Outcome-dependent sampling from existing cohorts with longitudinal binary response data: study planning and analysis

When novel scientific questions arise after longitudinal binary data have been collected, the subsequent selection of subjects from the cohort for whom further detailed assessment will be undertaken is often necessary to efficiently collect new information. Key examples of additional data collection include retrospective questionnaire data, novel data linkage, or evaluation of stored biological specimens. In such cases, all data required for the new analyses are available except for the new target predictor or exposure. We propose a class of longitudinal outcome-dependent sampling schemes and detail a design corrected conditional maximum likelihood analysis for highly efficient estimation of time-varying and time-invariant covariate coefficients when resource limitations prohibit exposure ascertainment on all participants. Additionally, we detail an important study planning phase that exploits available cohort data to proactively examine the feasibility of any proposed substudy as well as to inform decisions regarding the most desirable study design. The proposed designs and associated analyses are discussed in the context of a study that seeks to examine the modifying effect of an interleukin-10 cytokine single nucleotide polymorphism on asthma symptom regression in adolescents participating Childhood Asthma Management Program Continuation Study. Using this example we assume that all data necessary to conduct the study are available except subject-specific genotype data. We also assume that these data would be ascertained by analyzing stored blood samples, the cost of which limits the sample size.