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Annual Rare Disease Scientific Workshop
September 15, 2015
This workshop will focus on developing a practical framework for high quality, organized, patient-centered input for drug development that is systematic and objective, and representative of the patient population. Current methodologies for engaging patients across the entire drug development lifecycle will be discussed and evaluated, with emphasis on rare and serious diseases with the goal of identifying best practices and useful ideas for a set of standard approaches for getting patient input in development. Representatives from various rare disease stakeholder groups including industry, government, and patient advocacy organizations will have the opportunity to engage with one another in this interactive day-long forum.