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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

Controlled experiments are widely used in many applications to investigate the causal relationship between input factors and experimental outcomes. A completely randomized design is usually used to randomly assign treatment levels to…

统计方法学 · 统计学 2026-05-12 Yiou Li , Lulu Kang , Xiao Huang

Background: The development of classification methods for personalized medicine is highly dependent on the identification of predictive genetic markers. In survival analysis it is often necessary to discriminate between influential and…

统计方法学 · 统计学 2018-02-27 Thomas Welchowski , Verena Zuber , Matthias Schmid

Randomization, as a key technique in clinical trials, can eliminate sources of bias and produce comparable treatment groups. In randomized experiments, the treatment effect is a parameter of general interest. Researchers have explored the…

统计方法学 · 统计学 2023-12-05 Fuyi Tu , Wei Ma , Hanzhong Liu

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

统计方法学 · 统计学 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

Linear regression is arguably the most fundamental statistical model; however, the validity of its use in randomized clinical trials, despite being common practice, has never been crystal clear, particularly when stratified or…

统计方法学 · 统计学 2023-02-14 Wei Ma , Fuyi Tu , Hanzhong Liu

Sequential multiple assignment randomized trials mimic the actual treatment processes experienced by physicians and patients in clinical settings and inform the comparative effectiveness of dynamic treatment regimes. In such trials,…

统计方法学 · 统计学 2025-04-07 Zi Wang , Yu Cheng , Abdus S. Wahed

Covariate-adaptive randomization is widely employed to balance baseline covariates in interventional studies such as clinical trials and experiments in development economics. Recent years have witnessed substantial progress in inference…

统计方法学 · 统计学 2024-05-30 Jiahui Xin , Hanzhong Liu , Wei Ma

A sequential multiple assignment randomized trial (SMART) facilitates comparison of multiple adaptive treatment strategies (ATSs) simultaneously. Previous studies have established a framework to test the homogeneity of multiple ATSs by a…

统计方法学 · 统计学 2022-11-04 Liwen Wu , Junyao Wang , Abdus S. Wahed

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

We propose a framework for testing the homogeneity of conditional average treatment effects (CATEs) across multiple experimental and observational studies. Our approach leverages multiple randomized trials to assess whether treatment…

计量经济学 · 经济学 2026-02-25 Ana Armendariz , Martin Huber

To increase statistical efficiency in a randomized experiment, researchers often use stratification (i.e., blocking) in the design stage. However, conventional practices of stratification fail to exploit valuable information about the…

统计方法学 · 统计学 2025-10-28 Zikai Li

The identification of patient subgroups with comparable event-risk dynamics plays a key role in supporting informed decision-making in clinical research. In such settings, it is important to account for the inherent dependence that arises…

统计计算 · 统计学 2026-01-13 Alessandra Ragni , Lara Cavinato , Francesca Ieva

Randomized clinical trials (RCTs) are widely considered the gold standard for evaluating the effectiveness of new treatments or interventions in drug development. Still, they may not be feasible in certain cases, such as with rare diseases…

统计方法学 · 统计学 2025-08-05 Di Ran , Fanni Zhang , Sima Shahsavari , Kristine Broglio , Alasdair Henderson , Binbing Yu

We introduce a statistical procedure that integrates survival data from multiple biomedical studies, to improve the accuracy of predictions of survival or other events, based on individual clinical and genomic profiles, compared to models…

应用统计 · 统计学 2020-07-20 Steffen Ventz , Rahul Mazumder , Lorenzo Trippa

Frailty models are often the model of choice for heterogeneous survival data. A frailty model contains both random effects and fixed effects, with the random effects accommodating for the correlation in the data. Different estimation…

统计方法学 · 统计学 2019-09-17 Oodally Ajmal , Luc Duchateau , Estelle Kuhn

We propose a test-based elastic integrative analysis of the randomized trial and real-world data to estimate treatment effect heterogeneity with a vector of known effect modifiers. When the real-world data are not subject to bias, our…

统计方法学 · 统计学 2022-11-30 Shu Yang , Chenyin Gao , Donglin Zeng , Xiaofei Wang

Principal stratification is a causal framework to analyze randomized experiments with a post-treatment variable between the treatment and endpoint variables. Because the principal strata defined by the potential outcomes of the…

统计理论 · 数学 2015-07-22 Zhichao Jiang , Peng Ding , Zhi Geng

In heterogeneous cohorts and those where censoring by non-primary risks is informative many conventional survival analysis methods are not applicable; the proportional hazards assumption is usually violated at population level and the…

Stabilized dynamic treatment regimes are sequential decision rules for individual patients that not only adaptive throughout the disease progression but also remain consistent over time in format. The estimation of stabilized dynamic…

统计方法学 · 统计学 2019-03-05 Ying-Qi Zhao , Ruoqing Zhu , Guanhua Chen , Yingye Zheng