ABSTRACT
The objective of a phase II study is to evaluate whether a particular regimen has enough biologic activity in a given disease to warrant further investigation. We would like to have a mechanism in which the candidate agents or regimens can be screened relatively quickly, and for practical and ethical reasons, we would like to expose the minimal number of patients in order to evaluate activity. To plan an efficient phase II trial, there need to be at least three key considerations: What is the most appropriate endpoint? What is the optimal patient population, and what is the most reasonable statistical design?
