VRNN, Medicines and Healthcare products Regulatory Agency (MHRA), London, UK.
Pediatric Centre of Excellence, Pfizer S.r.l, Rome, Italy.
Arch Dis Child. 2021 Dec;106(12):1149-1154. doi: 10.1136/archdischild-2020-321433. Epub 2021 Apr 15.
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.
在婴儿、儿童和青少年中进行临床试验常常受到在试验开始之前本可以预见的问题的阻碍;也就是说,一些临床试验往往准备不足。为了确定一个良好的试验准备方法,欧洲药品管理局的儿科研究网络成立了一个工作组。该工作组包括来自监管机构、工业界、学术界、儿科临床研究网络和家长的代表。工作组就如何为儿科临床试验做准备进行了广泛的磋商。该小组的详细建议已经公布(https://www.ema.europa.eu/en/documents/other/preparedness-medicines-clinical-trials-paediatrics-recommendations-enpr-ema-working-group-trial_en.pdf)。本文是对关键建议的总结,包括以下内容:尽早开始,最好与药物开发计划和个别方案的设计同时进行;确定基本原理和临床需求;听取儿童和家庭以及患者权益组织的意见;确定有多少人有资格参加试验;确定所需的资源,如临床设施(包括游戏疗法)和参与者及其家属的自付费用;利用所有可用数据来估计可能性;以结构化的方式介绍准备情况;投入适当的资源支持试验准备工作。精心准备和设计的试验很可能在其过程中需要较少的更改,在更短的时间内进行,并实现预期的目标。