Paired versus two-sample design for a clinical trial of treatments with dichotomous outcome: power considerations

Biometrics. 1982 Sep;38(3):801-12.

Abstract

For the same number of observations in a small-sample clinical trial with dichotomous outcome, the statistical power associated with a two-sample design, analyzed by Fisher's exact test, is slightly greater than that associated with a matched design, analyzed by McNemar's test, and hence of the matched design, is monotone increasing in the within-pair correlation between the treatment responses. Power curves are presented which demonstrate that positive within-pair correlation, even when quite small, can result in a superiority in power for the matched design. Conversely, in the rare situations where there is a negative within-pair correlation, choice of a two-sample design can result in a substantial gain in power.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Clinical Trials as Topic*
  • Humans
  • Models, Biological
  • Probability
  • Research Design*
  • Statistics as Topic