Sample size/power calculation for case-cohort studies

Abstract

In epidemiologic studies and disease prevention trials, interest often involves estimation of the relationship between some disease endpoints and individual exposure. In some studies, due to the rarity of the disease and the cost in collecting the exposure information for the entire cohort, a case-cohort design, which consists of a small random sample of the whole cohort and all the diseased subjects, is often used. Previous work has focused on analyzing data from the case-cohort design and few have discussed the sample size issues. In this article, we describe two tests for the case-cohort design, which can be treated as a natural generalization of log-rank test in the full cohort design. We derive an explicit form for power/sample size calculation based on these two tests. A number of simulation studies have been used to illustrate the efficiency of the tests for the case-cohort design. An example is provided on how to use the formula.