ABSTRACT
In the previous chapters we noted there are some circumstances, particularly in the development of a new molecule, where it does not add much to the development process to use Phase 2 as a screen to see if there is sufficient activity to warrant a Phase 3 trial. In this chapter we show that a Phase 2 trial may still provide some benefit to clinical development in these circumstances when used as a pilot trial to optimize the design of a Phase 3 trial rather than as a screening trial. Using a Phase 2 trial in this way, one could ascertain whether dose strongly influences the drug’s effectiveness or whether certain subsets of patients have much better responses to treatment than others.
