将不同的协变量平衡方法与评估青少年药物使用治疗方案因果影响的应用程序进行比较的教程。

IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES
Andreas Markoulidakis, Khadijeh Taiyari, Peter Holmans, Philip Pallmann, Monica Busse, Mark D Godley, Beth Ann Griffin
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引用次数: 0

摘要

随机对照试验是衡量因果效应的黄金标准。然而,它们往往并不总是可行的,必须根据观察数据来估计因果治疗效果。观察性研究不允许对因果关系得出有力的结论,除非统计技术解释了各组预处理混杂因素的不平衡,并且关键假设成立。倾向评分和平衡加权(PSBW)是有用的技术,旨在通过对各组进行加权,使其在观察到的混杂因素上看起来相似,来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,对于给定的应用程序,哪种方法可以在协变量平衡和有效样本量之间实现最佳权衡,这一点尚不清楚。此外,评估对所需治疗效果进行稳健估计所需的关键假设的有效性至关重要,包括重叠和无未测量的混杂假设。我们提供了一个使用PSBW估计因果治疗效果的分步指南,其中包括如何在分析前评估重叠的步骤,使用多种方法获得PSBW的估计值并选择最佳方法,检查多个指标上的协变量平衡,并评估研究结果(估计的治疗效果和统计学意义)对未观察到的混杂因素的敏感性。我们通过一个案例研究来说明关键步骤,该案例研究检查了物质使用治疗计划的相对有效性,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的应用程序实施所提出的步骤。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A tutorial comparing different covariate balancing methods with an application evaluating the causal effects of substance use treatment programs for adolescents.

A tutorial comparing different covariate balancing methods with an application evaluating the causal effects of substance use treatment programs for adolescents.

A tutorial comparing different covariate balancing methods with an application evaluating the causal effects of substance use treatment programs for adolescents.

A tutorial comparing different covariate balancing methods with an application evaluating the causal effects of substance use treatment programs for adolescents.

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.

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来源期刊
Health Services and Outcomes Research Methodology
Health Services and Outcomes Research Methodology HEALTH CARE SCIENCES & SERVICES-
CiteScore
3.40
自引率
6.70%
发文量
28
期刊介绍: The journal reflects the multidisciplinary nature of the field of health services and outcomes research. It addresses the needs of multiple, interlocking communities, including methodologists in statistics, econometrics, social and behavioral sciences; designers and analysts of health policy and health services research projects; and health care providers and policy makers who need to properly understand and evaluate the results of published research. The journal strives to enhance the level of methodologic rigor in health services and outcomes research and contributes to the development of methodologic standards in the field. In pursuing its main objective, the journal also provides a meeting ground for researchers from a number of traditional disciplines and fosters the development of new quantitative, qualitative, and mixed methods by statisticians, econometricians, health services researchers, and methodologists in other fields. Health Services and Outcomes Research Methodology publishes: Research papers on quantitative, qualitative, and mixed methods; Case Studies describing applications of quantitative and qualitative methodology in health services and outcomes research; Review Articles synthesizing and popularizing methodologic developments; Tutorials; Articles on computational issues and software reviews; Book reviews; and Notices. Special issues will be devoted to papers presented at important workshops and conferences.
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