利用现实世界的数据为儿科临床试验的潜力:一项概念验证研究
Jens Declerck1, Joanne Lee2, Anando Sen2
1The European Institute for Innovation Through Health Data, Ghent, Belgium.
Journal of medical Internet research
|May 30, 2025
概括
欧洲医院缺乏结构化的儿科临床数据,这阻碍了研究中使用现实世界数据 (RWD). 这项研究评估了数据的可用性,发现了疾病特定信息中的差距,这限制了其在临床试验中的应用.
科学领域:
- 儿科临床研究 儿科临床研究
- 现实世界数据 (RWD) 的利用.
- 电子健康记录 (EHR) 数据标准化数据标准化
背景情况:
- 儿科临床研究,特别是罕见疾病的临床研究,在患者鉴定,招募和试验执行方面面临重大障碍,原因是小,分层的群体和碎片化的数据.
- 来自电子健康记录 (EHR) 的真实世界数据 (RWD) 为创新的试验设计提供了潜力,包括真实世界的比较器和营销后监测.
- 有效使用RWD取决于结构化和可重复使用的临床数据的常规可用性.
研究的目的:
- 评估欧洲儿科医院常规临床数据的可用性和结构.
- 评估与构建比较器臂和进行营销后监测研究相关的数据元素.
- 检查神经纤维素炎 (NF) 和亚托皮性皮肤炎 (AD) 的数据收集实践,作为代表性的儿科疾病.
主要方法:
- 根据对NF,AD和安全性研究协议的专家分析,开发了113个关键临床数据项的库存.
- 通过connect4children (c4c) 国家枢纽网络在欧洲儿科网站上进行了结构化的基于网络的调查.
- 评估的数据捕获方法:结构化/编码的EHR字段,自由文本,外部系统或纸质记录.
主要成果:
- 来自11个欧洲国家的24家医院的调查回答表明,数据捕获和存储的变化很大.
- 一般的临床和药物安全数据 (例如,人口统计,生命体征,药物) 经常是结构化的.
- 疾病特异性和上下文变量,生活方式和家族史数据经常被记录为自由文本或不一致,限制了EHR数据用于研究的重用性.
结论:
- 在欧洲医疗保健机构对儿科临床数据的结构化文档中存在重大差距.
- 缺乏标准化,编码的数据格式,阻碍了对EHR数据进行观察研究和比较研究的重复使用.
- 这项概念验证研究证实了评估EHR数据可用性的可行性,并为扩大RWD在儿科研究中的整合提供了基础.
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