A One-To-Two Arm Design for a Rare Disease

• Activity Number: 277
• Type: Topic Contributed
• Date/Time: Tuesday, August 5, 2014 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Todd Graves*+
• Companies: Berry Consultants
• Keywords: Adaptive design ; clinical trials ; Bayesian

Abstract

When a disease is devastating and rare and a new treatment has the potential to provide enormous benefits, a standard randomized clinical trial design may be inappropriate for ethical reasons. Instead, a design should strive to minimize, or possibly even eliminate, randomizing patients to standard treatment if early trial results for the new treatment are as promising as hoped. To accomplish this, the design must utilize historical data on standard treatment efficiently. The design can begin randomizing patients to placebo if early results are close enough so that ethics allow it and a precise comparison is called for. We illustrate these ideas with a design developed for spinal muscular atrophy.