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Abstract:
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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.
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