Markoulidakis Andreas, Taiyari Khadijeh, Holmans Peter, Pallmann Philip, Busse Monica, Godley Mark D, Griffin Beth Ann
Centre for Trials Research, Cardiff University, Cardiff, Wales UK.
School of Medicine, Cardiff University, Cardiff, Wales UK.
Health Serv Outcomes Res Methodol. 2023;23(2):115-148. doi: 10.1007/s10742-022-00280-0. Epub 2022 May 27.
Randomized controlled trials are the gold standard for measuring causal effects. However, they are often not always feasible, and causal treatment effects must be estimated from observational data. Observational studies do not allow robust conclusions about causal relationships unless statistical techniques account for the imbalance of pretreatment confounders across groups and key assumptions hold. Propensity score and balance weighting (PSBW) are useful techniques that aim to reduce the observed imbalances between treatment groups by weighting the groups to look alike on the observed confounders. Notably, there are many methods available to estimate PSBW. However, it is unclear a priori which will achieve the best trade-off between covariate balance and effective sample size for a given application. Moreover, it is critical to assess the validity of key assumptions required for robust estimation of the needed treatment effects, including the overlap and no unmeasured confounding assumptions. We present a step-by-step guide to the use of PSBW for estimation of causal treatment effects that includes steps on how to evaluate overlap before the analysis, obtain estimates of PSBW using multiple methods and select the optimal one, check for covariate balance on multiple metrics, and assess sensitivity of findings (both the estimated treatment effect and statistical significance) to unobserved confounding. We illustrate the key steps using a case study examining the relative effectiveness of substance use treatment programs and provide a user-friendly Shiny application that can implement the proposed steps for any application with binary treatments.
随机对照试验是衡量因果效应的金标准。然而,它们往往并不总是可行的,必须从观察数据中估计因果治疗效应。除非统计技术能够解决各组间治疗前混杂因素的不平衡问题且关键假设成立,否则观察性研究无法得出关于因果关系的可靠结论。倾向得分和平衡加权(PSBW)是有用的技术,旨在通过对组进行加权,使其在观察到的混杂因素上看起来相似,从而减少治疗组之间观察到的不平衡。值得注意的是,有许多方法可用于估计PSBW。然而,对于给定的应用,先验地不清楚哪种方法能在协变量平衡和有效样本量之间实现最佳权衡。此外,评估稳健估计所需治疗效应所需的关键假设的有效性至关重要,包括重叠性和无未测量混杂因素假设。我们提供了一份使用PSBW估计因果治疗效应的分步指南,其中包括在分析前如何评估重叠性、使用多种方法获得PSBW估计值并选择最优方法、检查多个指标上的协变量平衡以及评估研究结果(估计的治疗效应和统计显著性)对未观察到的混杂因素的敏感性等步骤。我们通过一个案例研究来说明关键步骤,该案例研究考察了物质使用治疗项目的相对有效性,并提供了一个用户友好的Shiny应用程序,该应用程序可以为任何二元治疗应用实施所提出的步骤。