Efficient analysis of case-control studies with sample weights

Analysis of population-based case–control studies with complex sampling designs is challenging because the sample selection probabilities (and, therefore, the sample weights) depend on the response variable and covariates. Commonly, the design-consistent (weighted) estimators of the parameters of the population regression model are obtained by solving (sample) weighted estimating equations. Weighted estimators, however, are known to be inefficient when the weights are highly variable as is typical for case–control designs. In this paper, we propose two alternative estimators that have higher efficiency and smaller finite sample bias compared with the weighted estimator. Both methods incorporate the information included in the sample weights by modeling the sample expectation of the weights conditional on design variables. We discuss benefits and limitations of each of the two proposed estimators emphasizing efficiency and robustness. We compare the finite sample properties of the two new estimators and traditionally used weighted estimators with the use of simulated data under various sampling scenarios. We apply the methods to the U.S. Kidney Cancer Case-Control Study to identify risk factors. Published 2012. This article is a US Government work and is in the public domain in the USA.