JAMA Network Open | 2019
Assessing the Justification, Funding, Success, and Survival Outcomes of Randomized Noninferiority Trials of Cancer Drugs
Abstract
Key Points Question Is the source of funding associated with the justification for using noninferiority design and success in claiming noninferiority in cancer drug trials, and are these trials associated with changes in patient survival? Findings In this systematic review and pooled analysis of 23 randomized noninferiority trials of cancer drugs, which used overall survival as the end point and enrolled 21\u2009437 patients, industry funding was associated with lack of justification but not with success in achieving noninferiority. No association of noninferiority trials with patient survival was found. Meaning Greater regulatory attention should be paid to randomized noninferiority trials of cancer drugs, especially regarding the justification for using such a design.