TL34: Non-inferiority in cancer trials
Zhiying (Cindy) Xu, Novartis  *Tingting Yi, Novartis  Lihui Zhao, Novartis 

Keywords: Noninferiority, cancer

To discuss various aspects of the non-inferiority (NI) trial design in oncology, including the background and rationale of using NI design (phase of the study, disease indication, active-controls involved, other benefits of the experiment drug, etc.), methods for selecting NI margin and estimating the active-control effect, choice of endpoints, sample size and power calculation, alpha level used, analysis populations, interpretation of the results, and outcome of the study. The draft FDA guidance on NI trials (Guidance for Industry. Non-Inferiority Clinical Trials. US Food and Drug Administration. March 2010. Available from: provides "advice on when NI studies can be interpretable, on how to choose the NI margin, and how to analyze the results." The review paper by Tanaka et al (Statistical issues and recommendations for noninferiority trials in oncology: a systematic review. Clin Cancer Res; 18(7); 1837–47) provides an overview of methodologic issues in NI trials in oncology and they conducted a systematic review of 72 trials in cancer. These can be the starting point of the discussion.