Communicating, and Justifying, Nonefficacy Benefits for New Drugs Approved via Noninferiority Trials

Noninferiority clinical trials are designed to determine whether an intervention is not “unacceptably worse” than a comparator by more than a prespecified difference, known as the noninferiority margin. The US Food and Drug Administration (FDA) has established guidance for industry to clarify when a superiority study design does not need to be used to establish new drug effectivene ss, usually because it would not be ethical to use a placebo or “no-treatment” control. Other justifications for noninferiority-designed trials include testing interventions with more favorable safety profiles or more convenient dosing, less patient burden, reduced cost, or other benefits for wh ich patients and clinicians might accept diminished effectiveness as a trade-off.
Source: JAMA Internal Medicine - Category: Internal Medicine Source Type: research