Qiu Hongxiang, Carone Marco, Sadikova Ekaterina, Petukhova Maria, Kessler Ronald C, Luedtke Alex
Dept. of Biostatistics, University of Washington.
Dept. of Health Care Policy, Harvard Medical School.
J Am Stat Assoc. 2021;116(533):174-191. doi: 10.1080/01621459.2020.1745814. Epub 2020 May 12.
There is an extensive literature on the estimation and evaluation of optimal individualized treatment rules in settings where all confounders of the effect of treatment on outcome are observed. We study the development of individualized decision rules in settings where some of these confounders may not have been measured but a valid binary instrument is available for a binary treatment. We first consider individualized treatment rules, which will naturally be most interesting in settings where it is feasible to intervene directly on treatment. We then consider a setting where intervening on treatment is infeasible, but intervening to encourage treatment is feasible. In both of these settings, we also handle the case that the treatment is a limited resource so that optimal interventions focus the available resources on those individuals who will benefit most from treatment. Given a reference rule, we evaluate an optimal individualized rule by its average causal effect relative to a prespecified reference rule. We develop methods to estimate optimal individualized rules and construct asymptotically efficient plug-in estimators of the corresponding average causal effect relative to a prespecified reference rule.
在能够观察到治疗对结局影响的所有混杂因素的情况下,关于估计和评估最优个体化治疗规则的文献极为丰富。我们研究在某些混杂因素可能未被测量,但对于二元治疗有一个有效的二元工具变量可用的情况下个体化决策规则的制定。我们首先考虑个体化治疗规则,在可行直接干预治疗的情况下,这自然会是最受关注的。然后我们考虑一种干预治疗不可行,但鼓励治疗可行的情况。在这两种情况下,我们还处理治疗是一种有限资源的情形,以便最优干预将可用资源集中于那些将从治疗中获益最大的个体。给定一个参考规则,我们通过相对于预先指定的参考规则的平均因果效应来评估最优个体化规则。我们开发方法来估计最优个体化规则,并构建相对于预先指定的参考规则的相应平均因果效应的渐近有效插件估计量。