Advanced Application of PFS to Accelerated Oncology Drug Development

• Activity Number: 387
• Type: Topic Contributed
• Date/Time: Tuesday, July 31, 2012 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Cong Chen*+ and Zhiping Sun
• Companies: Merck and Merck
• Keywords: seamless design ; surrogate ; decision analysis ; benefit-cost ratio

Abstract

Seamless Ph2/3 design holds the promise to accelerate an oncology drug development program. However, the Go-No Go (GNG) decision from Ph2 to Ph3 transition has to be made with limited clinical endpoint data. The difficulty to pre-specify such a decision rule is arguably the single most important reason why seamless designs are less used in practice than expected. In this presentation, we'd like to address the following issues: 1) how to effectively incorporate surrogate biomarker (e.g. PFS) data into the decision criteria; 2) how to derive objective GNG bars from a benefit-cost ratio perspective to streamline the decision making process; 3) how to fully realize the potential of a seamless design with proper risk mitigation. Our work is based on a real example in the oncology therapeutic area. However, the general approach is equally applicable to various other areas.