Overview/Mission
OPPTB supports research and research training on the development and use of safe and effective therapeutics for children and pregnant and lactating people.
The branch promotes basic, translational, and clinical research to evaluate and 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 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 for children during infancy, childhood, and the transition from adolescence to adulthood. Branch-supported research 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-medicine 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) legislation, which 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. The branch is also active in implementing the recommendations of the Task Force on Research Specific to Pregnant Women and Lactating Women (PRGLAC).
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: 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 people
- 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 people in the prevention, treatment, and management of diseases and disorders
- Translational Research in Maternal and Pediatric Pharmacology and Therapeutics: Supports translational and clinical research to advance precision medicine through the development of novel tools, models, and other technologies
- Enhancing Medical Devices for Children and Pregnant and Lactating People: 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
- Best Pharmaceuticals for Children Act (BPCA) : Seeks to improve pediatric therapeutics through non clinical and clinical research that improves knowledge of pediatric medications; examples of activities under this program includes the following:
- Pediatric Trials Network
: Conducts clinical research to improve the knowledge of dosing, safety, and effectiveness of therapeutics used in neonates and older children and inform drug labeling for these populations - T32 Principles of Pediatric Clinical Pharmacology and Therapeutics Lecture Series: Trains a diverse and highly skilled 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: Aggregates, presents, and expands available knowledge, tools, and expertise in maternal and pediatric therapeutics to the broader research, regulatory science, and drug development communities; this national resource comprises a Knowledge and Research Coordination Center and Centers of Excellence in Therapeutics
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:
- Pediatric Clinical and Developmental Pharmacology Training Network (PCDPTN): 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
- Postdoctoral Training in Pediatric Clinical Pharmacology (F32/T32) Program: Supports postdoctoral research and career development trainees at the clinical pharmacology-biomedical sciences interface to help ensure that a diverse and highly trained workforce is available in pediatric pharmacology
- 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.
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.
- OPPTB participates in NICHD’s activities related to COVID-19 and leads components related to drug dosing in pediatric COVID-19 patients .
- Developing a Framework to Address Issues for Research Specific to Pregnant and Lactating Persons (National Academies): This ongoing effort will develop a framework that enables researchers to address various issues related to research on pregnant and lactating people. 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.
- BPCA Stakeholders Meetings. Access minutes from recent meetings or access the meetings archive for older minutes.
- Rapid Acceleration of Diagnostics Technology (RADx® Tech) for Maternal Health Challenge. This challenge, part of the NIH Implementing a Maternal health and Pregnancy Outcomes Vision for Everyone (IMPROVE) Initiative, prioritizes home-based or point-of-care diagnostic devices, wearables, and other remote sensing technologies to extend postpartum care in regions lacking access to maternity care. Learn about the RADx Tech Challenge winners.
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