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Randomization inference for stepped-wedge designs with noncompliance with application to a palliative care pragmatic trial

存在不依从时阶梯楔形设计的随机化推断:姑息治疗试验应用

While palliative care is increasingly commonly delivered to hospitalized patients with serious illnesses, few studies have estimated its causal effects. Courtright et al. (2016) adopted a stepped-wedge cluster-randomized design to assess the effect of palliative care on a patient-centered outcome. The randomized intervention was a nudge to administer palliative care but did not guarantee receipt of palliative care, resulting in noncompliance. A subsequent analysis using…

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Powerful Multivariate Sensitivity Analysis via Sample Splitting in an Observational Study of the Effects of Poverty on Cardiovascular Disease Risk Factors

通过样本分割增强多变量敏感性分析:贫困与心血管疾病风险因素

When assessing the causal effect of an exposure on two or more outcomes in an observational study, a linear combination of outcomes may lessen the sensitivity of a test of the global null hypothesis to potential unmeasured biases. While all linear combinations of scored outcomes can be considered using Scheffe projections or constrained variants thereof, finding the combination that minimizes sensitivity to unmeasured biases requires corrections for multiple testing, which…

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A subgroup-aware scoring approach to the study of effect modification in observational studies

观察研究中效应修饰的子群感知评分方法

Effect modification means the size of a treatment effect varies with an observed covariate. Generally speaking, a larger treatment effect with more stable error terms is less sensitive to bias. Thus, we might be able to conclude that a study is less sensitive to unmeasured bias by using these subgroups experiencing larger treatment effects. Lee et al. (2018) proposed the submax method that leverages the joint distribution of test statistics from subgroups to draw a firmer…

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Optimal Sample Splitting for Observational Studies

观测研究中的最优样本拆分

In observational studies of treatment effects, estimates may be biased by unmeasured confounders, which can potentially affect the validity of the results. Understanding sensitivity to such biases helps assess how unmeasured confounding impacts credibility. The design of an observational study strongly influences its sensitivity to bias. Previous work has shown that the sensitivity to bias can be reduced by dividing a dataset into a planning sample and a larger analysis…

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A Differential Effect Approach to Partial Identification of Treatment Effects

用差异效应部分识别处理效应

We consider identification and inference for the average treatment effect and heterogeneous treatment effect conditional on observable covariates in the presence of unmeasured confounding. Since point identification of these treatment effects is not achievable without strong assumptions, we obtain bounds on these treatment effects by leveraging differential effects, a tool that allows for using a second treatment to learn the effect of the first treatment. The differential…

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Sensitivity Analysis for Attributable Effects in Case$^2$ Studies

病例—病例研究中可归因效应的敏感性分析

The case$^2$ study, also referred to as the case-case study design, is a valuable approach for conducting inference for treatment effects. Unlike traditional case-control studies, the case$^2$ design compares treatment in two types of cases with the same disease. A key quantity of interest is the attributable effect, which is the number of cases of disease among treated units which are caused by the treatment. Two key assumptions that are usually made for making inferences…

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On the mixed-model analysis of covariance in cluster-randomized trials

整群随机试验中的混合模型协方差分析

In the analyses of cluster-randomized trials, mixed-model analysis of covariance (ANCOVA) is a standard approach for covariate adjustment and handling within-cluster correlations. However, when the normality, linearity, or the random-intercept assumption is violated, the validity and efficiency of the mixed-model ANCOVA estimators for estimating the average treatment effect remain unclear. Under the potential outcomes framework, we prove that the mixed-model ANCOVA…

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Model-robust and efficient covariate adjustment for cluster-randomized experiments

整群随机实验中模型稳健且高效的协变量调整

Cluster-randomized experiments are increasingly used to evaluate interventions in routine practice conditions, and researchers often adopt model-based methods with covariate adjustment in the statistical analyses. However, the validity of model-based covariate adjustment is unclear when the working models are misspecified, leading to ambiguity of estimands and risk of bias. In this article, we first adapt two conventional model-based methods, generalized estimating equations…

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RobustIV and controlfunctionIV: Causal Inference for Linear and Nonlinear Models with Invalid Instrumental Variables

RobustIV 与 controlfunctionIV:可能无效工具变量下的因果推断软件

We present R software packages RobustIV and controlfunctionIV for causal inference with possibly invalid instrumental variables. RobustIV focuses on the linear outcome model. It implements the two-stage hard thresholding method to select valid instrumental variables from a set of candidate instrumental variables and make inferences for the causal effect in both low- and high-dimensional settings. Furthermore, RobustIV implements the high-dimensional endogeneity test and the…

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Randomization-based Test for Censored Outcomes: A New Look at the Logrank Test

删失结局的随机化检验:重新理解 Logrank 检验

Two-sample tests with censored outcomes are a classical topic in statistics with wide use even in cutting edge applications. There are at least two modes of inference used to justify two-sample tests. One is usual superpopulation inference assuming that units are independent and identically distributed (i.i.d.) samples from some superpopulation; the other is finite population inference that relies on the random assignments of units into different groups. When randomization…

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Structural mean models for instrumented difference-in-differences

工具变量双重差分的结构均值模型

In the standard difference-in-differences research design, the parallel trends assumption may be violated when the relationship between the exposure trend and the outcome trend is confounded by unmeasured confounders. Progress can be made if there is an exogenous variable that (i) does not directly influence the change in outcome means (i.e. the outcome trend) except through influencing the change in exposure means (i.e. the exposure trend), and (ii) is not related to the…

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