Sample size and power analysis for endometrial safety studies

J Biopharm Stat. 2005;15(3):491-9. doi: 10.1081/BIP-200056550.

Abstract

Endometrial safety studies are required for the approval of progestin components. The Committee for Proprietary Medicinal Products requirement is the actual percentage below 2% and the upper limit of the one-sided exact 95% confidence interval not more than 2% above the point estimate. The more recent U.S. Food and Drug Administration requirement is the actual percentage < or = 1% and the upper limit of the one-sided exact 95% confidence interval < or =4%. I studied the sample size and power needed to satisfy both requirements based on the exact confidence intervals for the binomial parameter and the Poisson parameter. I discovered that a larger sample size does not always lead to a higher power. I presented a best sample size that satisfies both requirements and recommended that the patient enrollment should be closely monitored during the study.

MeSH terms

  • Aged
  • Algorithms
  • Data Interpretation, Statistical
  • Endometrial Neoplasms / chemically induced*
  • Endometrial Neoplasms / epidemiology*
  • Estrogen Replacement Therapy / adverse effects*
  • Female
  • Humans
  • Middle Aged
  • Poisson Distribution
  • Risk Assessment
  • Sample Size