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Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)
Overview/Mission
OPPTB supports research and research training to advance the development and safe and effective use of therapeutics for children and for pregnant, postpartum, and lactating women.
The branch promotes basic, translational, and clinical research across a broad range of therapeutics and technologies, including pharmaceutical drugs, biologics, medical devices, drug-delivery systems, and regenerative medicine and tissue-engineered products. The branch develops and supports comprehensive national efforts to increase the knowledge base for understanding appropriate therapeutic treatment of diseases and conditions during pregnancy, the postpartum period, and lactation, and across pediatric development, from infancy through childhood and the transition from adolescence to adulthood.
OPPTB-supported research advances understanding of pharmacokinetics, pharmacodynamics, pharmacogenomics, developmental pharmacology, biomarkers, and variability in therapeutic response. The branch promotes innovative approaches that leverage genomics and multi-omics technologies, real-world data and real-world evidence, data science and artificial intelligence, advanced clinical trial designs, and pharmacometric and physiologically based pharmacokinetic modeling and simulation. These approaches can help identify therapeutic targets, improve prediction of treatment response and safety, inform dose selection and clinical trial design, and advance individualized and precision approaches to therapeutics. The goal of these efforts is to ensure that therapeutics are appropriately tested for dosing, safety, and effectiveness for individuals within the populations of interest. Significant evidence gaps remain regarding therapeutics and devices used in children and during pregnancy and lactation, contributing to limited product labeling and off-label use. OPPTB addresses these gaps through investigator-initiated research, targeted research programs, scientific resources and networks, research training, public-private and interagency collaborations, and implementation of congressional and NIH initiatives.
Branch Research Focus and Mission: Addresses gaps in knowledge about pharmacokinetics, pharmacodynamics, pharmacogenomics, outcome measures, and biomarkers of disease prevention and treatment for the safe and effective use of therapeutics for children and pregnant and lactating women
Advancing Knowledge in Pharmacology and Therapeutics in Pediatric, Obstetric, and Lactating Populations: Expands research to help ensure safety and effectiveness of medications and therapeutics for children and pregnant and lactating women in the prevention, treatment, and management of diseases and disorders
Translational Research in Maternal and Pediatric Pharmacology and Therapeutics: OPPTB supports translational and clinical research to: (1) advance precision medicine through the development, testing, and implementation of novel designs, tools, models, and other technologies that could have a direct clinical or health impact in pregnant, lactating and postpartum women, and in neonates, children, and adolescents; (2) discover and develop novel therapeutics or enhance the usage of existing drugs or drug repurposing for safer and more effective medications in pregnant, lactating, and postpartum women, and in neonates children, and adolescents; (3) enhance patient-provider communications about drug effectiveness and safety risks in pregnant, lactating and postpartum women, and in neonates, children, and adolescents; and, (4) optimize the dissemination, implementation, sustainment and scale-up of evidence-based interventions to pregnant, lactating and postpartum women, and in neonates, children, and adolescents. The overall goal is to improve safe and effective precision therapeutics for pregnant and lactating women, fetuses, neonates, and children, including those with disabilities.
Best Pharmaceuticals for Children Act (BPCA): One of the branch's major activities is implementation of the Best Pharmaceuticals for Children Act (BPCA) legislation, which promotes the prioritization of off-patent drugs and therapeutic areas that need further study in pediatrics. The BPCA allows NICHD to identify and prioritize needs in pediatric therapeutics; sponsor research on off-patent drugs and therapeutic areas requiring further study in children; submit resulting clinical study data to the U.S. Food and Drug Administration (FDA) for consideration of pediatric labeling changes; disseminate study results; and support research and training to address gaps in pediatric therapeutics. The program seeks to improve pediatric therapeutics through nonclinical and clinical research that improves knowledge of pediatric medications; examples of activities under this program include the following:
Pediatric Trials Network (PTN): Provides infrastructure and expertise for regulatory-rigorous clinical studies of therapeutics used in neonates, infants, children, and adolescents. PTN studies dosing, pharmacokinetics, safety, effectiveness, formulations, and other aspects of pediatric therapeutics and generates evidence that can inform FDA product labeling.
BPCA Prioritization and Research: Identifies high-priority gaps in pediatric therapeutics through collaboration with FDA, other NIH Institutes and Centers, researchers, clinicians, patients and families, and other stakeholders and supports research to address those needs.
BPCA Framework to Enable Pediatric Drug Development: Provides a curated resource for investigators and other stakeholders involved in pediatric drug development, including resources related to clinical trial design, biomarkers, pharmacokinetic modeling, pediatric formulations, pharmacoepidemiology, and quantitative systems pharmacology.
Clinical Pharmacology Training Network (CPTN): Supports development of a highly skilled clinical pharmacology and therapeutics workforce and provides training and career-development opportunities in pediatric clinical pharmacology, drug development, emerging technologies, and related disciplines.
Maternal and Pediatric Precision in Therapeutics (MPRINT) Hub: The MPRINT program serves as a national resource to advance therapeutics research for maternal, lactating, and pediatric populations. MPRINT brings together expertise, research platforms, data resources, modeling approaches, and educational resources to support investigator-initiated research, clinical studies, regulatory science, and therapeutic development.
Current and emerging areas of emphasis include coordination and support for multisite therapeutic studies; pharmacometrics and physiologically based pharmacokinetic modeling; use of real-world data and real-world evidence; mother-infant data linkage; genomics and other multi-omics approaches; research using human biospecimens, including human milk; and dissemination of tools and knowledge to the broader research community.
MPRINT resources, which comprise a Knowledge Portal, Peds Phecodes and COPPER, are intended to facilitate more informative and efficient studies and help address persistent evidence gaps in therapeutics used during pregnancy, lactation, and childhood.
Research Specific to Pregnant and Lactating Women: OPPTB supports NICHD and NIH efforts to advance research on therapeutics used during pregnancy and lactation and to facilitate the appropriate inclusion of pregnant and lactating women in clinical research.
The branch contributes to implementation of recommendations from the Task Force on Research Specific to Pregnant Women and Lactating Women (PRGLAC). These activities support efforts to address scientific, ethical, regulatory, and operational barriers to research; improve the evidence base for therapeutics used during pregnancy and lactation; expand use of real-world data and innovative research methods; and strengthen resources and infrastructure for studies involving these populations.
Transporter Elucidation Network (TEN): OPPTB supports the TEN to address critical knowledge gaps in the transport of drugs, nutrients, and other molecules across biological barriers important to maternal and pediatric health. Research focuses on transporters in tissues such as the placenta, lactating mammary gland, developing gastrointestinal tract, and blood-brain barrier
The network brings together expertise in transporter biology, pharmacology, structural biology, computational science, and modeling to generate broadly useful knowledge and research resources. Improved understanding of transporter function can inform drug disposition, nutrient transfer, therapeutic safety, and treatment strategies during pregnancy, lactation, infancy, and childhood.
Medical Devices and Technology Development: OPPTB supports research and innovation to improve medical devices and related technologies for children and for pregnant and lactating women. Research includes pediatric-specific and developmentally appropriate devices, diagnostic and point-of-care technologies, monitoring technologies, drug-delivery systems, biomaterials, implantable therapeutic constructs, and technologies for obstetric and gynecologic applications.
The branch is advancing multi-stakeholder efforts toward a National Pediatric Medical Device Ecosystem to strengthen the infrastructure needed to move pediatric medical technologies from innovation through development, clinical validation, regulatory evaluation, commercialization, and implementation. The initiative emphasizes coordination among stakeholders, efficient use of existing resources, access to specialized expertise, and public-private partnerships to help address longstanding gaps in pediatric-specific medical devices.
Training and Career Development: OPPTB supports development of the next generation of researchers and clinician-scientists in maternal and pediatric pharmacology and therapeutics through individual fellowships, institutional training programs, mentored career-development awards, research education programs, and specialized clinical pharmacology training.
The Clinical Pharmacology Training Network (CPTN) provides opportunities for interdisciplinary training and collaboration in clinical pharmacology and therapeutics. Training encompasses areas such as clinical trial design, pharmacokinetics and pharmacodynamics, pharmacometrics, drug development, regulatory science, multi-omics technologies, and precision therapeutics.
OPPTB also supports postdoctoral and career-development opportunities designed to strengthen the pipeline of investigators conducting rigorous maternal and pediatric therapeutics research.
OPPTB supports training and career development opportunities for researchers and clinicians who intend to focus on maternal and pediatric pharmacology.
Like other DER branches, OPPTB utilizes the following mechanisms:
Clinical Pharmacology Training Network (CPTN): Seeks to ensure a diverse and highly trained workforce is available in pediatric pharmacology-related clinical and biomedical fields by fostering entry points for aspiring clinician-scientists, cultivating early intellectual curiosity, and maintaining continuity, thus promoting and sustaining career development
Pediatric Clinical Pharmacology Research Career Development (K12) Program: Through a national network of mentors and scholars, the program allows scholars to conduct their research independently, while getting formal mentorship and support from national-level clinical pharmacologists, thus ensuring competitiveness for major grant support
Contact Lesly Samedy Bates with questions about OPPTB training and career development opportunities.
Access data from OPPTB-supported networks and initiatives, such as BPCA and the historic Pediatric Pharmacology Research Units Network in NICHD's Data and Specimen Hub (DASH)
PRGLAC Reports and Recommendations: Includes the PRGLAC Report to the HHS Secretary and Congress, the list of recommendations from the report, and the PRGLAC Report Implementation Plan
Division of Pediatric and Maternal Health: Describes FDA activities related to the study of drug and biological products in pediatric populations and the improvement of pregnancy and lactation-related information in product labeling
Aaron Pawlyk, Ph.D., Branch Chief Main Research Areas: ssay development and high throughput screening; novel drug and therapeutic discovery; biomarker discovery, validation, and qualification
Camille Fabiyi, Ph.D., Program Officer Main Research Areas: Medications and therapeutics for obstetric conditions and their role in lactation and breastfeeding; the inclusion of pregnant women in clinical research; dissemination and implementation research to support perinatal health outcomes
Antonello Pileggi, M.D., Ph.D., M.S.C.T.I., Program Officer and Associate Branch Chief Main Research Areas: Pediatric and obstetric devices; diagnostic devices; drug delivery systems; eHealth applications; novel biomaterials; neonatal pharmacology
Lesly Samedy Bates, Pharm.D., Ph.D., M.S., Program Officer Main Research Areas: Developmental pharmacology and therapeutics in pediatrics; translational and clinical research; pharmacogenomics and pharmacoepidemiology; health disparities research
Perdita Taylor-Zapata, M.D., Medical Officer Main Research Areas:Pediatric medicine; pharmacology: pediatric clinical trials design and implementation, including BPCA
Highlights
Now Available: HEAL KIDS (Knowledge, Innovation, and Discovery Studies) Pain Program. NICHD is leading activities for the KIDS Pain Program, part of NIH's Helping to End Addiction Long-term® Initiative (NIH HEAL Initiative®), through two Requests for Application (RFAs), RFA-HD-24-011 and RFA-HD-24-012. Check out these newly posted Frequently Asked Questions and the recording from a technical assistance webinar related to the KIDS Pain Program and the RFAs on the HEAL Initiative website.
Developing a Framework to Address Issues for Research Specific to Pregnant and Lactating Women (National Academies): This ongoing effort will develop a framework that enables researchers to address various issues related to research on pregnant and lactating women. Outcomes from this project will complement implementation of PRGLAC recommendations to improve research endeavors and evidence specific to this population. Visit the website to learn more about the project, upcoming and past workshops and meetings, publications, and other details.
Advancing Bioprinting and Regenerative Medicine Solutions for Obstetric, Gynecologic, and Pediatric Applications Workshop. This workshop, held November 16-17, 2021, was a transdisciplinary discussion on the state-of-the-art of tissue-construct manufacturing using 3D printing of biological, cellular, and tissue-based products (a.k.a., bioprinting) and regenerative medicine in the context of obstetric, gynecologic, and pediatric applications. Visit the meeting page to access recordings from Day 1 and Day 2.
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