THINKING OUTSIDE THE BOX: Recent Advances inthe Analysis and Presentation of Uncertainty inCost-Effectiveness Studies

As many more clinical trials collect economic information within theirstudy design, so health economics analysts are increasingly working with patient-leveldata on both costs and effects. In this paper, we review recent advances in the use ofstatistical methods for economic analysis of information collected alongside clinicaltrials. In particular, we focus on the handling and presentation of uncertainty, includingthe importance of estimation rather than hypothesis testing, the use of the net-benefitstatistic, and the presentation of cost-effectiveness acceptability curves.We also discussthe appropriate sample size calculations for cost-effectiveness analysis at the designstage of a study. Finally, we outline some of the challenges for future research in thisarea—particularly in relation to the appropriate use of Bayesian methods and methodsfor analyzing costs that are typically skewed and often incomplete.