Adaptive Designs with Sensitive Subgroup Selection

• Activity Number: 332
• Type: Topic Contributed
• Date/Time: Tuesday, August 3, 2010 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Yan Daniel Zhao*+ and Alex Dmitrienko
• Companies: Eli Lilly and Company and Eli Lilly and Company
• Keywords: Adaptive Design ; Taylored Therapy ; Multiple Testing ; Subgroup Selection ; Extended Fallback Test

Abstract

In tailored therapies it is essential to select sensitive subgroups of patients who may benefit more from the study treatment than the overall population. In the design phase of the study several potential sensitive subgroups are pre-defined based on previous studies and knowledge. Then, the objective of the study under consideration is to confirm or disregard these subgroups at pre-specified interim analyses or end of the study. In this paper we apply an extended fallback procedure to control the type I error level. Such a procedure is more powerful than existing approaches. We illustrate our approach using an oncology example and the operating characteristics are examined via simulation studies.