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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

Patients in clinical studies often exhibit heterogeneous treatment effect (HTE). Classical subgroup analyses provide inferential tools to test for effect modification, while modern machine learning methods estimate the Conditional Average…

Applications · Statistics 2026-01-05 Nan Miles Xi , Xin Huang , Lin Wang

Heterogeneous treatment effects (HTEs) are commonly identified during randomized controlled trials (RCTs). Identifying subgroups of patients with similar treatment effects is of high interest in clinical research to advance precision…

Machine Learning · Computer Science 2022-12-06 Peniel N. Argaw , Elizabeth Healey , Isaac S. Kohane

Precise estimation of treatment effects is crucial for evaluating intervention effectiveness. While deep learning models have exhibited promising performance in learning counterfactual representations for treatment effect estimation (TEE),…

Machine Learning · Computer Science 2024-01-24 Seungyeon Lee , Ruoqi Liu , Wenyu Song , Lang Li , Ping Zhang

There is strong interest in estimating how the magnitude of treatment effects of an intervention vary across sub-groups of the population of interest. In our paper, we propose a two-study approach to first propose and then test…

Methodology · Statistics 2020-06-23 Rahul Ladhania , Amelia Haviland , Neeraj Sood , Edward Kennedy , Ateev Mehrotra

Heterogeneous treatment effects can be very important in the analysis of randomized clinical trials. Heightened risks or enhanced benefits may exist for particular subsets of study subjects. When the heterogeneous treatment effects are…

Methodology · Statistics 2025-07-25 Richard A. Berk , Matthew Olson , Andreas Buja , Aurelie Ouss

Estimating heterogeneous treatment effect (HTE) for survival outcomes has gained increasing attention, as it captures the variation in treatment efficacy across patients or subgroups in delaying disease progression. However, most existing…

Methodology · Statistics 2025-11-27 Na Bo , Ying Ding

Deep learning models have demonstrated promising results in estimating treatment effects (TEE). However, most of them overlook the variations in treatment outcomes among subgroups with distinct characteristics. This limitation hinders their…

Machine Learning · Computer Science 2024-01-31 Seungyeon Lee , Ruoqi Liu , Wenyu Song , Ping Zhang

Treatment effect heterogeneity refers to the systematic variation in treatment effects across subgroups. There is an increasing need for clinical trials that aim to investigate treatment effect heterogeneity and estimate subgroup-specific…

Methodology · Statistics 2026-03-06 Xianglin Zhao , Shirin Golchi , Jean-Philippe Gouin , Kaberi Dasgupta

Recently, from the personalized medicine perspective, there has been an increased demand to identify subgroups of subjects for whom treatment is effective. Consequently, the estimation of heterogeneous treatment effects (HTE) has been…

Methodology · Statistics 2024-08-02 Ryoma Hieda , Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Heterogeneous treatment effects, which vary according to individual covariates, are crucial in fields such as personalized medicine and tailored treatment strategies. In many applications, rather than considering the heterogeneity induced…

Methodology · Statistics 2025-08-26 Peng Wu , Pengtao Zeng , Zhaoqing Tian , Shaojie Wei

Estimation of individualized treatment effects (ITE) from observational studies is a fundamental problem in causal inference and holds significant importance across domains, including healthcare. However, limited observational datasets pose…

Machine Learning · Computer Science 2024-02-14 Vinod Kumar Chauhan , Jiandong Zhou , Ghadeer Ghosheh , Soheila Molaei , David A. Clifton

An important task in early phase drug development is to identify patients, which respond better or worse to an experimental treatment. While a variety of different subgroup identification methods have been developed for the situation of…

Methodology · Statistics 2020-01-22 Marius Thomas , Björn Bornkamp , Heidi Seibold

In biomedical science, analyzing treatment effect heterogeneity plays an essential role in assisting personalized medicine. The main goals of analyzing treatment effect heterogeneity include estimating treatment effects in clinically…

Methodology · Statistics 2022-12-06 Waverly Wei , Maya Petersen , Mark J van der Laan , Zeyu Zheng , Chong Wu , Jingshen Wang

In personalised decision making, evidence is required to determine whether an action (treatment) is suitable for an individual. Such evidence can be obtained by modelling treatment effect heterogeneity in subgroups. The existing…

Methodology · Statistics 2022-06-24 Jiuyong Li , Lin Liu , Shisheng Zhang , Saisai Ma , Thuc Duy Le , Jixue Liu

In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

Machine Learning · Statistics 2022-06-08 Susan Athey , Guido Imbens

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…

Methodology · Statistics 2026-04-14 Yuan Bian , Donglin Zeng , Hyun-Joon Yang , Leanne M. Williams , Yuanjia Wang

Non-significant randomized control trials can hide subgroups of good responders to experimental drugs, thus hindering subsequent development. Identifying such heterogeneous treatment effects is key for precision medicine and many post-hoc…

Methodology · Statistics 2024-01-24 Valentine Perrin , Nathan Noiry , Nicolas Loiseau , Alex Nowak

Treatment effect heterogeneity occurs when individual characteristics influence the effect of a treatment. We propose a novel approach that combines prognostic score matching and conditional inference trees to characterize effect…

Estimating individualised treatment effect (ITE) -- that is the causal effect of a set of variables (also called exposures, treatments, actions, policies, or interventions), referred to as \textit{composite treatments}, on a set of outcome…

Machine Learning · Computer Science 2025-12-19 Vinod Kumar Chauhan , Lei Clifton , Gaurav Nigam , David A. Clifton
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