A0567
Title: Response-adaptive randomization for recurrent and terminal events data with a composite endpoint
Authors: Pei-Fang Su - National Cheng Kung University (Taiwan) [presenting]
Abstract: Recurrent event and terminal event data commonly arise in clinical and observational studies. A composite endpoint has been used as a possible assessment to evaluate the efficacy of a treatment effect for both types of events, particularly when faced with high costs and a more extended follow-up study. To model recurrent event processes complicated by the existence of a terminal event, joint frailty modelling has been typically employed. The objective was to develop target-driven response adaptive randomization strategies using a composite endpoint based on joint frailty modelling. First, a balanced, randomized design was implemented, and then the adaptive response randomization was investigated. The former is intuitively adopted first, while the latter is expected to be desirable and ethical in allocating more subjects to the more effective treatment. The results show that the proposed procedures using a composite endpoint are capable of reducing the number of trial participants who receive inferior treatment while simultaneously reaching a desired optimal target as compared to a balanced, randomized design. The R shiny application for calculating the sample size and allocation probabilities is also available.
