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Critically appraising noninferiority randomized controlled trials: a primer for emergency physicians

  • Mohammad Al Deeb (a1) (a2), Aftab Azad (a1) (a3) and David Barbic (a4)

Abstract

Noninferiority (NI) trials aim to show that a new treatment or drug is not inferior to a standard, accepted treatment. The rapid proliferation of NI trials within the literature makes it imperative for emergency physicians to be able to read, interpret, and appraise critically this type of research study. Using several emergency medicine examples from the recent literature, this article outlines the key differences between traditional, superiority randomized controlled trials and NI trials. We summarize four important points that an emergency physician should consider when critically appraising an NI trial: 1) Does the new treatment have tangible benefits over the standard treatment? 2) Was the choice of the NI margin appropriate? 3) Was the effect of the standard treatment preserved? Does the trial have assay sensitivity? and 4) What type of analysis strategy was employed: intention-to-treat (ITT) or per protocol (PP)?

Les essais de non-infériorité (ENI) visent à démontrer qu’un nouveau traitement ou un nouveau médicament n’est pas inférieur au traitement de référence, reconnu. La prolifération des ENI dans la documentation rend impérative la capacité des urgentologues à comprendre, à interpréter et à évaluer d’un oeil critique ce type de recherche. Ainsi, l’article fera ressortir, à l’aide de plusieurs exemples tirés de la documentation récente en médecine d’urgence, les principales différences qui existent entre le modèle classique d’essai comparatif, hasardisé , de supériorité , et le modèle d’ENI. Seront résumés quatre points importants qu’un urgentologue devrait envisager lorsqu’il évalue de façon critique un ENI: 1) Le nouveau traitement offre-t-il des avantages tangibles comparativement au traitement habituel? 2) Le choix de l’intervalle de non-infé riorité est-il pertinent? 3) L’effet du traitement de référence est-il conservé ? L’essai est-il sensible? 4) Quel type de straté gie d’analyse a été employé : selon l’intention de traiter ou selon le protocole?

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Copyright

Corresponding author

Correspondence to: Dr. David Barbic, Department of Emergency Medicine, Sunnybrook Health Sciences Centre, 2075 Bayview Avenue, Toronto,ON M4N 3N5; david.barbic@gmail.com

References

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Critically appraising noninferiority randomized controlled trials: a primer for emergency physicians

  • Mohammad Al Deeb (a1) (a2), Aftab Azad (a1) (a3) and David Barbic (a4)

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