Keywords: Pediatrics, Extrapolation, Adolescents, Pediatric Trial Networks
Significant advances have been made in pediatric drug development over the past 2 decades yet there continues to be a significant lag in labeling information between adults and children. Pediatric considerations span the life of a compound from early development through execution of clinical trials. The driving force for pediatric drug development should be the child’s medical need. That need should be fulfilled using all available data while exposing as few children as possible to get necessary data. Drugs can be developed for adults, all ages, or specifically for pediatrics. For those drugs developed for all ages, early considerations in adult programs are critical and can have significant positive impact on pediatric programs such as defining exposure-response relationships, biomarker or end-point determinations and considerations of including adolescents in early adult trials once the compound is assessed to be safe. This information can lead to the use of extrapolation of efficacy in the pediatric program helping to minimize exposure of pediatric patients to research and helping insure a more efficient assessment of drugs for children. Drugs should be developed for specific pediatric or neonatal needs. These indications are most commonly for rare diseases and often require innovative study designs to be successfully completed. Using MIDD and PBPK can be extremely effective and accurate in determining initial pediatric doses. We must not ask of children/parents to be involved in drug development programs that have little chance of success and thus an assessment of feasibility is a necessity. Once the indication, study design, and agreements with regulatory authorities are set, the biggest challenge is the execution of the studies. Development of sustainable, non-profit global pediatric clinical trial networks in the past year are critical steps in the successful and prompt completion of our trials to benefit our children.