A comparison of methods for sample size estimation for non-inferiority studies with binary outcomes

Author:

Julious Steven A1,Owen Roger J2

Affiliation:

1. Medical Statistics Group, Health Services Research, University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.

2. Biostatistics and Statistical Reporting, Novartis Research, Wimblehurst Road, Horsham, West Sussex, RH12 5AB, UK.

Abstract

Non-inferiority trials are motivated in the context of clinical research where a proven active treatment exists and placebo-controlled trials are no longer acceptable for ethical reasons. Instead, active-controlled trials are conducted where a treatment is compared to an established treatment with the objective of demonstrating that it is non-inferior to this treatment. We review and compare the methodologies for calculating sample sizes and suggest appropriate methods to use. We demonstrate how the simplest method of using the anticipated response is predominantly consistent with simulations. In the context of trials with binary outcomes with expected high proportions of positive responses, we show how the sample size is quite sensitive to assumptions about the control response. We recommend when designing such a study that sensitivity analyses be performed with respect to the underlying assumptions and that the Bayesian methods described in this article be adopted to assess sample size.

Publisher

SAGE Publications

Subject

Health Information Management,Statistics and Probability,Epidemiology

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