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In health technology assessment, decisions are based on complex cost-effectiveness models which, to be implemented, require numerous input parameters. When some of relevant estimates are not available the model may have to be simplified.…

Applications · Statistics 2019-01-23 Sze Huey Tan , Keith R Abrams , Sylwia Bujkiewicz

Composite endpoints, which combine two or more distinct outcomes, are frequently used in clinical trials to enhance the event rate and improve the statistical power. In the recent literature, the while-alive cumulative frequency measure…

Methodology · Statistics 2025-11-14 Xi Fang , Hajime Uno , Fan Li

Joint models are well suited to modelling linked data from laboratories and health registers. However, there are few examples of joint models that allow for (a) multiple markers, (b) multiple survival outcomes (including terminal events,…

The mean residual life function is a key functional for a survival distribution. It has a practically useful interpretation as the expected remaining lifetime given survival up to a particular time point, and it also characterizes the…

Methodology · Statistics 2018-10-11 Valerie Poynor , Athanasios Kottas

Use of historical data in clinical trial design and analysis has shown various advantages such as reduction of within-study placebo-treated number of subjects and increase of study power. The meta-analytic-predictive (MAP) approach accounts…

Applications · Statistics 2019-12-12 Sebastian Weber , Yue Li , John Seaman , Tomoyuki Kakizume , Heinz Schmidli

Survival analysis is one of the most important fields of statistics in medicine and the biological sciences. In addition, the computational advances in the last decades have favoured the use of Bayesian methods in this context, providing a…

Applications · Statistics 2020-07-28 Danilo Alvares , Elena Lázaro , Virgilio Gómez-Rubio , Carmen Armero

We propose a comprehensive Bayesian joint modeling framework for zero-inflated longitudinal count data and time-to-event outcomes, explicitly incorporating a cure fraction to account for subjects who never experience the event. The…

Methodology · Statistics 2025-08-27 Taban Baghfalaki , Mojtaba Ganjali

In epidemiological surveys, data missing not at random (MNAR) due to survey nonresponse may potentially lead to a bias in the risk factor estimates. We propose an approach based on Bayesian data augmentation and survival modelling to reduce…

Methodology · Statistics 2017-08-29 Juho Kopra , Juha Karvanen , Tommi Härkänen

The integration of data from multiple sources is increasingly used to achieve larger sample sizes and enhance population diversity. Our previous work established that, under random sampling from the same underlying population, integrating…

Methodology · Statistics 2026-01-01 Farimah Shamsi , Andriy Derkach

In early-phase cancer clinical trials, the limited availability of data presents significant challenges in developing a framework to efficiently quantify treatment effectiveness. To address this, we propose a novel utility-based Bayesian…

Methodology · Statistics 2025-06-10 Saurabh Bhandari , Michael J. Daniels , Chenguang Wang

Mid-study design modifications are becoming increasingly accepted in confirmatory clinical trials, so long as appropriate methods are applied such that error rates are controlled. It is therefore unfortunate that the important case of…

Applications · Statistics 2014-05-08 Dominic Magirr , Thomas Jaki , Franz Koenig , Martin Posch

A population-averaged additive subdistribution hazards model is proposed to assess the marginal effects of covariates on the cumulative incidence function and to analyze correlated failure time data subject to competing risks. This approach…

Methodology · Statistics 2023-08-23 Xinyuan Chen , Denise Esserman , Fan Li

Background: Incidence of adverse outcome events rises as patients with advanced illness approach end-of-life. Exposures that tend to occur near end-of-life, e.g., use of wheelchair, oxygen therapy and palliative care, may therefore be found…

Longitudinal observational patient data can be used to investigate the causal effects of time-varying treatments on time-to-event outcomes. Several methods have been developed for controlling for the time-dependent confounding that…

Methodology · Statistics 2021-10-08 Ruth H. Keogh , Jon Michael Gran , Shaun R. Seaman , Gwyneth Davies , Stijn Vansteelandt

The mean survival is the key ingredient of the decision process in several applications, notably in health economic evaluations. It is defined as the area under the complete survival curve, thus necessitating extrapolation of the observed…

Applications · Statistics 2026-03-10 Anastasios Apsemidis , Nikolaos Demiris

Leveraging external or historical data to improve the efficiency of randomized clinical trials without introducing bias or inflating the Type I error rate remains challenging. Recent work on externally trained prognostic scores, such as…

Methodology · Statistics 2026-05-28 Junyi Zhou , Qing Liu , May Mo , Amy Xia

Reliability updating refers to a problem that integrates Bayesian updating technique with structural reliability analysis and cannot be directly solved by structural reliability methods (SRMs) when it involves equality information. The…

Machine Learning · Computer Science 2023-04-19 Xiong Xiao , Zeyu Wang , Quanwang Li

Motivation: The discovery of relationships between gene expression measurements and phenotypic responses is hampered by both computational and statistical impediments. Conventional statistical methods are less than ideal because they either…

Methodology · Statistics 2019-07-16 Lei Ding , Daniel J. McDonald

We introduce a semi-parametric Bayesian model for survival analysis. The model is centred on a parametric baseline hazard, and uses a Gaussian process to model variations away from it nonparametrically, as well as dependence on covariates.…

Machine Learning · Statistics 2016-11-04 Tamara Fernández , Nicolás Rivera , Yee Whye Teh

Meta-analysis is widely used to integrate results from multiple experiments to obtain generalized insights. Since meta-analysis datasets are often heteroscedastic due to varying subgroups and temporal heterogeneity arising from experiments…

Methodology · Statistics 2026-01-19 Kohsuke Kubota , Shonosuke Sugasawa , Keiichi Ochiai , Takahiro Hoshino