Designing Phase 2 Trials Based on Program-Level Criteria

• Activity Number: 113
• Type: Topic Contributed
• Date/Time: Monday, July 30, 2012 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Nitin Patel*+ and James Bolognese and Christy Chuang-Stein and David Hewitt and Arnold Gammaitoni and Jose Carlos Pinheiro
• Companies: Cytel Inc. and Cytel Inc. and Pfizer Inc. and Merck and Nuvo Research and Johnson & Johnson
• Keywords: Probability of success ; Net present value ; Maximum utility dose selection ; Target efficacy dose selection

Abstract

Traditionally, sample size considerations for phase 2 trials are based on the desired properties of the design and response information from the trials. In this talk, we propose to design phase 2 trials based on program-level optimization. We present a framework to evaluate the impact that several phase 2 design features have on the probability of phase 3 success and the expected net present value of the product. These factors include the phase 2 sample size, decision rules to select a dose for phase 3 trials and the sample size for phase 3 trials. Using neuropathic pain as an example, we use simulations to illustrate the framework and show the benefit of including these factors in the overall decision process.