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Cole Beck

Publications and source records attributed to Cole Beck.

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Sharp Bounds for Treatment Effect Generalization under Outcome Distribution Shift

Generalizing treatment effects from a randomized trial to a target population requires the assumption that potential outcome distributions are invariant across populations after conditioning on observed covariates. This assumption fails when unmeasured effect modifiers are distributed differently between trial participants and the target population. We develop a sensitivity analysis framework that bounds how much conclusions can change when this transportability assumption is violated. Our approach constrains the likelihood ratio between target and trial outcome densities by a scalar parameter $\Lambda \geq 1$, with $\Lambda = 1$ recovering standard transportability. For each $\Lambda$, we derive sharp bounds on the target average treatment effect -- the tightest interval guaranteed to contain the true effect under all data-generating processes compatible with the observed data and the sensitivity model. We show that the optimal likelihood ratios have a simple threshold structure, leading to a closed-form greedy algorithm that requires only sorting trial outcomes and redistributing probability mass. The resulting estimator runs in $O(n \log n)$ time and is consistent under standard regularity conditions. Simulations demonstrate that our bounds achieve nominal coverage when the true outcome shift falls within the specified $\Lambda$, provide substantially tighter intervals than worst-case bounds, and remain informative across a range of realistic violations of transportability.

stat.ME

Improving Precision of RCT-Based CATE Estimation using Data Borrowing with Double Calibration

Understanding how treatment effects vary across patient characteristics is essential for personalized medicine, yet randomized controlled trials (RCTs) are often underpowered to detect heterogeneous treatment effects (HTEs). We propose a framework that improves the efficiency of conditional average treatment effect (CATE) estimation in RCTs by leveraging large observational studies (OS) while preserving RCT unbiasedness. Framing CATE estimation as a supervised learning problem, we show that estimation variance is minimized using the counterfactual mean outcome (CMO) as an augmentation function. We derive finite-sample error bounds and give conditions under which OS data improves CMO estimation, and thus CATE efficiency, even under confounding in the OS or outcome distribution shift between populations. We introduce R-OSCAR (Robust Observational Studies for CMO-Augmented RCT), a two-stage estimator that calibrates OS outcome predictions to the RCT population and corrects residual bias through regularized regression. For any OS-derived nuisance, R-OSCAR is consistent for the RCT-population CATE, and is efficient relative to RCT-only estimators when the RCT-OS outcome mean discrepancy is estimable from the RCT at lower complexity than the full RCT outcome model. A cross-fitted RCT diagnostic determines, from observable data alone, whether borrowing from a given OS is supported. Simulations show R-OSCAR can reduce the RCT sample size needed for HTE detection by up to 75%, while remaining robust to misspecification. We validate on two case studies: a semi-synthetic analysis of the Tennessee STAR study with constructed observational confounding, and the Greenlight Plus pediatric-obesity trial linked with external electronic-health-record controls, where borrowing improves control-arm estimation for small trials and the diagnostic certifies it only where the records cover the trial population.

stat.ME