Study design and simulation approach

Modeling and simulation techniques are a mainstay of clinical drug development and are particularly useful to support clinical trials in children. If a pediatrician wants to use these tools most efficiently, a basic understanding of the principles and methods of classical and novel techniques of modeling and simulation is essential. Key elements comprise the definition and description of terms like deterministic simulation, Monte Carlo simulation, classical "top down" or novel "bottom up" approach, as well as the term "virtual world simulation." The illustrated examples in this chapter from pediatric clinical trials will help to understand and demonstrate these key elements. The importance of the understanding of developmental physiology and pharmacokinetics will become visible when explaining novel "bottom up" approaches like physiologically based pharmacokinetic simulations which also bridge to current research tools from other areas such as systems biology using mathematical models to describe biological systems.