SEARCH

SEARCH BY CITATION

Keywords:

  • Exact test;
  • Likelihood ratio test;
  • Optimal sampling ratio;
  • Penalized likelihood;
  • Score test;
  • Wald test

Abstract

Summary

We reexamine the subject of sample size determination (SSD) when testing logarithm of odds ratio (OR) against zero in two independent binomials. Four common approaches are considered: a closed-form SS formula based on the Wald test (inline image), closed-form formulas that meet SS requirement by score and exact tests respectively (inline image and inline image), and a numerical approach to calculating SS based on likelihood ratio (LR) tests (inline image). Several practically useful findings are presented. First, inline image is a strictly convex function of OR for OR inline image and OR inline image, respectively, implying that SS calculated by inline image does not necessarily decrease as OR gets further away from 1. However, minimum SS often occurs at OR values that are deemed relatively extreme and rare in real life. inline image, and inline image decrease monotonically as OR diverges from 1. Secondly, the optimal sampling ratio (OSR) between two independent binomials that yields maximum power for a given total SS is not always 1:1 but depends on the odds of outcome in each arm. inline image benefits the most from the application of OSR in that total SS can be significantly reduced as compared to the commonly used 1:1 sampling ratio. Savings in SS by OSR in inline image, inline image and inline image are relatively immaterial from a practical perspective. Finally, we use simulation studies to examine the power loyalty of each SS approach and explore penalized likelihood as a remedy for undermined power loyalty.