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AI 根据所提供的论文材料归类:摘要直接讨论随机分配与治疗偏好选择并存时的因果效应估计和试验设计。

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关注这篇论文的作者
Qi Long
Roderick J Little
Xihong Lin

包含治疗选择的混合干预试验中的因果推断

研究问题
当部分参与者随机接受治疗、另一些人按偏好选择治疗时,如何估计偏好定义子群的因果效应,并区分相关偏好效应?
方法与思路
作者为混合试验建立因果效应模型,采用EM算法计算最大似然估计,并分析女性心脏病干预试验的健康影响评分与依从性。
材料陈述的贡献
摘要称偏好治疗与评分改善存在一定证据,与依从性存在较强偏好效应;同时提出比较混合试验设计及其假设要求的框架。
阅读关注点
应核对随机分配与自主选择的设计层次、偏好子群的识别假设和效应定义,并查阅摘要提示的更正对模型及结论的影响。
阅读边界
仅据版本未注明的索引摘要,不能推断刊本变化;摘要提示另有更正,但材料未提供其内容,不能判断更正后的公式、估计或结论。

原文摘要

Randomized allocation of treatments is a cornerstone of experimental design but has drawbacks when a limited set of individuals are willing to be randomized, or the act of randomization undermines the success of the treatment. Choice-based experimental designs allow a subset of the participants to choose their treatments. We discuss here causal inferences for hybrid experimental designs in which some participants are randomly allocated to treatments and others receive their treatment preference. This work was motivated by the Women Take Pride (WTP) study, a doubly randomized preference trial (DRPT) that assessed behavioral interventions for women with heart disease. We propose a model for estimating the causal effects in the subpopulations defined by treatment preferences, and thus preference effects. We describe an EM algorithm for computing maximum likelihood estimates of the model parameters. We illustrate the method by analyzing sickness impact profile (SIP) scores and treatment adherence in the WTP data. Our results show some evidence that SIP scores were improved when women received their preferred treatment and strong preference effects on program adherence; that is, women assigned to their preferred treatment were more likely to adhere to the program. We also provide a framework for assessing the DRPT and other hybrid trial designs and discuss some alternative designs from the perspective of the strength of assumptions required to make causal inferences. Please see the supplemental material for a correction to this article.

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