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Misclassified binary outcome or mediator variables can cause unpredictable bias in resulting parameter estimates. As more datasets that were not originally collected for research purposes are being used for studies in the social and health…

Computation · Statistics 2025-01-15 Kimberly A. Hochstedler Webb , Martin T. Wells

Covariate adjustment can improve precision in analyzing randomized experiments. With fully observed data, regression adjustment and propensity score weighting are asymptotically equivalent in improving efficiency over unadjusted analysis.…

Methodology · Statistics 2024-03-06 Anqi Zhao , Peng Ding , Fan Li

Recent advances have shown that statistical tests for the rank of cross-covariance matrices play an important role in causal discovery. These rank tests include partial correlation tests as special cases and provide further graphical…

Machine Learning · Computer Science 2025-06-13 Xinshuai Dong , Ignavier Ng , Boyang Sun , Haoyue Dai , Guang-Yuan Hao , Shunxing Fan , Peter Spirtes , Yumou Qiu , Kun Zhang

Longitudinal observational patient data can be used to investigate the causal effects of time-varying treatments on time-to-event outcomes. Several methods have been developed for controlling for the time-dependent confounding that…

Methodology · Statistics 2021-10-08 Ruth H. Keogh , Jon Michael Gran , Shaun R. Seaman , Gwyneth Davies , Stijn Vansteelandt

Classical randomized experiments, equipped with randomization-based inference, provide assumption-free inference for treatment effects. They have been the gold standard for drawing causal inference and provide excellent internal validity.…

Methodology · Statistics 2021-09-22 Zihao Yang , Tianyi Qu , Xinran Li

Fisher's randomization test (FRT) delivers exact $p$-values under the strong null hypothesis of no treatment effect on any units whatsoever and allows for flexible covariate adjustment to improve the power. Of interest is whether the…

Methodology · Statistics 2021-05-03 Anqi Zhao , Peng Ding

External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized…

Methodology · Statistics 2025-05-08 Ke Zhu , Shu Yang , Xiaofei Wang

We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling…

Methodology · Statistics 2023-02-27 Imke Mayer , Julie Josse , Traumabase Group

Randomized pre-post designs, with outcomes measured at baseline and follow-ups, have been commonly used to compare the clinical effectiveness of two competing treatments. There are vast, but often conflicting, amount of information in…

Applications · Statistics 2020-07-16 Fei Wan

We propose novel estimators for categorical and continuous treatments by using an optimal covariate balancing strategy for inverse probability weighting. The resulting estimators are shown to be consistent and asymptotically normal for…

Methodology · Statistics 2025-09-08 Seong-ho Lee , Yanyuan Ma , Xavier de Luna

There is a dearth of robust methods to estimate the causal effects of multiple treatments when the outcome is binary. This paper uses two unique sets of simulations to propose and evaluate the use of Bayesian Additive Regression Trees…

Methodology · Statistics 2020-01-22 Liangyuan Hu , Chenyang Gu , Michael Lopez , Jiayi Ji , Juan Wisnivesky

Semi-competing risks data arise when both non-terminal and terminal events are considered in a model. Such data with multiple events of interest are frequently encountered in medical research and clinical trials. In this framework, terminal…

Methodology · Statistics 2022-11-21 Fatemeh Mahmoudi , Xuewen Lu

Randomized controlled trials (RCTs) can be used to generate guarantees on treatment effects. However, RCTs often spend unnecessary resources exploring sub-optimal treatments, which can reduce the power of treatment guarantees. To address…

Computers and Society · Computer Science 2024-10-16 Santiago Cortes-Gomez , Naveen Raman , Aarti Singh , Bryan Wilder

When testing for superiority in a parallel-group setting with a continuous outcome, adjusting for covariates (e.g., baseline measurements) is usually recommended, in order to reduce bias and increase power. For this purpose, the analysis of…

Methodology · Statistics 2020-04-28 Georg Zimmermann , Meinhard Kieser , Arne Bathke

A practical limitation of cluster randomized controlled trials (cRCTs) is that the number of available clusters may be small, resulting in an increased risk of baseline imbalance under simple randomization. Constrained randomization…

Methodology · Statistics 2022-01-19 Yunji Zhou , Elizabeth L. Turner , Ryan A. Simmons , Fan Li

We propose the covariate-balanced-and-adjusted response-adaptive randomization (CBARA) procedure for adaptive design in clinical trials, which integrates the complementary strengths of covariate-adjusted response-adaptive randomization…

Methodology · Statistics 2026-05-08 Hengjia Fang , Wei Ma

Nonparametric covariate adjustment is considered for log-rank type tests of treatment effect with right-censored time-to-event data from clinical trials applying covariate-adaptive randomization. Our proposed covariate-adjusted log-rank…

Methodology · Statistics 2023-01-23 Ting Ye , Jun Shao , Yanyao Yi

The proximal causal inference framework enables the identification and estimation of causal effects in the presence of unmeasured confounding by leveraging two disjoint sets of observed strong proxies: negative control treatments and…

Methodology · Statistics 2025-12-16 Antonio Olivas-Martinez , Peter B. Gilbert , Andrea Rotnitzky

Personalized medicine seeks to identify the causal effect of treatment for a particular patient as opposed to a clinical population at large. Most investigators estimate such personalized treatment effects by regressing the outcome of a…

Machine Learning · Statistics 2021-09-02 Eric V. Strobl , Shyam Visweswaran

What can be considered an appropriate statistical method for the primary analysis of a randomized clinical trial (RCT) with a time-to-event endpoint when we anticipate non-proportional hazards owing to a delayed effect? This question has…

Methodology · Statistics 2023-04-18 José L. Jiménez , Isobel Barrott , Francesca Gasperoni , Dominic Magirr
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