Considerations Regarding Noninferiority: A Case Study In Multiple Sclerosis

Non-inferiority (NI) study designs are intended to determine whether a proposed (new) intervention is no worse than a reference intervention by a prespecified margin. Such designs frequently are encountered in development programs sponsored by the pharmaceutical industry, particularly in studies in which it would be clinically inappropriate to include an untreated control group and where the placebo effect (or standard of care effect) can be inferred reliably from existing historical data.

As with any study design, though, NI designs have specific attributes that must be accounted for to ensure that the design can adequately and justifiably address the research questions at hand. To illustrate the seven key elements that should be considered before selecting an NI study design, this paper presents a case study examining the potential use of an NI design in a study involving relapsing remitting multiple sclerosis (RRMS).