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The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

Estimating individualized treatment rules (ITRs) is crucial for tailoring interventions in precision medicine. Typical ITR estimation methods rely on conditional average treatment effects (CATEs) to guide treatment assignments. However,…

Methodology · Statistics 2025-10-20 Peng Wu , Qing Jiang , Shanshan Luo , Zhi Geng

Estimation of conditional average treatment effects (CATEs) plays an essential role in modern medicine by informing treatment decision-making at a patient level. Several metalearners have been proposed recently to estimate CATEs in an…

Applications · Statistics 2022-09-07 Yizhe Xu , Nikolaos Ignatiadis , Erik Sverdrup , Scott Fleming , Stefan Wager , Nigam Shah

To estimate the causal effect of an endogenous treatment using clustered data, the canonical two-stage least squares (2sls) estimates a linear regression of the outcome on treatment status using an instrumental variable (IV) and conducts…

Methodology · Statistics 2026-04-03 Anqi Zhao , Peng Ding , Fan Li

Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyze them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level.…

Methodology · Statistics 2021-08-06 Fangzhou Su , Peng Ding

Recently, many researchers have advanced data-driven methods for modeling heterogeneous treatment effects (HTEs). Even still, estimation of HTEs is a difficult task -- these methods frequently over- or under-estimate the treatment effects,…

Methodology · Statistics 2022-03-28 Yizhe Xu , Steve Yadlowsky

A crucial task for a randomized controlled trial (RCT) is to specify a statistical method that can yield an efficient estimator and powerful test for the treatment effect. A novel and effective strategy to obtain efficient and powerful…

Background: Target trial emulation (TTE) that applies trial design principles to improve the analysis of non-randomized studies is increasingly being used. Applications of TTE to emulate cluster randomized trials (RCTs) have been limited.…

Applications · Statistics 2025-12-17 Shomoita Alam , Nathaniel Dyrkton , Susan Shepherd , Ibrahim Sana , Kevin Phelan , Jay JH Park

Estimating how a treatment affects units individually, known as heterogeneous treatment effect (HTE) estimation, is an essential part of decision-making and policy implementation. The accumulation of large amounts of data in many domains,…

Machine Learning · Computer Science 2022-06-28 Christopher Tran , Elena Zheleva

Sample size determination for cluster randomised trials (CRTs) is challenging as it requires robust estimation of the intra-cluster correlation coefficient (ICC). Typically, the sample size is chosen to provide a certain level of power to…

Applications · Statistics 2023-08-23 S. Faye Williamson , Svetlana V. Tishkovskaya , Kevin J. Wilson

Early risk diagnosis and driving anomaly detection from vehicle stream are of great benefits in a range of advanced solutions towards Smart Road and crash prevention, although there are intrinsic challenges, especially lack of ground truth,…

Machine Learning · Computer Science 2024-10-01 Xiupeng Shi , Yiik Diew Wong , Chen Chai , Michael Zhi-Feng Li , Tianyi Chen , Zeng Zeng

In health and social sciences, it is critically important to identify subgroups of the study population where there is notable heterogeneity of treatment effects (HTE) with respect to the population average. Decision trees have been…

Methodology · Statistics 2024-05-28 Falco J. Bargagli-Stoffi , Riccardo Cadei , Kwonsang Lee , Francesca Dominici

The increasing scientific attention given to precision medicine based on real-world data has led many recent studies to clarify the relationships between treatment effects and patient characteristics. However, this is challenging because of…

Methodology · Statistics 2022-06-20 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

We consider experiments for comparing treatments using units that are ordered linearly over time or space within blocks. In addition to the block effect, we assume that a trend effect influences the response. The latter is modeled as a…

Statistics Theory · Mathematics 2008-12-18 Dibyen Majumdar , John Stufken

The Predictive Approaches to Treatment Effect Heterogeneity statement focused on baseline risk as a robust predictor of treatment effect and provided guidance on risk-based assessment of treatment effect heterogeneity in the RCT setting.…

Sample size calculations can be challenging with skewed continuous outcomes in randomized controlled trials (RCTs). Standard t-test-based calculations may require data transformation, which may be difficult before data collection.…

Randomized controlled trials (RCTs) frequently utilize covariate-adaptive randomization (CAR) (e.g., stratified block randomization) and commonly suffer from imperfect compliance. This paper studies the identification and inference for the…

Econometrics · Economics 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

The conclusions of randomized controlled trials may be biased when the outcome of one unit depends on the treatment status of other units, a problem known as interference. In this work, we study interference in the setting of one-sided…

Methodology · Statistics 2022-11-01 Jennifer Brennan , Vahab Mirrokni , Jean Pouget-Abadie

We consider a real-world chemotherapy scheduling template design problem, where we cluster patient types into groups and find a representative time-slot duration for each group to accommodate all patient types assigned to that group, aiming…

Optimization and Control · Mathematics 2025-10-14 Qing Zhu , Xian Yu , Yu-Li Huang

In stepped wedge cluster randomized trials (SW-CRTs), the intervention is rolled out to clusters over multiple periods. A standard approach for analyzing SW-CRTs utilizes the linear mixed model, where the treatment effect is only present…