Case-control designs in the study of common diseases: updates on the demise of the rare disease assumption and the choice of sampling scheme for controls

Abstract

In recent years the use of case-control designs has been extended to the study of common diseases. It has been shown that the rare disease assumption is not necessary, and that by a suitable choice of sampling scheme for controls, it is possible to obtain direct estimates of relative risk and relative rate, instead of relying on the odds ratio as an indirect estimate. The majority of papers addressing these issues are theoretical, and the arguments have been couched in mathematical terms. As such they are not readily accessible to many practising epidemiologists. This paper summarizes the discussion in a simplified manner. It describes the three different measures of relative incidence, namely the relative risk, the relative rate and the odds ratio, together with their corresponding case-control designs. The discussion is extended to show that the choice of the appropriate measure of relative incidence depends on the mode of action of the risk factor, as well as on characteristics of disease. We propose a classification scheme comprising five different categories of situation, and make recommendations regarding study designs for each.