How to optimise drug study design: pharmacokinetics and pharmacodynamics studies introduced to paediatricians

Objectives: In children, there is often lack of sufficient information concerning the pharmacokinetics (PK) and pharmacodynamics (PD) of a study drug to support dose selection and effective evaluation of efficacy in a randomised clinical trial (RCT). Therefore, one should consider the relevance of relatively small PKPD studies, which can provide the appropriate data to optimise the design of an RCT. Methods: Based on the experience of experts collaborating in the EU-funded Global Research in Paediatrics con