Idle thoughts of a 'well-calibrated' Bayesian in clinical drug development

Abstract

The use of Bayesian approaches in the regulated world of pharmaceutical drug development has not been without its difficulties or its critics. The recent Food and Drug Administration regulatory guidance on the use of Bayesian approaches in device submissions has mandated an investigation into the operating characteristics of Bayesian approaches and has suggested how to make adjustments in order that the proposed approaches are in a sense calibrated. In this paper, I present examples of frequentist calibration of Bayesian procedures and argue that we need not necessarily aim for perfect calibration but should be allowed to use procedures, which are well-calibrated, a position supported by the guidance.

Keywords: bayesian statistics; calibration; drug development; operating characteristics; planning; simulation.