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In the fight against hard-to-treat diseases such as cancer, it is often difficult to discover new treatments that benefit all subjects. For regulatory agency approval, it is more practical to identify subgroups of subjects for whom the…

Methodology · Statistics 2014-10-09 Wei-Yin Loh , Xu He , Michael Man

An important task in drug development is to identify patients, which respond better or worse to an experimental treatment. Identifying predictive covariates, which influence the treatment effect and can be used to define subgroups of…

Methodology · Statistics 2018-11-27 Marius Thomas , Björn Bornkamp , Katja Ickstadt

While studying response trajectory, often the population of interest may be diverse enough to exist distinct subgroups within it and the longitudinal change in response may not be uniform in these subgroups. That is, the timeslope and/or…

Methodology · Statistics 2020-06-30 Madan Gopal Kundu , Jaroslaw Harezlak

Cluster or group randomized trials (CRTs) are increasingly used for both behavioral and system-level interventions, where entire clusters are randomly assigned to a study condition or intervention. Apart from the assigned cluster-level…

Methodology · Statistics 2024-11-19 Shubhadeep Chakraborty , Bo Wang , Ram Tiwari , Samiran Ghosh

Precision medicine is an emerging field that takes into account individual heterogeneity to inform better clinical practice. In clinical trials, the evaluation of treatment effect heterogeneity is an important component, and recently, many…

Methodology · Statistics 2023-02-24 Yuejia Xu , Angela M. Wood , Brian D. M. Tom

Understanding effect modification -- how treatment effects vary across subpopulations -- is practically important in observational studies, as it helps identify which subgroups are likely to benefit from a given treatment. In this paper, we…

Methodology · Statistics 2026-05-12 Yu Gui , Dylan S Small , Zhimei Ren

Medical studies frequently require to extract the relationship between each covariate and the outcome with statistical confidence measures. To do this, simple parametric models are frequently used (e.g. coefficients of linear regression)…

Machine Learning · Computer Science 2023-05-02 Zachary Izzo , Ruishan Liu , James Zou

Some patients benefit from a treatment while others may do so less or do not benefit at all. We have previously developed a two-stage network meta-regression prediction model that synthesized randomized trials and evaluates how treatment…

In this study, we address the interpretability issue in complex, black-box Machine Learning models applied to sequence data. We introduce the Model-Based tree Hidden Semi-Markov Model (MOB-HSMM), an inherently interpretable model aimed at…

Machine Learning · Computer Science 2023-10-31 Chan Hsu , Wei-Chun Huang , Jun-Ting Wu , Chih-Yuan Li , Yihuang Kang

Physical activity is crucial for human health. With the increasing availability of large-scale mobile health data, strong associations have been found between physical activity and various diseases. However, accurately capturing this…

Methodology · Statistics 2026-01-19 Xiaojing Sun , Bingxin Zhao , Fei Xue

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

Methodology · Statistics 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Causal discovery uncovers complex relationships between variables, enhancing predictions, decision-making, and insights into real-world systems, especially in nonlinear multivariate time series. However, most existing methods primarily…

Machine Learning · Computer Science 2025-10-30 Wasim Ahmad , Joachim Denzler , Maha Shadaydeh

In evidence synthesis, multilevel modeling approaches (MMAs) are commonly employed to combine aggregate data (AD) and individual participant data (IPD). These approaches rely on an aggregate outcome model that is ideally obtained by…

Methodology · Statistics 2026-02-18 Tran Trong Khoi Le , Marie-Felicia Béclin , Sivem Afach , Tat-Thang Vo

Individualized treatment decisions can improve health outcomes, but using data to make these decisions in a reliable, precise, and generalizable way is challenging with a single dataset. Leveraging multiple randomized controlled trials…

Model-based trees are used to find subgroups in data which differ with respect to model parameters. In some applications it is natural to keep some parameters fixed globally for all observations while asking if and how other parameters vary…

Computation · Statistics 2025-10-07 Heidi Seibold , Torsten Hothorn , Achim Zeileis

Subgroup analysis is a frequently used tool for evaluating heterogeneity of treatment effect and heterogeneity in treatment harm across observed baseline patient characteristics. While treatment efficacy and adverse event measures are often…

Applications · Statistics 2018-08-14 Nicholas C. Henderson , Ravi Varadhan

A stepped wedge design is a unidirectional crossover design where clusters are randomized to distinct treatment sequences. While model-based analysis of stepped wedge designs is standard practice to evaluate treatment effects accounting for…

Methodology · Statistics 2024-09-13 Bingkai Wang , Xueqi Wang , Fan Li

Do expert-defined or diagnostically-labeled data groups align with clusters inferred through statistical modeling? If not, where do discrepancies between predefined labels and model-based groupings occur and why? In this work, we introduce…

Methodology · Statistics 2026-03-18 Patricia Puchhammer , Ines Wilms , Peter Filzmoser

Estimation of heterogeneous treatment effects is an active area of research. Most of the existing methods, however, focus on estimating the conditional average treatment effects of a single, binary treatment given a set of pre-treatment…

Methodology · Statistics 2025-05-30 Max Goplerud , Kosuke Imai , Nicole E. Pashley

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