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ABSTRACT: Background
Cost-effectiveness models for the treatment of long-term conditions often require information on survival beyond the period of available data.Objectives
This paper aims to identify a robust and reliable method for the extrapolation of overall survival (OS) in patients with radioiodine-refractory differentiated thyroid cancer receiving lenvatinib or placebo.Methods
Data from 392 patients (lenvatinib: 261, placebo: 131) from the SELECT trial are used over a 34-month period of follow-up. A previously published criterion-based approach is employed to ascertain credible estimates of OS beyond the trial data. Parametric models with and without a treatment covariate and piecewise models are used to extrapolate OS, and a holistic approach, where a series of statistical and visual tests are considered collectively, is taken in determining the most appropriate extrapolation model.Results
A piecewise model, in which the Kaplan-Meier survivor function is used over the trial period and an extrapolated tail is based on the Exponential distribution, is identified as the optimal model.Conclusion
In the absence of long-term survival estimates from clinical trials, survival estimates often need to be extrapolated from the available data. The use of a systematic method based on a priori determined selection criteria provides a transparent approach and reduces the risk of bias. The extrapolated OS estimates will be used to investigate the potential long-term benefits of lenvatinib in the treatment of radioiodine-refractory differentiated thyroid cancer patients and populate future cost-effectiveness analyses.
SUBMITTER: Tremblay G
PROVIDER: S-EPMC4934460 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
Tremblay Gabriel G Livings Christopher C Crowe Lydia L Kapetanakis Venediktos V Briggs Andrew A
ClinicoEconomics and outcomes research : CEOR 20160630
<h4>Background</h4>Cost-effectiveness models for the treatment of long-term conditions often require information on survival beyond the period of available data.<h4>Objectives</h4>This paper aims to identify a robust and reliable method for the extrapolation of overall survival (OS) in patients with radioiodine-refractory differentiated thyroid cancer receiving lenvatinib or placebo.<h4>Methods</h4>Data from 392 patients (lenvatinib: 261, placebo: 131) from the SELECT trial are used over a 34-mo ...[more]