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100 * ! Mon, 8/3/2020, 1:00 PM - 2:50 PM Virtual
Statistical Innovations for Drug Approval and Reimbursement for Rare Disease — Invited Papers
International Indian Statistical Association, Biopharmaceutical Section, Health Policy Statistics Section
Organizer(s): Amit Bhattacharyya, Alexion Pharmaceuticals
Chair(s): Amit Bhattacharyya, Alexion Pharmaceuticals
1:05 PM On Analysis of Single Arm Trial with Natural History Controls
Qing Liu, Quantitative and Regulatory Medical Science, LLC
1:25 PM Challenges in Rare Disease Clinical Trials: The PREVENT Trial for NMOSD
Fanny O'Brien, Alexion
1:45 PM Predictive Models in Rare Diseases: Risks and Opportunities in Clinical Trials, with Examples in Amyotrophic Lateral Sclerosis (ALS)
Alex Karanevich, EMB Statistical Solutions
2:05 PM Innovative Designs and Rare Diseases: Bridging Information Through Evidence Synthesis
Satrajit Roychoudhury, Pfizer Inc.
2:25 PM Use Case Scenario: Indirect Comparisons to Enhance Reimbursement Negotiations in Rare Disease
Jinesh Shah, CSL Behring
2:45 PM Floor Discussion