Non-inferiority trials: the 'at least as good as' criterion with dichotomous data

The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the...

Full description

Saved in:
Bibliographic Details
Published in:Statistics in medicine 2006-04, Vol.25 (7), p.1115-1130
Main Authors: Laster, Larry L., Johnson, Mary F., Kotler, Mitchell L.
Format: Article
Language:English
Subjects:
adaptive versus fixed margins
at least as good as
Clinical trials
Data Interpretation, Statistical
Drug Evaluation - methods
Humans
Models, Statistical
non-inferiority trials
Randomized Controlled Trials as Topic - methods
Research Design
Sample Size
Statistical analysis
statistical efficiency
Statistical methods
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:The ‘at least as good as’ criterion, introduced by Laster and Johnson for a continuous response variate, is developed here for applications with dichotomous data. This approach is adaptive in nature, as the margin of non‐inferiority is not taken as a fixed difference; it varies as a function of the positive control response. When the non‐inferiority margin is referenced as a high fraction of the positive control response, the procedure is seen to be uniformly more efficient than the fixed margin approach, yielding smaller sample sizes when sizing non‐inferiority trials under identically specified conditions. Extending this method to proportions is straightforward, but highlights special considerations in the design of non‐inferiority trials versus superiority trials, including potential trade‐offs in statistical efficiency and interpretability. Copyright © 2005 John Wiley & Sons, Ltd.
ISSN:0277-6715
1097-0258
DOI:10.1002/sim.2476