评估使用观察方法和现实世界的数据来模拟正在进行的随机对照试验
Joshua D Wallach1, Yihong Deng2, Eric C Polley3
1Department of Epidemiology, Rollins School of Public Health, Emory University, Atlanta, GA, USA.
Clinical trials (London, England)
|August 17, 2023
概括
使用真实世界数据的观察性研究可以模拟随机对照试验,提供对药物有效性的及时见解. 虽然数据确定存在挑战,但关键结果显示一致,支持其在医疗产品评估中的使用.
科学领域:
- 医疗保健服务研究 医疗服务研究
- 临床试验方法论 临床试验方法论
- 现实世界的证据.
背景情况:
- 越来越多的人对医学产品评估的观察研究方法感兴趣.
- 两项随机对照试验 (RCT) 的先前模拟,使用链接索赔和电子健康记录.
- 需要将模拟试验群体和结果与已发表的RCT进行比较.
研究的目的:
- 将人口和模拟试验的结果与已发表的PRONOUNCE和GRADERCT进行比较.
- 评估使用现实世界数据用于基线参与者特征的可行性.
- 评估基线特征和初级/二级终点的一致性.
主要方法:
- 使用OptumLabs数据仓库结构化数据模拟了两个RCT (PRONOUNCE,GRADE).
- 基线特征的评估可确定性 (可确定性,部分可确定性,不可确定性).
- 基线特征 (SMD<0.20) 和终点结果 (方向,意义) 的模拟与发表的RCT进行了比较.
主要成果:
- 发音:9/31 (29%) 可以确定基线特征;6/9 (66.7%) 不一致. 主要/次要终点一致.
- 级别:可以确定8/34 (23.5%) 的基线特征;4/8 (50%) 不一致. 主要终点大多是一致的.
- 与已发表的RCT相比,模拟试验在参与者数量上有所不同.
结论:
- 现实世界的数据和观察方法显示了及时药物评估的潜力.
- 数据确定存在挑战,但并不排除实用性.
- 这些方法可以评估多样化和代表性的患者群体.
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