Sample size calculation in three-arm equivalence trials including a placebo for binary clinical endpoints

When assessing the equivalence between a generic drug (T) and its innovator drug (R) based on clinical endpoints, three-arm equivalence trials that include a placebo (P) are recommended by regulatory agencies whenever ethically justifiable. In such trials, equivalence of T and R is established only if the equivalence test (T vs. R) and both superiority tests (T vs. P and R vs. P) are simultaneously satisfied. Existing sample size calculation methods have typically considered only the equivalence test and one of the superiority tests (T vs. P), overlooking the other superiority test (R vs. P). We propose two methods for sample size calculation in three-arm equivalence trials with binary endpoints. These methods simultaneously account for the equivalence test based on the ratio of two rates and both superiority tests. Additionally, a grid search approach is proposed to determine the optimal allocation ratio for the sample sizes among the three arms.

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Publication Details

Journal
iScience
Published
2026-01-13
DOI
https://doi.org/10.1016/j.isci.2026.114686
Primary Topic
Statistical Methods in Clinical Trials
Type
article
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article

Sample size calculation in three-arm equivalence trials including a placebo for binary clinical endpoints

Shuming Ji, Xinyi Zhao, Fei Yu, Zaiyun Xiao et al.
iScience
Statistical Methods in Clinical Trials
article

Sample size calculation in three-arm equivalence trials including a placebo for binary clinical endpoints

Shuming Ji, Xinyi Zhao, Fei Yu, Zaiyun Xiao, Jie Zhong
article en

Abstract

When assessing the equivalence between a generic drug (T) and its innovator drug (R) based on clinical endpoints, three-arm equivalence trials that include a placebo (P) are recommended by regulatory agencies whenever ethically justifiable. In such trials, equivalence of T and R is established only if the equivalence test (T vs. R) and both superiority tests (T vs. P and R vs. P) are simultaneously satisfied. Existing sample size calculation methods have typically considered only the equivalence test and one of the superiority tests (T vs. P), overlooking the other superiority test (R vs. P). We propose two methods for sample size calculation in three-arm equivalence trials with binary endpoints. These methods simultaneously account for the equivalence test based on the ratio of two rates and both superiority tests. Additionally, a grid search approach is proposed to determine the optimal allocation ratio for the sample sizes among the three arms.

iScienceVol. 29(2)
Sichuan University (CN), West China Medical Center of Sichuan University (CN), West China Hospital of Sichuan University (CN), Sichuan Cancer Hospital (CN)
Peace, Justice and strong institutions
Openalex Percentile: Top 91%
Statistical Methods in Clinical Trials
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