How to use a noninferiority trial: users' guides to the medical literature

Mulla, Sohail M., Scott, Ian A., Jackevicius, Cynthia A., You, John J. and Guyatt, Gordon H. (2012) How to use a noninferiority trial: users' guides to the medical literature. JAMA: Journal of the American Medical Association, 308 24: 2605-2611. doi:10.1001/2012.jama.11235

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Author Mulla, Sohail M.
Scott, Ian A.
Jackevicius, Cynthia A.
You, John J.
Guyatt, Gordon H.
Title How to use a noninferiority trial: users' guides to the medical literature
Journal name JAMA: Journal of the American Medical Association   Check publisher's open access policy
ISSN 0098-7484
Publication date 2012-12-26
Sub-type Critical review of research, literature review, critical commentary
DOI 10.1001/2012.jama.11235
Volume 308
Issue 24
Start page 2605
End page 2611
Total pages 7
Place of publication Chicago, United States
Publisher American Medical Association
Language eng
Abstract Clinical investigators are increasingly testing treatments that have the primary benefit of decreased burden or harms relative to an existing standard. The goal of the resulting randomized trials—called noninferiority trials—is to establish that the novel treatment's effectiveness is not substantially less than the existing standard. Conclusions from these trials are, however, based on noninferiority thresholds specified by authors whose judgments may not coincide with those of patients and clinicians. This article highlights issues related to validity, interpretation, and applicability of results specific to noninferiority trials. Suboptimal administration of standard treatment or exclusive reliance on the analyze-as-randomized approach that is standard for conventional superiority trials may produce misleading results in noninferiority trials. Clinicians should judge whether the novel treatment's impact on effectiveness outcomes—the prime reason for wanting to prescribe it—is sufficiently close to that of standard treatment that they are comfortable substituting it for the existing standard. Trading off desirable and undesirable consequences is an individual decision: given the benefits of a novel treatment, some patients may perceive the uncertainty regarding a reduction in treatment effectiveness as acceptable while others may not.
Keyword Nonvalvular atrial-fibrillation
Equivalence randomized-trials
Good enough
Q-Index Code C1
Q-Index Status Provisional Code
Institutional Status Non-UQ

Document type: Journal Article
Sub-type: Critical review of research, literature review, critical commentary
Collection: School of Medicine Publications
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Citation counts: TR Web of Science Citation Count  Cited 31 times in Thomson Reuters Web of Science Article | Citations
Scopus Citation Count Cited 32 times in Scopus Article | Citations
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