메뉴 건너뛰기




Volumn 12, Issue 1, 2002, Pages 37-58

Proving non-inferiority or equivalence of two treatments with dichotomous endpoints using exact methods

Author keywords

[No Author keywords available]

Indexed keywords

ANTIINFECTIVE AGENT; ANTINEOPLASTIC AGENT; HEPATITIS A VACCINE; IMMUNOGLOBULIN; INFLUENZA VACCINE; PLACEBO;

EID: 0037280386     PISSN: 09622802     EISSN: None     Source Type: Journal    
DOI: 10.1191/0962280203sm314ra     Document Type: Review
Times cited : (53)

References (83)
  • 1
    • 0017643592 scopus 로고
    • Significance testing to establish equivalence between treatments with special reference to data in the form of 2 × 2 tables
    • Dunnett CW, Gent M. Significance testing to establish equivalence between treatments with special reference to data in the form of 2 × 2 tables. Biometrics 1977, 33: 593-602.
    • (1977) Biometrics , vol.33 , pp. 593-602
    • Dunnett, C.W.1    Gent, M.2
  • 2
    • 0020308864 scopus 로고
    • Proving the null hypothesis in clinical trials
    • Blackwelder WC. Proving the null hypothesis in clinical trials. Controlled Clinical Trials 1982; 3: 345-53.
    • (1982) Controlled Clinical Trials , vol.3 , pp. 345-353
    • Blackwelder, W.C.1
  • 4
    • 0024241319 scopus 로고
    • A statistical assessment of clinical equivalence
    • Mau J. A statistical assessment of clinical equivalence. Statistics in Medicine 1988, 7: 1267-77.
    • (1988) Statistics in Medicine , vol.7 , pp. 1267-1277
    • Mau, J.1
  • 5
    • 0025633361 scopus 로고
    • Test statistics and sample size formulae for comparative binomial trials with null hypothesis of non-zero risk difference or non-unity relative risk
    • Farrington CP, Manning G. Test statistics and sample size formulae for comparative binomial trials with null hypothesis of non-zero risk difference or non-unity relative risk. Statistics in Medicine 1990; 9: 1447-54.
    • (1990) Statistics in Medicine , vol.9 , pp. 1447-1454
    • Farrington, C.P.1    Manning, G.2
  • 6
    • 0027973846 scopus 로고
    • Mantel-Haenszel-type tests for testing equivalence or more than equivalence in comparative clinical trials
    • Yanagawa T, Tango T, Hiejima Y. Mantel-Haenszel-type tests for testing equivalence or more than equivalence in comparative clinical trials. Biometrics 1994; 50: 859-64.
    • (1994) Biometrics , vol.50 , pp. 859-864
    • Yanagawa, T.1    Tango, T.2    Hiejima, Y.3
  • 7
    • 0029089457 scopus 로고
    • Sample size determination in stratified trials to establish the equivalence of two treatments
    • Nam JM. Sample size determination in stratified trials to establish the equivalence of two treatments. Statistics in Medicine 1995; 14: 2037-49.
    • (1995) Statistics in Medicine , vol.14 , pp. 2037-2049
    • Nam, J.M.1
  • 8
    • 0029760614 scopus 로고    scopus 로고
    • An alternative to the use of two-sided tests in clinical trials
    • Dunnett CW, Gent M. An alternative to the use of two-sided tests in clinical trials. Statistics in Medicine 1996; 15: 1729-38.
    • (1996) Statistics in Medicine , vol.15 , pp. 1729-1738
    • Dunnett, C.W.1    Gent, M.2
  • 9
    • 0032442459 scopus 로고    scopus 로고
    • Testing a specified difference between proportions
    • Falk RW, Koch GG. Testing a specified difference between proportions. Biometrics 1998; 54: 1602-14.
    • (1998) Biometrics , vol.54 , pp. 1602-1614
    • Falk, R.W.1    Koch, G.G.2
  • 10
    • 0034114838 scopus 로고    scopus 로고
    • Tests for equivalence or non-inferiority between two proportions
    • Chen JJ, Tsong Y, Kang SH. Tests for equivalence or non-inferiority between two proportions. Drug Information Journal 2000; 34: 569-78.
    • (2000) Drug Information Journal , vol.34 , pp. 569-578
    • Chen, J.J.1    Tsong, Y.2    Kang, S.H.3
  • 11
    • 0032581130 scopus 로고    scopus 로고
    • Exact tests of equivalence and efficacy with a non-zero lower bound for comparative studies
    • Chan ISF. Exact tests of equivalence and efficacy with a non-zero lower bound for comparative studies. Statistics in Medicine 1998; 17: 1403-13.
    • (1998) Statistics in Medicine , vol.17 , pp. 1403-1413
    • Chan, I.S.F.1
  • 12
    • 0039165517 scopus 로고    scopus 로고
    • Unconditional non-asymptotic one-sided tests for independent binomial proportions when the interest lies in showing non-inferiority and/or superiority
    • Röhmel J, Mansmann U. Unconditional non-asymptotic one-sided tests for independent binomial proportions when the interest lies in showing non-inferiority and/or superiority. Biometrical Journal 1999; 2: 149-70.
    • (1999) Biometrical Journal , vol.2 , pp. 149-170
    • Röhmel, J.1    Mansmann, U.2
  • 13
    • 84972545970 scopus 로고
    • A survey of exact inference for contingency tables (with discussion)
    • Agresti A. A survey of exact inference for contingency tables (with discussion). Statistical Science 1992; 7: 131-77.
    • (1992) Statistical Science , vol.7 , pp. 131-177
    • Agresti, A.1
  • 14
    • 0028188622 scopus 로고
    • The exact analysis of contingency tables in medical research
    • Mehta CR. The exact analysis of contingency tables in medical research. Statistical Methods in Medical Research 1994; 3: 135-56.
    • (1994) Statistical Methods in Medical Research , vol.3 , pp. 135-156
    • Mehta, C.R.1
  • 15
    • 0004198427 scopus 로고    scopus 로고
    • Cambridge, MA
    • Cytel Software Corporation. StatXact 5. Cambridge, MA, 2001.
    • (2001) StatXact 5
  • 17
    • 84872639295 scopus 로고
    • Significance test for 2 × 2 tables
    • Barnard GA. Significance test for 2 × 2 tables. Biometrika 1947; 34: 123-38.
    • (1947) Biometrika , vol.34 , pp. 123-138
    • Barnard, G.A.1
  • 18
    • 84987224470 scopus 로고
    • Raised conditional level of significance for the 2 × 2-table when testing the equality of two probabilities
    • Boschloo RD. Raised conditional level of significance for the 2 × 2-table when testing the equality of two probabilities. Statistica Neerlandica 1970; 24: 1-35.
    • (1970) Statistica Neerlandica , vol.24 , pp. 1-35
    • Boschloo, R.D.1
  • 19
    • 0000800267 scopus 로고
    • A nonrandomized unconditional test for comparison two proportions in 2 × 2 contingency tables
    • McDonald LL, Davis BM, Milliken GA. A nonrandomized unconditional test for comparison two proportions in 2 × 2 contingency tables. Technometrics 1977; 19: 145-57.
    • (1977) Technometrics , vol.19 , pp. 145-157
    • McDonald, L.L.1    Davis, B.M.2    Milliken, G.A.3
  • 21
    • 0022586210 scopus 로고
    • An exact unconditional test for the 2 × 2 comparative trials
    • Haber M. An exact unconditional test for the 2 × 2 comparative trials. Psychological Bulletin 1986; 99: 129-32.
    • (1986) Psychological Bulletin , vol.99 , pp. 129-132
    • Haber, M.1
  • 23
    • 0023615056 scopus 로고
    • A comparison of the two one-sided tests procedure and the power approach for assessing the equivalence of average bioavailability
    • Schuirmann DJ. A comparison of the two one-sided tests procedure and the power approach for assessing the equivalence of average bioavailability. Journal of Pharmacokinetics and Biopharmaceutics 1987; 15: 657-80.
    • (1987) Journal of Pharmacokinetics and Biopharmaceutics , vol.15 , pp. 657-680
    • Schuirmann, D.J.1
  • 25
    • 0030367916 scopus 로고    scopus 로고
    • Problems in interpreting active control equivalence trials
    • Temple R. Problems in interpreting active control equivalence trials. Accountability in Research 1996; 4: 267-75.
    • (1996) Accountability in Research , vol.4 , pp. 267-275
    • Temple, R.1
  • 27
    • 0032528867 scopus 로고    scopus 로고
    • Practical issues in equivalence trials
    • Ebbutt AF, Frith L. Practical issues in equivalence trials. Statistics in Medicine 1998; 17: 1691-1701.
    • (1998) Statistics in Medicine , vol.17 , pp. 1691-1701
    • Ebbutt, A.F.1    Frith, L.2
  • 28
    • 0032529072 scopus 로고    scopus 로고
    • Therapeutic equivalence investigations: Statistical considerations
    • Röhmel J. Therapeutic equivalence investigations: Statistical considerations. Statistics in Medicine 1998; 17: 1703-14.
    • (1998) Statistics in Medicine , vol.17 , pp. 1703-1714
    • Röhmel, J.1
  • 29
    • 0344672882 scopus 로고    scopus 로고
    • ICH E10 Guideline. Choice of control group and related issues in clinical trials
    • ICH E10 Guideline. Choice of control group and related issues in clinical trials. International Conference on Harmonization (ICH), July 2000.
    • International Conference on Harmonization (ICH), July 2000
  • 31
    • 0034734436 scopus 로고    scopus 로고
    • An approximate unconditional test of non-inferiority between two proportions
    • Kang SH, Chen JJ. An approximate unconditional test of non-inferiority between two proportions. Statistics in Medicine 2000, 19: 2089-2100.
    • (2000) Statistics in Medicine , vol.19 , pp. 2089-2100
    • Kang, S.H.1    Chen, J.J.2
  • 33
    • 0032756788 scopus 로고    scopus 로고
    • Test-based exact confidence intervals for the difference of two binomial proportions
    • Chan ISF, Zhang Z. Test-based exact confidence intervals for the difference of two binomial proportions. Biometrics 1999; 55: 1202-9.
    • (1999) Biometrics , vol.55 , pp. 1202-1209
    • Chan, I.S.F.1    Zhang, Z.2
  • 35
    • 38149147384 scopus 로고
    • Choosing the optimal unconditional test for comparing two independent proportions
    • Martin Andres A, Silva Mato A Choosing the optimal unconditional test for comparing two independent proportions. Computational Statistics and Data Analysis 1994; 17: 555-74.
    • (1994) Computational Statistics and Data Analysis , vol.17 , pp. 555-574
    • Martin Andres, A.1    Silva Mato, A.2
  • 36
    • 0034364626 scopus 로고    scopus 로고
    • Undominated p-value and property C for unconditional one-sided two-sample binomial tests
    • Frick H. Undominated p-value and property C for unconditional one-sided two-sample binomial tests. Biometrical Journal 2000; 42: 715-28.
    • (2000) Biometrical Journal , vol.42 , pp. 715-728
    • Frick, H.1
  • 37
    • 0033566014 scopus 로고    scopus 로고
    • Author's reply on 'Exact tests of equivalence and efficacy with a non-zero lower bound for comparative trials.'
    • Chan ISF. Author's reply on 'Exact tests of equivalence and efficacy with a non-zero lower bound for comparative trials.' Statistics in Medicine 1999; 18: 1735-37.
    • (1999) Statistics in Medicine , vol.18 , pp. 1735-1737
    • Chan, I.S.F.1
  • 38
    • 0001072895 scopus 로고
    • The use of confidence or fiducial limits illustrated in the case of the binomial
    • Clopper CJ, Pearson ES. The use of confidence or fiducial limits illustrated in the case of the binomial. Biometrika 1934; 26: 404-13.
    • (1934) Biometrika , vol.26 , pp. 404-413
    • Clopper, C.J.1    Pearson, E.S.2
  • 39
    • 84972971163 scopus 로고
    • Exact confidence intervals, based on the Z statistic, for the difference between two proportions
    • Soms AP. Exact confidence intervals, based on the Z statistic, for the difference between two proportions. Communications in Statistics, Part B - Simulation and Computation 1989; 18: 1325-41.
    • (1989) Communications in Statistics, Part B - Simulation and Computation , vol.18 , pp. 1325-1341
    • Soms, A.P.1
  • 40
    • 0000695799 scopus 로고
    • Some recent results for exact confidence intervals for the difference between two proportions
    • Soms AP. Some recent results for exact confidence intervals for the difference between two proportions. Communications in Statistics, Part B - Simulation and Computation 1989; 18: 1343-57
    • (1989) Communications in Statistics, Part B - Simulation and Computation , vol.18 , pp. 1343-1357
    • Soms, A.P.1
  • 41
    • 0034869336 scopus 로고    scopus 로고
    • On small-sample confidence intervals for parameters in discrete distributions
    • Agresti A, Min Y. On small-sample confidence intervals for parameters in discrete distributions. Biometrics 2001; 57: 963-71.
    • (2001) Biometrics , vol.57 , pp. 963-971
    • Agresti, A.1    Min, Y.2
  • 42
    • 0037196902 scopus 로고    scopus 로고
    • A quasi-exact method for the confidence intervals of the difference of two independent binomial proportions in small sample cases
    • Chen X. A quasi-exact method for the confidence intervals of the difference of two independent binomial proportions in small sample cases. Statistics in Medicine 2002; 21(6): 943-56.
    • (2002) Statistics in Medicine , vol.21 , Issue.6 , pp. 943-956
    • Chen, X.1
  • 43
    • 0001277701 scopus 로고
    • Small sample confidence intervals for the difference, ratio and odds ratio of two success probabilities
    • Coe PR, Tamhane AC. Small sample confidence intervals for the difference, ratio and odds ratio of two success probabilities. Communications in Statistics, Part B - Simulation and Computation 1993; 22: 925-38.
    • (1993) Communications in Statistics, Part B - Simulation and Computation , vol.22 , pp. 925-938
    • Coe, P.R.1    Tamhane, A.C.2
  • 46
    • 0004075615 scopus 로고    scopus 로고
    • Evaluation of new anti-bacterial medicinal products
    • CPMP EWP. Evaluation of new anti-bacterial medicinal products, 1997.
    • (1997)
  • 49
    • 0030328302 scopus 로고    scopus 로고
    • More powerful tests from confidence interval p-value
    • Berger RL. More powerful tests from confidence interval p-value. American Statistician 1996; 50: 314-18.
    • (1996) American Statistician , vol.50 , pp. 314-318
    • Berger, R.L.1
  • 50
    • 0001372312 scopus 로고
    • The comparison of proportions: A review of significance tests, confidence intervals and adjustments for stratification
    • Gart JJ. The comparison of proportions: A review of significance tests, confidence intervals and adjustments for stratification. Review of the International Statistical Institute 1971; 39: 148-69.
    • (1971) Review of the International Statistical Institute , vol.39 , pp. 148-169
    • Gart, J.J.1
  • 51
    • 0000038809 scopus 로고
    • Practical tests of 2 × 2 contingency tables
    • Liddell D. Practical tests of 2 × 2 contingency tables. The Statistician 1978; 25: 295-304.
    • (1978) The Statistician , vol.25 , pp. 295-304
    • Liddell, D.1
  • 52
    • 0000275179 scopus 로고
    • Exact properties of some exact test statistics for comparing two binomial proportions
    • Storer BE, Kim C. Exact properties of some exact test statistics for comparing two binomial proportions. Journal of the American Statistical Association 1990; 85: 146-55.
    • (1990) Journal of the American Statistical Association , vol.85 , pp. 146-155
    • Storer, B.E.1    Kim, C.2
  • 53
    • 0024500576 scopus 로고
    • How to establish equivalence between treatments: A one-sided clinical trial in paediatric oncology
    • Rodary C, Com-Nougue C, Tournade M. How to establish equivalence between treatments: A one-sided clinical trial in paediatric oncology. Statistics in Medicine 1989; 8: 593-98.
    • (1989) Statistics in Medicine , vol.8 , pp. 593-598
    • Rodary, C.1    Com-Nougue, C.2    Tournade, M.3
  • 54
    • 0027459268 scopus 로고
    • Safety and immunogenicity of a recombinant protein influenza A vaccine in adult human volunteers and protective efficacy against wild-type H1N1 virus challenge
    • Fries LF et al. Safety and immunogenicity of a recombinant protein influenza A vaccine in adult human volunteers and protective efficacy against wild-type H1N1 virus challenge. The Journal of Infectious Diseases 1993; 167: 593-601.
    • (1993) The Journal of Infectious Diseases , vol.167 , pp. 593-601
    • Fries, L.F.1
  • 56
    • 0021548948 scopus 로고
    • Confidence intervals for the ratio of two binomial proportions
    • Koopman PAR. Confidence intervals for the ratio of two binomial proportions. Biometrics 1984; 40: 513-17.
    • (1984) Biometrics , vol.40 , pp. 513-517
    • Koopman, P.A.R.1
  • 57
    • 0022035942 scopus 로고
    • Estimation of a common effect parameter from sparse follow-up data
    • Greenland S, Robins JM. Estimation of a common effect parameter from sparse follow-up data. Biometrics 1985; 41: 55-68.
    • (1985) Biometrics , vol.41 , pp. 55-68
    • Greenland, S.1    Robins, J.M.2
  • 59
    • 0027253889 scopus 로고
    • Sample size and power for prospective analysis of relative risk
    • Blackwelder WC. Sample size and power for prospective analysis of relative risk. Statistics in Medicine 1993; 12: 691-98.
    • (1993) Statistics in Medicine , vol.12 , pp. 691-698
    • Blackwelder, W.C.1
  • 60
    • 0031946427 scopus 로고    scopus 로고
    • Power and sample size for stratified prospective studies using the score method for testing relative risk
    • Nam JM. Power and sample size for stratified prospective studies using the score method for testing relative risk. Biometrics 1998; 54: 331-36.
    • (1998) Biometrics , vol.54 , pp. 331-336
    • Nam, J.M.1
  • 62
    • 0016255002 scopus 로고
    • Power computations for designing comparative poisson trials
    • Gail M. Power computations for designing comparative poisson trials. Biometrics 1974; 30: 231-37.
    • (1974) Biometrics , vol.30 , pp. 231-237
    • Gail, M.1
  • 63
    • 0023154434 scopus 로고
    • Epidemiologic programs for computers and calculators - Exact binomial confidence intervals for the relative risk in follow-up studies with sparsely stratified incidence density data
    • Guess HA, Lydick EG, Small RD, Miller LP. Epidemiologic programs for computers and calculators - Exact binomial confidence intervals for the relative risk in follow-up studies with sparsely stratified incidence density data. American Journal of Epidemiology 1987; 125: 340-47.
    • (1987) American Journal of Epidemiology , vol.125 , pp. 340-347
    • Guess, H.A.1    Lydick, E.G.2    Small, R.D.3    Miller, L.P.4
  • 66
    • 0030023875 scopus 로고    scopus 로고
    • Exact estimates for a rate ratio
    • Martin DO, Austin H: Exact estimates for a rate ratio. Epidemiology 1996; 7: 29-33.
    • (1996) Epidemiology , vol.7 , pp. 29-33
    • Martin, D.O.1    Austin, H.2
  • 67
    • 0034050768 scopus 로고    scopus 로고
    • An exact method for meta-analysis of case-control and follow-up studies
    • Martin DO, Austin H. An exact method for meta-analysis of case-control and follow-up studies. Epidemiology 2000; 11: 255-60.
    • (2000) Epidemiology , vol.11 , pp. 255-260
    • Martin, D.O.1    Austin, H.2
  • 68
    • 0026643021 scopus 로고
    • A controlled trial of a formalin-inactivated hepatitis A vaccine in healthy children
    • Werzberger A et al. A controlled trial of a formalin-inactivated hepatitis A vaccine in healthy children. New England Journal of Medicine 1992; 327: 453-57.
    • (1992) New England Journal of Medicine , vol.327 , pp. 453-457
    • Werzberger, A.1
  • 70
    • 0029743994 scopus 로고    scopus 로고
    • Duration of protection from clinical hepatitis A disease after vaccination with VAQTA®
    • Wiens BL, Bohidar NR, Pigeon JG et al. Duration of protection from clinical hepatitis A disease after vaccination with VAQTA®. Journal of Medical Virology 1996; 49: 235-41.
    • (1996) Journal of Medical Virology , vol.49 , pp. 235-241
    • Wiens, B.L.1    Bohidar, N.R.2    Pigeon, J.G.3
  • 74
    • 84950756067 scopus 로고
    • Computing an exact confidence interval for the common odds ratio in several 2 by 2 contingency tables
    • Mehta CR, Patel NR, Gray R. Computing an exact confidence interval for the common odds ratio in several 2 by 2 contingency tables. Journal of the American Statistical Association 1985; 80: 969-73.
    • (1985) Journal of the American Statistical Association , vol.80 , pp. 969-973
    • Mehta, C.R.1    Patel, N.R.2    Gray, R.3
  • 75
    • 0000575708 scopus 로고
    • Fast computation of exact confidence limits for the common odds ratio in a series of 2 × 2 tables
    • Vollset SE, Hirji KF, Elashoff RM. Fast computation of exact confidence limits for the common odds ratio in a series of 2 × 2 tables. Journal of the American Statistical Association 1991; 86: 404-409.
    • (1991) Journal of the American Statistical Association , vol.86 , pp. 404-409
    • Vollset, S.E.1    Hirji, K.F.2    Elashoff, R.M.3
  • 76
    • 0342809446 scopus 로고
    • Comparison of exact, mid-p, and Mantel-Haenszel confidence intervals for the common odds ratio across several 2 × 2 contingency tables
    • Mehta CR, Walsh SJ. Comparison of exact, mid-p, and Mantel-Haenszel confidence intervals for the common odds ratio across several 2 × 2 contingency tables. The American Statistician 1992; 46: 146-50.
    • (1992) The American Statistician , vol.46 , pp. 146-150
    • Mehta, C.R.1    Walsh, S.J.2
  • 77
    • 0028250757 scopus 로고
    • Combining estimates of the odds ratio: The state of the art
    • Emerson JD. Combining estimates of the odds ratio: The state of the art. Statistical Methods in Medical Research 1994; 3: 157-78.
    • (1994) Statistical Methods in Medical Research , vol.3 , pp. 157-178
    • Emerson, J.D.1
  • 78
    • 0029098036 scopus 로고
    • On the sample size for one-sided equivalence of sensitivities based upon McNemar's test
    • Lu Y, Bean JA. On the sample size for one-sided equivalence of sensitivities based upon McNemar's test. Statistics in Medicine 1995; 14: 1831-39.
    • (1995) Statistics in Medicine , vol.14 , pp. 1831-1839
    • Lu, Y.1    Bean, J.A.2
  • 79
    • 0031467706 scopus 로고    scopus 로고
    • Establishing equivalence of two treatments and sample size requirements in matched-pairs design
    • Nam J. Establishing equivalence of two treatments and sample size requirements in matched-pairs design. Biometrics 1997; 53: 1422-30.
    • (1997) Biometrics , vol.53 , pp. 1422-1430
    • Nam, J.1
  • 80
    • 0032580373 scopus 로고    scopus 로고
    • Equivalence test and confidence interval for the difference in proportions for the paired-sample design
    • Tango T. Equivalence test and confidence interval for the difference in proportions for the paired-sample design. Statistics in Medicine 1998; 17: 891-908.
    • (1998) Statistics in Medicine , vol.17 , pp. 891-908
    • Tango, T.1
  • 81
    • 0032531910 scopus 로고    scopus 로고
    • Assessing screening tests: Extensions of McNemar's test
    • Lachenbruch PA, Lynch CJ. Assessing screening tests: Extensions of McNemar's test. Statistics in Medicine 1998; 17: 2207-17.
    • (1998) Statistics in Medicine , vol.17 , pp. 2207-2217
    • Lachenbruch, P.A.1    Lynch, C.J.2
  • 82
    • 0038753902 scopus 로고    scopus 로고
    • On tests of equivalence via non-unity relative risk for matched-pairs design
    • in press
    • Tang NS, Tang ML, Chan ISF. On tests of equivalence via non-unity relative risk for matched-pairs design. Statistics in Medicine, in press.
    • Statistics in Medicine
    • Tang, N.S.1    Tang, M.L.2    Chan, I.S.F.3
  • 83
    • 0035009459 scopus 로고    scopus 로고
    • Unconditional exact tests for equivalence or noninferiority for paired binary endpoints
    • Hsueh HM, Liu JP, Chen JJ. Unconditional exact tests for equivalence or noninferiority for paired binary endpoints. Biometrics 2001; 57: 478-83.
    • (2001) Biometrics , vol.57 , pp. 478-483
    • Hsueh, H.M.1    Liu, J.P.2    Chen, J.J.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.