title: A targeted maximum likelihood estimator for two-stage designs.
abstract: We consider two-stage sampling designs, including so-called nested case control studies, where one takes a random sample from a target population and completes measurements on each subject in the first stage. The second stage involves drawing a subsample from the original sample, collecting additional data on the subsample. This data structure can be viewed as a missing data structure on the full-data structure collected in the second-stage of the study. Methods for analyzing two-stage designs include parametric maximum likelihood estimation and estimating equation methodology. We propose an inverse probability of censoring weighted targeted maximum likelihood estimator (IPCW-TMLE) in two-stage sampling designs and present simulation studies featuring this estimator.
authors.affiliation: University of California, Berkeley, USA.
authors.forename: Sherri
authors.initials: S
authors.lastname: Rose
authors.forename: Mark J
authors.initials: MJ
authors.lastname: van der Laan
mesh_terms: D016022:Case-Control Studies; D006801:Humans; D016013:Likelihood Functions; D015233:Models, Statistical; D012107:Research Design; D018401:Sample Size