New Advance on Bayesian Superiority Trial Design with Application to Rare Disease Drug Development

Music City Center 

In this paper, we explore the Bayesian design of a two-arm superiority clinical trial. There is a
significant amount of existing literature on the subject already, but also growing interest in new
or expanded applications. Likewise, our paper aims to simultaneously address both the past
and the future of this Bayesian design. We expose and formally prove a number of desirable
properties of the superiority trial design that previously have not been expounded on, which
helps further demonstrate that the design is statistically sound. We also expand the framework
of our design into the frontier of dynamically borrowing historical data. In particular, we use the
Overlapping Index (OVI) as a means to quantify the similarity between current and historical
data, and hence determine an appropriate level of borrowing. We present simulation studies to
show the results and operating characteristics of our proposed new design. Finally, we conclude
with a comparison of our results with other priors followed by a summary and discussion of
future work.

Power Prior

Historical Borrowing

Overlapping Index 

Section on Bayesian Statistical Science