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We consider a joint survival and mixed-effects model to explain the survival time from longitudinal data and high-dimensional covariates in a population. The longitudinal data is modeled using a non linear mixed-effects model to account for…

Statistics Theory · Mathematics 2025-08-06 Antoine Caillebotte , Estelle Kuhn , Sarah Lemler

Prevalent cohort sampling is commonly used to study the natural history of a disease when the disease is rare or it usually takes a long time to observe the failure event. It is known, however, that the collected sample in this situation is…

Methodology · Statistics 2022-09-05 Omidali Aghababaei Jazi

Nonresponse after probability sampling is a universal challenge in survey sampling, often necessitating adjustments to mitigate sampling and selection bias simultaneously. This study explored the removal of bias and effective utilization of…

Methodology · Statistics 2025-11-13 Kosuke Morikawa , Kenji Beppu , Wataru Aida

Machine learning applications for longitudinal electronic health records often forecast the risk of events at fixed time points, whereas survival analysis achieves dynamic risk prediction by estimating time-to-event distributions. Here, we…

Machine Learning · Computer Science 2024-11-26 Munib Mesinovic , Peter Watkinson , Tingting Zhu

In this paper we consider two-stage adaptive dose-response study designs, where the study design is changed at an interim analysis based on the information collected so far. In a simulation study, two approaches will be compared for these…

Methodology · Statistics 2016-02-08 Emma McCallum , Björn Bornkamp

In studies based on electronic health records (EHR), the frequency of covariate monitoring can vary by covariate type, across patients, and over time. This can lead to major challenges: first, the difference in monitoring protocols may…

The hazard function is central to the formulation of commonly used survival regression models such as the proportional hazards and accelerated failure time models. However, these models rely on a shared baseline hazard, which, when…

Methodology · Statistics 2025-12-19 J. A. Christen , F. J. Rubio

The Cox proportional hazards model is widely used in survival analysis to model time-to-event data. However, it faces significant computational challenges in the era of large-scale data, particularly when dealing with time-dependent…

Methodology · Statistics 2025-01-14 Miaomiao Su , Ruoyu Wang

Background: Well-designed phase II trials must have acceptable error rates relative to a pre-specified success criterion, usually a statistically significant p-value. Such standard designs may not always suffice from a clinical perspective…

Applications · Statistics 2020-02-10 Satrajit Roychoudhury , Nicolas Scheuer , Beat Neuenschwander

Regression calibration as developed by Rosner, Spiegelman and Willet is used to correct the bias in effect estimates due to measurement error in continuous exposures. The method involves two models: a measurement error model (MEM) relating…

Methodology · Statistics 2026-02-24 Wenze Tang , Donna Spiegelman , Xiaomei Liao , Molin Wang

Electronic Health Record (EHR) systems provide critical, rich and valuable information at high frequency. One of the most exciting applications of EHR data is in developing a real-time mortality warning system with tools from survival…

Machine Learning · Computer Science 2021-11-12 Zhale Nowroozilarki , Arash Pakbin , James Royalty , Donald K. K. Lee , Bobak J. Mortazavi

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

Medical studies that depend on electronic health records (EHR) data are often subject to measurement error, as the data are not collected to support research questions under study. These data errors, if not accounted for in study analyses,…

Methodology · Statistics 2020-03-10 Eric J. Oh , Bryan E. Shepherd , Thomas Lumley , Pamela A. Shaw

Accelerated degradation tests are used to provide accurate estimation of lifetime characteristics of highly reliable products within a relatively short testing time. Data from particular tests at high levels of stress (e.g., temperature,…

Applications · Statistics 2021-06-07 Helmi Shat , Rainer Schwabe

Two-phase designs involve measuring extra variables on a subset of the cohort where some variables are already measured. The goal of two-phase designs is to choose a subsample of individuals from the cohort and analyse that subsample…

Applications · Statistics 2020-10-12 Tong Chen , Thomas Lumley

Period-prevalent cohorts are often used for their cost-saving potential in epidemiological studies of survival outcomes. Under this design, prevalent patients allow for evaluations of long-term survival outcomes without the need for long…

Methodology · Statistics 2024-10-28 Nicholas Hartman

We propose a general approach for encouraging fairness in survival analysis models based on minimizing a worst-case error across all subpopulations that occur with at least a user-specified probability. This approach can be used to convert…

Machine Learning · Statistics 2024-09-18 Shu Hu , George H. Chen

Randomized clinical trials are often designed to assess whether a test treatment prolongs survival relative to a control treatment. Increased patient heterogeneity, while desirable for generalizability of results, can weaken the ability of…

Methodology · Statistics 2020-04-30 Devan V. Mehrotra , Rachel Marceau West

We consider planning longitudinal covariate measurements in follow-up studies where covariates are time-varying. We assume that the entire cohort cannot be selected for longitudinal measurements due to financial limitations and study how a…

Methodology · Statistics 2024-07-03 Jaakko Reinikainen , Juha Karvanen

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