Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)

Overview/Mission

OPPTB supports research and research training on the development and use of safe and effective therapeutic drugs and medical devices for children and pregnant and lactating people, including during the postpartum period.

The branch promotes basic, translational, and clinical research to improve the safety and efficacy of multiple types of therapeutics, such as, but not limited to, pharmaceutical drugs, biologics, medical devices, and regenerative tissue constructs. The branch is responsible for developing and supporting comprehensive national efforts to increase the knowledge base for understanding how to treat diseases and conditions appropriately for people during pregnancy, the postpartum period, and lactation, and for children during infancy, childhood, adolescence, and the transition from adolescence to adulthood. This work includes expanding the genomic understanding and phenotypic characterization of drug responses, and leveraging advanced multi-omics technologies, novel clinical trial approaches, data science and artificial intelligence methods, and pharmacometric modeling tools to inform prevention and treatment strategies. 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. Individualized and precision approaches are an important component to treating diseases of these populations.

Multiple knowledge gaps regarding the use of therapeutics in children and pregnant and lactating people have led to inadequate labeling and frequent off-label use of prescription drugs and devices. 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. The BPCA allows NICHD to sponsor clinical research of the prioritized therapeutics and to disseminate results to improve pediatric drug labeling.

We are interested in applications that align with the following research priorities. For more information about NICHD’s research themes, cross-cutting topics, and aspirational goals, visit the plan’s Scientific Research Themes and Objectives.

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 pregnant people 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 people 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 pregnant and lactating people. 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 people.

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 and lactating people. 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 people. 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 prevention and treatment
3D Printing for Creation of Implantable Devices: Encourages research that will lead to development of medical devices that will adapt to the needs of growing children as well as for obstetric and gynecologic applications
BPCA: Aims to improve pediatric therapeutics through preclinical and clinical research that improves knowledge of pediatric medications; this research area includes the following:
- Pediatric Trials Network : Aims to improve the knowledge of dosing, safety, and effectiveness of therapeutics used in neonates and older children through clinical trials that lead to improvements in drug labeling for these populations
- T32 Principles of Pediatric Clinical Pharmacology and Therapeutics Lecture Series: Helps ensure a diverse and highly trained workforce in new 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
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.

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
BPCA Pediatric Clinical Trials: Includes a listing of current BPCA clinical trials
Food and Drug Administration (FDA) Office of Pediatric Therapeutics: Highlights FDA activities related to pediatric pharmacology, therapeutics, and safety
FDA Division of Pediatric and Maternal Health: Lists information about FDA activities related to the study of drug and biological products in the pediatric population and the improvement of pregnancy and lactation-related information in product labeling
Obstetric-Fetal Pharmacology Research Centers: Aimed to improve the safety and effective use of therapeutic drugs in pregnant and lactating people
Pediatric Pharmacology Research Units Network (Historical/For Reference Only): Describes a now-concluded research network that facilitated and promoted pediatric labeling of new drugs or drugs already on the market and fostered cooperative and complementary research efforts
Specialized Centers in Research in Pediatric Developmental Pharmacology: Investigated mechanisms of changes in drug disposition and response throughout development, provided an arena for multidisciplinary interactions between basic and clinical scientists, and served as a resource for training and career development of new scientists

Aaron Pawlyk, Branch Chief
Main Research Areas: Assay development and high throughput screening; novel drug and therapeutic discovery; biomarker discovery, validation, and qualification
Dominique Davis, Administrative Assistant
Josh Lorenzo, Program Analyst
Main Research Areas: Clinical trials; regulatory affairs; human subjects research; program analysis
Antonello Pileggi, Program Officer
Main Research Areas: Pediatric and obstetric devices; diagnostic devices; drug delivery systems; eHealth applications; novel biomaterials; neonatal pharmacology
Zhaoxia Ren, Program Director
Main Research Areas: Translational research in pediatrics and obstetrics; pharmacogenomics, pharmacoepidemiology; obstetric-fetal pharmacology; medications in pregnant and lactating people; 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
Katie Vance, Program Officer
Main Research Areas: Clinical trial design and implementation; drug discovery and translational research; pharmacology; data management and sharing; electronic health records

Highlights

Our branch is hiring! Visit our Jobs page to learn more.
Advancing Bioprinting and Regenerative Medicine Solutions for Obstetric, Gynecologic, and Pediatric Applications Workshop. Watch the saved recordings of both days of this workshop, held November 16-17, 2021, as multidisciplinary experts in regenerative medicine research came together for 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. As a corollary to the workshop, stakeholders can still add ideas to the conversation about priority areas for future research, technology development, and resources in the 3D bioprinting and regenerative medicine fields through the Advancing Bioprinting and Regenerative Medicine Solutions for Obstetric, Gynecologic, and Pediatric Applications IdeaScale campaign .
OPPTB participates in NICHD’s activities related to COVID-19 and leads components related to drug dosing in pediatric COVID-19 patients .
News Release: NIH announces winners of data challenge to identify risk factors for first-time pregnancies. Awarded projects of the Decoding Maternal Morbidity Data Challenge seek to reduce pregnancy complications and prevent maternal deaths.
OPPTB intends to support 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. The MPRINT Hub will comprise a Knowledge and Research Coordination Center and Centers of Excellence in Therapeutics.
Pediatric Pharmacology Training in the United States: At the most recent T32 Fellows' meeting, former OPPTB member Dr. George Giacoia discussed NICHD's efforts to enhance the pediatric pharmacology program through effective collaborations and innovative research projects.
2020 BPCA Stakeholders Meeting (PDF 218 KB): OPPTB held a stakeholder meeting/webinar on December 14-15, 2020. The meeting’s goals were to: (1) provide updates to NIH on the BPCA clinical program; and (2) spearhead the development of a roadmap for identifying needs, prioritizing those needs, and closing knowledge gaps in pediatric therapeutics that are applicable to a variety of stakeholders involved in pediatric drug development.