Richardson Amy, Hudgens Michael G, Gilbert Peter B, Fine Jason P
Quantitative Analyst, Google Inc., Mountain View, California 94043, USA.
Associate Professor, Department of Biostatistics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, USA.
Stat Sci. 2014 Nov;29(4):596-618. doi: 10.1214/14-STS499.
This paper considers conducting inference about the effect of a treatment (or exposure) on an outcome of interest. In the ideal setting where treatment is assigned randomly, under certain assumptions the treatment effect is identifiable from the observable data and inference is straightforward. However, in other settings such as observational studies or randomized trials with noncompliance, the treatment effect is no longer identifiable without relying on untestable assumptions. Nonetheless, the observable data often do provide some information about the effect of treatment, that is, the parameter of interest is partially identifiable. Two approaches are often employed in this setting: (i) bounds are derived for the treatment effect under minimal assumptions, or (ii) additional untestable assumptions are invoked that render the treatment effect identifiable and then sensitivity analysis is conducted to assess how inference about the treatment effect changes as the untestable assumptions are varied. Approaches (i) and (ii) are considered in various settings, including assessing principal strata effects, direct and indirect effects and effects of time-varying exposures. Methods for drawing formal inference about partially identified parameters are also discussed.
本文考虑对一种治疗(或暴露)对感兴趣的结局的影响进行推断。在理想的随机分配治疗的情况下,在某些假设下,治疗效果可从可观测数据中识别出来,推断也很直接。然而,在其他情况下,如观察性研究或存在不依从性的随机试验中,不依赖不可检验的假设,治疗效果就不再可识别。尽管如此,可观测数据通常确实提供了一些关于治疗效果的信息,即感兴趣的参数是部分可识别的。在这种情况下通常采用两种方法:(i)在最小假设下推导治疗效果的界值,或(ii)引入额外的不可检验假设以使治疗效果可识别,然后进行敏感性分析,以评估随着不可检验假设的变化,关于治疗效果的推断如何改变。在各种情况下都考虑了方法(i)和(ii),包括评估主要分层效应、直接和间接效应以及随时间变化的暴露的效应。还讨论了对部分识别参数进行形式化推断的方法。