The challenges of determining noninferiority margins: A case study of noninferiority randomized controlled trials of novel oral anticoagulants

摘要

A randomized controlled trial (RCT) can have either a superiority design or a noninferiority design. A superiority design aims to show that a new drug is better than placebo or an active comparator, whereas a noninferiority design aims to show that a new drug is not worse than its comparator, which is typically an active drug. Noninferiority trials can be used when a new drug is anticipated to have an efficacy profile similar to its comparator and could offer advantages over the existing drug, such as a novel method of administration.