Non-inferiority trial
A trial designed to show a new treatment is not meaningfully worse than the standard, rather than better: used when the new option is shorter, cheaper, less toxic or easier (five radiotherapy fractions instead of 25, six months of trastuzumab instead of twelve).
The sponsor pre-specifies a non-inferiority margin (the largest loss of efficacy considered acceptable, e.g. a hazard ratio upper confidence limit below 1.2) and the trial succeeds if the confidence interval excludes that margin. Because a bad trial (poor adherence, wrong population) tends toward no difference, non-inferiority trials must be run rigorously and are analysed per-protocol as well as intention-to-treat. They underpin most de-escalation (hypofractionation, IDEA 3 vs 6 months FOLFOX, PERSEPHONE, segmentectomy vs lobectomy, SANO active surveillance) and biosimilar approvals. Choosing the margin is contentious: a 'non-inferior' result can still mean some patients are harmed.
Pages like this
not linked directly; found by shared links- TermBlinding (double-blind, open-label, placebo-controlled)
Shares Intention-to-treat (ITT) and per-protocol analysis, Control arm and comparator (investigator's choice).
- TermPre-specified vs post-hoc analysis
Shares Intention-to-treat (ITT) and per-protocol analysis, Statistical significance (P values, alpha, multiplicity).
- TermBackbone, add-on and monotherapy
Shares Control arm and comparator (investigator's choice), De-escalation, escalation and response-adapted therapy.