Design of clinical trials for recurrent events with periodic monitoring.

摘要: This paper presents a design for randomized clinical trials in which incomplete data are collected on the occurrence of potentially recurrent events through periodic monitoring. In particular, assumed to arise according point process, but information is available at times monitoring only if one or more has occurred since preceding point. The event process modelled via piecewise Poisson and proportional rates model introduced represent difference between treatment groups. was developed basis Wald-type test derived from generalized estimating equations Liang Zeger (Biometrika 73, 13-22 (1986)). Robustification variance estimator effect considered under random effects with semi-parametric mixture distribution. adopted address issues arose an osteoporosis trial conducted Japan.