Methods for equivalence and noninferiority testing

Biol Blood Marrow Transplant. 2009 Jan;15(1 Suppl):120-7. doi: 10.1016/j.bbmt.2008.10.004.


Classical hypothesis testing focuses on testing whether treatments have differential effects on outcome. However, sometimes clinicians may be more interested in determining whether treatments are equivalent or whether one has noninferior outcomes. We review the hypotheses for these noninferiority and equivalence research questions, consider power and sample size issues, and discuss how to perform such a test for both binary and survival outcomes. The methods are illustrated on 2 recent studies in hematopoietic cell transplantation.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Hematopoietic Stem Cell Transplantation / methods
  • Hematopoietic Stem Cell Transplantation / standards*
  • Humans
  • Methods
  • Odds Ratio
  • Probability*
  • Research Design
  • Therapeutic Equivalency*
  • Treatment Outcome