Due to the innovations in the field of oncological treatment that have profoundly changed our perception of modern cancer therapy, the design of clinical trials had to be modified appropriately with the purpose of allowing for the assessment of the distinct effects of new drugs. This means that long-standing paradigms had to be abandoned, and new principles took their place. In light of continuing research, which happens at breathtaking speed, this process is still ongoing.
As the size and complexity of phase I trials have been increasing over time, investigators are facing considerable challenges these days. On the other hand, the investigational process can be shortened significantly if patient selection and assessment of treatment efficacy are conducted from the very beginning. Phase II studies used to provide initial assessment of efficacy, but adaptive trial designs in the setting of targeted therapy often lead to amalgamation of phases I and II.
Endpoints play a major role in the design of clinical studies and should therefore be selected carefully. The use of quality-of-life assessments adds valuable information to the data delineating the strengths and weaknesses of a certain treatment.
Given the recent developments, the articles presented in this paper are aimed at updating the reader on aspects of the contemporary designs of phase I and II trials, with an emphasis on endpoint selection and the use of quality of life as a trial outcome. We hope to provide useful information that might contribute to supporting scientific efforts on our way towards finding a cure for cancer.
Author: Chia-Chi (Josh) Lin, MD, PhD, National Taiwan University Hospital, National Taiwan University College of Medicine, Taipei, Taiwan
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