Sharp symbolic nonparametric bounds for measures of benefit in observational and imperfect randomized studies with ordinal outcomes

IF 2.4 2区 数学 Q2 BIOLOGY Biometrika Pub Date : 2024-04-11 DOI:10.1093/biomet/asae020
Erin E Gabriel, Michael C Sachs, Andreas Kryger Jensen
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Abstract

Summary The probability of benefit is a valuable and meaningful measure of treatment effect, which has advantages over the average treatment effect. Particularly for an ordinal outcome, it has a better interpretation and can make apparent different aspects of the treatment impact. Unfortunately, this measure, and variations of it, are not identifiable even in randomized trials with perfect compliance. There is, for this reason, a long literature on nonparametric bounds for unidentifiable measures of benefit. These have primarily focused on perfect randomized trial settings and one or two specific estimands. We expand these bounds to observational settings with unmeasured confounders and imperfect randomized trials for all three estimands considered in the literature: the probability of benefit, the probability of no harm, and the relative treatment effect.
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具有序数结果的观察性研究和不完全随机研究中收益测量的锐利符号非参数界限
摘要 受益概率是衡量治疗效果的一个有价值、有意义的指标,它比平均治疗效果更有优势。特别是对于序数结果,它有更好的解释,并能使治疗效果的不同方面显而易见。遗憾的是,即使是在完全符合要求的随机试验中,也无法识别这种测量方法及其变体。因此,关于无法识别的收益测量的非参数界限的文献有很长的篇幅。这些文献主要集中于完美随机试验环境和一两个特定的估计值。我们将这些界限扩展到具有未测量混杂因素和不完全随机试验的观察环境中,适用于文献中考虑的所有三种估计值:获益概率、无害概率和相对治疗效果。
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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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