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Ability to quantify and predict progression of a disease is fundamental for selecting an appropriate treatment. Many clinical metrics cannot be acquired frequently either because of their cost (e.g. MRI, gait analysis) or because they are…

Machine Learning · Statistics 2019-05-28 Guanyang Wang , Yumeng Zhang , Yong Deng , Xuxin Huang , Łukasz Kidziński

Adaptive enrichment allows for pre-defined patient subgroups of interest to be investigated throughout the course of a clinical trial. Many trials which measure a long-term time-to-event endpoint often also routinely collect repeated…

Methodology · Statistics 2024-02-26 Abigail J. Burdon , Richard D. Baird , Thomas Jaki

Epidemics are inherently stochastic, and stochastic models provide an appropriate way to describe and analyse such phenomena. Given temporal incidence data consisting of, for example, the number of new infections or removals in a given time…

Methodology · Statistics 2024-05-24 Sam A. Whitaker , Andrew Golightly , Colin S. Gillespie , Theodore Kypraios

Survival analysis has been developed and applied in the number of areas including manufacturing, finance, economics and healthcare. In healthcare domain, usually clinical data are high-dimensional, sparse and complex and sometimes there…

Machine Learning · Computer Science 2018-08-13 Milad Zafar Nezhad , Najibesadat Sadati , Kai Yang , Dongxiao Zhu

Few Bayesian methods for analyzing high-dimensional sparse survival data provide scalable variable selection, effect estimation and uncertainty quantification. Such methods often either sacrifice uncertainty quantification by computing…

Methodology · Statistics 2022-07-06 Michael Komodromos , Eric Aboagye , Marina Evangelou , Sarah Filippi , Kolyan Ray

We consider the optimal value of information (VoI) problem, where the goal is to sequentially select a set of tests with a minimal cost, so that one can efficiently make the best decision based on the observed outcomes. Existing algorithms…

Artificial Intelligence · Computer Science 2017-07-18 Yuxin Chen , Jean-Michel Renders , Morteza Haghir Chehreghani , Andreas Krause

We introduce a statistical procedure that integrates survival data from multiple biomedical studies, to improve the accuracy of predictions of survival or other events, based on individual clinical and genomic profiles, compared to models…

Applications · Statistics 2020-07-20 Steffen Ventz , Rahul Mazumder , Lorenzo Trippa

Background Survival extrapolation is essential in the cost-effectiveness analysis to quantify the lifetime survival benefit associated with a new intervention, due to the restricted duration of randomized controlled trials (RCTs). Current…

Methodology · Statistics 2022-06-02 Zhaojing Che , Nathan Green , Gianluca Baio

In this article, we develop nonparametric inference methods for comparing survival data across two samples, which are beneficial for clinical trials of novel cancer therapies where long-term survival is a critical outcome. These therapies,…

Methodology · Statistics 2024-09-05 Yi-Cheng Tai , Weijing Wang , Martin T. Wells

Applications of machine learning in healthcare often require working with time-to-event prediction tasks including prognostication of an adverse event, re-hospitalization or death. Such outcomes are typically subject to censoring due to…

Machine Learning · Computer Science 2022-08-04 Chirag Nagpal , Willa Potosnak , Artur Dubrawski

Health policy decisions are often informed by estimates of long-term survival based primarily on short-term data. A range of methods are available to include longer-term information, but there has previously been no comprehensive and…

Methodology · Statistics 2025-05-05 Christopher Jackson

In order for clinicians to manage disease progression and make effective decisions about drug dosage, treatment regimens or scheduling follow up appointments, it is necessary to be able to identify both short and long-term trends in…

Quantitative Methods · Quantitative Biology 2016-12-06 Norman Poh , Simon Bull , Santosh Tirunagari , Nicholas Cole , Simon de Lusignan

The assessment of safety is an important aspect of the evaluation of new therapies in clinical trials, with analyses of adverse events being an essential part of this. Standard methods for the analysis of adverse events such as the…

In healthcare, risk assessment of patient outcomes has been based on survival analysis for a long time, i.e. modeling time-to-event associations. However, conventional approaches rely on data from a single time-point, making them suboptimal…

Machine Learning · Computer Science 2026-02-20 Mine Öğretir , Miika Koskinen , Juha Sinisalo , Risto Renkonen , Harri Lähdesmäki

Given a collection of features available for inclusion in a predictive model, it may be of interest to quantify the relative importance of a subset of features for the prediction task at hand. For example, in HIV vaccine trials, participant…

Methodology · Statistics 2025-03-27 Charles J. Wolock , Peter B. Gilbert , Noah Simon , Marco Carone

Biomarker is a critically important tool in modern clinical diagnosis, prognosis, and classification/prediction. However, there are fiscal and analytical barriers to biomarker research. Selective Genotyping is an approach to increasing…

Methodology · Statistics 2022-08-02 A. Adam Ding , Natalie DelRocco , Samuel Wu

In this paper we develop a very efficient approach to the Monte Carlo estimation of the expected value of partial perfect information (EVPPI) that measures the average benefit of knowing the value of a subset of uncertain parameters…

Numerical Analysis · Mathematics 2019-12-09 Michael B. Giles , Takashi Goda

A platform trial with a master protocol provides an infrastructure to ethically and efficiently evaluate multiple treatment options in multiple diseases. Given that certain study drugs can enter or exit a platform trial, the randomization…

Methodology · Statistics 2025-07-15 Tianyu Zhan , Jane Zhang , Lei Shu , Yihua Gu

Most statistical tests for treatment effects used in randomized clinical trials with survival outcomes are based on the proportional hazards assumption, which often fails in practice. Data from early exploratory studies may provide evidence…

Statistics Theory · Mathematics 2020-05-28 Andrea Arfé , Brian Alexander , Lorenzo Trippa

Utilizing established risk factors and prognostic models can often improve the construction of a newer risk model that uses novel biomarkers in a smaller, internal study. However, directly borrowing information from an established…

Methodology · Statistics 2026-03-12 Nicholas C. Henderson