导航现实世界:对结构化框架的全面审查,以有效地识别,评估和选择适合目的研究的现实世界数据源
Sonia Zebachi1, Julien Tanniou1, Elisabeth Bakker2
1Quinten Health, Paris, France.
Clinical pharmacology and therapeutics
|July 2, 2025
概括
选择适当的真实世界数据 (RWD) 来源对于监管提交至关重要. 本综述分析了九个框架,如REQueST和SPIFD2,以指导利益相关者选择适合目的的RWD用于证据生成.
科学领域:
- 医疗信息学 医疗信息学
- 监管科学 监管科学
- 数据科学数据科学数据科学
背景情况:
- 现实世界数据 (RWD),特别是来自患者登记册的数据,对于评估产品的有效性和支持监管提交越来越重要.
- 像EMA和FDA这样的监管机构已经发布了关于利用RWD进行证据生成的指导.
- 现在有许多结构化框架存在,以帮助利益相关者评估RWD来源.
研究的目的:
- 为评估和选择"适合目的"真实世界数据源提供结构化框架的概述.
- 识别和描述关键工具,包括REQueST和SPIFD2.
- 帮助利益相关者选择适当的RWD用于监管和健康技术评估 (HTA) 目的.
主要方法:
- 对RWD源选择的结构化框架进行了范围审查.
- 确定了九个关键的评估工具.
- 描述每个框架,并对其评估标准进行比较分析.
主要成果:
- 确定并分析了九个用于RWD源评估的结构化框架.
- 框架标准包括研究设计,数据可靠性,相关性,伦理,成本和可行性.
- 每个框架都有其独特的优势和局限性,这取决于其应用环境.
结论:
- 结构化框架提供了可靠的方法来确保RWD源的适用性.
- 了解这些框架有助于利益相关者选择最佳的RWD来源以获得高质量的证据.
- 这一审查有助于在监管和HTA流程中利用RWD进行知情决策.
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