Assessing treatment effect heterogeneity in the presence of missing effect modifier data in cluster-randomized trials
整群随机试验中效应修饰变量缺失时的治疗效应异质性评估AI 解读 ↓
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Understanding whether and how treatment effects vary across subgroups is crucial to inform clinical practice and recommendations. Accordingly, the assessment of heterogeneous treatment effects (HTE) based on pre-specified potential effect modifiers has become a common goal in modern randomized trials. However, when one or more potential effect modifiers are missing, complete-case analysis may lead to bias and under-coverage. While statistical methods for handling missing data have been proposed and compared for individually randomized trials with missing effect modifier data, few guidelines exist for the cluster-randomized setting, where intracluster correlations in the effect modifiers, outcomes, or even missingness mechanisms may introduce further threats to accurate assessment of HTE. In this article, the performance of several missing data methods are compared through a simulation study of cluster-randomized trials with continuous outcome and missing binary effect modifier data, and further illustrated using real data from the Work, Family, and Health Study. Our results suggest that multilevel multiple imputation (MMI) and Bayesian MMI have better performance than other available methods, and that Bayesian MMI has lower bias and closer to nominal coverage than standard MMI when there are model specification or compatibility issues.
自适应实验中策略改进价值的推断
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Randomized experiments with ordinal outcomes are common across scientific applications, but conventional causal estimands such as the average treatment effect are difficult to interpret because ordinal categories lack meaningful numerical spacing. Two interpretable estimands, the probabilities that the treatment is beneficial and strictly beneficial, depend on the joint distribution of the potential outcomes and are therefore not identifiable from observed data. The sharp…
图分割贝叶斯因果森林:空间异质性处理效应估计
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利用 SIMEX 估计真实效应量分布
Large-scale online experimentation produces noisy effect estimates, which can overstate gains and complicate decisions about launches and testing policies. We propose a nonparametric method based on SIMulation-EXtrapolation (SIMEX) to estimate the latent distribution of true effects from estimated average treatment effects with known variances. The method evaluates quantiles after adding progressively more simulated measurement noise and extrapolates the resulting inverse…