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Adherence can be defined as "the extent to which patients take their medications as prescribed by their healthcare providers"[Osterberg and Blaschke, 2005]. World Health Organization's reports point out that, in developed countries, only…

机器学习 · 计算机科学 2018-11-30 Thomas Janssoone , Clémence Bic , Dorra Kanoun , Pierre Hornus , Pierre Rinder

The STEP 1 randomized trial evaluated the effect of taking semaglutide vs placebo on body weight over a 68 week duration. As with any study evaluating an intervention delivered over a sustained period, non-adherence was observed. This was…

An early phase clinical trial is the first step in evaluating the effects in humans of a potential new anti-disease agent or combination of agents. Usually called "phase I" or "phase I/II" trials, these experiments typically have the…

统计方法学 · 统计学 2010-12-01 Peter F. Thall

There has been significant attention given to developing data-driven methods for tailoring patient care based on individual patient characteristics. Dynamic treatment regimes formalize this through a sequence of decision rules that map…

统计方法学 · 统计学 2022-02-22 Eric J. Rose , Erica E. M. Moodie , Susan Shortreed

This study addresses the challenge of estimating average treatment effects (ATEs) for advertising campaigns in online marketplaces where complete randomized experimentation is infeasible. We propose two key innovations: (1) a shrinkage…

统计方法学 · 统计学 2026-04-01 Yen-Chun Liu , Alexander Volfovsky , German Schnaidt , Cristobal Garib , Eric Laber

From personalised medicine to targeted advertising, it is an inherent task to provide a sequence of decisions with historical covariates and outcome data. This requires understanding of both the dynamics and heterogeneity of treatment…

统计方法学 · 统计学 2022-06-22 Oscar Hernan Madrid Padilla , Yi Yu

Developing new drugs for target diseases is a time-consuming and expensive task, drug repurposing has become a popular topic in the drug development field. As much health claim data become available, many studies have been conducted on the…

机器学习 · 计算机科学 2023-02-22 Yaobin Ling , Pulakesh Upadhyaya , Luyao Chen , Xiaoqian Jiang , Yejin Kim

To effectively optimize and personalize treatments, it is necessary to investigate the heterogeneity of treatment effects. With the wide range of users being treated over many online controlled experiments, the typical approach of manually…

统计方法学 · 统计学 2022-11-07 John Cai , Weinan Wang

When evaluating a two-phase intervention, the cumulative average treatment effect (ATE) is often the primary causal estimand of interest. However, some individuals who do not respond well to the Phase I treatment may subsequently display…

统计方法学 · 统计学 2026-02-02 Guanglei Hong , Xu Qin , Zhengyan Xu , Fan Yang

Benchmark dose analysis aims to estimate the level of exposure to a toxin that results in a clinically-significant adverse outcome and quantifies uncertainty using the lower limit of a confidence interval for this level. We develop a novel…

This paper studies treatment effect models in which individuals are classified into unobserved groups based on heterogeneous treatment rules. Using a finite mixture approach, we propose a marginal treatment effect (MTE) framework in which…

计量经济学 · 经济学 2022-05-24 Tadao Hoshino , Takahide Yanagi

Propensity score weighting approaches have been widely implemented in clinical research to estimate the effects of a treatment or exposure while mitigating the risk of confounding in the absence of random assignment. In practice, when…

统计方法学 · 统计学 2026-04-17 Emma K. Mackay , Amol A. Verma , Fahad Razak , Surain B. Roberts

Response-adaptive randomization (RAR) has been studied extensively in conventional, single-stage clinical trials, where it has been shown to yield ethical and statistical benefits, especially in trials with many treatment arms. However, RAR…

统计方法学 · 统计学 2024-01-09 Peter Norwood , Marie Davidian , Eric Laber

Numerous algorithms have been developed for Conditional Average Treatment Effect (CATE) estimation. In this paper, we first highlight a common issue where many algorithms exhibit inconsistent learning behavior for the same instance across…

机器学习 · 计算机科学 2025-07-08 Yi-Fu Fu , Keng-Te Liao , Shou-De Lin

Aims: Combinations of treatments can offer additional benefit over the treatments individually. However, trials of these combinations are lower priority than the development of novel therapies, which can restrict funding, timelines and…

Estimating the Conditional Average Treatment Effect (CATE) is often constrained by the high cost of obtaining outcome measurements, making active learning essential. However, conventional active learning strategies suffer from a fundamental…

机器学习 · 统计学 2025-09-29 Erdun Gao , Jake Fawkes , Dino Sejdinovic

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

Estimation and inference for the Average Treatment Effect (ATE) is a cornerstone of causal inference and often serves as the foundation for developing procedures for more complicated settings. Although traditionally analyzed in a batch…

机器学习 · 统计学 2025-02-10 Ojash Neopane , Aaditya Ramdas , Aarti Singh

Electroencephalography (EEG) foundation models have shown strong potential for learning generalizable representations from large-scale neural data, yet their clinical deployment is hindered by distribution shifts across clinical settings,…

机器学习 · 计算机科学 2026-04-21 Gabriel Jason Lee , Jathurshan Pradeepkumar , Jimeng Sun

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