The value of explicitly emulating a target trial when using real world evidence: an application to colorectal cancer screening
The value of explicitly emulating a target trial when using real world evidence: an application to colorectal cancer screening
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DOI：
10.1007/s10654-017-0287-2
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发表时间：
2017-06-01
影响因子：
13.6
通讯作者：
Hernan, Miguel A.
中科院分区：
文献类型：
--
作者：
Garcia-Albeniz, Xabier;Hsu, John;Hernan, Miguel A.
因果推断的观察分析通常依赖于为研究目的以外的目的收集的真实的世界数据。这些观察分析的一个常见目标是使用数据来模拟假设的随机实验，即，目标试验，模拟真实实验的设计特征，包括明确定义时间零点，同步治疗分配和确定合格性。我们回顾了最近的一项观察性分析，该分析明确模拟了使用美国医疗保险索赔进行结肠镜检查筛查的目标试验。然后，我们将这种明确的模拟与替代的、更简单的观察性分析进行比较，这些分析在零时不同步治疗分配和资格确定和/或不允许重复资格。这种实证比较表明，缺乏明确的模拟目标试验导致有偏估计，并表明，允许重复的资格增加了统计效率的估计。
Observational analyses for causal inference often rely on real world data collected for purposes other than research. A frequent goal of these observational analyses is to use the data to emulate a hypothetical randomized experiment, i.e., the target trial, that mimics the design features of a true experiment, including a clear definition of time zero with synchronization of treatment assignment and determination of eligibility. We review a recent observational analysis that explicitly emulated a target trial of screening colonoscopy using insurance claims from U.S. Medicare. We then compare this explicit emulation with alternative, simpler observational analyses that do not synchronize treatment assignment and eligibility determination at time zero and/or do not allow for repeated eligibility. This empirical comparison suggests that lack of an explicit emulation of the target trial leads to biased estimates, and shows that allowing for repeated eligibility increases the statistical efficiency of the estimates.