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Personalized medicine, a paradigm of medicine tailored to a patient's characteristics, is an increasingly attractive field in health care. An important goal of personalized medicine is to identify a subgroup of patients, based on baseline…

机器学习 · 统计学 2023-01-31 Hengrui Cai , Wenbin Lu , Rachel Marceau West , Devan V. Mehrotra , Lingkang Huang

Given two possible treatments, there may exist subgroups who benefit greater from one treatment than the other. This problem is relevant to the field of marketing, where treatments may correspond to different ways of selling a product. It…

机器学习 · 统计学 2016-05-16 Derek Feng , Xiaofei Wang

A plethora of disparate statistical methods have been proposed for subgroup identification to help tailor treatment decisions for patients. However a majority of them do not have corresponding R packages and the few that do pertain to…

统计计算 · 统计学 2018-11-14 Jared D. Huling , Menggang Yu

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…

统计方法学 · 统计学 2014-10-09 Wei-Yin Loh , Xu He , Michael Man

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…

统计方法学 · 统计学 2018-06-22 Jon Arni Steingrimsson , Jiabei Yang

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…

统计方法学 · 统计学 2021-12-08 Jiabei Yang , Issa J. Dahabreh , Jon A. Steingrimsson

Precision medicine is an approach for disease treatment that defines treatment strategies based on the individual characteristics of the patients. Motivated by an open problem in cancer genomics, we develop a novel model that flexibly…

统计方法学 · 统计学 2023-09-04 Matteo Pedone , Raffaele Argiento , Francesco C. Stingo

Identifying patient subgroups with different treatment responses is an important task to inform medical recommendations, guidelines, and the design of future clinical trials. Existing approaches for treatment effect estimation primarily…

统计方法学 · 统计学 2025-12-10 Vincent Jeanselme , Chang Ho Yoon , Fabian Falck , Brian Tom , Jessica Barrett

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…

统计方法学 · 统计学 2020-01-22 Marius Thomas , Björn Bornkamp , Heidi Seibold

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…

机器学习 · 统计学 2026-02-05 Zhongming Xie , Joseph Giorgio , Jingshen Wang

Pharmaceutical companies continue to seek innovative ways to explore whether a drug under development is likely to be suitable for all or only an identifiable stratum of patients in the target population. The sooner this can be done during…

统计方法学 · 统计学 2019-12-10 Thomas O. Jemielita , Devan V. Mehrotra

We consider the problem of selecting the optimal subgroup to treat when data on covariates is available from a randomized trial or observational study. We distinguish between four different settings including (i) treatment selection when…

统计方法学 · 统计学 2018-02-28 Tyler J. VanderWeele , Alex R. Luedtke , Mark J. van der Laan , Ronald C. Kessler

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…

统计方法学 · 统计学 2018-07-11 Debashis Ghosh , Youngjoo Cho

Problem definition: Personalized medicine (PM) seeks the best treatment for each patient among a set of available treatment methods. Since a specific treatment does not work well on all patients, traditionally, the best treatment was…

最优化与控制 · 数学 2023-08-03 Jianzhong Du , Siyang Gao , Chun-Hung Chen

Precision oncology aims to prescribe the optimal cancer treatment to the right patients, maximizing therapeutic benefits. However, identifying patient subgroups that may benefit more from experimental cancer treatments based on randomized…

统计方法学 · 统计学 2026-01-06 Xingyu Li , Qing Liu , Tony Jiang , Hong Amy Xia , Peng Wei , Brian P. Hobbs

This paper extends my research applying statistical decision theory to treatment choice with sample data, using maximum regret to evaluate the performance of treatment rules. The specific new contribution is to study as-if optimization…

计量经济学 · 经济学 2021-10-05 Charles F. Manski

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…

计量经济学 · 经济学 2023-12-21 Jann Spiess , Vasilis Syrgkanis , Victor Yaneng Wang

Discovering subgroups with the maximum average treatment effect is crucial for targeted decision making in domains such as precision medicine, public policy, and education. While most prior work is formulated in the potential outcome…

机器学习 · 计算机科学 2025-11-26 Lincen Yang , Zhong Li , Matthijs van Leeuwen , Saber Salehkaleybar

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…

统计计算 · 统计学 2016-06-28 Marius Thomas , Björn Bornkamp

We propose a prognostic stratum matching framework that addresses the deficiencies of Randomized trial data subgroup analysis and transforms ObservAtional Data to be used as if they were randomized, thus paving the road for precision…

应用统计 · 统计学 2023-11-06 Dimitris Bertsimas , Angelos G. Koulouras , Georgios Antonios Margonis
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