Model-Informed Pediatric Drug Development: Application of Pharmacometrics to Define the Right Dose for Children.

Abstract

One of the biggest challenges in pediatric drug development is defining a safe and effective dose in pediatric populations, which span across a wide age and development range from neonates to adolescents. Model-informed drug development approaches are particularly suited to address knowledge gaps including data leveraging to increase the success of pediatric studies. Considering the often limited number of patients available for study and logistic difficulties to collect the necessary data in pediatric populations, the application of pharmacometrics and modeling and simulation techniques can improve clinical trial efficiency, increase the probability of regulatory success, and optimize therapeutic individualization in support of dedicated trials. This review describes the state of pediatric model-informed drug development to define the right dose for children and provides suggestions for future development.