilization of advanced data science techniques to assess health outcomes, therapeutic safety and effectiveness, patient engagement and health care delivery in datasets that include children, pregnant or lactating women, 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 programmatic research areas to expand knowledge in pharmacokinetics, pharmacodynamics, pharmacogenomics, biomarkers, new drug development, and drug repurposing for the treatment of pediatric and obstetric diseases.
Program Contact
Camile Fabiyi
Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)
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