Cost-effectiveness analysis of a multinational RCT with a binary measure of effectiveness and an interacting...

In a recent multinational randomized clinical trial, 1356 patients from 14 countries were randomized between two arms. The primary measure of effectiveness was 30-day survival. Health care utilization was collected on all patients and was combined with a single country's price weights to provide patient-level cost data. The purpose of this paper is to report the results of the cost-effectiveness analysis for the country that provided the cost weights, so as to provide a case study for illustrating recently proposed methodologies that account for skewed cost data, the between-country variation in treatment effects, possible interactions between treatment and baseline covariates, and the difficulty of estimated adjusted risk differences. A hierarchal model is used to account for the two sources of variation (between country and between patients, within a country). The model, which uses gamma distributions for cost data and recent methods for estimating adjusted risk differences, provides overall and country-specific estimates of treatment effects. Model estimation is facilitated by Markov chain Monte Carlo methods using the WinBUGS software. In addition, the theory of expected value of information is used to determine if the data provided by the trial are sufficient for decision making.