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Methods of Analysis in Randomised Non-Inferiority...
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Methods of Analysis in Randomised Non-Inferiority Trials: A Methodological Survey Protocol (Preprint)

Abstract

BACKGROUND

Non-inferiority (NI) trial designs that investigate whether an experimental intervention is no worse than standard of care have been used increasingly in recent years. The robustness of the conclusions are in part dependent on the analysis population set used for the analysis. The intention-to-treat (ITT) analysis has been thought to be anti-conservative compared with the per-protocol (PP) analysis in the NI setting.

OBJECTIVE

We aim to conduct a methodological review assessing the analysis population set used in NI trials.

METHODS

A comprehensive electronic search strategy will be used to identify studies indexed in Medline, Embase, Emcare, and Cochrane Central Register of Controlled Trials (CENTRAL) databases. Studies will be included if they are non-inferiority trials published in 2024. The primary outcome is the analysis population used in the primary analysis of the trial (ITT or PP). Secondary outcomes will be the NI margin, effect estimates, point estimates, and corresponding confidence intervals of the analysis. Analysis will be done using descriptive statistics.

RESULTS

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CONCLUSIONS

This methodological survey of NI trials will describe the population analysis set used in primary analysis and assess factors which could be associated with each analysis

CLINICALTRIAL

Authors

Xie D; McKechnie T; Staibano P; Arvikar E; Gou D; Longia S; Maatallah A; Maatallah M; Wang B; Wang Y

Publication date

May 6, 2025

DOI

10.2196/preprints.76876

Preprint server

JMIR Preprints
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