Statistical inference for a two-stage outcome-dependent sampling design with a continuous outcome.

ABSTRACT: The two-stage case-control design has been widely used in epidemiology studies for its cost-effectiveness and improvement of the study efficiency (White, 1982,?American Journal of Epidemiology?115, 119-128; Breslow and Cain, 1988,?Biometrika?75, 11-20). The evolution of modern biomedical studies has called for cost-effective designs with a continuous outcome and exposure variables. In this article, we propose a new two-stage outcome-dependent sampling (ODS) scheme with a continuous outcome variable, where both the first-stage data and the second-stage data are from ODS schemes. We develop a semiparametric empirical likelihood estimation for inference about the regression parameters in the proposed design. Simulation studies were conducted to investigate the small-sample behavior of the proposed estimator. We demonstrate that, for a given statistical power, the proposed design will require a substantially smaller sample size than the alternative designs. The proposed method is illustrated with an environmental health study conducted at National Institutes of Health.

SUBMITTER: Zhou H 

PROVIDER: S-EPMC4106685 | biostudies-literature | 2011 Mar

REPOSITORIES: biostudies-literature