Type I error and power in noninferiority/equivalence trials with correlated multiple endpoints: An example from vaccine development trials

Lan Kong, Robert C. Kohberger, Gary G. Koch

Research output: Contribution to journalReview article

10 Citations (Scopus)

Abstract

Clinical trials necessary for the development of new treatment often require testing of multiple endpoints for equivalence or noninferiority relative to an existing effective standard therapy. An example is a vaccine study with multiple antibody measurements in sera of subjects receiving a combination vaccine such as a pneumococcal vaccine, which contains many different serotypes of the pneumococcal organism. This article describes testing methods for the demonstration of simultaneous marginal equivalence or noninferiority of two treatments on each component of the response vector that follows a multivariate normal distribution. Systematic simulation studies are conducted to evaluate the performance of the testing method and to examine under what conditions the power is substantially different if the multiple endpoints are assumed to be independent when they are actually strongly correlated. Data from an illustrative example are used to describe how the study power can be evaluated in the design of the trials.

Original languageEnglish (US)
Pages (from-to)893-907
Number of pages15
JournalJournal of Biopharmaceutical Statistics
Volume14
Issue number4
DOIs
StatePublished - Dec 7 2004

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Multiple Endpoints
Non-inferiority
Type I error
Vaccine
Vaccines
Equivalence
Combined Vaccines
Pneumococcal Vaccines
Testing
Normal Distribution
Multivariate Normal Distribution
Clinical Trials
Antibody
Therapy
Antibodies
Serum
Simulation Study
Necessary
Evaluate
Therapeutics

All Science Journal Classification (ASJC) codes

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

Cite this

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Type I error and power in noninferiority/equivalence trials with correlated multiple endpoints : An example from vaccine development trials. / Kong, Lan; Kohberger, Robert C.; Koch, Gary G.

In: Journal of Biopharmaceutical Statistics, Vol. 14, No. 4, 07.12.2004, p. 893-907.

Research output: Contribution to journalReview article

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