Phase I and II Trials: Designs and Strategies

Abstract

Phase I and II studies should be designed for decision making and to begin to fill in the labeling that will be applied to a new molecular entity (NME) when it is ultimately approved for marketing. Therefore, the design of these trials is critical to the medical and commercial success of the NME. Phase I and II studies should be designed to capture both pharmacokinetic and pharmacodynamic information as well as the traditional safety and tolerance data. In general, these studies reach the largest doses that will ever be given to humans. Therefore, if they are not designed to capture this important information, it is an opportunity lost. For traditional synthetic drugs, Phase II can establish the doses and endpoints for large Phase III studies; for biotechnology products, Phase II can represent the pivotal studies for product licensing application (PLA) filing.