arXiv subjects

Bosen Cui

Publications and source records attributed to Bosen Cui.

3 recordsLinked to original sources

arXiv2026

Power and sample size calculations for causal mediation analysis with a binary mediator in randomized trials

Mediation analyses are increasingly conducted in randomized trials, but a sample size adequate for the total treatment effect may leave the natural indirect effect (NIE) or natural direct effect (NDE) substantially underpowered. Randomization does not extend to the mediator, so precision depends on the conditional mediator distribution and the mediator-outcome association, neither of which enters a total-effect calculation. Planning outside linear structural equation models is largely based on simulation under a fully specified data-generating mechanism rarely available at the design stage. This paper develops analytic power and sample size formulas for the NIE and NDE with a binary mediator and a continuous or binary outcome. Under standard identification assumptions, we focus on the ratio-of-mediator-probability weighting (RMPW) estimator that does not require an outcome model for effect estimation. We decompose the oracle variances of the RMPW estimators into components capturing mediator-probability-ratio variability, outcome variation, and their association, with an additional shared-arm covariance term for the NIE. Under a probit latent-index mediator and a working outcome model, these components are determined by a small number of interpretable design inputs rather than by the full joint distribution of covariates, mediator, and outcome. Simulations show that the analytic sample sizes closely match simulation-based benchmarks, attain the target power, and maintain type I error near the nominal level. An ACTG175 illustration shows how pilot data can calibrate the inputs.

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arXiv2026

Transporting Trial Evidence Under Posterior Drift and Possible Hidden Confounding

Randomized trials provide internally valid treatment-effect evidence, but trial participants may not represent the target population. In contrast, observational studies are often closer to the target population, but their treatment assignment may be affected by possible hidden confounding. We develop a robust posterior-drift framework for estimating the average treatment effect in an observational target population when exact conditional-effect transportability may fail. The framework represents observational conditional potential-outcome regressions as their randomized-trial counterparts plus source-specific drifts. The randomized trial serves as an internally valid anchor, while the observational study supplies the target covariate distribution and partial information about the target causal contrast. To account for possible hidden confounding, we consider a Rosenbaum-type uncertainty set induced by a sensitivity parameter on the generalized propensity score and estimate the drift through a minimax worst-case risk criterion. We derive efficiency results in auxiliary regimes, establish uniform concentration and near-optimality guarantees for the minimax estimator, and handle general parametric and smooth nonparametric drift classes. Simulations and an ACTG 175--WIHS application show that the proposed analysis yields more cautious and interpretable target-population effect estimates than exact-transportability analyses.

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arXiv2026

Assessing Estimate of CATE from Observational Data via an RCT Study

Conditional average treatment effects (CATEs) are increasingly estimated from observational data and used to guide policy and individualized treatment decisions. Before such estimates can be trusted in practice, their predictive fitness needs to be assessed, yet observational data alone offer limited opportunities for doing so. We propose CATE Assessment via Fitness Evaluation (CAFE), a formal framework for directly assessing the goodness-of-fit of a CATE estimate learned from observational data, rather than the full underlying outcome model, using evidence from a randomized trial. CAFE partitions the trial covariate space according to estimated propensity scores (or the like) and compares observationally derived conditional treatment effects with group-level experimental averages. The framework accommodates a broad class of CATE learners, including parametric models and flexible machine learning methods such as causal forest and boosting. We establish theoretical guarantees under both the null and alternative hypotheses, and introduce a maximum-type extension to improve sensitivity to localized lack of fit. When both randomized trial and observational data are available, we further develop a two-stage procedure to detect the existence of unobserved confounders. Extensive numerical studies show the utility of the CAFE approach when assessing observational-derived CATE estimates.

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