监管决策的现实证据:来自世界各地的最新指南
Leah Burns1, Nadege Le Roux2, Robert Kalesnik-Orszulak3
1Worldwide Health Economics and Outcomes Research, Bristol Myers Squibb, Princeton, NJ, United States.
Frontiers in medicine
|November 29, 2023
概括
现实世界的证据 (RWE) 对医疗保健监管决策越来越重要. 本次审查更新了RWE的全球框架,包括新的程序指导和卫生技术评估的调整.
科学领域:
- 监管科学 监管科学
- 卫生经济学 卫生经济学
- 数据科学数据科学数据科学
背景情况:
- 现实世界的证据 (RWE) 对于明智的医疗监管决策和及时的治疗批准至关重要.
- 之前的工作审查了基于框架,数据质量和研究方法的RWE的全球监管格局,截至2021年5月.
- 在医疗监管中利用RWE的势头继续增长,强调患者的结果和经验.
研究的目的:
- 为监管现实世界的证据 (RWE) 环境中最近的发展提供一个更新和详细的审查.
- 评估区域在三个关键要素上的进展:监管RWE框架,数据质量指导和研究方法指导.
- 介绍和分析一个新的类别:程序指导,并讨论差距和协调的机会.
主要方法:
- 对有关现实世界的证据 (RWE) 的区域监管发展进行系统的审查和分析.
- 评估更新至2021年5月,重点关注监管框架,数据质量,研究方法和程序指导.
- 对全球不同地区的RWE政策进行比较分析,包括卫生技术评估 (HTA) 机构的发展.
主要成果:
- 自2021年5月以来,全球范围内在监管RWE框架,数据质量和研究方法指导方面取得了重大进展.
- 程序指导的引入是RWE监管领域的一个关键发展.
- 确定的差距和协调的机会存在,特别是在缺乏先前存在的RWE政策的地区.
结论:
- 现实世界证据 (RWE) 的全球监管环境正在迅速发展,越来越关注协调和程序方面.
- 监管机构与医疗技术评估 (HTA) 机构之间关于RWE的协调是未来发展的关键领域.
- 持续发展和协调RWE政策对于支持全球高效和有效的监管决策至关重要.
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