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570 * ! Thu, 8/6/2020, 3:00 PM - 4:50 PM Virtual
Addressing the Unique Analytic Challenges of Drug Development in Rare Diseases with the Use of Innovative Designs and Real World Evidence in Clinical Trials — Topic Contributed Papers
ENAR, Health Policy Statistics Section, International Chinese Statistical Association
Organizer(s): Junjing Lin , AbbVie
Chair(s): Margaret Gamalo-Siebers, Eli Lilly & Co
3:05 PM Case Studies of Clinical Trials in Rare Diseases with Innovative Designs Utilizing Historical Data
Bingzhi Zhang, Sanofi
3:25 PM On Randomized Delayed-Start Design with Integrated Analysis of Efficacy for Drug Development in Rare Diseases
Qing Liu, Quantitative and Regulatory Medical Science, LLC; Fred Holdbrook, Amicus Therapeutics, Inc.
3:45 PM Statistical Issues in Trials for Rare Diseases Using Innovative Design and Real-World Evidence – Regulatory Experiences
Aloka Chakravarty, Office of Biostatistics in CDER, FDA
4:05 PM Clinical Data Augmentation Strategy via Propensity-Score-Based Bayesian Analysis
Junjing Lin, Takeda; Margaret Gamalo-Siebers, Eli Lilly & Co; Ram Tiwari, FDA
4:25 PM Use of historical controls to assess emerging interventions in cystic fibrosis: a comparison of methods and a look forward
Amalia Magaret, University of Washington
4:45 PM Floor Discussion