Accelerating proof of concept by smart early clinical trials

The time required to develop a new drug takes over 12 years and often much longer, and its development is very expensive. This dilemma was the main reason for launching translational medicine as a potential remedy. From the beginning, it was clear that a pivotal early step in clinical development is the proof-of-concept (PoC) study, typically obtained in the phase IIb clinical trial. It is only a natural consequence to adapt the development strategy for a new drug to obtain a positive PoC as early as possible. This goal was one of the major objectives of the Critical Path Initiative in the United States and similar constructs elsewhere. In this chapter, methods and pathways to accelerate PoC are discussed and examples given.