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.
- 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.