Keywords: Extrapolation; Pediatric Drug Development; Bayesian Hierarchical Modeling; PK/PD Data
Extrapolation may be generally defined as extending information and conclusions available from studies in the source population (or in related conditions or medicinal products) to make inferences for the target population (or condition or product), thus reducing or eliminating the need to generate additional information to reach conclusions for the target population. The need is especially acute in pediatrics, where small sample size and other challenges mean that pediatric drug development is often delayed or abandoned. In this presentation, we will discuss the use of Bayesian hierarchical modeling approaches as a powerful and systematic tool for incorporating prior information from the adult population for pediatric drug development. Furthermore, we will describe an innovative approach by jointly modeling of both PK/PD and clinical data and systematically determining how much information we can borrow from adult data based on the evidence in PK/PD studies.