Design, Identification, and Sensitivity Analysis for Patient Preference Trials
Design, Identification, and Sensitivity Analysis for Patient Preference Trials
复制标题
患者偏好试验的设计、识别和敏感性分析
DOI：
10.1080/01621459.2019.1585248
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发表时间：
2019
影响因子：
3.7
通讯作者：
Adam J. Berinsky
中科院分区：
文献类型：
--
作者：
D. Knox;Teppei Yamamoto;M. Baum;Adam J. Berinsky
社会和医学科学家经常担心实验结果的外部有效性可能因治疗效果的异质性而受到损害。如果一种治疗方法对愿意接受治疗的人和不愿意接受治疗的人有不同的效果，那么在标准随机对照试验（RCT）中估计的平均治疗效果可能会对其在研究样本之外的影响产生误导。患者偏好试验（PPTs）提供了一种可能的解决方案，其中参与者对治疗方案的偏好被纳入研究设计。在本文中，我们基于因果推理的潜在结果框架对ppt进行了系统的分析。我们提出了一种多值治疗的一般设计，参与者陈述他们喜欢的治疗方法，然后随机分为标准随机对照试验或自我选择条件。在提出的设计下，我们推导出每个基于选择的参与者亚群之间的平均因果效应的非参数锐界。我们还提出了对关键可忽略性假设的违反的敏感性分析，该假设足以确定目标因果量。提出的设计和方法是通过对党派新闻媒体及其行为影响的原始研究来说明的。本文的补充材料，包括可用于复制作品的材料的标准化描述，可作为在线补充。
Abstract Social and medical scientists are often concerned that the external validity of experimental results may be compromised because of heterogeneous treatment effects. If a treatment has different effects on those who would choose to take it and those who would not, the average treatment effect estimated in a standard randomized controlled trial (RCT) may give a misleading picture of its impact outside of the study sample. Patient preference trials (PPTs), where participants’ preferences over treatment options are incorporated in the study design, provide a possible solution. In this paper, we provide a systematic analysis of PPTs based on the potential outcomes framework of causal inference. We propose a general design for PPTs with multi-valued treatments, where participants state their preferred treatments and are then randomized into either a standard RCT or a self-selection condition. We derive nonparametric sharp bounds on the average causal effects among each choice-based subpopulation of participants under the proposed design. We also propose a sensitivity analysis for the violation of the key ignorability assumption sufficient for identifying the target causal quantity. The proposed design and methodology are illustrated with an original study of partisan news media and its behavioral impact. Supplementary materials for this article, including a standardized description of the materials available for reproducing the work, are available as an online supplement.