Applications of 'Learn' Phase Adaptive Designs

• Activity Number: 221
• Type: Topic Contributed
• Date/Time: Monday, August 2, 2010 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Jose Carlos Pinheiro*+ and Chyi-Hung Hsu
• Companies: Johnson & Johnson and Novartis Pharmaceuticals Corporation
• Keywords: Dose finding ; Proof-of-concept ; Model-based drug development ; Dose-ranging ; Flexible designs

Abstract

Innovative designs and data analysis methods, such as adaptive designs, play a critical role in the ongoing efforts to modernize and improve the efficiency of clinical drug development. This is especially true for clinical studies in the "Learn" phase of development, such as proof-of-concept and dose-ranging trials, in which sponsors typically have more freedom (from a regulatory oversight point of view) to experiment with new statistical approaches. This talk will discuss and illustrate the use of adaptive, model-based methods in the context of seamless Phase IIa/IIb studies, including comparisons to more traditional methods based on pairwise hypothesis testing.