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Cluster analysis methods are used to identify homogeneous subgroups in a data set. In biomedical applications, one frequently applies cluster analysis in order to identify biologically interesting subgroups. In particular, one may wish to…

Methodology · Statistics 2016-09-23 Sheila Gaynor , Eric Bair

With medical tests becoming increasingly available, concerns about over-testing and over-treatment dramatically increase. Hence, it is important to understand the influence of testing on treatment selection in general practice. Most…

Methodology · Statistics 2020-08-11 Yun Li , Irina Bondarenko , Michael R. Elliott , Timothy P. Hofer , Jeremy M. G. Taylor

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

Personalized medicine aims at identifying best treatments for a patient with given characteristics. It has been shown in the literature that these methods can lead to great improvements in medicine compared to traditional methods…

Machine Learning · Statistics 2018-11-26 Oleg Sysoev , Krzysztof Bartoszek , Eva-Charlotte Ekstrom , Katarina Ekholm Selling

Biomarker subpopulations have become increasingly important for drug development in targeted therapies. The use of biomarkers has the potential to facilitate more effective outcomes by guiding patient selection appropriately, thus enhancing…

Methodology · Statistics 2020-08-07 Ting-Yu Chen , Jing Zhao , Linda Sun , Keaven Anderson

In high-stakes settings where machine learning models are used to automate decision-making about individuals, the presence of algorithmic bias can exacerbate systemic harm to certain subgroups of people. These biases often stem from the…

Machine Learning · Computer Science 2026-04-07 Erin Tan , Judy Hanwen Shen , Irene Y. Chen

Randomized Controlled Trials (RCTs) are the gold standard for comparing the effectiveness of a new treatment to the current one (the control). Most RCTs allocate the patients to the treatment group and the control group by uniform…

Machine Learning · Statistics 2018-10-22 Onur Atan , William R. Zame , Mihaela van der Schaar

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

In regression and causal inference, controlled subgroup selection aims to identify, with inferential guarantees, a subgroup (defined as a subset of the covariate space) on which the average response or treatment effect is above a given…

Methodology · Statistics 2025-10-31 Nathan Cheng , Asher Spector , Lucas Janson

Distribution shifts remain a fundamental problem for the safe application of machine learning systems. If undetected, they may impact the real-world performance of such systems or will at least render original performance claims invalid. In…

Machine Learning · Computer Science 2023-03-10 Lisa M. Koch , Christian M. Schürch , Christian F. Baumgartner , Arthur Gretton , Philipp Berens

Finding patient subgroups with similar characteristics is crucial for personalized decision-making in various disciplines such as healthcare and policy evaluation. While most existing approaches rely on unsupervised clustering methods,…

Machine Learning · Statistics 2026-03-06 Luwei Wang , Nazir Lone , Sohan Seth

Investigators often use multi-source data (e.g., multi-center trials, meta-analyses of randomized trials, pooled analyses of observational cohorts) to learn about the effects of interventions in subgroups of some well-defined target…

Methodology · Statistics 2024-02-06 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

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

In many clinical trials, individuals in different subgroups have experience differential treatment effects. This leads to individualized differences in treatment benefit. In this article, we introduce the general concept of predictive…

Methodology · Statistics 2018-07-11 Debashis Ghosh , Youngjoo Cho

Deriving insights from high-dimensional data is one of the core problems in data mining. The difficulty mainly stems from the fact that there are exponentially many variable combinations to potentially consider, and there are infinitely…

Machine Learning · Statistics 2021-11-08 Jefrey Lijffijt , Bo Kang , Wouter Duivesteijn , Kai Puolamäki , Emilia Oikarinen , Tijl De Bie

Out of the participants in a randomized experiment with anticipated heterogeneous treatment effects, is it possible to identify which subjects have a positive treatment effect? While subgroup analysis has received attention, claims about…

Methodology · Statistics 2024-05-14 Boyan Duan , Larry Wasserman , Aaditya Ramdas

For testing the statistical significance of a treatment effect, we usually compare between two parts of a population, one is exposed to the treatment, and the other is not exposed to it. Standard parametric and nonparametric two-sample…

Computation · Statistics 2012-11-02 Bikram Karmakar , Kumaresh Dhara , Kushal Kumar Dey , Analabha Basu , Anil Ghosh

If the assumed model does not accurately capture the underlying structure of the data, a statistical method is likely to yield sub-optimal results, and so model selection is crucial in order to conduct any statistical analysis. However, in…

Methodology · Statistics 2023-06-21 Vasilis Chasiotis , Dimitris Karlis

Machine learning approaches have been effective in predicting adverse outcomes in different clinical settings. These models are often developed and evaluated on datasets with heterogeneous patient populations. However, good predictive…

Machine Learning · Computer Science 2018-06-11 Harini Suresh , Jen J. Gong , John Guttag

We present the motivation, experience and learnings from a data challenge conducted at a large pharmaceutical corporation on the topic of subgroup identification. The data challenge aimed at exploring approaches to subgroup identification…