Some Considerations on Adaptive Design with a Long-Term Assessment of Primary Efficacy Endpoint

• Activity Number: 350
• Type: Contributed
• Date/Time: Tuesday, August 5, 2014 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Junliang Chen*+ and Chunqin Deng and Jaume Ayguasanosa and Sandra Camprubi
• Companies: Grifols Therapeutics and Grifols Therapeutics and Instituto Grifols S.A. and Instituto Grifols S.A.
• Keywords: Adaptive Design ; Dose Selection ; Endpoint ; Clinical Trial

Abstract

Abstract: Adaptive design is getting more and more attractive among clinical community for clinical trial design nowadays after the FDA issued the draft guidance on adaptive design clinical trials for drugs and biologics. Most adaptive designs utilize the short term assessment of endpoints so that a timely adjustment can be implemented after the interim analysis, such as dose selection in a seamless phase 2/3 design. For a study with long term assessment of endpoint, challenges will arise when the study team tries to shorten the gap between the last subject included in the interim analysis and the adjustment(e.g., dose selection) for the next phase. In this presentation, some practical considerations are proposed when utilizing a long term assessment of primary efficacy endpoint (1 year assessment after the baseline) in a dose selection of seamless phase 2/3 design, which is accepted by a regulatory agency. The over running issue, under running issue, and the impact of enrollment rate will be assessed.