Avoiding ambiguity with the Type i error rate in noninferiority trials

Research output: Contribution to journalArticle

Abstract

This review article sets out to examine the Type I error rates used in noninferiority trials. Most papers regarding noninferiority trials only state Type I error rate without mentioning clearly which Type I error rate is evaluated. Therefore, the Type I error rate in one paper is often different from the Type I error rate in another paper, which can confuse readers and makes it difficult to understand papers. Which Type I error rate should be evaluated is related directly to which paradigm is employed in the analysis of noninferiority trial, and to how the historical data are treated. This article reviews the characteristics of the within-trial Type I error rate and the unconditional across-trial Type I error rate which have frequently been examined in noninferiority trials. The conditional across-trial Type I error rate is also briefly discussed. In noninferiority trials comparing a new treatment with an active control without a placebo arm, it is argued that the within-trial Type I error rate should be controlled in order to obtain approval of the new treatment from the regulatory agencies. I hope that this article can help readers understand the difference between two paradigms employed in noninferiority trials.

Original languageEnglish
Pages (from-to)452-465
Number of pages14
JournalJournal of Biopharmaceutical Statistics
Volume26
Issue number3
DOIs
Publication statusPublished - 2016 May 3

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Non-inferiority
Type I Error Rate
Error Rate
Placebos
Therapeutics
Paradigm
Ambiguity
Active Control
Historical Data

All Science Journal Classification (ASJC) codes

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

Cite this

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Avoiding ambiguity with the Type i error rate in noninferiority trials. / Kang, Seung Ho.

In: Journal of Biopharmaceutical Statistics, Vol. 26, No. 3, 03.05.2016, p. 452-465.

Research output: Contribution to journalArticle

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