ABSTRACT

Platform trials have the potential to drive tremendous efficiencies in the development of new therapeutics. These efficiencies can be operational or statistical in nature. Statistical efficiencies are derived from many sources including the use of a shared control arm and adaptive randomization. In rare or difficult to enroll populations, platform trials offer the opportunity to steer important new therapies into a standing clinical trials infrastructure, potentially expediting the availability of badly needed new therapies. Efficiency can also be gained from statistical modeling to formally leverage both historical information and concurrent information in related populations. In this chapter, the various sources of efficiency will be described in more detail and examples will be provided to demonstrate their relative contribution to a more economical paradigm for clinical trials.