Randomized clinical trial design for assessing noninferiority when superiority is expected.


Journal Article

The randomized clinical trial (RCT) is the gold standard for definitive evaluation of new therapies. RCTs designed to show that the therapeutic efficacy of a new therapy is not unacceptably inferior to that of standard therapy are called noninferiority trials. Traditionally, noninferiority trials have required very large sample sizes. Sometimes, a new treatment regimen with a favorable toxicity and/or tolerability profile is also expected to have some modest improvement in efficacy. In such specialized settings we describe a hybrid trial-design approach that requires a dramatically smaller sample size than that of a standard noninferiority design. This hybrid design can naturally incorporate a formal test of superiority as well as noninferiority.

Full Text

Duke Authors

Cited Authors

  • Freidlin, B; Korn, EL; George, SL; Gray, R

Published Date

  • November 1, 2007

Published In

Volume / Issue

  • 25 / 31

Start / End Page

  • 5019 - 5023

PubMed ID

  • 17971602

Pubmed Central ID

  • 17971602

Electronic International Standard Serial Number (EISSN)

  • 1527-7755

Digital Object Identifier (DOI)

  • 10.1200/JCO.2007.11.8711


  • eng

Conference Location

  • United States