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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

Across a wide array of disciplines, many researchers use machine learning (ML) algorithms to identify a subgroup of individuals who are likely to benefit from a treatment the most (``exceptional responders'') or those who are harmed by it.…

Methodology · Statistics 2025-09-03 Michael Lingzhi Li , Kosuke Imai

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

Triple difference designs have become increasingly popular in empirical economics. The advantage of a triple difference design is that, within a treatment group, it allows for another subgroup of the population -- potentially less impacted…

Econometrics · Economics 2025-06-04 Laura Caron

Developing targeted therapies based on patients' baseline characteristics and genomic profiles such as biomarkers has gained growing interests in recent years. Depending on patients' clinical characteristics, the expression of specific…

Applications · Statistics 2019-02-26 Yanxun Xu , Florica Constantine , Yuan Yuan , Yili L. Pritchett

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

This paper develops a nonparametric model that represents how sequences of outcomes and treatment choices influence one another in a dynamic manner. In this setting, we are interested in identifying the average outcome for individuals in…

Econometrics · Economics 2019-01-16 Sukjin Han

Analyzing data from multiple sources offers valuable opportunities to improve the estimation efficiency of causal estimands. However, this analysis also poses many challenges due to population heterogeneity and data privacy constraints.…

Methodology · Statistics 2025-10-23 Rong Zhao , Jason Falvey , Xu Shi , Vernon M. Chinchilli , Chixiang Chen

Developing tools for estimating heterogeneous treatment effects (HTE) and individualized treatment effects has been an area of active research in recent years. While these tools have proven to be useful in many contexts, a concern when…

Methodology · Statistics 2025-03-07 Mahsa Ashouri , Nicholas C. Henderson

Previous works on Treatment Effect Estimation (TEE) are not in widespread use because they are predominantly theoretical, where strong parametric assumptions are made but untractable for practical application. Recent work uses multilayer…

Machine Learning · Computer Science 2022-10-18 Yi-Fan Zhang , Hanlin Zhang , Zachary C. Lipton , Li Erran Li , Eric P. Xing

To effectively optimize and personalize treatments, it is necessary to investigate the heterogeneity of treatment effects. With the wide range of users being treated over many online controlled experiments, the typical approach of manually…

Methodology · Statistics 2022-11-07 John Cai , Weinan Wang

Estimating treatment effects is of great importance for many biomedical applications with observational data. Particularly, interpretability of the treatment effects is preferable for many biomedical researchers. In this paper, we first…

Machine Learning · Statistics 2022-06-28 Kan Chen , Qishuo Yin , Qi Long

Machine learning (ML) models may suffer from significant performance disparities between patient groups. Identifying such disparities by monitoring performance at a granular level is crucial for safely deploying ML to each patient.…

Machine Learning · Computer Science 2025-03-14 Alceu Bissoto , Trung-Dung Hoang , Tim Flühmann , Susu Sun , Christian F. Baumgartner , Lisa M. Koch

An important aspect of precision medicine focuses on characterizing diverse responses to treatment due to unique patient characteristics, also known as heterogeneous treatment effects (HTE), and identifying beneficial subgroups with…

Methodology · Statistics 2024-07-03 Na Bo , Jong-Hyeon Jeong , Erick Forno , Ying Ding

In this paper we review recent advances in statistical methods for the evaluation of the heterogeneity of treatment effects (HTE), including subgroup identification and estimation of individualized treatment regimens, from randomized…

Methodology · Statistics 2024-10-22 Ilya Lipkovich , David Svensson , Bohdana Ratitch , Alex Dmitrienko

Recent methodological developments have introduced new black-box approaches to better estimate heterogeneous treatment effects; however, these methods fall short of providing interpretable characterizations of the underlying individuals who…

Methodology · Statistics 2025-08-19 Melody Huang , Tiffany M. Tang , Ana M. Kenney

Given a dataset of individuals each described by a covariate vector, a treatment, and an observed outcome on the treatment, the goal of the individual treatment effect (ITE) estimation task is to predict outcome changes resulting from a…

Machine Learning · Computer Science 2024-06-07 Lokesh Nagalapatti , Pranava Singhal , Avishek Ghosh , Sunita Sarawagi

We consider the problem of estimating the average treatment effect (ATE) in a semi-supervised learning setting, where a very small proportion of the entire set of observations are labeled with the true outcome but features predictive of the…

Methodology · Statistics 2020-10-27 David Cheng , Ashwin Ananthakrishnan , Tianxi Cai

A lack of understanding of human biology creates a hurdle for the development of precision medicines. To overcome this hurdle we need to better understand the potential synergy between a given investigational treatment (vs. placebo or…

Applications · Statistics 2017-08-17 Jia Jia , Qi Tang , Wangang Xie , Richard Rode

Inferring unbiased treatment effects has received widespread attention in the machine learning community. In recent years, our community has proposed numerous solutions in standard settings, high-dimensional treatment settings, and even…

Machine Learning · Computer Science 2024-03-19 Krzysztof Kacprzyk , Samuel Holt , Jeroen Berrevoets , Zhaozhi Qian , Mihaela van der Schaar