Evidence produced by multinational trial-based cost-effectiveness studies is often used to inform decisions concerning the adoption of new healthcare technologies. A key issue relating to the use of this type of evidence is the extent to which trial-wide economic results are applicable to every...

The debate surrounding whether the findings of efficacy studies are applicable to real-world treatment situations is ongoing. The issue of lack of applicability due to a lack of clinical heterogeneity could be addressed by employing less restrictive inclusion criteria. Given that health economic...

Sample size and power for cost-effectiveness analysis depend on assumptions about the difference in cost and effect, the standard deviations of cost and effect, the correlation of the difference in cost and effect, the α and β errors and maximum willingness to pay (W). The first seven of these...

This article deals with the question of how to handle costs to enhance medication adherence in trial-based pharmacoeconomic analyses. It argues that resources to improve patient adherence have a clearly distinguishable impact on costs and utility and thus are relatively easy to exclude when...

Methods for determining sample size requirements for cost-effectiveness studies are reviewed and illustrated. Traditional methods based on tests of hypothesis and power arguments are given for the incremental cost-effectiveness ratio and incremental net benefit (INB). In addition, a full...

Basic sample size and power formulae for cost-effectiveness analysis have been established in the literature. These formulae are reviewed and the similarities and differences between sample size and power for cost-effectiveness analysis and for the analysis of other continuous variables such as...

DOI: 10.2165/0019053-200826100-00009

There has been much recent debate in the health economics literature as to the (near) equivalence of cost-benefit analysis (CBA) and cost-effectiveness analysis (CEA). The aim of this paper is to demonstrate that whether such a (near) equivalence exists depends on whether one defines economic...

Pharmacoeconomic data may be obtained within the context of randomised clinical trials (RCTs) and from effectiveness studies in the `real world'. The differences between the 2 types of study design have implications for the types of data that can be obtained and the interpretation of the...

Background: Missing data are potentially an extensive problem in cost-effectiveness analyses conducted alongside randomised clinical trials, where prospective collection of both resource use and health outcome information is required. There are several possible reasons for the presence of...