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Activity Number: 490
Type: Contributed
Date/Time: Wednesday, August 3, 2016 : 8:30 AM to 10:20 AM
Sponsor: Biometrics Section
Abstract #318623 View Presentation
Title: A Small N Sequential Multiple Assignment Randomized Trial for Use in Rare Disease Research
Author(s): Roy Tamura*
Companies: University of South Florida
Keywords: re-randomization ; binary data ; weighted Z statistic

Clinical research in rare diseases is difficult for a number of reasons, including limited number of patients, and patient attitudes toward active and placebo therapy. Oftentimes, a number of available drugs are used for a rare disease with limited information about the efficacy of any of the drugs. In such situations, it is logical to first design a trial which would determine if any of the drugs shows promising efficacy. In cooperation with the Vasculitis Clinical Research Consortium under the NIH, we have recently developed a small n sequential multiple assignment randomized trial (snSMART) for rare vasculitis diseases. The goal of the design is to determine the best out of a number of potential drugs and to compare the chosen drug to the best of the remaining drugs. The design, analysis, and operating characteristics of the snSMART will be presented.

Authors who are presenting talks have a * after their name.

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