STATISTICS COLLOQUIUM
Bradley P. Carlin
Mayo Professor of Public Health
University of Minnesota
Bayesian Adaptive Clinical Trial Methods for Incorporating Auxiliary Data and Identifying Interesting Subgroups, with Application to Orphan Diseases
ABSTRACT
Bayesian methods have a long history of success in clinical trial settings where patients and other resources are scarce, where good reliable external information is available, or both. In this talk we review several real-life settings where Bayesian methods have paid such dividends. First, we describe recent Bayesian advances in the adaptive incorporation of historical information in clinical trials through what are known as commensurate priors, showing connections with traditional meta-analytic methods and illustrating their potential for improved power while maintaining acceptable Type I error. We then extend the approach to the cases of survival and functional data, utilizing penalized splines in both cases, and illustrating with a dataset from diagnostic radiology. We then describe our experiences to date using adaptive Bayesian methods in the context of recent work with the University of Minnesota Center for Orphan Drug Research. Applications include trials for drugs treating epilepsy and neurodegenerative diseases such as Parkinson's and adrenoleukodystrophy (ALD), in the latter case with Lorenzo's Oil. We briefly describe current research in the cautious combination of randomized and nonrandomized data sources, and also introduce methods for selection of patient subsets for which it is very likely the treatment is effective. While like Nebraska's Platte River this talk will be "a mile wide and an inch deep," all of our methods will be illustrated in the context of real trial settings arising in both academics and industry.
This talk represents joint work with Drs. Brian Hobbs and Tom Murray of the University of Texas M.D. Anderson Cancer Center, and Dr. Jim Cloyd, Ms. Cynthia Basu, Ms. Hong Zhao, and Mr. Patrick Schnell of
the University of Minnesota.
DATE: Wednesday, November 11, 2015
TIME: 4:00 p.m. -5:00 p.m.
PLACE: Philip E. Austin Bldg., Rm. 105
Coffee will be served at 3:30 p.m. in the Noether Lounge (AUST 326)
For more information, contact: Tracy Burke at tracy.burke@uconn.edu