Cost-effectiveness analysis has gained status over the last 15 years as an important tool for assisting resource allocation decisions in a budget-limited environment such as healthcare. Randomised (multicentre) multinational controlled trials are often the main vehicle for collecting primary...

Objective: There are multiple reasons for missing data in observational studies; excluding patients with missing data can lead to significant bias. In this study, we evaluated several methods for assigning missing values to health service utilisation. Design and setting: Cancer of the Prostate...

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...

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...

Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinical condition, or to improve the delivery of care. The purpose of this article is to compare - within...

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...

Many research designs and statistical methodologies will be used to conduct comparative effectiveness research (CER). In particular, it is almost certainly the case that the demand for real-world evidence will drive increased demand for CER analyses of observational data. Although a great deal...