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A platform trial with a master protocol provides an infrastructure to ethically and efficiently evaluate multiple treatment options in multiple diseases. Given that certain study drugs can enter or exit a platform trial, the randomization…

统计方法学 · 统计学 2025-07-15 Tianyu Zhan , Jane Zhang , Lei Shu , Yihua Gu

Evaluating the treatment effects has become an important topic for many applications. However, most existing literature focuses mainly on the average treatment effects. When the individual effects are heavy-tailed or have outlier values,…

统计方法学 · 统计学 2023-05-11 Yongchang Su , Xinran Li

Estimating individualized treatment rules (ITRs) is fundamental to precision medicine, where the goal is to tailor treatment decisions to individual patient characteristics. While numerous methods have been developed for ITR estimation,…

统计方法学 · 统计学 2026-05-15 Eun Jeong Oh , Min Qian

A treatment benefit predictor (TBP) is a function that maps patient characteristics to an estimate of the treatment benefit for that patient. Such predictors support optimizing individualized treatment decisions, which are central to…

统计方法学 · 统计学 2025-09-30 Yuan Xia , Mohsen Sadatsafavi , Paul Gustafson

Consider a setup in which a decision maker is informed about the population by a finite sample and based on that sample has to decide whether or not to apply a certain treatment. We work out finite sample minimax regret treatment rules…

计量经济学 · 经济学 2026-01-08 Patrik Guggenberger , Nihal Mehta , Nikita Pavlov

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

The aim of clinical effectiveness research using repositories of electronic health records is to identify what health interventions 'work best' in real-world settings. Since there are several reasons why the net benefit of intervention may…

统计方法学 · 统计学 2020-06-19 Jie Zhu , Blanca Gallego

In observational studies, balancing covariates in different treatment groups is essential to estimate treatment effects. One of the most commonly used methods for such purposes is weighting. The performance of this class of methods usually…

统计方法学 · 统计学 2021-07-07 Ruoqi Yu , Shulei Wang

This paper provides a solution to the evaluation of treatment effects in selective samples when neither instruments nor parametric assumptions are available. We provide sharp bounds for average treatment effects under a conditional…

计量经济学 · 经济学 2024-12-17 Phillip Heiler , Asbjørn Kaufmann , Bezirgen Veliyev

The empirical risk minimization approach to data-driven decision making requires access to training data drawn under the same conditions as those that will be faced when the decision rule is deployed. However, in a number of settings, we…

统计方法学 · 统计学 2025-09-17 Roshni Sahoo , Lihua Lei , Stefan Wager

There is a fast-growing literature on estimating optimal treatment rules directly by maximizing the expected outcome. In biomedical studies and operations applications, censored survival outcome is frequently observed, in which case the…

统计方法学 · 统计学 2026-03-12 Yifan Cui , Junyi Liu , Tao Shen , Zhengling Qi , Xi Chen

Personalized medicine has received increasing attention among statisticians, computer scientists, and clinical practitioners. A major component of personalized medicine is the estimation of individualized treatment rules (ITRs). Recently,…

统计方法学 · 统计学 2015-08-14 Xin Zhou , Nicole Mayer-Hamblett , Umer Khan , Michael R. Kosorok

The beneficial effects of treatments vary across individuals in most studies. Treatment heterogeneity motivates practitioners to search for the optimal policy based on personal characteristics. A long-standing common practice in policy…

统计理论 · 数学 2025-01-06 Xuqiao Li , Ying Yan

The individualized treatment rule (ITR), which recommends an optimal treatment based on individual characteristics, has drawn considerable interest from many areas such as precision medicine, personalized education, and personalized…

统计方法学 · 统计学 2023-09-28 Qi Xu , Haoda Fu , Annie Qu

Policy learning can be used to extract individualized treatment regimes from observational data in healthcare, civics, e-commerce, and beyond. One big hurdle to policy learning is a commonplace lack of overlap in the data for different…

机器学习 · 统计学 2020-12-04 Nathan Kallus

Although there is now a large literature on policy evaluation and learning, much of the prior work assumes that the treatment assignment of one unit does not affect the outcome of another unit. Unfortunately, ignoring interference can lead…

统计方法学 · 统计学 2025-04-02 Yi Zhang , Kosuke Imai

Health policy decisions regarding patient treatment strategies require consideration of both treatment effectiveness and cost. Optimizing treatment rules with respect to effectiveness may result in prohibitively expensive strategies; on the…

统计方法学 · 统计学 2021-10-20 Nicholas Illenberger , Andrew J. Spieker , Nandita Mitra

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…

统计方法学 · 统计学 2024-06-06 Junwei Shen , Erica E. M. Moodie , Shirin Golchi

Machine learning-based decision support systems are increasingly deployed in clinical settings, where probabilistic scoring functions are used to inform and prioritize patient management decisions. However, widely used scoring rules, such…

机器学习 · 计算机科学 2025-07-01 Gerardo A. Flores , Alyssa H. Smith , Julia A. Fukuyama , Ashia C. Wilson

There is a fast-growing literature on estimating optimal treatment regimes based on randomized trials or observational studies under a key identifying condition of no unmeasured confounding. Because confounding by unmeasured factors cannot…

统计方法学 · 统计学 2020-08-12 Yifan Cui , Eric Tchetgen Tchetgen