Recommendations by the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) Working Group on preparedness of clinical trials about paediatric medicines process.



Siapkara, Angeliki, Fracasso, Claudio, Egger, Gunter F, Rizzari, Carmelo, Trasorras, Cristina Serén, Athanasiou, Dimitrios, Turner, Mark A ORCID: 0000-0002-5299-8656 and Working Group Membership,
(2021) Recommendations by the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) Working Group on preparedness of clinical trials about paediatric medicines process. Archives of Disease in Childhood, 106 (12). pp. 1149-1154.

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Abstract

Conduct of clinical trials in babies, children and young people is often hindered by issues that could have been foreseen before the trial opened; that is, some clinical trials are often underprepared. In order to identify a good approach to trial preparedness, the European Network of Paediatric Research at the European Medicines Agency formed a working group. The Working Group included representation from regulators, industry, academics, paediatric clinical research networks and parents.The Working Group consulted widely about how to prepare for paediatric clinical trials. The Group's detailed recommendations have been published (https://www.ema.europa.eu/en/documents/other/preparedness-medicines-clinical-trials-paediatrics-recommendations-enpr-ema-working-group-trial_en.pdf).This paper is a summary of the key recommendations including the following: start early, preferably in parallel to designing the medicine's development plan and individual protocols; identify the rationale and clinical need; listen to the perspectives of children and families, and of patient advocacy groups; identify how many people will be eligible for the trial; identify the resources needed, such as clinical facilities (including play therapy) and out-of-pocket expenditure by participants and their families; use all available data to estimate what is possible; present information about preparedness in a structured way; deploy proportionate resources to support the preparation of trials.A well-prepared, well-designed trial is likely to require fewer changes during its course, be run in a shorter time frame and achieve expected objectives.

Item Type: Article
Uncontrolled Keywords: Working Group Membership
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User: Symplectic Admin
Date Deposited: 11 May 2021 10:27
Last Modified: 18 Jan 2023 22:47
DOI: 10.1136/archdischild-2020-321433
Open Access URL: http://doi.org/10.1136/archdischild-2020-321433
Related URLs:
URI: https://livrepository.liverpool.ac.uk/id/eprint/3122326