Session 4: October 19-22, 2009 (San Francisco, CA)

Confirming Trials: Methodology and Biostatistics

Session Co-Leaders: Christy Chuang-Stein, PhD, Pfizer; Robert O'Neill, PhD, Food and Drug Administration; and Stephen Senn, MSc, PhD, CStat, University of Glasgow

Session Chairs: Keaven Anderson, PhD, Merck; James Cross, PhD, University of Washington; Robert O'Neill, PhD, Food and Drug Administration

The different tests and methods of biostatistics are discussed. The application of different trial designs is simulated, within-trial decisions, data management, extraction, manipulation and storage of data.

Frontiers in Drug Development and Regulatory Science Series: Value-Benefit-Risk Equation on October 22.

Lectures

• Biostatistical Concepts and Principles for Confirmatory Clinical Trials
• Choice of Endpoints
• Non-inferiority Trials
• Sample Size Analysis
• Choosing a Clinical Trial Design
• The Role of the Statistical Analysis Plan in Confirmatory Clinical Trials
• Attrition in Randomized Controlled Clinical Trials
• Choice of Endpoints
• Exploratory vs. Confirmatory Trials
• Multiple Comparisons/Endpoints
• Statistical Issues in the Use Imaging Technology in Clinical Studies
• Sequential Design and Analysis
• Adaptive Designs: An Overview
• Adaptive Designs: Regulatory Perspective
• Data Safety Monitoring Boards
• Safety Analysis: Points to Consider
• Meta Analysis: How to Get It Right?
  
  Frontiers Workshop
• Risk Evaluation and Mitigation Strategies ("REMS"): FDA, Industry, and Patient Perspectives
• Quantifying Pharmaceutical Benefit-Risk: Promises and Perils of Proposed New Methods
• Patient Benefit-Risk Preferences for New Pharmaceuticals: A Case Study of Maximum Acceptable Risk and Minimum Acceptable Benefit
• Epidemiological Approach to the Benefit/Risk Assessment
• Insights on US Payer Process for Product Valuation
• Valuing Drug Benefits and Risks from a CMS Perspective: Future Trends in Medicare Coverage and Reimbursement
• A European Perspective on Product Value and Coverage Decisions

Case Studies

• LIFE Study
• Surrogate Endpoints
• Seamless Phase II/III Design

Teaching Faculty
Keaven Anderson, PhD, Merck
H. Michael Arrighi, PhD, MSPH, JANSSEN Alzheimer Immunotherapy
Wade M. Aubry, MD, CEnter for Medical Technology Policy
Jane A. Axelrad, JD, Food and Drug Administration
Alex C. Bajamonde, PhD, Independent Consultant
John FP Bridges, PhD, Johns Hopkins Bloomberg School of Public Health
James Cross, PhD, University of Washington
Scott Emerson, MD, PhD, University of Washington
Robert Erwin, MSc, Marti Nelson Cancer Foundation
Paul Flyer, Pacific Northwest Statistical Consulting
Rekha Garg, MD, MS, Amgen
Joseph F. Heyse, PhD, Merck
Ying Lu, PhD, Stanford University
Jeff Maca, PhD, Novartis
Ralph O'Brien, PhD, Case Western Reserve University
Robert T. O'Neill, PhD, Food and Drug Administration
Thomas D. Szucs, MD, MBA, University of Basel
Steven M. Snappin, PhD, Amgen
Roy N. Tamura, PhD, Eli Lilly
Lu Tian, PhD, Stanford University
Joachim Vollmar, PhD, International Clinical Development Consultants
John B. Watkins, RPh, MPH, BCPS, Premera Blue Cross

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