Legend:
CC = Vancouver Convention Centre
F = Fairmont Waterfront Vancouver
* = applied session ! = JSM meeting theme
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511
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Wed, 8/1/2018,
10:30 AM -
12:00 PM
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CC-West 212
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Statistical Considerations for Rare Disease Clinical Development: Opportunities and Challenges — Topic Contributed Papers
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Biopharmaceutical Section
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Organizer(s): Yang Song, Vertex Pharmaceuticals Inc.
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Chair(s): Xihao Li, Harvard T.H. Chan School of Public Health
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10:35 AM
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What Constitutes Scientific Evidence - Controversies in Rare Disease Trial Designs and Personalized Medicine
Mark Chang, Veristat
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10:55 AM
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A Statistical Framework on Clinical Trials for Information Integration Across Data Sources with Applications to Rare Disease Clinical Development
Yang Song, Vertex Pharmaceuticals Inc.; Xihao Li, Harvard T.H. Chan School of Public Health
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11:15 AM
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On Randomized Controlled Trials with Integrated Real World Evidence for Drug Development in Rare Diseases
Qing Liu, Amicus Therapeutics, Inc
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11:35 AM
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Bayes in Drug Development for Rare Diseases
John Scott, FDA
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11:55 AM
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Integrative Statistical Learning with Real World Healthcare Data: Towards a Data Driven Suicide Prevention Framework
Kun Chen, University of Connecticut
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12:15 PM
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Floor Discussion
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