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Background: Assessment of long-term survival for health technology assessment often necessitates extrapolation beyond the duration of a clinical trial. Without robust methods and external data, extrapolations are unreliable. Flexible…

Introduction: Modelling of relative treatment effects is an important aspect to consider when extrapolating the long-term survival outcomes of treatments. Flexible parametric models offer the ability to accurately model the observed data,…

Methodology · Statistics 2024-01-02 Philip Cooney , Arthur White

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

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

Objective: We aim to utilise real world data in evidence synthesis to optimise an evidence base for the effectiveness of biologic therapies in rheumatoid arthritis in order to allow for evidence on first-line therapies to inform second-line…

Decisions based upon pairwise comparisons of multiple treatments are naturally performed in terms of the mean survival of the selected study arms or functions thereof. However, synthesis of treatment comparisons is usually performed on…

Applications · Statistics 2025-12-11 Anastasios Apsemidis , Dimitris Mavridis , Nikolaos Demiris

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

While randomised controlled trials (RCTs) are the gold standard for estimating causal treatment effects, their limited sample sizes and restrictive criteria make it difficult to extrapolate to a broader population. Observational data, while…

Methodology · Statistics 2025-09-09 Stephanie Riley , Ricardo Silva , Matthew Sperrin

Time to an event of interest over a lifetime is a central measure of the clinical benefit of an intervention used in a health technology assessment (HTA). Within the same trial, multiple end-points may also be considered. For example,…

Applications · Statistics 2026-01-13 Nathan Green , Murat Kurt , Andriy Moshyk , James Larkin , Gianluca Baio

Objectives To investigate the use of a Bayesian joint modelling approach to predict overall survival (OS) from immature clinical trial data using an intermediate biomarker. To compare the results with a typical parametric approach of…

Comparing survival experiences of different groups of data is an important issue in several applied problems. A typical example is where one wishes to investigate treatment effects. Here we propose a new Bayesian approach based on…

Methodology · Statistics 2024-07-17 Alan Riva-Palacio , Fabrizio Leisen , Antonio Lijoi

An aspect of interest in surveillance of diseases is whether the survival time distribution changes over time. By following data in health registries over time, this can be monitored, either in real time or retrospectively. With relevant…

Applications · Statistics 2025-03-10 Jimmy Huy Tran , Jan Terje Kvaløy , Hartwig Kørner

Randomized Controlled Trials (RCTs) represent a gold standard when developing policy guidelines. However, RCTs are often narrow, and lack data on broader populations of interest. Causal effects in these populations are often estimated using…

Machine Learning · Computer Science 2023-03-07 Zeshan Hussain , Michael Oberst , Ming-Chieh Shih , David Sontag

Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

The difference in restricted mean survival time (RMST) is a clinically meaningful measure to quantify treatment effect in randomized controlled trials, especially when the proportional hazards assumption does not hold. Several frequentist…

Patients with metastatic breast cancer (mBC) undergo repeated computed tomography (CT) imaging during treatment to monitor disease progression. Accurate longitudinal tracking of individual lesions across scans from multiple radiologists is…

Restricted mean survival time (RMST) is an intuitive summary statistic for time-to-event random variables, and can be used for measuring treatment effects. Compared to hazard ratio, its estimation procedure is robust against the…

Methodology · Statistics 2023-05-25 Ruizhe Chen , Sanjib Basu , Qian Shi

Survival data is encountered in a range of disciplines, most notably health and medical research. Although Bayesian approaches to the analysis of survival data can provide a number of benefits, they are less widely used than classical (e.g.…

Computation · Statistics 2020-02-25 Samuel L. Brilleman , Eren M. Elci , Jacqueline Buros Novik , Rory Wolfe

While randomized controlled trials (RCTs) are the gold standard for estimating treatment effects in medical research, there is increasing use of and interest in using real-world data for drug development. One such use case is the…

Methodology · Statistics 2021-10-11 Devin Incerti , Michael T Bretscher , Ray Lin , Chris Harbron

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment…

Methodology · Statistics 2022-10-05 Dasom Lee , Shu Yang , Xiaofei Wang
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