The use of randomisation-based efficacy estimators in non-inferiority trials.

ABSTRACT:

Background

In a non-inferiority (NI) trial, analysis based on the intention-to-treat (ITT) principle is anti-conservative, so current guidelines recommend analysing on a per-protocol (PP) population in addition. However, PP analysis relies on the often implausible assumption of no confounders. Randomisation-based efficacy estimators (RBEEs) allow for treatment non-adherence while maintaining a comparison of randomised groups. Fischer et al. have developed an approach for estimating RBEEs in randomised trials with two active treatments, a common feature of NI trials. The aim of this paper was to demonstrate the use of RBEEs in NI trials using this approach, and to appraise the feasibility of these estimators as the primary analysis in NI trials.

Methods

Two NI trials were use

SUBMITTER: Gillespie D 

PROVIDER: S-EPMC5343391 | biostudies-literature | 2017 Mar

REPOSITORIES: biostudies-literature