|
Abstract:
|
Phase 3 umbrella/platform trials with common control could increase operational efficiency, save patient resources, and reduce enrollment competition. A few phase 3 platform trials have been conducted so far, initiated by non-profit organizations that allow various companies to participate. However, individual company-led phase 3 umbrella trials, especially those intended for regulatory registration, is rare. As a natural extension of a conventional multi-arm trial design, a three-arm umbrella trial design is more appealing and practical than a general platform trial design. Besides, a three-arm umbrella trial design is a building block of a general platform trial design. It is important to build a solid foundation around it before taking a leap. We present detailed statistical properties of a phase 3 three-arm umbrella design including Type I error control and power as well as important practical issues such as the optimal allocation ratio. We intend to not only complement the existing literature, but more importantly to provide practical guidance to pave the way for its implementation by individual companies.
|
