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Hypertrophic cardiomyopathy (HCM) requires accurate risk stratification to inform decisions regarding ICD therapy and follow-up management. Current established models, such as the European Society of Cardiology (ESC) score, exhibit moderate…

Desirability Of Outcome Ranking (DOOR) methodology accounts for problems that conventional benefit:risk analyses in clinical trials ignore, such as competing risks and the trade-off relationship between efficacy and toxicity. DOOR levels…

Methodology · Statistics 2025-05-19 Shiyu Shu , Guoqing Diao , Toshimitsu Hamasaki , Scott Evans

Experimentation is widely utilized for causal inference and data-driven decision-making across disciplines. In an A/B experiment, for example, an online business randomizes two different treatments (e.g., website designs) to their customers…

Methodology · Statistics 2025-01-15 Wenxuan Guo , JungHo Lee , Panos Toulis

Recent work has focused on nonparametric estimation of conditional treatment effects, but inference has remained relatively unexplored. We propose a class of nonparametric tests for both quantitative and qualitative treatment effect…

Methodology · Statistics 2026-04-07 Oliver Dukes , Mats J. Stensrud , Riccardo Brioschi , Aaron Hudson

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

The electronic health record (EHR) provides an unprecedented opportunity to build actionable tools to support physicians at the point of care. In this paper, we investigate survival analysis in the context of EHR data. We introduce deep…

Machine Learning · Statistics 2016-09-20 Rajesh Ranganath , Adler Perotte , Noémie Elhadad , David Blei

Background: Survival prediction models are often less reliable in clinical groups with limited sample sizes or few outcome events. Target-only models may be unstable, whereas models from larger cohorts may transfer poorly when risk-factor…

Methodology · Statistics 2026-05-18 Junhan Yu , Yurui Chen , Juan Delgado-SanMartin , Dennis Wang , Hong Pan , Doudou Zhou

Randomization is a common technique used in clinical trials to eliminate potential bias and confounders in a patient population. Equal allocation to treatment groups is the standard due to its optimal efficiency in many cases. However, in…

Applications · Statistics 2020-04-09 Thevaa Chandereng , Xiaodan Wei , Rick Chappell

There is strong interest in estimating how the magnitude of treatment effects of an intervention vary across sub-groups of the population of interest. In our paper, we propose a two-study approach to first propose and then test…

Methodology · Statistics 2020-06-23 Rahul Ladhania , Amelia Haviland , Neeraj Sood , Edward Kennedy , Ateev Mehrotra

Hazard ratios are ubiquitously used in time to event analysis to quantify treatment effects. Although hazard ratios are invaluable for hypothesis testing, other measures of association, both relative and absolute, may be used to fully…

Methodology · Statistics 2020-11-02 Federico Ambrogi , Simona Iacobelli , Per Kragh Andersen

Time-to-event outcomes are commonly used as primary endpoints in randomized clinical trials. Despite this, relatively little work incorporates baseline covariate information while also accounting for stratified randomization, a common form…

Methodology · Statistics 2026-05-01 Raphael C. Kim , Brian Gilbert , Ramin Zabih , Michele Santacatterina , Ivan Diaz

Globally, the incidence of heart failure is increasing, and its principal treatment involves drug therapy. However, widespread non-adherence to therapies is prevalent among heart failure patients and often results in worsening health…

Applications · Statistics 2023-12-29 Nicole Fontana , Laura Savaré , Francesca Ieva

We propose a novel personalized concept for the optimal treatment selection for a situation where the response is a multivariate vector, that could contain right-censored variables such as survival time. The proposed method can be applied…

Methodology · Statistics 2022-10-03 Chathura Siriwardhana , K. B. Kulasekera , Somnath Datta

In survival analysis, longitudinal information on the health status of a patient can be used to dynamically update the predicted probability that a patient will experience an event of interest. Traditional approaches to dynamic prediction…

Methodology · Statistics 2025-06-16 Mirko Signorelli

Variable selection for optimal treatment regime in a clinical trial or an observational study is getting more attention. Most existing variable selection techniques focused on selecting variables that are important for prediction, therefore…

Methodology · Statistics 2014-05-22 Ailin Fan , Wenbin Lu , Rui Song

When confronted with an undesired cell population, such as bacterial infections or tumors, we seek the most effective treatment, designed to eliminate the population as rapidly as possible. A common practice is to monitor the cells…

Biological Physics · Physics 2025-01-23 Uzi Harush , Ravid Straussman , Baruch Barzel

In 2023, the U.S. Food and Drug Administration issued guidance for adjustment of covariates in randomized clinical trials, emphasizing its role in enhancing precision and power through prognostic baseline variables. Despite its potential,…

Methodology · Statistics 2026-05-28 Kelly Van Lancker , Iván Díaz , Stijn Vansteelandt

For survival data with high-dimensional covariates, results generated in the analysis of a single dataset are often unsatisfactory because of the small sample size. Integrative analysis pools raw data from multiple independent studies with…

Methodology · Statistics 2015-01-13 Qingzhao Zhang , Sanguo Zhang , Jin Liu , Jian Huang , Shuangge Ma

This research was motivated by studying anti-drug antibody (ADA) formation and its potential impact on long-term benefit of a biologic treatment in a randomized controlled trial, in which ADA status was not only unobserved in the control…

Methodology · Statistics 2021-01-13 Shengchun Kong , Dominik Heinzmann , Sabine Lauer , Tian Lu

Multi-Regional Clinical Trials (MRCTs) play a central role in the development of new therapies by enabling the simultaneous evaluation of drug efficacy and safety across diverse global populations. Assessing the consistency of treatment…

Applications · Statistics 2026-02-10 Kunhai Qing , Xinru Ren , Jin Xu , Menggang Yu