Nonstop Drug Development: Strategies for a Quick Transition from Phase II to Phase III

• Activity Number: 12
• Type: Topic Contributed
• Date/Time: Sunday, August 8, 2004 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Ronald W. Helms*+ and Karen Kesler
• Companies: Rho, Inc. and Rho, Inc.
• Keywords: cllinical trials ; accelerated drug development ; NonStop ; biopharmaceutical

Abstract

The NonStop drug development strategy, as introduced by Helms (2001), can be used to make dramatic reductions in the time and cost of Phase II, under appropriate circumstances. This presentation focuses on the transition from Phase II to Phase III, addressing some issues not addressed in the original presentation. Briefly, a Phase II implementation of the NonStop strategy is based on one or more Phase II studies with the objective of selecting a treatment regimen to be carried forward into Phase III. A Phase II study design includes a control treatment and a set of "many" (e.g., 4 - 12) "active" treatment regimens or "treatment arms," covering a spectrum that has a high probability of including at least one that can successfully be carried to Phase III. Frequent interim analyses are conducted with the objective of "pruning" most of the treatment arms as quickly as possible, either for low efficacy or a poor safety profile. The goal is quickly to reduce the number of remaining treatment arms to one and, as soon as this happens, immediately transition to Phase III. This presentation addresses solutions to issues that arise in the Phase II - III transition.