Journal
STATISTICAL METHODS IN MEDICAL RESEARCH
Volume 20, Issue 6, Pages 595-612Publisher
SAGE PUBLICATIONS LTD
DOI: 10.1177/0962280210378945
Keywords
sample size; power; non-inferiority; parallel group; clinical trials; sensitivity analysis; Bayesian methods
Ask authors/readers for more resources
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.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available