Justification, margin values, and analysis populations for oncologic noninferiority and equivalence trials: a meta-epidemiological study.

ABSTRACT:

Background

Noninferiority and equivalence trials evaluate whether an experimental therapy's effect on the primary endpoint is contained within an acceptable margin compared with standard of care. The reliability and impact of this conclusion, however, is largely dependent on the justification for this design, the choice of margin, and the analysis population used.

Methods

A meta-epidemiological study was performed of phase 3 randomized noninferiority and equivalence oncologic trials registered at ClinicalTrials.gov. Data were extracted from each trial's registration page and primary manuscript.

Results

We identified 65 noninferiority and 10 equivalence trials that collectively enrolled 61 632 patients. Of these, 61 (81%) trials demonstrated noninferiority or equivalen

SUBMITTER: Kleber TJ 

PROVIDER: S-EPMC12058270 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature