Europe – EMA : Preparedness of medicines’ clinical trials in paediatrics

This document was developed by a Working Group of the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) 2 and sets out recommendations for discussions about trial preparedness in paediatrics.
We define trial preparedness as a structured assessment of the key factors that could increase the likelihood of a smooth and timely course of a paediatric clinical trial, integrating information from multiple stakeholders on what is possible within a development plan3 and/or individual studies. This includes several activities that are labelled as study “feasibility”. However, here a global assessment of all aspects of trial preparation is addressed which goes beyond “feasibility” aspects and therefore this
term is not used.
The design of drug development plans and individual studies in paediatrics overlaps with preparedness to some extent. By design, we mean the selection of methods to answer a research question (or set of questions such as biostatistics, Model Informed Drug Discovery and Development (MID3), extrapolation). Design needs to take account of the specificities of infants, children and young people while maximising the use of extant data (including preclinical data such as toxicity) and minimising the burden of research in these populations. There are many sources describing details specific to the design of paediatric development programmes and studies. Section 5 on methodology provides an overview of the activity undertaken by the Enpr-EMA working group on trial preparedness in collecting
stakeholders’ points to consider during preparation of a study as basis for this document.
Trial preparation should be initiated before and conducted in parallel to the designing of the
development plan and the individual studies, and in parallel to sponsor readiness.
The recommendations in this document target both sponsors as well as principal investigators and trialists. However, it should be noted that this document does not describe all aspects of ‘sponsor readiness’, such as operational aspects within sponsors and intermediary organizations, e.g. Contract Research Organizations (CRO), or strategic factors, such as patient need and economic opportunities.
Furthermore activities to support marketing of products using data about market size are out of scope of this document.
Standards for site readiness and practical arrangements for sites and participants in public and patient involvement relating to the preparation of programmes and studies are under development by other initiatives…