A more powerful exact test of noninferiority from binary matched-pairs data

Chris J. Lloyd, Max Moldovan

Research output: Contribution to journalArticle

25 Citations (Scopus)

Abstract

Assessing the therapeutic noninferiority of one medical treatment compared with another is often based on the difference in response rates from a matched binary pairs design. This paper develops a new exact unconditional test for noninferiority that is more powerful than available alternatives. There are two new elements presented in this paper. First, we introduce the likelihood ratio statistic as an alternative to the previously proposed score statistic of Nam (Biometrics 1997; 53:1422-1430). Second, we eliminate the nuisance parameter by estimation followed by maximization as an alternative to the partial maximization of Berger and Boos (Am. Stat. Assoc. 1994; 89:1012-1016) or traditional full maximization. Based on an extensive numerical study, we recommend tests based on the score statistic, the nuisance parameter being controlled by estimation followed by maximization.

LanguageEnglish
Pages3540-3549
Number of pages10
JournalStatistics in Medicine
Volume27
Issue number18
DOIs
Publication statusPublished - 15 Aug 2008

Keywords

  • Exact tests
  • Noninferiority
  • Nuisance parameters

ASJC Scopus subject areas

  • Epidemiology
  • Statistics and Probability

Cite this

@article{d89242d6dd03412c972db58523c2a9c3,
title = "A more powerful exact test of noninferiority from binary matched-pairs data",
abstract = "Assessing the therapeutic noninferiority of one medical treatment compared with another is often based on the difference in response rates from a matched binary pairs design. This paper develops a new exact unconditional test for noninferiority that is more powerful than available alternatives. There are two new elements presented in this paper. First, we introduce the likelihood ratio statistic as an alternative to the previously proposed score statistic of Nam (Biometrics 1997; 53:1422-1430). Second, we eliminate the nuisance parameter by estimation followed by maximization as an alternative to the partial maximization of Berger and Boos (Am. Stat. Assoc. 1994; 89:1012-1016) or traditional full maximization. Based on an extensive numerical study, we recommend tests based on the score statistic, the nuisance parameter being controlled by estimation followed by maximization.",
keywords = "Exact tests, Noninferiority, Nuisance parameters",
author = "Lloyd, {Chris J.} and Max Moldovan",
year = "2008",
month = "8",
day = "15",
doi = "10.1002/sim.3229",
language = "English",
volume = "27",
pages = "3540--3549",
journal = "Statistics in Medicine",
issn = "0277-6715",
publisher = "John Wiley and Sons Ltd",
number = "18",

}

A more powerful exact test of noninferiority from binary matched-pairs data. / Lloyd, Chris J.; Moldovan, Max.

In: Statistics in Medicine, Vol. 27, No. 18, 15.08.2008, p. 3540-3549.

Research output: Contribution to journalArticle

TY - JOUR

T1 - A more powerful exact test of noninferiority from binary matched-pairs data

AU - Lloyd, Chris J.

AU - Moldovan, Max

PY - 2008/8/15

Y1 - 2008/8/15

N2 - Assessing the therapeutic noninferiority of one medical treatment compared with another is often based on the difference in response rates from a matched binary pairs design. This paper develops a new exact unconditional test for noninferiority that is more powerful than available alternatives. There are two new elements presented in this paper. First, we introduce the likelihood ratio statistic as an alternative to the previously proposed score statistic of Nam (Biometrics 1997; 53:1422-1430). Second, we eliminate the nuisance parameter by estimation followed by maximization as an alternative to the partial maximization of Berger and Boos (Am. Stat. Assoc. 1994; 89:1012-1016) or traditional full maximization. Based on an extensive numerical study, we recommend tests based on the score statistic, the nuisance parameter being controlled by estimation followed by maximization.

AB - Assessing the therapeutic noninferiority of one medical treatment compared with another is often based on the difference in response rates from a matched binary pairs design. This paper develops a new exact unconditional test for noninferiority that is more powerful than available alternatives. There are two new elements presented in this paper. First, we introduce the likelihood ratio statistic as an alternative to the previously proposed score statistic of Nam (Biometrics 1997; 53:1422-1430). Second, we eliminate the nuisance parameter by estimation followed by maximization as an alternative to the partial maximization of Berger and Boos (Am. Stat. Assoc. 1994; 89:1012-1016) or traditional full maximization. Based on an extensive numerical study, we recommend tests based on the score statistic, the nuisance parameter being controlled by estimation followed by maximization.

KW - Exact tests

KW - Noninferiority

KW - Nuisance parameters

UR - http://www.scopus.com/inward/record.url?scp=48249136982&partnerID=8YFLogxK

U2 - 10.1002/sim.3229

DO - 10.1002/sim.3229

M3 - Article

VL - 27

SP - 3540

EP - 3549

JO - Statistics in Medicine

T2 - Statistics in Medicine

JF - Statistics in Medicine

SN - 0277-6715

IS - 18

ER -