Futility stopping in non-inferiority trials

摘要

In a non-inferiority trial, the goal is to demonstrate that the effect ofa new treatment is not inferior to the effect of the active control by aspecified amount, called the non-inferiority margin. There can beflexibility in selecting the margin depending on the new treatment'spotential advantages (e.g. fewer serious adverse effects or less frequentdosing) over the active control. The Food and Drug Administration(FDA) issued a guidance document on non-inferiority trials in 2016,which covers some major aspects of their design and analysis [1].