Bayesian adaptive design for concurrent trials involving biologically related diseases

BIOSTATISTICS(2022)

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摘要
We develop a Bayesian design method for a clinical program where an investigational product is to be studied concurrently in a set of clinical trials involving related diseases with the goal of demonstrating superiority to a control in each. The approach borrows information on treatment effectiveness using correlated mixture priors using an analysis procedure that is closely related Bayesian model averaging. Mixture priors are constructed by eliciting conjugate priors based on pessimistic and enthusiastic predictions for the data to be observed for each disease and then by eliciting mixture weights for all possible configurations of the pessimistic and enthusiastic priors across the diseases to be studied. The proposed approach provides a robust framework for information borrowing in settings where the diseases may have endpoints based on different data types. We show via simulation that operating characteristics based on the proposed design framework are favorable compared to those based on information borrowing designs using the Bayesian hierarchical model which is poorly suited for information borrowing when there are different data types underpinning the endpoints across which information is to be borrowed.
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关键词
Bayesian model averaging, Clinical trial design, Conjugate prior, Mixture prior
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