A perspective on principles of comparative cost-effectiveness studies for pharmacotherapy of chronic diseases

Abstract

Comparative cost-effectiveness drug studies can support decisionmaking for allocation of health care resources if principles of clinical pharmacology and pharmacoepidemiology are considered. Use of constant or milligramequivalent doses instead of bio-equivalent doses, reliance on placebo-controlled instead of head-to-head randomized trials, disparities in community-based distribution of disease burden, lack of clinically important endpoint data, and absence of adherence data can limit the applicability of such studies. This article highlights methodological issues that should be incorporated in comparative cost-effectiveness drug studies, using statins as an example.