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Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

统计方法学 · 统计学 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

In multicenter randomized trials, when effect modifiers have a different distribution across centers, comparisons between treatment groups that average over centers may not apply to any of the populations underlying the individual centers.…

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

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

统计计算 · 统计学 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

Heterogeneous treatment effects are of major interest in economics. For example, a poverty reduction measure would be best evaluated by its effects on those who would be poor in the absence of the treatment, or by the share among the poor…

计量经济学 · 经济学 2025-08-07 Tetsuya Kaji , Jianfei Cao

A fundamental principle of clinical medicine is that a treatment should only be administered to those patients who would benefit from it. Treatment strategies that assign treatment to patients as a function of their individual…

应用统计 · 统计学 2025-06-13 Nicholas Williams , Kara Rudolph , Iván Díaz

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…

统计理论 · 数学 2022-07-13 David Azriel , Yosef Rinott , Martin Posch

Many scientific and engineering challenges -- ranging from personalized medicine to customized marketing recommendations -- require an understanding of treatment effect heterogeneity. In this paper, we develop a non-parametric causal forest…

统计方法学 · 统计学 2017-07-11 Stefan Wager , Susan Athey

Identification of treatment effects in the presence of unmeasured confounding is a persistent problem in the social, biological, and medical sciences. The problem of unmeasured confounding in settings with multiple treatments is most common…

统计方法学 · 统计学 2022-07-12 Wang Miao , Wenjie Hu , Elizabeth L. Ogburn , Xiaohua Zhou

The heterogeneity of treatment effect (HTE) lies at the heart of precision medicine. Randomized controlled trials are gold-standard for treatment effect estimation but are typically underpowered for heterogeneous effects. In contrast, large…

统计方法学 · 统计学 2024-11-14 Shu Yang , Siyi Liu , Donglin Zeng , Xiaofei Wang

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…

统计方法学 · 统计学 2016-11-26 Michael Rosenblum , Han Liu , and En-Hsu Yen

Heterogeneous treatment effect models allow us to compare treatments at subgroup and individual levels, and are of increasing popularity in applications like personalized medicine, advertising, and education. In this talk, we first survey…

统计方法学 · 统计学 2022-01-28 Zijun Gao , Trevor Hastie

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

统计方法学 · 统计学 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

When considering the effect a treatment will cause in a population of interest, we often look to evidence from randomized controlled trials. In settings where multiple trials on a treatment are available, we may wish to synthesize the…

统计方法学 · 统计学 2023-09-08 Nicole Schnitzler , Eloise Kaizar

We are interested in estimating the effect of a treatment applied to individuals at multiple sites, where data is stored locally for each site. Due to privacy constraints, individual-level data cannot be shared across sites; the sites may…

机器学习 · 计算机科学 2023-04-04 Ruoxuan Xiong , Allison Koenecke , Michael Powell , Zhu Shen , Joshua T. Vogelstein , Susan Athey

We combine two recently proposed nonparametric difference-in-differences methods, extending them to enable the examination of treatment effect heterogeneity in the staggered adoption setting using machine learning. The proposed method,…

计量经济学 · 经济学 2023-10-19 Julia Hatamyar , Noemi Kreif , Rudi Rocha , Martin Huber

Heterogeneous treatment effects (HTEs) are commonly identified during randomized controlled trials (RCTs). Identifying subgroups of patients with similar treatment effects is of high interest in clinical research to advance precision…

机器学习 · 计算机科学 2022-12-06 Peniel N. Argaw , Elizabeth Healey , Isaac S. Kohane

Multistate process data are common in studies of chronic diseases such as cancer. These data are ideal for precision medicine purposes as they can be leveraged to improve more refined health outcomes, compared to standard survival outcomes,…

统计方法学 · 统计学 2022-11-28 Giorgos Bakoyannis

As clinical decision-making increasingly moves toward individualized and context-specific treatment recommendations, reliance on any single evidence source, randomized or observational, may be insufficient. Principled integration of…

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…

统计方法学 · 统计学 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa