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Model-based recursive partitioning (MOB) and its extension, metaMOB, are potent tools for identifying subgroups with differential treatment effects. In the metaMOB approach random effects are used to model heterogeneity of the treatment…

Methodology · Statistics 2023-11-06 Cynthia Huber , Tim Friede

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

Model-based recursive partitioning (MOB) is a semi-parametric statistical approach allowing the identification of subgroups that can be combined with a broad range of outcome measures including continuous time-to-event outcomes. When time…

Methodology · Statistics 2022-09-15 Cynthia Huber , Matthias Schmid , Tim Friede

Growth curve models are popular tools for studying the development of a response variable within subjects over time. Heterogeneity between subjects is common in such models, and researchers are typically interested in explaining or…

Methodology · Statistics 2025-10-07 Marjolein Fokkema , Achim Zeileis

We consider the problem of identifying sub-groups of participants in a clinical trial that have enhanced treatment effect. Recursive partitioning methods that recursively partition the covariate space based on some measure of between groups…

Methodology · Statistics 2018-06-22 Jon Arni Steingrimsson , Jiabei Yang

Identifying and making statistical inferences on differential treatment effects (commonly known as subgroup analysis in clinical research) is central to precision health. Subgroup analysis allows practitioners to pinpoint populations for…

Machine Learning · Statistics 2026-02-05 Zhongming Xie , Joseph Giorgio , Jingshen Wang

The identification of patient subgroups with differential treatment effects is the first step towards individualised treatments. A current draft guideline by the EMA discusses potentials and problems in subgroup analyses and formulated…

Methodology · Statistics 2020-01-22 Heidi Seibold , Achim Zeileis , Torsten Hothorn

Subgroup selection in clinical trials is essential for identifying patient groups that react differently to a treatment, thereby enabling personalised medicine. In particular, subgroup selection can identify patient groups that respond…

Researchers are increasingly turning to machine learning (ML) algorithms to investigate causal heterogeneity in randomized experiments. Despite their promise, ML algorithms may fail to accurately ascertain heterogeneous treatment effects…

Methodology · Statistics 2024-04-23 Kosuke Imai , Michael Lingzhi Li

Case-mix heterogeneity across studies complicates meta-analyses. As a result of this, treatments that are equally effective on patient subgroups may appear to have different effectiveness on patient populations with different case mix. It…

Applications · Statistics 2021-08-25 Tat-Thang Vo , Raphael Porcher , Anna Chaimani , Stijn Vansteelandt

The idea of "stratified medicine" is an important driver of methodological research on the identification of predictive biomarkers. Most methods proposed so far for this purpose have been developed for the use on randomized data only.…

Methodology · Statistics 2022-12-19 Julia Krzykalla , Axel Benner , Annette Kopp-Schneider

Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple…

Methodology · Statistics 2024-07-10 Noorie Hyun , Abisola E. Idu , Andrea J. Cook , Jennifer F. Bobb

Subgroup analysis of treatment effects plays an important role in applications from medicine to public policy to recommender systems. It allows physicians (for example) to identify groups of patients for whom a given drug or treatment is…

Machine Learning · Statistics 2020-10-20 Hyun-Suk Lee , Yao Zhang , William Zame , Cong Shen , Jang-Won Lee , Mihaela van der Schaar

Identifying subgroups, which respond differently to a treatment, both in terms of efficacy and safety, is an important part of drug development. A well-known challenge in exploratory subgroup analyses is the small sample size in the…

Computation · Statistics 2016-06-28 Marius Thomas , Björn Bornkamp

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

During drug development, evidence can emerge to suggest a treatment is more effective in a specific patient subgroup. Whilst early trials may be conducted in biomarker-mixed populations, later trials are more likely to enrol…

Methodology · Statistics 2023-06-07 Lorna Wheaton , Dan Jackson , Sylwia Bujkiewicz

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

Subgroup identification (clustering) is an important problem in biomedical research. Gene expression profiles are commonly utilized to define subgroups. Longitudinal gene expression profiles might provide additional information on disease…

Methodology · Statistics 2016-09-27 Jiehuan Sun , Jose D. Herazo-Maya , Naftali Kaminski , Hongyu Zhao , Joshua L. Warren

We propose Causal Interaction Trees for identifying subgroups of participants that have enhanced treatment effects using observational data. We extend the Classification and Regression Tree algorithm by using splitting criteria that focus…

Methodology · Statistics 2021-12-08 Jiabei Yang , Issa J. Dahabreh , Jon A. Steingrimsson

When a subgroup is identified from the data, it must be evaluated in a replicable way. The usual in-sample approach, which evaluates the post-hoc identified subgroup as predefined, might suffer from selection bias. This issue of in-sample…

Methodology · Statistics 2026-05-06 Shuoxun Xu , Xinzhou Guo
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