Handling incomplete outcomes and covariates in cluster-randomized trials: doubly-robust estimation, efficiency considerations, and sensitivity analysis
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In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the various types of missing data in CRTs, missing outcomes have attracted the most attention. However, no existing methods simultaneously address all aforementioned types of missing data in CRTs. To fill in this gap, we propose a doubly-robust estimator for the average treatment effect on a variety of effect measure scales. The proposed estimator simultaneously handles missing outcomes under missingness at random, missing covariates without constraining the missingness mechanism, and missing cluster-population sizes via a uniform sampling mechanism. Furthermore, we detail key considerations to improve precision by specifying the optimal weights, leveraging machine learning, and modeling the treatment assignment mechanism. Finally, to evaluate the impact of violating missing data assumptions, we contribute a new sensitivity analysis framework tailored to CRTs. We assess the performance of the proposed methods through simulations and illustrate their use in a real data application.
The conditional average treatment effect (CATE) is frequently estimated in clinical studies to refute a homogeneous treatment effect hypothesis. Under this regime, all patients making up the population experience identical benefit from a given treatment relative to a comparator. Uncovering heterogeneous treatment effects through inference about the CATE, however, requires that covariates truly modifying the treatment effect be reliably collected at baseline. CATE-based…
嵌套工具变量分析:转换者平均处理效应及可推广性
Instrumental variables (IVs) are widely used to estimate causal effects from non-randomized data. A canonical example is a randomized trial with noncompliance, in which the randomized treatment assignment serves as an IV for the non-ignorable treatment received. Under a monotonicity assumption, a valid IV nonparametrically identifies the average treatment effect among a latent complier subgroup, whose generalizability is often under debate. In many studies, there exist…
Traditional machine learning relies on explicit models and domain assumptions, often limiting flexibility and interpretability. We introduce a "model-free" framework of instance-based learning using surprisal (information theoretic uncertainty) to directly analyze and perform inferences from raw data, eliminating distribution modeling, reducing bias, and enabling efficient updates including direct edits and deletion of training data. By quantifying relevance through…
自删失模型下信息性缺失的纵向因果推断
Longitudinal observational data are often subject to informative missingness. For example, in healthcare research using electronic health records, covariates and outcomes are measured only when patients interact with the healthcare system. Whether these data are observed usually depends on the unobserved variable values themselves, introducing a common form of data missing not at random. We develop identification and estimation methods for the effects of longitudinal…
通过因果数据融合衡量礼品卡项目的增量收入
Businesses regularly offer gift card programs to drive customer spending and increase engagement. A central question is how much incremental revenue these programs generate, and which channels drive it most efficiently. Measuring the incremental revenue associated with a gift card program is a challenging problem in causal inference, requiring a firm to infer how much each customer would have spent if they never received a gift card. Observational data on past customer…
计数数据的去偏机器学习:基于神经网络的部分线性 Poisson 模型
We develop a novel debiased machine learning (DML) estimator to analyze count data, which are common in clinical or biomedical studies. Specifically, we estimate the effect of a binary treatment on count outcomes using a partially linear Poisson model. A key advantage of this model is that it represents the complex covariate structure through a nuisance function, which is estimated flexibly using neural networks. We use a Neyman-orthogonal score function to construct the…