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Ratio-based biomarkers (RBBs), such as the proportion of necrotic tissue within a tumor, are widely used in clinical practice to support diagnosis, prognosis, and treatment planning. These biomarkers are typically estimated from…

计算机视觉与模式识别 · 计算机科学 2026-04-03 Jiameng Li , Teodora Popordanoska , Aleksei Tiulpin , Sebastian G. Gruber , Frederik Maes , Matthew B. Blaschko

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

Optimal performance is critical for decision-making tasks from medicine to autonomous driving, however common performance measures may be too general or too specific. For binary classifiers, diagnostic tests or prognosis at a timepoint,…

The family-wise error rate (FWER) has been widely used in genome-wide association studies. With the increasing availability of functional genomics data, it is possible to increase the detection power by leveraging these genomic functional…

统计方法学 · 统计学 2020-12-25 Huijuan Zhou , Xianyang Zhang , Jun Chen

To maximize clinical benefit, clinicians routinely tailor treatment to the individual characteristics of each patient, where individualized treatment rules are needed and are of significant research interest to statisticians. In the…

统计方法学 · 统计学 2021-11-23 Trinetri Ghosh , Yanyuan Ma , Rui Song , Pingshou Zhong

Estimation of the high-dimensional banded covariance matrix is widely used in multivariate statistical analysis. To ensure the validity of estimation, we aim to test the hypothesis that the covariance matrix is banded with a certain…

统计方法学 · 统计学 2022-04-26 Xiaoyi Wang , Gongjun Xu , Shurong Zheng

Companies offering web services routinely run randomized online experiments to estimate the causal impact associated with the adoption of new features and policies on key performance metrics of interest. These experiments are used to…

统计方法学 · 统计学 2023-07-13 Lorenzo Masoero , Doug Hains , James McQueen

In cancer biomarker development, a key objective is to evaluate whether a new biomarker, when combined with an established one, improves early cancer detection compared to using the established biomarker alone. Incremental value is often…

统计方法学 · 统计学 2025-11-21 Indrila Ganguly , Ying Huang

The ROC curve is a statistical tool that analyses the accuracy of a diagnostic test in which a variable is used to decide whether an individual is healthy or not. Along with that diagnostic variable it is usual to have information of some…

In observational studies, propensity scores are commonly estimated by maxi- mum likelihood but may fail to balance high-dimensional pre-treatment covariates even after specification search. We introduce a general framework that unifies and…

统计方法学 · 统计学 2017-03-22 Qingyuan Zhao

Background: It has long been advised to account for baseline covariates in the analysis of confirmatory randomised trials, with the main statistical justifications being that this increases power and, when a randomisation scheme balanced…

统计方法学 · 统计学 2021-12-09 Tim P. Morris , A. Sarah Walker , Elizabeth J. Williamson , Ian R. White

An inference procedure is proposed to provide consistent estimators of parameters in a modal regression model with a covariate prone to measurement error. A score-based diagnostic tool exploiting parametric bootstrap is developed to assess…

统计方法学 · 统计学 2024-07-02 Qingyang Liu , Xianzheng Huang

Identifying reliable biomarkers for predicting clinical events in longitudinal studies is important for accurate disease prognosis and for guiding development of new treatments. However, prognostic studies are often observational, making it…

统计方法学 · 统计学 2025-08-12 Ainesh Sewak , Vanda Inacio , Joanne Wuu , Michael Benatar , Torsten Hothorn

Time-dependent Receiver Operating Characteristics (ROC) analysis is a standard method to evaluate the discriminative performance of biomarkers or risk scores for time-to-event outcomes. Extensions of this useful method to left-truncated…

统计方法学 · 统计学 2025-09-09 Kendrick Li , Mithun Kumar Acharjee

Inferring the causal effect of a treatment on an outcome in an observational study requires adjusting for observed baseline confounders to avoid bias. However, adjusting for all observed baseline covariates, when only a subset are…

统计方法学 · 统计学 2021-02-04 Wen Wei Loh , Stijn Vansteelandt

In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

Weighting methods are popular tools for estimating causal effects; assessing their robustness under unobserved confounding is important in practice. In the following paper, we introduce a new set of sensitivity models called "variance-based…

统计方法学 · 统计学 2023-03-14 Melody Huang , Samuel D. Pimentel

The paper introduces a novel approach to global sensitivity analysis, grounded in the variance-covariance structure of random variables derived from random measures. The proposed methodology facilitates the application of…

统计方法学 · 统计学 2025-10-20 Caleb Deen Bastian , Herschel Rabitz , Grzegorz A Rempala

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

Adjusting for covariates is a well established method to estimate the total causal effect of an exposure variable on an outcome of interest. Depending on the causal structure of the mechanism under study there may be different adjustment…

统计理论 · 数学 2021-04-27 Jack Kuipers , Giusi Moffa