Superiority trials are conducted to test the hypothesis that a treatment or strategy A is superior to (i.e., better than) treatment strategy B in reducing the impact of disease. However, A may be considerably safer, more convenient, or cheaper than B. These features may make A more attractive than B even if the burden of disease is reduced comparably by the two treatments, or even a bit worse by A over B. In this context, non-inferiority trials have become increasingly popular to test the hypothesis that a new treatment is not ‘unacceptably worse’ than an active comparator by more than a predefined non-inferiority margin. Non-inferiority trials have unique design features and methodology and require a different analysis than traditional superiority trials. The main aim of this overview is to analyze the role of non-inferiority trials in the development of new treatments, involving some scientific, statistical, and practical considerations. We elucidate some aspects of non-inferiority trials that contribute to the validity of the results. The unique design features and methodology of non-inferiority trials are summarized and key findings to consider when evaluating a non-inferiority trial are illustrated.
%0 Journal Article
%1 Angeli2020
%A Angeli, Fabio
%A Verdecchia, Paolo
%A Vaudo, Gaetano
%A Masnaghetti, Sergio
%A Reboldi, Gianpaolo
%D 2020
%I Adis
%J Pharmaceutical Medicine
%K DataAccuracy DataInterpretation EndpointDetermination EquivalenceTrialsasTopic* FabioAngeli GianpaoloReboldi Humans MEDLINE NCBI NIH NLM NationalCenterforBiotechnologyInformation NationalInstitutesofHealth NationalLibraryofMedicine PaoloVerdecchia PubMedAbstract ResearchDesign* Review Statistical TreatmentOutcome doi:10.1007/s40290-020-00334-z pmid:32277352
%N 3
%P 159-165
%R 10.1007/s40290-020-00334-z
%T Optimal Use of the Non-Inferiority Trial Design
%U https://pubmed.ncbi.nlm.nih.gov/32277352/
%V 34
%X Superiority trials are conducted to test the hypothesis that a treatment or strategy A is superior to (i.e., better than) treatment strategy B in reducing the impact of disease. However, A may be considerably safer, more convenient, or cheaper than B. These features may make A more attractive than B even if the burden of disease is reduced comparably by the two treatments, or even a bit worse by A over B. In this context, non-inferiority trials have become increasingly popular to test the hypothesis that a new treatment is not ‘unacceptably worse’ than an active comparator by more than a predefined non-inferiority margin. Non-inferiority trials have unique design features and methodology and require a different analysis than traditional superiority trials. The main aim of this overview is to analyze the role of non-inferiority trials in the development of new treatments, involving some scientific, statistical, and practical considerations. We elucidate some aspects of non-inferiority trials that contribute to the validity of the results. The unique design features and methodology of non-inferiority trials are summarized and key findings to consider when evaluating a non-inferiority trial are illustrated.
@article{Angeli2020,
abstract = {Superiority trials are conducted to test the hypothesis that a treatment or strategy A is superior to (i.e., better than) treatment strategy B in reducing the impact of disease. However, A may be considerably safer, more convenient, or cheaper than B. These features may make A more attractive than B even if the burden of disease is reduced comparably by the two treatments, or even a bit worse by A over B. In this context, non-inferiority trials have become increasingly popular to test the hypothesis that a new treatment is not ‘unacceptably worse’ than an active comparator by more than a predefined non-inferiority margin. Non-inferiority trials have unique design features and methodology and require a different analysis than traditional superiority trials. The main aim of this overview is to analyze the role of non-inferiority trials in the development of new treatments, involving some scientific, statistical, and practical considerations. We elucidate some aspects of non-inferiority trials that contribute to the validity of the results. The unique design features and methodology of non-inferiority trials are summarized and key findings to consider when evaluating a non-inferiority trial are illustrated.},
added-at = {2023-02-03T11:44:35.000+0100},
author = {Angeli, Fabio and Verdecchia, Paolo and Vaudo, Gaetano and Masnaghetti, Sergio and Reboldi, Gianpaolo},
biburl = {https://www.bibsonomy.org/bibtex/263cfb46412fcb5806c56f5a03847dd12/jepcastel},
doi = {10.1007/s40290-020-00334-z},
interhash = {384adeec1f812ff253e1b9a5a5492f0c},
intrahash = {63cfb46412fcb5806c56f5a03847dd12},
issn = {11791993},
journal = {Pharmaceutical Medicine},
keywords = {DataAccuracy DataInterpretation EndpointDetermination EquivalenceTrialsasTopic* FabioAngeli GianpaoloReboldi Humans MEDLINE NCBI NIH NLM NationalCenterforBiotechnologyInformation NationalInstitutesofHealth NationalLibraryofMedicine PaoloVerdecchia PubMedAbstract ResearchDesign* Review Statistical TreatmentOutcome doi:10.1007/s40290-020-00334-z pmid:32277352},
month = {6},
note = {Tests d'equivalència; Introductori; Critical appraisal},
number = 3,
pages = {159-165},
pmid = {32277352},
publisher = {Adis},
timestamp = {2023-02-03T11:44:35.000+0100},
title = {Optimal Use of the Non-Inferiority Trial Design},
url = {https://pubmed.ncbi.nlm.nih.gov/32277352/},
volume = 34,
year = 2020
}