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Sample size formula for a win ratio endpoint.

Ron Xiaolong Yu1, Jitendra Ganju2

  • 1Biostatistics, Gilead Sciences, Foster City, California, USA.

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|January 27, 2022
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Summary
This summary is machine-generated.

A new formula simplifies sample size calculations for win ratio composite endpoints in clinical trials. This method, unlike complex simulations, uses summary data and improves trial design efficiency.

Keywords:
composite endpointsphase 3 clinical trialspowersample size

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Area of Science:

  • Clinical Trials
  • Biostatistics
  • Medical Research

Background:

  • The win ratio composite endpoint is increasingly used in late-stage clinical trials.
  • Current sample size calculations for win ratio endpoints often rely on complex simulations.
  • A simple, accessible formula for sample size determination is lacking in the literature.

Purpose of the Study:

  • To derive and validate a simple formula for calculating sample sizes for win ratio composite endpoints.
  • To compare confidence intervals derived from the new formula with those from patient-level data.
  • To provide insights into trial design and power considerations using the win ratio method.

Main Methods:

  • A novel sample size formula was developed based on probabilities of patient outcomes and ties.
  • The formula's performance was evaluated by comparing confidence intervals with those calculated from patient-level data across 17 endpoints.
  • Simulations were conducted to assess the formula's accuracy and utility.

Main Results:

  • The formula provides accurate confidence intervals comparable to those requiring patient-level data.
  • Simulations demonstrated the formula's effectiveness in sample size calculations.
  • The formula offers insights into how hierarchical endpoint inclusion impacts statistical power.

Conclusions:

  • The developed formula offers a practical and efficient alternative to complex simulations for win ratio sample size calculations.
  • This formula can aid researchers, including non-specialists, in determining appropriate trial sizes.
  • The findings support the broader adoption and understanding of the win ratio method in clinical research.