7 - Study design and methodology

Abstract

Making the study design right and ensuring an adequate study methodology are among the key factors to ensure a successful paediatric clinical study, probably to a greater extent than for adult studies. In this chapter, we will cover some basic principles of paediatric clinical study design and methodology based on the ICH E11 principles. The example of paediatric psychopharmacological studies in major depressive disorders will illustrate the inherent challenges and the necessity of finding innovative approaches. We will also emphasise the consequences of unfinished paediatric studies on an ethical and scientific standpoint. Study design and methodology are per nature evolving topics, and three chapters will cover several of their aspects. This first chapter is more general and will be followed by a specific chapter about recruitment as the study design probably affects paediatric recruitment to a greater extent compared with the adult experience. The last chapter will address the issue of the use of placebo focusing on indiscriminative, false negative efficacy studies in paediatric psychopharmacology.