Abstract

ObjectiveIn economic evaluation, a commonly used outcome measure for the treatment effect is the between-arm difference in restricted mean survival time (rmstD). This study illustrates how different survival analysis methods can be used to estimate the rmstD for economic evaluation using individual patient data (IPD) meta-analysis. Our aim was to study if/how the choice of a method impacts on cost-effectiveness results.MethodsWe used IPD from the Meta-Analysis of Radiotherapy in Lung Cancer concerning 2,000 patients with locally advanced non-small cell lung cancer, included in ten trials. We considered methods either used in the field of meta-analysis or in economic evaluation but never applied to assess the rmstD for economic evaluation using IPD meta-analysis. Methods were classified into two approaches. With the first approach, the rmstD is estimated directly as the area between the two pooled survival curves. With the second approach, the rmstD is based on the aggregation of the rmstDs estimated in each trial.ResultsThe average incremental cost-effectiveness ratio (ICER) and acceptability curves were sensitive to the method used to estimate the rmstD. The estimated rmstDs ranged from 1.7 month to 2.5 months, and mean ICERs ranged from € 24,299 to € 34,934 per life-year gained depending on the chosen method. At a ceiling ratio of € 25,000 per life year-gained, the probability of the experimental treatment being cost-effective ranged from 31% to 68%.ConclusionsThis case study suggests that the method chosen to estimate the rmstD from IPD meta-analysis is likely to influence the results of cost-effectiveness analyses.

Highlights

  • The individual patient data meta-analysis (IPD-MA) has become the gold standard for obtaining the best evidence for treatment effects

  • The average incremental cost-effectiveness ratio (ICER) and acceptability curves were sensitive to the method used to estimate the rmstD

  • The estimated rmstDs ranged from 1.7 month to 2.5 months, and mean ICERs ranged from € 24,299 to € 34,934 per life-year gained depending on the chosen method

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Summary

Introduction

The individual patient data meta-analysis (IPD-MA) has become the gold standard for obtaining the best evidence for treatment effects (e.g. see [1,2]). The aim of an IPD-MA is to estimate a pooled treatment effect from the aggregation of data from all randomised controlled trials (RCTs) that investigated the same clinical question [3,4,5]. Economic evaluation uses an absolute outcome measure such as the number of life-years gained associated with the experimental treatment [6] It is estimated as the between-arm difference in the restricted mean survival time (rmstD) and corresponds to the area between the two survival curves for the experimental arm and the control arm restricted to a certain time horizon [7]. In a recent paper, Wei and colleagues [18] compared three different methods to estimate the rmstD from IPD-MA but they did not study costeffectiveness

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