Keyword Search
Legend:
CC = Vancouver Convention Centre F = Fairmont Waterfront Vancouver
* = applied session ! = JSM meeting theme
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Keyword Search Criteria: Rare Disease returned 15 record(s)
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Tuesday, 07/31/2018
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An Evaluation of Statistical Methods with Missing Data in Small Clinical Trials
Takayuki Abe, Yokohama City University, School of Data Science; Kazuhito Shiosakai, Daiichi Sankyo Co., Ltd.; Manabu Iwasaki, Yokohama City University, School of Data Science
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A Bayesian Analysis of Small N Sequential Multiple Assignment Randomized Trials (SnSMARTs)
Boxian Wei, University of Michigan, Ann Arbor; Kelley M Kidwell, University of Michigan; Thomas M Braun, University of Michigan; Roy N Tamura, University of South Florida
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Wednesday, 08/01/2018
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A Novel Blind Start Study Design to Investigate Vestronidase Alfa for Mucopolysaccharidosis VII, an Ultra-Rare Genetic Disease
Wenjie Song, Ultragenyx Pharmaceutical Inc; Chao-Yin Chen, Ultragenyx Pharmaceutical Inc; Christine Haller, Ultragenyx Pharmaceutical Inc; Emil Kakkis, Ultragenyx Pharmaceutical Inc
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A Novel Blind Start Study Design to Investigate Vestronidase Alfa for Mucopolysaccharidosis VII, an Ultra-Rare Genetic Disease
Wenjie Song, Ultragenyx Pharmaceutical Inc; Chao-Yin Chen, Ultragenyx Pharmaceutical Inc; Christine Haller, Ultragenyx Pharmaceutical Inc; Emil Kakkis, Ultragenyx Pharmaceutical Inc
8:40 AM
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What Constitutes Scientific Evidence - Controversies in Rare Disease Trial Designs and Personalized Medicine
Mark Chang, Veristat
10:35 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
10:55 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
11:15 AM
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Bayes in Drug Development for Rare Diseases
John Scott, FDA
11:35 AM
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Simulation Study in Handing Missing Data Due to Use of Rescue Therapy in Rare Disease
Yiwei Zhang, Biogen; Peng Sun, Biogen; Baoguang Han, Biogen; John Zhong, Biogen
2:20 PM
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Achieving Regulatory Approvals Without a Randomized Control Study in Rare Infectious Disease
Masanori Ito, Astellas Pharma; Misun Yu Lee, Astellas Pharma
2:30 PM
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Thursday, 08/02/2018
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Utilization of Historical Data and Real World Evidence in Clinical Trial Development - Case Studies in Rare Disease and Oncology
Florence H Yong, Pfizer Inc.; Ray Li, Pfizer Inc.; Steven Y Hua, Celgene - Receptos; Jeffery Palmer, Pfizer Inc.; Roberto Bugarini, Pfizer Inc.
8:50 AM
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Statistical Challenges and Opportunities in Drug Development for Rare Diseases
Guowen Sun, BioMarin; Keith Gregg, BioMarin; Peter Slasor, BioMarin; Chito Hernandez, BioMarin Pharmaceutical Inc.
9:50 AM
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