Module232025

09/05/2025

Decision tree for pediatric studies

Equally aplicable in EU and US (safety extrapolation)

Modelingand simulation(M&S) can help optimize paediatricdevelopment

The Organisation for Professionals in Regulatory Affairs

21

M&S in paediatric drug development Modeling and Simulation approaches examples:

• to assess similarity of disease and response to treatment, • examine and inform study design, • derive dosing recommendations, • test assumptions, and • predict the effects of the drug in the target population Important to provide clear reports! models are fit for simulation purposes • model assumptions • input data quality • simulation set up • Detail • Equations • Interrelations (e.g. parameter correlations) • Assumption testing • Distinguish between sources of variability and uncertainties

The Organisation for Professionals in Regulatory Affairs

22