Considerations of Practical Study Designs for Rare Disease Drug Development
Mingxiu Hu, Takeda Pharmaceuticals Wei Huang, RedBudTek Inc Serap Sankoh, Takeda Pharmaceuticals *Huyuan Yang, Takeda Pharmaceuticals International Co Keywords: Rare Disease drug development, Frequentist method, Bayesian design With the rapidly growing development in rare disease drug, the adaptation of sensible clinical trial designs becomes the most challenging task to both investigator/sponsors and regulatory. Given the sample size limit of rare disease patient population, the conventional statistical designs and rules of clinical trials are not suitable anymore. Frequentist method based on historical control or crossover patient self-control have been considered to perform hypothesis testing in controlling type I/II error. However, the underline assumptions for these designs can be easily violated. Bayesian adaptive design, as been used more and more in early phases in drug development, has now been considered in confirmatory trials for rare disease clinical trials. In this talk we will go over these statistical considerations and illustrate with real and simulated examples.
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Key Dates
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November 1 - December 17, 2013
Online proposal submission for a session, short course and Town Hall Open -
January 6 - March 11, 2014
Online proposal submission for Roundtables Open -
April 30 - May 28, 2014
Abstract Submission Open -
June 4, 2014
Online Registration Opens -
August 8 - August 22, 2014
Invited Abstract Editing -
August 11, 2014
Short Course materials due from Instructors -
September 1, 2014
Housing Deadline -
September 15, 2014
Cancellation Deadline and Registration Closes @ 11:59 pm EDT -
September 22 - September 24, 2014
Marriott Wardman Park, Washington, DC