The European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) has invited relevant stakeholders, and anyone involved in the preparation and conduct of paediatric clinical trials, to send comments on the draft framework released about paediatric clinical trial preparedness. This framework includes recommendations for sponsors, principal investigators and trialists on the key factors that can increase the likelihood of a smooth and timely course of a paediatric clinical trial.
Trial preparation should begin before and run in parallel to the designing of a drug development plan and sponsor readiness. Paediatric studies can be difficult to undertake for many reasons. There are often a limited number of eligible paediatric patients and patients may be reluctant to enrol into a trial due to the type and number of assessments, which may not be acceptable to their families. Failing to consider patient needs and perspectives can lead to delays and further expenses, sometimes leading to trial failure. Furthermore, paediatric data on natural history and epidemiology are very limited, with inadequate allocation of resources. There is often no resource allocated to support paediatric trial preparation.
The framework addresses these problems with principles of good preparation, such as collecting relevant information, involving relevant contributors, following a structured process and using appropriate resources. It also outlines approaches to prepare plans and studies, including a flow diagram about participant availability during preparation of a medicine’s development plan, and a table exhibiting a structured outline of a preparedness concept.
The methodology for preparing this document has been laid out to support the objectives and conclusions achieved, as well as a useful list of published literature which can be used to identify the main barriers to paediatric clinical trial preparedness.
To view the full framework for public consultation, click here.