Bypassing phase 1 trials in the cancer drug development paradigm: Generally unwise and potentially dangerous

Abstract

Although the primary purpose of phase 1 trials is the determination of safety, these early clinical investigative efforts are also associated with several important secondary endpoints that are essential to future drug development. Unfortunately, there has been a recent trend in which some oncology investigators have suggested bypassing formal phase 1 testing of presumably theoretically attractive new/novel antineoplastic drug combinations. This commentary challenges the need for, and wisdom of, this approach and highlights the potential danger to research subjects participating in later stage trials that are not supported by rigorous phase 1 data. © 2010 American Cancer Society.