In clinical trials, using placebo is unethical in many therapeutic areas. In turn, non-inferiority studies are common. An experimental drug is compared against an active-control, that is, a gold standard. Simply said, we move from hypothesis testing to demonstrate that a new drug is superior to placebo to the use of confidence intervals to demonstrate that a new drug is not-inferior to the standard. However, these studies are challenging, especially in the necessary sample size, the choice of the standard and the margin itself, plus doing so in an extremely regulated environment.

This non-technical presentation includes the basics of superiority and non-inferiority study designs, their pros and cons, their methodologies, a non-inferiority story, plus an opportunity for continued research.