Approximately 90% of neonates in intensive care receive at least one unlicensed or off-label medicinal product, clearly emphasising the fact that more clinical trials are needed in neonates to study the effects of medicines as well as to improve evidence-based pharmacotherapy. These trials include, for example, pharmacokinetic and dose-finding studies as well as randomised controlled trials to evaluate efficacy and safety. However, this age group is very vulnerable and poses additional ethical and scientific issues. Such ethical considerations and aspects of clinical trial methodology in neonates will be highlighted and discussed.
In order to facilitate development and availability of medicines for all paediatric age groups the European Paediatric Regulation entered into force in 2007. First experiences with studies submitted for neonates since then will be discussed.