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In the realm of precision medicine, effective patient stratification and disease subtyping demand innovative methodologies tailored for multi-omics data. Clustering techniques applied to multi-omics data have become instrumental in…

Machine Learning · Computer Science 2024-01-30 Bastian Pfeifer , Christel Sirocchi , Marcus D. Bloice , Markus Kreuzthaler , Martin Urschler

Treatment effect estimation is a fundamental problem in causal inference. We focus on designing efficient randomized controlled trials, to accurately estimate the effect of some treatment on a population of $n$ individuals. In particular,…

Machine Learning · Computer Science 2022-10-14 Raghavendra Addanki , David Arbour , Tung Mai , Cameron Musco , Anup Rao

Individualized decision rules (IDRs) have become increasingly prevalent in societal applications such as personalized marketing, healthcare, and public policy design. However, a critical ethical concern arises from the potential…

Machine Learning · Statistics 2026-03-06 Wenhai Cui , Wen Su , Donglin Zeng , Xingqiu Zhao

A treatment regime is a rule that assigns a treatment to patients based on their covariate information. Recently, estimation of the optimal treatment regime that yields the greatest overall expected clinical outcome of interest has…

Methodology · Statistics 2022-03-07 Kevin Gunn , Wenbin Lu , Rui Song

We propose new, optimal methods for analyzing randomized trials, when it is suspected that treatment effects may differ in two predefined subpopulations. Such sub-populations could be defined by a biomarker or risk factor measured at…

Methodology · Statistics 2016-11-26 Michael Rosenblum , Han Liu , and En-Hsu Yen

Individualized treatment rules tailor treatments to patients based on clinical, demographic, and other characteristics. Estimation of individualized treatment rules requires the identification of individuals who benefit most from the…

Methodology · Statistics 2024-06-06 Junwei Shen , Erica E. M. Moodie , Shirin Golchi

Individualized treatment rules (ITRs) are considered a promising recipe to deliver better policy interventions. One key ingredient in optimal ITR estimation problems is to estimate the average treatment effect conditional on a subject's…

Methodology · Statistics 2021-03-16 Hongming Pu , Bo Zhang

In biomedical Subgroup Discovery, practitioners are interested in discovering interpretable and homogeneous subgroups within a group of patients. In this paper, assuming that healthy subjects (i.e., controls) share common but irrelevant…

Machine Learning · Computer Science 2026-05-21 Robin Louiset , Edouard Duchesnay , Benoit Dufumier , Antoine Grigis , Pietro Gori

Existing strategies for determining the optimal treatment or monitoring strategy typically assume unlimited access to resources. However, when a health system has resource constraints, such as limited funds, access to medication, or…

Applications · Statistics 2019-03-18 Ellen C Caniglia , Eleanor J Murray , Miguel A Hernan , Zach Shahn

Identifying relationships between molecular variations and their clinical presentations has been challenged by the heterogeneous causes of a disease. It is imperative to unveil the relationship between the high dimensional molecular…

Methodology · Statistics 2021-09-02 Wennan Chang , Changlin Wan , Yong Zang , Chi Zhang , Sha Cao

Estimation and evaluation of individualized treatment rules have been studied extensively, but real-world treatment resource constraints have received limited attention in existing methods. We investigate a setting in which treatment is…

Methodology · Statistics 2022-11-24 Hongxiang Qiu , Marco Carone , Alex Luedtke

Optimal treatment rules are mappings from individual patient characteristics to tailored treatment assignments that maximize mean outcomes. In this work, we introduce a conditional potential benefit (CPB) metric that measures the expected…

Methodology · Statistics 2024-08-29 Alexander W. Levis , Eli Ben-Michael , Edward H. Kennedy

Individualized treatment rules (ITR) can improve health outcomes by recognizing that patients may respond differently to treatment and assigning therapy with the most desirable predicted outcome for each individual. Flexible and efficient…

Methodology · Statistics 2017-09-25 Brent R. Logan , Rodney Sparapani , Robert E. McCulloch , Purushottam W. Laud

An optimal dynamic treatment regime (DTR) consists of a sequence of decision rules in maximizing long-term benefits, which is applicable for chronic diseases such as HIV infection or cancer. In this paper, we develop a novel angle-based…

Methodology · Statistics 2020-01-15 Fei Xue , Yanqing Zhang , Wenzhuo Zhou , Haoda Fu , Annie Qu

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

Researchers often run resource-intensive randomized controlled trials (RCTs) to estimate the causal effects of interventions on outcomes of interest. Yet these outcomes are often noisy, and estimated overall effects can be small or…

Econometrics · Economics 2023-12-21 Jann Spiess , Vasilis Syrgkanis , Victor Yaneng Wang

An important objective in the development of targeted therapies is to identify the populations where the treatment under consideration has positive benefit risk balance. We consider pivotal clinical trials, where the efficacy of a treatment…

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

As the COVID-19 pandemic progresses, researchers are reporting findings of randomized trials comparing standard care with care augmented by experimental drugs. The trials have small sample sizes, so estimates of treatment effects are…

Econometrics · Economics 2020-06-02 Charles F. Manski , Aleksey Tetenov

We study the design of multi-armed parallel group clinical trials to estimate personalized treatment rules that identify the best treatment for a given patient with given covariates. Assuming that the outcomes in each treatment arm are…

Statistics Theory · Mathematics 2022-07-13 David Azriel , Yosef Rinott , Martin Posch