Adaptive Enrichment Designs for Confirmatory Randomized Trials: Statistical Methods and Software Tools 1608 Rhode Island Avenue, NW, Washington, DC 20036 Tuesday, June 13, 2017 12:00pm – 4:00pm |
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Instructors:Michael Rosenblum, PhD, Associate Professor of Biostatistics, Johns Hopkins Bloomberg School of Public Health mrosen@jhu.edu BackgroundMost randomized trials are designed to determine average treatment effects for a population. This results in trials that may fail to detect important differences in benefits and harms for subpopulations. For example, standard trial designs are not targeted to determine whether a treatment benefits most patients, benefits only a select few, or benefits some patients and harms others. The impact is that treatment recommendations based on the results of standard trial designs may be suboptimal, leading to poor patient outcomes and wasting healthcare resources. This problem affects virtually all disease areas, since it stems from how randomized trials, the gold standard for evaluating treatments, are currently designed and analyzed. Course DescriptionThis course presents an overview of the strengths and limitations of these designs, explains recent advances in statistical methods for these designs, and presents a software tool for optimizing these designs. Two case studies are presented in the context of treatments for stroke and Alzheimer’s disease.
Target AudienceAnyone involved in or interested in the planning and/or analyzing of randomized trials. Individuals from industry, government, and academia are all welcome. |