A mixed approach for proving non-inferiority in clinical trials with binary endpoints

Biometrical Journal

Volumn 50, Issue 2, 2008, Pages 190-204

  Rousson, Valentin ^a   Seifert, Burkhardt ^b  

^a UNIVERSITY OF LAUSANNE   (Switzerland)

^b UNIVERSITY OF ZURICH   (Switzerland)

Author keywords

Active control; Non inferiority margin; Odds ratio, difference of proportions; Sample size calculation; Test for non inferiority

Indexed keywords

ACTIVE CONTROL; NON-INFERIORITY; NON-INFERIORITY MARGIN; ODD RATIOS; ODD-RATIO, DIFFERENCE OF PROPORTION; RATIO DIFFERENCES; SAMPLE SIZE CALCULATION; SAMPLE SIZES; SIZE CALCULATIONS; TEST FOR NON-INFERIORITY;

MEDICAL APPLICATIONS;

CLINDAMYCIN;

ARTICLE; BIOASSAY; FEMALE; HUMAN; METHODOLOGY; PHARMACOKINETICS; RANDOMIZED CONTROLLED TRIAL; RISK; SAMPLE SIZE; STATISTICAL ANALYSIS; VAGINITIS;

CLINDAMYCIN; DATA INTERPRETATION, STATISTICAL; ENDPOINT DETERMINATION; FEMALE; HUMANS; ODDS RATIO; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SAMPLE SIZE; THERAPEUTIC EQUIVALENCY; VAGINOSIS, BACTERIAL;

EID: 43249104707     PISSN: 03233847     EISSN: 15214036     Source Type: Journal    
DOI: 10.1002/bimj.200710410     Document Type: Article

References (29)

1. Agresti, A. (1980). Generalized odds-ratio for ordinal data. Biometrics 36, 59-67.
2. Agresti, A. (1990). Categorical Data Analysis. Wiley, New York.
3. Amsel, R., Totten, P. A., Spiegel, C. A., Chen, K. C. S., and Eschenbach, D. A. (1983). Nonspecific vaginitis: Diagnostic criteria and microbial and epidemiologic associations. American Journal of Medicine 74, 14-22.
4. Blackwelder, W. C. (1982). Proving the null hypothesis in clinical trials. Controlled Clinical Trials 3, 345-353.
5. Brunner, E., Domhof, S., and Langer, F. (2002). Nonparametric Analysis of Longitudinal Data in Factorial Experiments. Wiley.
6. Campbell, M. J., Julious, S. A., and Altman, D. G. (1995). Estimating sample sizes for binary, ordered categorical, and continuous outcomes in two group comparisons. British Medical Journal 311, 1145-1148.
7. Chan, I. S. F. (2003). Proving non-inferiority or equivalence of two treatments with dichotomous endpoints using exact methods. Statistical Methods in Medical Research 12, 37-58.
8. Cohen, J. (1987). Statistical Power Analysis for the Behavioral Sciences. Second Edition. Lawrence Erlbaum Associates, Hillsdale.
9. CPMP (2000). Points to consider on switching between superiority and non-inferiority. Available at http://www.emea.europa.eu/pdfs/human/ewp/048299en. pdf [last accessed October 24, 2007].
10. CPMP (2005). Guideline on the choice of the non-inferiority margin. Available at http://www.emea.europa.eu/pdfs/human/ewp/215899en.pdf [last accessed October 24, 2007].
11. Dunnett, C. W. and Gent, M. (1977). Significance testing to establish equivalence between treatments, with special reference to data in the form of 2 x 2 tables. Biometrics 33, 593-602.
12. Edwardes, M. D. (1995). A confidence interval for Pr (X < Y) - Pr (X > Y) estimated from simple cluster samples. Biometrics 51, 571-578.
13. Edwardes, M. D. and Baltzan, M. (2000). The generalization of the odds-ratio, risk ratio and risk difference to r x k tables. Statistics in Medicine 19, 1901-1914.
14. Farrington, C. P. and Manning, G. (1990). 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 9, 1447-1454.
15. FDA (1997). Guidance for industry. Evaluating clinical studies of antimicrobials in the division of anti-infective drug products. Available at http://www.fda.gov/cder/guidance/draft9al.pdf [last accessed October 24, 2007].
16. Garrett, A. D. (2003). Therapeutic equivalence: fallacies and falsification. Statistics in Medicine 22, 741-762.
17. ICH (1998). Guidance for industry. E9 Statistical principles for clinical trials. Available at http://www.fda.gov/cder/guidance/ICH_E9-fnl.pdf [last accessed October 24, 2007].
18. ICH (2001). Guidance for industry. E10 Choice of control groups and related issues in a clinical trial. Available at http://www.fda.gov/cder/ guidance/4155fnl.pdf [last accessed October 24, 2007].
19. Joesoef, M. R., Schmid, G. P., and Hillier, S. L. (1999). Bacterial vaginosis: Review of treatment options and potential clinical indications for therapy. Clinical Infectious Diseases 28 (Suppl. 1), S57-S65.
20. Machin, D., Campbell, M., Fayers, P., and Pinol, A. (1997). Sample Size Tables for Clinical Studies, Second Edition. Blackwell Science.
21. Miettinen, O. and Nurminen, M. (1985). Comparative analysis of two rates. Statistics in Medicine 4, 213-226.
22. Phillips, K. F. (2003). A new test of non-inferiority for anti-infective trials. Statistics in Medicine 22, 201-212.
23. Rodary, C., Com-Nougue, C., and Tournade, M. F. (1989). How to establish equivalence between treatments: A one-sided clinical trial in paediatric oncology. Statistics in Medicine 8, 593-598.
24. Röhmel, J. (2001). Statistical considerations of FDA and CPMP rules for the investigation of new anti-bacterial products. Statistics in Medicine 20, 2561-2571.
25. Schuirmann, D. J. (1987). A comparison of the two one-sided tests procedure and the power approach for assessing the equivalence of bioavailability. Journal of Pharmacokinetics and Biopharmaceutics 15, 657-680.
26. Senn, S. (2000). Consensus and controversy in pharmaceutical statistics (with discussion). Journal of the Royal Statistical Society, Series D, 49, 135-176.
27. Tu, D. (1998). On the use of the ratio or the odds ratio of cure rates in therapeutic equivalence clinical trials with binary endpoints. Journal of Biopharmaceutical Statistics 8, 263-282.
28. Wellek, S. (2003). Testing Statistical Hypotheses of Equivalence. Chapman and Hall.
29. Wellek, S. (2005). Statistical methods for the analysis of two-arm non-inferiority trials with binary outcomes. Biometrical Journal 47, 48-61.