A Bayesian Approach to Rare Disease Trials
*Scott M Berry, Berry Consultants Keywords: bayesian, adaptive design, rare disease In this talk a Bayesian approach to trials of rare diseases are discussed. In rare disease situations the ability to do “type I error” controlled trials can be impossible—more patients than exist! There is no single idea that will transform rare disease scenarios—many situations are unique: some have historical data available, some have related diseases, and some have multiple experimental drugs available to test—an even harder problem! In this talk I provide example scenarios with innovations that can be used to provide a way forward. All these approaches include adaptive designs, may include borrowing from historical data, borrowing from other diseases, or running platform trials with multiple experimental treatments.
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Key Dates
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April 30 - May 22, 2013
Invited Abstract Submission Open -
June 4, 2013
Online Registration Opens -
August 9 - August 23, 2013
Invited Abstract Editing -
August 23, 2013
Short Course materials due from Instructors -
August 26, 2013
Housing Deadline -
September 9, 2013
Cancellation Deadline and Registration Closes @ 11:59 pm EDT -
September 16 - September 18, 2013
Marriott Wardman Park, Washington, DC