Systemic Utilization of Phase II Interim Efficacy Analysis to Expedite Drug Development

• Activity Number: 345
• Type: Topic Contributed
• Date/Time: Tuesday, August 11, 2015 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Jie Huang*
• Companies: Genentech, Inc./Roche
• Keywords: benefit-risk assessment ; phase 2 interim ananlysis ; phase 2 development decision ; progression-free survival ; time-to-event endpoints

Abstract

Routine and systematic utilization of interim efficacy analyses from comparative phase 2 trials could streamline the development decision making, expedite the development process, and reduce the overall drug development cost. This is a practical approach to shorten the drug development timeline without complicating the trial design and compromising the sponsor's ability to identify gaps in knowledge and thoughtfully design the phase 3 trial.

I will first examine the theoretical basis as well as the empirical evidence from 35 Roche/Genentech oncology trials for using the phase 2 interim analysis to facilitate earlier development decisions that is consistent with the final phase 2 readout, and then address issues related to the benefit, cost and implementation details through recent Roche/Genentech oncology trial experience.