Noninferiority Trials

Patrick P. J. Phillips () and David V. Glidden ()
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Patrick P. J. Phillips: University of California San Francisco, UCSF Center for Tuberculosis
David V. Glidden: University of California San Francisco, Department of Epidemiology and Biostatistics

Chapter 69 in Principles and Practice of Clinical Trials, 2022, pp 1297-1323 from Springer

Abstract: Abstract In this chapter we provide an overview of non-inferiority trials. We first introduce two motivating examples and describe scenarios for when a non-inferiority trial is appropriate. We next describe the procedures for defining the margin of non-inferiority from both regulatory and public health perspectives and then provide practical guidance for how to design a non-inferiority trial and analyze the resulting data, paying particular attention to regulatory and other published guidelines. We go on to discuss particular challenges unique to non-inferiority trials including the importance of assay sensitivity, the enigma of effect preservation, switching between non-inferiority and superiority, the interpretation of results when event rate assumptions are incorrect, and the place of interim analyses and safety monitoring. We conclude the chapter by addressing alternative methodologies and innovative perspectives on non-inferiority trials that have been proposed in an attempt to mitigate these challenges, including Bayesian approaches, alternative three-arm and pragmatic designs, and methods that address different treatment durations and the averted infections ratio.

Keywords: Noninferiority; Margin of noninferiority; Assay sensitivity; Effect preservation; Active control trial; Biocreep (search for similar items in EconPapers)
Date: 2022
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Persistent link: https://EconPapers.repec.org/RePEc:spr:sprchp:978-3-319-52636-2_98

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DOI: 10.1007/978-3-319-52636-2_98

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