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
Strategic Plan Theme 5: Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities
Strategic Plan Aspirational Goal: Facilitate application of precision medicine approaches in children
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
Strategic Plan Theme 5: Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities
Strategic Plan Aspirational Goal: Facilitate application of precision medicine approaches in children
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
Strategic Plan Theme 5: Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities
Strategic Plan Aspirational Goal: Facilitate application of precision medicine approaches in children
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
- Pediatric Trials Network
- 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.
Highlights
- OPPTB participates in NICHD’s activities related to COVID-19 and leads components related to drug dosing in pediatric COVID-19 patients .
- 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.
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