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CC = Vancouver Convention Centre   F = Fairmont Waterfront Vancouver
* = applied session       ! = JSM meeting theme

Activity Details

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