Obstetric and Pediatric Pharmacology and Therapeutics Branch (OPPTB)

Overview/Mission

OPPTB promotes basic, translational, and clinical research to improve the safety and efficacy of therapeutics (primarily pharmaceuticals) and to ensure centralization and coordination of research, clinical trials, and drug development activities for obstetric and pediatric populations.

The branch is responsible for developing and supporting a comprehensive national effort to increase the knowledge base for understanding how to appropriately treat disease during pregnancy, infancy, childhood, and adolescence using evidence-based therapeutic approaches. The goal of this effort is to assure that medications are appropriately tested for dosing, safety, and effectiveness within their target populations.

Multiple gaps in knowledge regarding the use of therapeutics in children and pregnant women have resulted in lack of or inadequate labeling and frequent off-label use of prescription drugs in these two populations. 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 that need further study in pediatrics and allows NICHD to sponsor clinical research of the prioritized therapeutics and disseminate results to improve drug labeling.

New Drug Development and Drug Repurposing

Gap: There is currently a lack of safe and efficient medications for children and pregnant women. In fact, both populations often have been considered pharmaceutical "orphans."

Priority: Support projects to identify drug targets for children and pregnant women for conditions specifically relevant to these populations, including neonatal/pediatric pain, rare diseases, infectious diseases, cardiovascular disease (including hypertension), type 2 and gestational diabetes, pulmonary disease (including bronchopulmonary dysplasia), preterm labor, and preeclampsia. These targets can be used for the development of new drugs or to repurpose appropriate existing drugs.

Novel Alternatives to Traditional Pediatric and Obstetric Clinical Trials

Gap: Significant hardship exists in the design and execution of pediatric and obstetric clinical trials, secondary to many obstacles including recruitment, retention, and ethical concerns.

Priority: Support initiatives to develop innovative approaches and algorithms to predict drug dosing, safety, and effectiveness in children and in women during pregnancy and lactation. This includes modeling and simulation methods and advanced methods of utilizing existing data, such as electronic medical records.

Developmental Pharmacology and Pathophysiology of Pregnancy

Gap: A requirement for the development of safe and efficient medications for pregnant and lactating women is understanding the mechanisms of drug action (pharmacokinetics, pharmacodynamics, pharmacogenomics) in these populations and in their preclinical models to decrease the risks of adverse events and toxicity.

Priority: Support developmental pharmacology initiatives and initiatives that explore mechanisms of drug action in pregnant and lactating women. Critical areas include pain management in neonates and pregnant women, treatment of type 2 and gestational diabetes, and preterm birth.

Outcome Measures for Pediatric and Obstetric Clinical Trials

Gap: Outcome measures and biomarkers that reflect diseases/conditions and predict drug safety and effectiveness in children and pregnant and lactating women are lacking.

Priority: Support initiatives for the identification of outcome measures and biomarkers in this population. Outcome measures and biomarkers related to pain, sedation, type 2 and gestational diabetes, and acute kidney injury would be highly relevant.

Pediatric Formulations

Gap: Appropriate formulations for pediatric populations remain elusive or absent.

Priority: Support initiatives for the development of palatable and safe (i.e., without harmful additives) formulations for children.

Therapeutic Devices

Gap: Development of non-drug therapeutics, such as devices, is needed to improve therapeutic treatment for the fetus and for children.

Priority: Support development of non-invasive drug delivery systems and devices to measure drug safety or efficacy non-invasively.

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
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
George Giacoia, Medical Officer
Main Research Areas: Developmental pharmacology; pharmacology biomarkers; drug delivery systems and formulations; mechanisms of adverse drug reactions in children; training
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
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
Perdita Taylor-Zapata, Medical Officer
Main Research Areas: Pediatric medicine; pharmacology: pediatric clinical trials design and implementation

Highlights

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.