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One of the main goals of sequential, multiple assignment, randomized trials (SMART) is to find the most efficacious design embedded dynamic treatment regimes. The analysis method known as multiple comparisons with the best (MCB) allows…

统计方法学 · 统计学 2020-08-07 William J. Artman , Ashkan Ertefaie , Kevin G. Lynch , James R. McKay

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

应用统计 · 统计学 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li

While machine learning algorithms hold promise for personalised medicine, their clinical adoption remains limited, partly due to biases that can compromise the reliability of predictions. In this paper, we focus on sample selection bias…

Estimating nonlinear functionals of probability distributions from samples is a fundamental statistical problem. The "plug-in" estimator obtained by applying the target functional to the empirical distribution of samples is biased.…

统计理论 · 数学 2026-02-20 Florian Schäfer

In the field of operational modal analysis (OMA), obtained modal information is frequently used to assess the current state of aerospace, mechanical, offshore and civil structures. However, the stochasticity of operational systems and the…

机器学习 · 统计学 2025-07-04 Brandon J. O'Connell , Max D. Champneys , Timothy J. Rogers

Background Most methods of adjusting for multiplicity focus primarily on controlling type I errors and rarely consider type II errors. We propose a new method that considers controlling for false-positive findings while ensuring sufficient…

应用统计 · 统计学 2025-07-31 Jiale Li , Zimu Wei

Machine Learning (ML) algorithms are vital for supporting clinical decision-making in biomedical informatics. However, their predictive performance can vary across demographic groups, often due to the underrepresentation of historically…

机器学习 · 计算机科学 2025-03-04 Ioannis Bilionis , Ricardo C. Berrios , Luis Fernandez-Luque , Carlos Castillo

The win ratio (WR) statistic is increasingly used to evaluate treatment effects based on prioritized composite endpoints, yet existing Bayesian adaptive designs are not directly applicable because the WR is a summary statistic derived from…

统计方法学 · 统计学 2026-02-20 Di Zhu , Yong Zang

In epidemiological research, causal models incorporating potential mediators along a pathway are crucial for understanding how exposures influence health outcomes. This work is motivated by integrated epidemiological and blood biomarker…

统计方法学 · 统计学 2024-11-28 Youngho Bae , Chanmin Kim , Fenglei Wang , Qi Sun , Kyu Ha Lee

A typical random effects meta-analysis of odds-ratios assumes binomially distributed numbers of events in a treatment and control group and requires the proportion of deaths to be extracted from published papers. This data is often not…

统计方法学 · 统计学 2014-10-13 Shemra Rizzo , Robert E. Weiss

Missing data is a pervasive problem in epidemiology, with multiple imputation (MI) a commonly used analysis method. MI is valid when data are missing at random (MAR). However, definitions of MAR with multiple incomplete variables are not…

统计方法学 · 统计学 2025-04-14 Paul Madley-Dowd , Rachael A. Hughes , Maya B. Mathur , Jon Heron , Kate Tilling

Observational studies are frequently used to estimate the effect of an exposure or treatment on an outcome. To obtain an unbiased estimate of the treatment effect, it is crucial to measure the exposure accurately. A common type of exposure…

统计方法学 · 统计学 2024-07-02 Suhwan Bong , Kwonsang Lee , Francesca Dominici

Meta-analysis is a powerful tool for assessing drug safety by combining treatment-related toxicological findings across multiple studies, as clinical trials are typically underpowered for detecting adverse drug effects. However, incomplete…

统计方法学 · 统计学 2024-02-12 Xinyue Qi , Shouhao Zhou , Christine B. Peterson , Yucai Wang , Xinying Fang , Michael L. Wang , Chan Shen

Randomized clinical trials are considered the gold standard for informing treatment guidelines, but results may not generalize to real-world populations. Generalizability is hindered by distributional differences in baseline covariates and…

统计方法学 · 统计学 2025-06-03 Rachael K. Ross , Ivan Diaz , Amy J. Pitts , Elizabeth A. Stuart , Kara E. Rudolph

For many cancer sites low-dose risks are not known and must be extrapolated from those observed in groups exposed at much higher levels of dose. Measurement error can substantially alter the dose-response shape and hence the extrapolated…

定量方法 · 定量生物学 2024-03-15 Mark P Little , Nobuyuki Hamada , Lydia B Zablotska

Researchers are often faced with evaluating the effect of a policy or program that was simultaneously initiated across an entire population of units at a single point in time, and its effects over the targeted population can manifest at any…

Instrumental variables are a popular tool to infer causal effects under unobserved confounding, but choosing suitable instruments is challenging in practice. We propose gIVBMA, a Bayesian model averaging procedure that addresses this…

统计方法学 · 统计学 2026-03-02 Gregor Steiner , Mark Steel

Background: Pairwise and network meta-analyses using fixed effect and random effects models are commonly applied to synthesise evidence from randomised controlled trials. The models differ in their assumptions and the interpretation of the…

统计方法学 · 统计学 2017-08-04 Shijie Ren , Jeremy E. Oakley , John W. Stevens

Hybrid controlled trials (HCTs), which augment randomized controlled trials (RCTs) with external controls (ECs), are increasingly receiving attention as a way to address limited power, slow accrual, and ethical concerns in clinical…

统计方法学 · 统计学 2025-05-02 Jiajun Liu , Ke Zhu , Shu Yang , Xiaofei Wang

We consider the problem of variance reduction in randomized controlled trials, through the use of covariates correlated with the outcome but independent of the treatment. We propose a machine learning regression-adjusted treatment effect…

机器学习 · 统计学 2022-01-07 Yongyi Guo , Dominic Coey , Mikael Konutgan , Wenting Li , Chris Schoener , Matt Goldman