Biostatistics Advance Access originally published online on March 27, 2008
Biostatistics 2008 9(4):735-749; doi:10.1093/biostatistics/kxn006
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On outcome-dependent sampling designs for longitudinal binary response data with time-varying covariates
Department of Biostatistics, Vanderbilt University School of Medicine, S-2323 Medical Center North, 1161 21st Avenue South, Nashville, TN 37232-2158, USA
jonathan.schildcrout{at}vanderbilt.edu
Department of Biostatistics, University of Washington, F-600 Health Sciences Building, Campus Mail Stop 357232, Seattle, WA 98105-7232, USA
* To whom correspondence should be addressed.
A typical longitudinal study prospectively collects both repeated measures of a health status outcome as well as covariates that are used either as the primary predictor of interest or as important adjustment factors. In many situations, all covariates are measured on the entire study cohort. However, in some scenarios the primary covariates are time dependent yet may be ascertained retrospectively after completion of the study. One common example would be covariate measurements based on stored biological specimens such as blood plasma. While authors have previously proposed generalizations of the standard case–control design in which the clustered outcome measurements are used to selectively ascertain covariates (Neuhaus and Jewell, 1990) and therefore provide resource efficient collection of information, these designs do not appear to be commonly used. One potential barrier to the use of longitudinal outcome-dependent sampling designs would be the lack of a flexible class of likelihood-based analysis methods. With the relatively recent development of flexible and practical methods such as generalized linear mixed models (Breslow and Clayton, 1993) and marginalized models for categorical longitudinal data (see Heagerty and Zeger, 2000, for an overview), the class of likelihood-based methods is now sufficiently well developed to capture the major forms of longitudinal correlation found in biomedical repeated measures data. Therefore, the goal of this manuscript is to promote the consideration of outcome-dependent longitudinal sampling designs and to both outline and evaluate the basic conditional likelihood analysis allowing for valid statistical inference.
Keywords: Binary data; Longitudinal data analysis; Marginal models; Marginalized models; Outcome-dependent sampling; Time-dependent covariates
Received July 5, 2007; revised December 3, 2007; accepted for publication January 4, 2008.