Recurrent events are often important endpoints in randomized clinical trials. For example, the number of recurrent disease-related hospitalizations may be considered as a clinically meaningful endpoint in cardiovascular studies. In some settings, the recurrent event process may be terminated by an event such as death, which makes it more challenging to define and estimate a causal treatment effect on recurrent event endpoints. In this talk, I will briefly introduce the estimands framework and then focus on the principal stratum estimand, where the treatment effect of interest on recurrent events is defined among subjects who would be alive regardless of the assigned treatment. We propose a Bayesian approach based on a joint model of the recurrent event and death processes with a frailty term accounting for within-subject correlation to estimate the principal stratum effect in randomized clinical trials. We also present Bayesian posterior predictive check procedures for assessing the model fit. The randomized Phase III chronic heart failure trial PARAGON-HF demonstrated the proposed approaches
Speaker: Tianmeng Lyu, PhD, Associate Director Statistical Consultant in Statistical Methodology, Novartis
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