Program seeks Council approval for an initiative titled “Catalyzing Innovation in Pediatric Pharmacology Clinical Trial Design by Resource Access”.
Clinical trials are essential in overcoming knowledge gaps in the prevention, diagnosis and treatment of various diseases in the pediatric population. Clinical trials represent a significant investment of resources — human, financial and material. Effective vision, planning, oversight and objective standards for the safe, timely and cost-efficient conduct of clinical trials, particularly in the special population of pediatrics, are integral in maximizing their impact on health and return on investment to all stakeholders. For many clinical trials in pediatrics, the failure rate can be high. Analyses by stakeholders across various fields have shown that failure rates can be attributed to many factors, including poorly executed recruitment plans, poor dose selection, limitations in validated outcome measures and many other design-related factors. The design and planning of regulatory rigorous clinical trials in pediatrics require specific expertise that is difficult to locate and access.
The purpose of this initiative is to provide investigators with access to consultative expertise from the Best Pharmaceutical for Children Act (BPCA) Clinical Program. The initiative would include (1) access to Pediatric Trials Network (PTN) and Data Coordinating Center (DCC) available resources for pediatric clinical trial implementation and support and (2) access to expertise in clinical trial design for both pilot studies and full scale pediatric therapeutic clinical trials that focus on dosing, safety, and effectiveness of medications used in children. By providing greater access to these expert resources, the goal of the initiative is to facilitate design of multidisciplinary research approaches to pediatric drug development clinical trials that can ultimately lead to drug label changes for medications lacking such data or advance novel drugs.
This initiative aligns with the NICHD’s Strategic Plan 2020, particularly Theme 5, which promotes the goal of advancing safe and effective therapeutics and devices for children, pregnant and lactating women, and people with disabilities.
This initiative aligns with the OPPTB’s research priorities to expand the knowledge and training in pharmacokinetics, pharmacodynamics, pharmacogenomics, outcome measures, and biomarkers.
Program Contact
Perdita Taylor-Zapata
Obstetric & Pediatric Pharmacology & Therapeutics Branch (OPPTB)
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