The effects of outcome misclassification and measurement error on the design and analysis of therapeutic equivalence trials

Mimi Kim, Judith D. Goldberg

Research output: Contribution to journalArticle

22 Citations (Scopus)

Abstract

In any clinical trial, the use of imperfect diagnostic procedures or laboratory techniques may lead to misclassification and measurement error in the primary outcome. Although the effects of non-differential outcome misclassification and measurement error on conventional superiority trials have been extensively investigated, less is known about the impact of these errors on the results and interpretation of therapeutic equivalence trials. In this paper we formally investigate the effects of outcome misclassification and measurement error on the estimates of treatment effects, type I error rate, and power of equivalence trials. Our results indicate that, contrary to what one may expect based on the well known attenuating effects of non-differential error in conventional studies, these errors do not always favour the goal of demonstrating equivalence. The magnitude and direction of the influence depend on a number of factors including the nature of the outcome variable, specific formulation of equivalence, size of the error rates, and assumptions regarding the true treatment effect.

Original languageEnglish (US)
Pages (from-to)2065-2078
Number of pages14
JournalStatistics in Medicine
Volume20
Issue number14
DOIs
StatePublished - Jul 30 2001
Externally publishedYes

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Misclassification Error
Measurement Error
Equivalence
Treatment Effects
Clinical Trials
Type I Error Rate
Therapeutics
Imperfect
Error Rate
Diagnostics
Design
Formulation
Estimate
Direction compound

ASJC Scopus subject areas

  • Epidemiology

Cite this

The effects of outcome misclassification and measurement error on the design and analysis of therapeutic equivalence trials. / Kim, Mimi; Goldberg, Judith D.

In: Statistics in Medicine, Vol. 20, No. 14, 30.07.2001, p. 2065-2078.

Research output: Contribution to journalArticle

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