It is unacceptable that many drugs prescribed to children have not been proven safe and effective for them. Yet some of the necessary drug studies do not provide the participating children direct benefit. From an ethical point of view, involving children in such studies is complex: in view of the absence of direct advantages, the possible disadvantages must be strictly limited. A European ethical framework that provides guidance regarding these limits and that can and will be consistently applied by all European countries is essential for facilitating a harmonised approach to paediatric clinical trials across Europe.
Currently, limits for paediatric research without direct benefit are defined in two European documents. According to the Council of Europe’s European Convention on Human Rights and Biomedicine such research may only be approved if it entails ‘minimal risk and minimal burden’. In contrast, in a more recent document aimed to provide guidance on the application of the Clinical Trials Directive with regard to trials with minors, the EU recommends to allow ‘a minor increase over minimal risk’ in case of benefit for the group of children with the same disease.
On the basis of an example, this paper shows that the inconsistency between these two documents may either prohibit important research or expose participants to unjustified risks of harm. We recommend that both documents use the same terminology and that the EU Recommendations emphasise that exposing children to higher levels of risk and burden that cannot be compensated by direct benefits, must always be regarded as a last resort.