Use of modeling and simulation in the design and conduct of pediatric clinical trials and the optimization of individualized dosing regimens

Abstract

Mathematical models of drug action and disease progression can inform pediatric pharmacotherapy. In this tutorial, we explore the key issues that differentiate pediatric from adult pharmacokinetic (PK) / pharmacodynamic (PD) studies, describe methods to calculate the number of participants to be enrolled and the optimal times at which blood samples should be collected, and therapeutic drug monitoring methods for individualizing pharmacotherapy. The development of pediatric-specific drug dosing dashboards is also highlighted, with an emphasis on clinical-relevance and ease of use.