Characteristics and Principles of Adaptive Trial Designs

This chapter discusses the property and principles of adaptive trial designs. Adaptive trial designs refer to trial designs that offer pre-planned opportunities to modify the design of an ongoing trial based on accumulating trial data. Decisions for potential adaptations are made during the trial based on interim data, but flexibilities in adaptive trial designs are established and outlined in the study documents before any patient is recruited. For statistical planning, a simulation-guided approach is often used to evaluate the statistical properties of the design. It is generally required to demonstrate control of false positive rates for the regulatory, ethics, and funding bodies. Measures to mitigate and plan for operational bias and complexity in adaptive trial designs are needed.