Design Considerations in Phase IIa and IIb for a Symptomatic Alzheimer's Treatment

• Activity Number: 605
• Type: Contributed
• Date/Time: Thursday, August 7, 2014 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Christopher Assaid*+ and James Kost and Jang-Ho Cha and Ying Zhang
• Companies: Merck and Merck and Merck and Merck
• Keywords: Adaptive Design ; Alzheimer's Disease ; Seamless IIa/IIb

Abstract

Given the staggering failure rate of late-stage clinical trials in Alzheimer's disease (AD), sponsors of AD development programs are understandably much more cautious in their development strategies and plans for spending precious dollars from R&D budgets. This is true not only for agents that are expected to be potentially disease-modifying, but also for compounds that are expected to have efficacy associated only with chronic exposure to the drug (i.e., symptomatic treatments). Development of AD drugs is complicated by the fact that regulatory agencies require evidence of effectiveness for both cognitive and functional measures, where the efficacy on functional outcomes typically lags behind that of the cognitive measures and also tend to be less sensitive. We present a range of development scenarios for consideration that may address some of the investment and resource concerns associated with this historically risky disease area as well as some of the tradeoffs associated with seamless Phase IIa/IIb adaptive designs.