Model-robust standardization in stepped wedge cluster randomized trials
阶梯楔形集群随机试验中的模型稳健标准化AI 解读 ↓
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阶梯楔形集群随机试验中的模型稳健标准化AI 解读 ↓
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Stepped-wedge cluster-randomized trials (SW-CRTs) are widely used in healthcare and implementation science, enabling all clusters to receive the intervention through a staggered rollout. Traditional model-based methods, including generalized estimating equations and mixed models, yield estimates that depend on implicit weighting schemes and parametric assumptions, and therefore may target ambiguous estimands under model misspecification. In this article, we propose a model-robust standardization framework for SW-CRTs that generalizes existing methods from parallel-arm CRTs to address informative sizes. We define causal estimands including horizontal-individual, horizontal-cluster, vertical-individual, and vertical-cluster average treatment effects under a super population framework and introduce a simple procedure that standardizes parametric and semiparametric working models for estimand-aligned analysis. For any specified working model, the resulting estimators remain consistent for their target estimands even if the working regression model is misspecified; moreover, their efficiency improves as the working model more closely approximates the true data-generating process. We evaluate the finite-sample properties of our proposed estimators through extensive simulations. Finally, we illustrate the application of our methods through reanalyses of two real-world SW-CRTs.
We develop a semiparametric continuous time inferential framework for testing cumulative treatment effects, including both immediate and carryover effects. The latent state process is modeled by an ordinary differential equation (ODE) whose drift depends on state and treatment, while the observed data are noisy measurements of this latent state at irregular, multi resolution time points. We define the average treatment effect (ATE) as the difference of expected cumulative…
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…
We study nonparametric inference for a continuous-treatment causal dose-response curve and its first derivative under unmeasured confounding, using treatment- and outcome-inducing proxies. We establish a proximal doubly robust pseudo-outcome whose conditional mean given treatment equals the dose-response curve whenever either bridge function is correctly specified. Their (smoothed) efficient influence function representations lead to cross-fitted local-polynomial estimators…
前门模型下因果效应的灵活非参数推断
Abstract Evaluating causal treatment effects in observational studies requires addressing confounding. While the back-door criterion enables identification through adjustment for observed covariates, it fails in the presence of unmeasured confounding. The front-door criterion offers an alternative by leveraging variables that fully mediate the treatment effect and are unaffected by unmeasured confounders of the treatment-outcome pair. We develop novel one-step and targeted…
使用 AI 学习表征的实用双重机器学习
Text, images, and other rich covariates are increasingly compressed into AI-learned representations and then used as controls in causal analysis. We study when this approach is valid and develop a practical framework for causal inference with learned representations. For a broad class of estimands, an imperfect representation distorts the target causal parameter by the product of two representation errors: one in the outcome regression and one in the balancing weight (or…
The bootstrap is widely used to construct confidence intervals but may perform poorly for some estimators. We consider semiparametric estimators that use cross-validation to estimate nuisance pa- rameters and can be computationally expensive in large datasets. We illustrate potential problems with bootstrapping with replacement when cross-validation is used and extend the cheap bootstrap confidence interval to subsampling without replacement for asymptotically linear…