Adaptive Designs: An Industry Perspective

• Activity Number: 216
• Type: Invited
• Date/Time: Monday, August 1, 2011 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Jose Carlos Pinheiro*+
• Companies: Johnson & Johnson Pharmaceutical R&D, LLC
• Keywords: Response-adaptive ; flexible designs ; simulation ; modeling ; dose finding

Abstract

The release of the FDA draft guidance on adaptive designs has provided long awaited clarification on the Agency's position with regard to the use of these methods in clinical drug development programs. The encouraging tone of the draft guidance, particularly with regard to exploratory clinical studies, has led to renewed interest across the pharmaceutical industry in the use of response-adaptive approaches for improving the information efficiency of drug development. Considerable interest is currently focused on the quantification of the potential benefit of adaptive and other innovative approaches in comparison to more traditional strategies. Such evaluations are generally simulation-based and take into account not only the statistical properties of the approach (e.g., probability of success), but also its operational characteristics and costs (e.g., drug supply, duration). This talk will discuss key concepts related to the quantitative evaluation of alternative clinical development approaches, using real trial examples to illustrate and motivate the ideas and methods.