Current issues in oncology drug development, with a focus on Phase II trials.

In this commentary we discuss several challenges that are of current relevance to the design of clinical trials in oncology. We argue that the compartmentalization of trials into the three standard phases, with non overlapping aims, is not necessary and in fact may slow the clinical development of agents. Combined Phase I/II trials and/or Phase I trials that at minimum collect efficacy data and more optimally include a preliminary measure of efficacy in dosing determination should be more widely utilized. Similarly, we posit that randomized Phase II trials should be used more frequently, as opposed to the traditional historical single arm Phase II trial that usually does not have a valid comparison group. The use of non binary endpoints is a simple modification that can improve the efficiency of early phase trials. The heterogeneity in scientific goals and contexts in early phase oncology trials is considerable, and the potential to improve the design to match these goals is great. We review these and other issues in the context of 5 manuscripts related to Phase II trials published in this volume. Our overall premise is that the potential benefits associated with the oncology clinical trial community moving away from the one size fits all paradigm of trial design are great, and that more flexible and efficient designs tailored to match the goals of each study are currently available and being used successfully.