Much has been written over the past decade regarding the ethics of phase 1 studies in patients with malignant disease.1- 8 A primary focus of this literature has been on the expressed concern that patients, and often their physicians, “misinterpret” the fundamental aims of such studies, believing either consciously, or unconsciously, that participation will be of direct clinical benefit to the research subject. Rather, the argument goes, the purpose of phase 1 trials is to “determine the metabolism and pharmacologic actions of the drug in humans, the adverse effects associated with increasing doses, and, if possible, to gain early evidence on effectiveness,”9 information that will hopefully be of benefit to future patients.
Markman M. “Therapeutic Intent” in Phase 1 Oncology TrialsA Justifiable Objective. Arch Intern Med. 2006;166(14):1446-1448. doi:10.1001/archinte.166.14.1446