分层随机实验和配对观察研究中的治疗效果量化

IF 2.4 2区 数学 Q2 BIOLOGY
Biometrika Pub Date : 2022-08-24 DOI:10.1093/biomet/asad030
Yongchang Su, Xinran Li
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引用次数: 2

摘要

评价处理效果已成为许多应用领域的重要课题。然而,大多数现有文献主要集中在平均治疗效果上。当个体效应是重尾效应或有离群值时,不仅平均效应可能不适合用于总结治疗效果,而且由于大样本近似性差,常规推断可能是敏感的,并且可能无效。在本文中,我们关注个体治疗效果的分位数,在存在极端个体效应的情况下,它可能更稳健。此外,我们对它们的推断是纯粹基于随机化的,避免了对单位的任何分布假设。我们首先考虑分层随机实验中的推理,将最近的工作扩展到?Caughey et al.(2021)。我们证明了检验个体效应分位数上的零假设的有效p值的计算可以转化为多项选择背包问题的实例,该问题可以有效地精确或稍微保守地解决。然后,我们将我们的方法扩展到匹配的观察性研究,并提出敏感性分析,以调查我们对个体效应分位数的推断在多大程度上对未测量的混淆是稳健的。所提出的随机化推理和敏感性分析对个体效应的所有分位数同时有效,注意到对最大或最小个体效应的分析与假设恒定治疗效应的传统分析一致。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Treatment Effect Quantiles in Stratified Randomized Experiments and Matched Observational Studies
Evaluating the treatment effect has become an important topic for many applications. However, most existing literature focuses mainly on average treatment effects. When the individual effects are heavy-tailed or have outlier values, not only may the average effect not be appropriate for summarizing treatment effects, but also the conventional inference for it can be sensitive and possibly invalid due to poor large-sample approximations. In this paper we focus on quantiles of individual treatment effects, which can be more robust in the presence of extreme individual effects. Moreover, our inference for them is purely randomization-based, avoiding any distributional assumptions on the units. We first consider inference in stratified randomized experiments, extending the recent work by? Caughey et al. (2021). We show that the computation of valid p-values for testing null hypotheses on quantiles of individual effects can be transformed into instances of the multiple-choice knapsack problem, which can be efficiently solved exactly or slightly conservatively. We then extend our approach to matched observational studies and propose a sensitivity analysis to investigate to what extent our inference on quantiles of individual effects is robust to unmeasured confounding. The proposed randomization inference and sensitivity analysis are simultaneously valid for all quantiles of individual effects, noting that the analysis for the maximum or minimum individual effect coincides with the conventional analysis assuming constant treatment effects.
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来源期刊
Biometrika
Biometrika 生物-生物学
CiteScore
5.50
自引率
3.70%
发文量
56
审稿时长
6-12 weeks
期刊介绍: Biometrika is primarily a journal of statistics in which emphasis is placed on papers containing original theoretical contributions of direct or potential value in applications. From time to time, papers in bordering fields are also published.
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