Woolson Lecture

A non-inferiority (NI) trial is a type of clinical trial designed to show that a new treatment is not inferior to an existing standard treatment by some pre-specified amount (the non-inferiority margin). It is evident that NI trials have an important place in the lexicon of clinical trial design and analysis. These trials may be the only way and only opportunity to answer certain questions, so they must be designed and conducted with rigor. The US Food and Drug Administration released a Guidance Document on NI Trials in 2016 and the CONSORT (Consolidated Standards of Reporting Trials) Statement was updated in 2012 to include information on the reporting of NI trials. Nonetheless, several important challenges in the approach to NI trials still exist. This talk will describe the NI landscape based on my experience in the design, coordination and reporting of 3 different federally-funded NI trials over the past 20 years, each one having a unique challenge including choice of active control, a life-threatening disease and a blinded sample size re-estimation.