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Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as…

统计方法学 · 统计学 2026-04-16 Qinwei Yang , Jingyi Li , Peng Wu , Shu Yang

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…

统计方法学 · 统计学 2024-04-15 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

Randomized controlled trials (RCTs) often exhibit limited inferential efficiency in estimating treatment effects due to small sample sizes. In recent years, the combination of external controls has gained increasing attention as a means of…

统计方法学 · 统计学 2025-10-06 Qinwei Yang , Jingyi Li , Peng Wu

Randomized controlled trials (RCTs) often suffer from limited sample sizes due to high costs and lengthy recruitment periods, compromising precision in treatment effect estimation. External real-world control data offer a valuable…

应用统计 · 统计学 2026-05-05 Peng Wu , Jile Chaoge , Shu Yang

An individualized treatment rule (ITR) tailors treatments to a patient's specific characteristics. However, randomized controlled trials (RCTs) are often underpowered to detect the treatment effect heterogeneity needed for reliable ITR…

统计方法学 · 统计学 2026-04-14 Yuan Bian , Donglin Zeng , Hyun-Joon Yang , Leanne M. Williams , Yuanjia Wang

Inferring causal individual treatment effect (ITE) from observational data is a challenging problem whose difficulty is exacerbated by the presence of treatment assignment bias. In this work, we propose a new way to estimate the ITE using…

机器学习 · 计算机科学 2021-03-16 Abhin Shah , Kartik Ahuja , Karthikeyan Shanmugam , Dennis Wei , Kush Varshney , Amit Dhurandhar

Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…

应用统计 · 统计学 2026-05-26 Yifei Xu , Hwiyoung Lee , Zhenyao Ye , Yezhi Pan , Jingsong Zhou , Yun Yang , Chixiang Chen , Shuo Chen

Estimating conditional average treatment effects (CATE) from randomized controlled trials (RCTs) and generalizing them to broader populations is essential for personalizing treatment rules but is complicated by selection bias due to trial…

统计方法学 · 统计学 2026-05-15 Rikuta Hamaya , Etsuji Suzuki , Konan Hara

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…

计量经济学 · 经济学 2025-05-02 Federico A. Bugni , Mengsi Gao , Filip Obradovic , Amilcar Velez

Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

应用统计 · 统计学 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu

Estimating conditional average treatment effects (CATE) is challenging, especially when treatment information is missing. Although this is a widespread problem in practice, CATE estimation with missing treatments has received little…

机器学习 · 统计学 2023-04-19 Milan Kuzmanovic , Tobias Hatt , Stefan Feuerriegel

We develop estimators that improve precision of heterogeneous treatment effect estimates that allow borrowing information from observational studies when the available covariates in each data source do not perfectly match. Standard…

统计方法学 · 统计学 2026-03-19 Samhita Pal , Jared D. Huling , Amir Asiaee

Augmented inverse probability weighting and G-computation with canonical generalized linear models have become increasingly popular for estimating average treatment effects (ATEs) in randomized experiments. These methods leverage outcome…

统计方法学 · 统计学 2026-03-13 Muluneh Alene , Stijn Vansteelandt , Kelly Van Lancker

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…

统计方法学 · 统计学 2025-07-22 Amir Asiaee , Chiara Di Gravio , Cole Beck , Yuting Mei , Samhita Pal , Jared D. Huling

We consider the problem of variance reduction in randomized controlled trials, through the use of covariates correlated with the outcome but independent of the treatment. We propose a machine learning regression-adjusted treatment effect…

机器学习 · 统计学 2022-01-07 Yongyi Guo , Dominic Coey , Mikael Konutgan , Wenting Li , Chris Schoener , Matt Goldman

Accurately quantifying uncertainty of individual treatment effects (ITEs) across multiple decision points is crucial for personalized decision-making in fields such as healthcare, finance, education, and online marketplaces. Previous work…

统计方法学 · 统计学 2025-12-10 Swaraj Bose , Walter Dempsey

Confounding is a significant obstacle to unbiased estimation of causal effects from observational data. For settings with high-dimensional covariates -- such as text data, genomics, or the behavioral social sciences -- researchers have…

人工智能 · 计算机科学 2024-02-01 Katherine A. Keith , Sergey Feldman , David Jurgens , Jonathan Bragg , Rohit Bhattacharya

Machine learning is increasingly used to select which individuals receive limited-resource interventions in domains such as human services, education, development, and more. However, it is often not apparent what the right quantity is for…

机器学习 · 计算机科学 2025-03-20 Vibhhu Sharma , Bryan Wilder

Randomized controlled trials are the standard method for estimating causal effects, ensuring sufficient statistical power and confidence through adequate sample sizes. However, achieving such sample sizes is often challenging. This study…

统计方法学 · 统计学 2025-03-28 Keisuke Hanada , Masahiro Kojima

The proliferation of data has sparked significant interest in leveraging findings from one study to estimate treatment effects in a different target population without direct outcome observations. However, the transfer learning process is…

统计方法学 · 统计学 2025-05-15 Seok-Jin Kim , Hongjie Liu , Molei Liu , Kaizheng Wang
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