Exact tests using binary data in adaptive two or multi-stage designs. (August 2020)

• Record Type: Journal Article
• Authors: Yin, Huan; Wang, Weizhen; Zhang, Zhongzhan
• Is Part Of: Statistical methods in medical research. Volume 29:Number 8(2020)
• Page Start: 2269
• Page End: 2281
• Publication Date: 2020-08
• Subjects: Binomial distribution -- phase II clinical trial -- power -- sample size -- Type I error rate; Medicine -- Research -- Statistical methods -- Periodicals; Research -- Periodicals; Review Literature -- Periodicals; Statistics -- methods -- Periodicals; Médecine -- Recherche -- Méthodes statistiques -- Périodiques; 610.727
• DOI: 10.1177/0962280219892299
• Languages: English
• ISSNs: 0962-2802

Abstract

When establishing an effective treatment with binary data in a two-stage design, one-sided tests for a proportion p are employed. Researchers use the parameter configuration at the boundary of the null hypothesis space to determine a rejection region and an optimal design. However, it is unclear whether the (maximum) Type I error rate is achieved at the boundary especially when the sample size in stage 2 varies. In this paper, we first prove that this is true for a large family of tests in adaptive two-stage designs by showing that any test in the family has a nondecreasing power in p and then derive optimal designs. Second, similar results are established for m -stage designs with m > 2. Supplementary materials for this article are available online.