Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)

Overview/Mission

OPPTB aims to assure that there are safe and effective therapeutics for children and pregnant and lactating women and that these medications are used optimally according to individual needs.
The branch promotes basic, translational, and clinical research to improve the safety and efficacy of therapeutics, primarily pharmaceuticals. It is responsible for developing and supporting a comprehensive national effort to increase the knowledge base for understanding how to appropriately treat disease during pregnancy, lactation, infancy, childhood, and adolescence using evidence-based therapeutic approaches. This includes expanding the genomic understanding, phenotypic characterization, and use of advanced ’omics technologies to inform prevention and treatment strategies. The goal of these efforts is to assure that medications are appropriately tested for dosing, safety, and effectiveness for individuals within their target populations.

Multiple gaps in knowledge regarding the use of therapeutics in children and pregnant and lactating women have led to inadequate labeling and frequent off-label use of prescription drugs. One of the branch's major activities is implementation of the Best Pharmaceuticals for Children Act (BPCA). The BPCA legislation promotes the prioritization of off-patent drugs and therapeutic areas that need further study in pediatrics and allows NICHD to sponsor clinical research of the prioritized therapeutics and disseminate results to improve drug labeling.

We are interested in applications that align with the following research priorities. For more information about NICHD’s research themes and aspirational goals, see NICHD Strategic Plan 2020 (PDF 2.3 MB).

Understanding Differences and Heterogeneity in Pediatric Disease Treatment

Gap: There is a lack of safe and effective therapies for many pediatric conditions. This is driven by a poor understanding of how drug action and pathophysiology differ for pediatrics versus similar conditions in adults and how individual factors (e.g., developmental stage genomics and microbiome) contribute.

Priority: Research to quantitatively understand differences in drug action and related pathophysiology between childhood and adult disease and conditions unique to pediatrics. This includes developing tools (e.g., biomarkers, outcome measures, and physiologically based pharmacokinetic/pharmacodynamic models) to support pediatric drug discovery and development and to facilitate the application of precision medicine approaches in children. NICHD considers applications for pediatric conditions that have significant efforts at other NIH institutes (e.g., pediatric oncology, juvenile diabetes) to be of lower programmatic priority.

Pharmacology and Pathophysiology of Pregnancy

Strategic Plan Theme 5: Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities
Strategic Plan Aspirational Goals: Identify genomic changes and exposure risks that explain or predict fetal loss; Use understanding of immune factors in pregnancy and placental development to determine reasons for pregnancy rejection

Gaps: There is a lack of effective and safe therapies for conditions that arise during pregnancy or for existing co-morbidities that contribute to high rates of maternal morbidity and mortality. A key requirement for the advancement of therapeutics that can restore the foundation for healthy pregnancies is understanding how drug action is altered during normal pregnancy, the post-partum period, and lactation.

Priority: Developmental pharmacology research and approaches that explore the intersections of physiological changes in women and during fetal development with drug action (e.g., pharmacokinetic, pharmacodynamics, and pharmacogenomics) and with molecular pathways that may serve as novel therapeutic targets for disease-modifying therapies specific to these populations. Critical areas include pain management in pregnant and lactating women and treatment of gestational diabetes, preeclampsia, and prevention of preterm delivery.

Novel Alternatives to Traditional Pediatric and Obstetric Clinical Trials

Gap: Significant challenges exist in the design and execution of pediatric and obstetric clinical trials, including recruitment, retention, and ethical concerns, among others.

Priority: Development of innovative approaches and algorithms to determine drug dosing, safety, and effectiveness in children and in women during pregnancy and lactation. This includes artificial intelligence-driven modeling and simulation methods, novel approaches to utilizing existing data and archived biosamples/biospecimens, and pragmatic trials.

Population- and Individual-Specific Diagnostic and Therapeutic Devices

Gap: There is a lack of devices to support diagnosis, drug delivery, and non-drug therapy appropriate for use in neonates, children, and pregnant and lactating women.

Priority: Development of devices specific to these populations, accounting for individual differences, that can advance precision medicine through individualized diagnosis, drug delivery, and non-drug therapy. This may include 3D bioprinting, AI-driven diagnostic and decision-making tools, novel drug delivery devices, and formulations.

New Uses for Drugs, Biologics, and Other Therapeutics

Strategic Plan Theme 5: Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities

Gap: There is a lack of safe and effective medications for children and pregnant women. This is partially due to the historical lack of inclusion of these populations across the drug development process.

Priority: Research and the development of approaches and technologies to identify drugs, biologics, pharmaceutical assets, etc., that may have new therapeutic uses for pediatric conditions or in pregnant and lactating women. This includes the development and use of preclinical experimental models (e.g., animal models and human biomimetics) and computation models or the accumulation of real-world evidence in support of new therapeutic uses.

Branch Research Focus and Mission: Expand the knowledge of pharmacokinetics, pharmacodynamics, pharmacogenomics, outcome measures, and biomarkers of disease and treatment.
3D Printing for Creation of Implantable Devices: Encourages research that will lead to development of minimally invasive/non-implantable flexible medical devices that will adapt to the needs of growing children
BPCA: Aims to improve pediatric therapeutics through preclinical and clinical research that improves knowledge of pediatric medications
Maternal and Pediatric Precision in Therapeutics (MPRINT) Hub: Seeks to aggregate, present, and expand the available knowledge, tools, and expertise in maternal and pediatric therapeutics to the broader research, regulatory science, and drug development communities.
Pediatric Trials Network : Aims to improve the dosing, safety, and effective use of therapeutic drugs in neonates and older children through clinical trials that lead to drug labeling changes for these populations
Obstetric-Fetal Pharmacology Research Centers: Aims to improve the safety and effective use of therapeutic drugs in women during pregnancy and lactation
Specialized Centers in Research in Pediatric Developmental Pharmacology: Investigates mechanisms of changes in drug disposition and response throughout development, provides an arena for multidisciplinary interactions between basic and clinical scientists, and serves as a resource for training and career development of new scientists
T32 Pediatric Pharmacology Training and Lecture Series : Helps ensure a diverse and highly trained workforce in new "-omics" technologies and their application to pediatric therapeutics, integration, and synergistic interaction; includes a weekly webinar (from October to June) on topics in pediatric clinical pharmacology

Branch-Funded Projects

Now in NICHD's Data and Specimen Hub (DASH): BPCA Data
BPCA Findings and Resources: Describes findings from BPCA research and lists resources for pediatric pharmacology researchers
Current BPCA Clinical Trials: Provides a listing of current BPCA clinical trials (PDF 267 KB)
Food and Drug Administration (FDA) Office of Pediatric Therapeutics: Highlights FDA activities related to pediatric pharmacology, therapeutics, and safety
FDA Pediatric Drug Development related to BPCA Lists information about FDA activities related to pediatric product development
Healthcare Preparedness for Children in Disasters: 2015 NACCD Report (PDF 655 KB): National Advisory Committee on Children and Disasters report on healthcare preparedness

Aaron Pawlyk, Branch Chief
Cenia Davis, Administrative Assistant
June Lee, Clinical Pharmacologist/Program Director
Main Research Areas: Artificial intelligence (AI) for medical treatments; digital biomarkers; AI drug delivery systems and formulations; AI treatment algorithm models for pediatric/obstetric patients; AI pharmacokinetic/pharmacodynamic models for pharmacology and therapeutics
Antonello Pileggi, Program Officer
Main Research Areas: Pediatric and obstetric devices; diagnostic devices; drug delivery systems; eHealth applications; novel biomaterials
Zhaoxia Ren, Program Director
Main Research Areas: Translational research in pediatrics and obstetrics; pharmacogenomics, pharmacoepidemiology; obstetric-fetal pharmacology; medications in pregnant and lactating women; new drug development
Lesly Samedy Bates, Program Officer
Main Research Areas: Developmental pharmacology and therapeutics in pediatrics; translational and clinical research; pharmacogenomics and pharmacoepidemiology; health disparities research; underserved, under-represented, and underreported populations
Perdita Taylor-Zapata, Medical Officer
Main Research Areas: Pediatric medicine; pharmacology: pediatric clinical trials design and implementation

Highlights

OPPTB invites applications to form the MPRINT Hub, which aims to aggregate, present, and expand the available knowledge, tools, and expertise in maternal and pediatric therapeutics to the broader research, regulatory science, and drug development communities.
Pediatric Pharmacology Training in the United States: At the most recent T32 Fellows' meeting, OPPTB's Dr. George Giacoia discussed NICHD's efforts to enhance the pediatric pharmacology program through effective collaborations and innovative research projects.
2019 BPCA Stakeholders Meeting : OPPTB held a stakeholder meeting/webinar on March 22, 2019. The meeting’s goals were to: provide an update on current studies, recent label changes, and scientific advances of the BPCA program; identify continuing gaps in pediatric drug development in select therapeutic areas; and discuss the program’s scientific direction.

COVID-19 is an emerging, rapidly evolving situation.

Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)

Overview/Mission

Branch Research Priorities