Bayesian Innovations in Clinical Trial Design and Analysis

• Activity Number: 142
• Type: Invited
• Date/Time: Monday, August 9, 2004 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Donald Berry*+
• Companies: University of Texas M.D. Anderson Cancer Center
• Keywords: adaptive designs ; sequential designs ; clinical trials ; Bayesian approach

Abstract

I will describe recent innovations in the design and analysis of clinical trials. The goals are creating more efficient clinical trials and clinical development programs and treating patients more effectively, both those in and those outside of trials. These designs are developed from the Bayesian perspective but are frequentist in the sense that they meet the usual standards for operating characteristics. These designs have been effected at my home institution, in national oncology studies. Or in pharmaceutical and medical device industry-sponsored trials. The revolutionary aspect is hardly earth-shattering in nonmedical science: we pay heed to the accumulating data and let it guide the course of the trial! I will give case studies showing actual designs and in analyses presented to the FDA. They include the possibility of early stopping, assigning patients to better performing therapies, and variations on themes such as seamless phase II/III trials with sequential sampling and using early endpoints. The savings of such an approach in terms of effectively using patient resources are substantial.