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With more and better clinical data being captured outside of clinical studies and greater data sharing of clinical studies, external controls may become a more attractive alternative to randomized clinical trials. Both industry and…

When incorporating historical control data into the analysis of current randomized controlled trial data, it is critical to account for differences between the datasets. When the cause of the difference is an unmeasured factor and…

Methodology · Statistics 2025-09-10 Tomohiro Ohigashi , Kazushi Maruo , Takashi Sozu , Masahiko Gosho

The weighted average treatment effect (WATE) is a causal measure for the comparison of interventions in a specific target population, which may be different from the population where data are sampled from. For instance, when the goal is to…

Methodology · Statistics 2018-04-17 Yebin Tao , Haoda Fu

This paper proposes Bayesian Adaptive Trials (BAT) as both an efficient method to conduct trials and a unifying framework for evaluation social policy interventions, addressing limitations inherent in traditional methods such as Randomized…

There is growing interest in exploring causal effects in target populations via data combination. However, most approaches are tailored to specific settings and lack comprehensive comparative analyses. In this article, we focus on a typical…

Methodology · Statistics 2024-09-17 Peng Wu , Shanshan Luo , Zhi Geng

The conditional average treatment effect (CATE) is the best measure of individual causal effects given baseline covariates. However, the CATE only captures the (conditional) average, and can overlook risks and tail events, which are…

Machine Learning · Statistics 2025-06-05 Nathan Kallus , Miruna Oprescu

Consider the problem of improving the estimation of conditional average treatment effects (CATE) for a target domain of interest by leveraging related information from a source domain with a different feature space. This heterogeneous…

Machine Learning · Computer Science 2022-10-13 Ioana Bica , Mihaela van der Schaar

Machine learning models used in medical applications often face challenges due to the covariate shift, which occurs when there are discrepancies between the distributions of training and target data. This can lead to decreased predictive…

Machine Learning · Computer Science 2024-12-24 Mingyang Cai , Thomas Klausch , Mark A. van de Wiel

Estimating individualized treatment rules (ITRs) is fundamental to precision medicine, where the goal is to tailor treatment decisions to individual patient characteristics. While numerous methods have been developed for ITR estimation,…

Methodology · Statistics 2026-05-15 Eun Jeong Oh , Min Qian

Randomized controlled trials (RCT's) allow researchers to estimate causal effects in an experimental sample with minimal identifying assumptions. However, to generalize or transport a causal effect from an RCT to a target population,…

Methodology · Statistics 2022-02-08 Melody Huang

In many areas, practitioners seek to use observational data to learn a treatment assignment policy that satisfies application-specific constraints, such as budget, fairness, simplicity, or other functional form constraints. For example,…

Statistics Theory · Mathematics 2020-09-08 Susan Athey , Stefan Wager

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

Methodology · Statistics 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

Extrapolating treatment effects from related studies is a promising strategy for designing and analyzing clinical trials in situations where achieving an adequate sample size is challenging. Bayesian methods are well-suited for this…

Methodology · Statistics 2025-11-25 Tristan Fauvel , Julien Tanniou , Pascal Godbillot , Marie Génin , Billy Amzal

From scientific experiments to online A/B testing, the previously observed data often affects how future experiments are performed, which in turn affects which data will be collected. Such adaptivity introduces complex correlations between…

Machine Learning · Statistics 2018-01-03 Xinkun Nie , Xiaoying Tian , Jonathan Taylor , James Zou

While sample sizes in randomized clinical trials are large enough to estimate the average treatment effect well, they are often insufficient for estimation of treatment-covariate interactions critical to studying data-driven precision…

Machine Learning · Statistics 2020-04-22 Steve Yadlowsky , Fabio Pellegrini , Federica Lionetto , Stefan Braune , Lu Tian

We propose a practical and robust method for making inferences on average treatment effects estimated by synthetic controls. We develop a $K$-fold cross-fitting procedure for bias correction. To avoid the difficult estimation of the…

Econometrics · Economics 2025-05-26 Victor Chernozhukov , Kaspar Wuthrich , Yinchu Zhu

In this work, we proposed a novel inferential procedure assisted by machine learning based adjustment for randomized control trials. The method was developed under the Rosenbaum's framework of exact tests in randomized experiments with…

Methodology · Statistics 2024-07-23 Han Yu , Alan D. Hutson , Xiaoyi Ma

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…

Methodology · Statistics 2026-05-15 Rikuta Hamaya , Etsuji Suzuki , Konan Hara

Randomized controlled trials (RCTs) yield internally valid causal effect estimates, but generalizing these results to target populations with different characteristics requires an untestable selection ignorability assumption: conditional on…

Methodology · Statistics 2026-03-31 Amir Asiaee , Samhita Pal , Jared D. Huling

We develop new semiparametric methods for estimating treatment effects. We focus on settings where the outcome distributions may be thick tailed, where treatment effects may be small, where sample sizes are large and where assignment is…

Methodology · Statistics 2023-08-24 Susan Athey , Peter J. Bickel , Aiyou Chen , Guido W. Imbens , Michael Pollmann