Cohort study design for illness-death processes with disease status under intermittent observation

Cohort studies are routinely conducted to learn about the incidence or progression rates of chronic diseases. The illness-death model offers a natural framework for joint consideration of non-fatal events in the semi-competing risks setting. We consider the design of prospective cohort studies where the goal is to estimate the effect of a marker on the risk of a non-fatal event which is subject to interval-censoring due to an intermittent observation scheme. The sample size is shown to depend on the effect of interest, the number of assessments, and the duration of follow-up. Minimum-cost designs are also developed to account for the different costs of recruitment and follow-up examination. We also consider the setting where the event status of individuals is observed subject to misclassification; the consequent need to increase the sample size to account for this error is illustrated through asymptotic calculations.

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