Noninferiority testing beyond simple two-sample comparison

Journal of Biopharmaceutical Statistics

Volumn 17, Issue 2, 2007, Pages 289-308

  Tsong, Yi ^a,b   Chen, Wen Jen ^a  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^b Office of Biostatistics   (United States)

Author keywords

Cross trial comparison; Generalized historical control; Noninferiority test

Indexed keywords

ANAMNESIS; ARTICLE; CLINICAL TRIAL; COMPARATIVE STUDY; CROSSOVER PROCEDURE; DATA ANALYSIS; MATHEMATICAL COMPUTING; METHODOLOGY; NONINFERIORITY TRIAL; PRIORITY JOURNAL; SAMPLING; STANDARD; STATISTICAL ANALYSIS;

CONFIDENCE INTERVALS; CONTROLLED CLINICAL TRIALS; DATA INTERPRETATION, STATISTICAL; DRUG EVALUATION; HUMANS; MODELS, STATISTICAL; RESEARCH DESIGN; SAMPLE SIZE;

EID: 33947206730     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543400601177368     Document Type: Article

References (44)

1. Chen, L., Tsong, Y. (2004). Letter to editor. Stat. Med. 23:3713-3721.
2. D'Agostino Sr, R. B., Massaro, J. M., Sullivan, L. M. (2003). Noninferiority trials: design concepts and issues-the encounters of academic consultants in statistics. Stat. Med. 22:169-186.
3. Dunnet, C. W., Gent, M. (1977). Significance testing to establish equivalence between treatments, with special reference to data in the form of 2 x 2 tables. Biometric. J. 33:593-602.
4. Durrleman, S., Simon, R. (1990). Planning and monitoring of equivalence studies. Biometric. J. 46:329-336.
5. Durreleman, S., Chaikin, P. (2003). The use of putative placebo in active control trials: two applications in a regulatory setting. Stat. Med. 22:941-952.
6. Ellenberg, S. S., Temple, R. (2000). Placebo-controlled trials and active controlled trials in the evaluation of new treatment. Part 2: practical issues and scientific issues. Annals Intern. Med. 133:455-463.
7. Fisher, L. D., Gent, M., Buller, H. R. (2001). Active-control trials: How would a new agent compare with placebo? A method illustrated with clopidogrel, aspirin, and placebo. Am. Heart J. 141:26-32.
8. Garrett, A. D. (2003). Therapeutic equivalence: fallacies and falsification. Stat. Med. 22:741-762.
9. Hasselblad, V., Kong, D. F. (2001). Statistical methods for comparison to placebo in active-control trials. Drug Inf. J. 35:435-449.
10. Hauck, W. W., Anderson, S. (1999). Some issues in the design and analysis of equivalence trials. Drug Inf. J. 33:109-118.
11. Holmgren, E. B. (1999). Establishing equivalence by showing that a prespecified percentage of the effect of the active control over placebo is maintained. J. Biopharm. Stat. 9:651-659.
12. Hung, H.-M. J., Wang, S. J. (2004). Multiple testing of noninferiority hypotheses in active control trials. J. Biopharm. Stat. 14:327-335.
13. Hung, H.-M. J., Wang, S.-J., Tsong, Y., Lawrence, J., O'Neill, R. T. (2003). Some fundamental issues for noninferiority testing in active controlled trials. Stat. Med. 22:213-225.
14. Hung, H. M., Wang, S. J., O'Neill, R. T. (2006). Issues with statistical risks for testing methods in noninferiority trial without a placebo arm. J. Biopharm. Stat. 17(2):201-213.
15. Iglewicz, B., Wiens, B. L. (2002). Multi-treatment equivalence trials: design and analysis considerations. Proceedings of the Biopharm. Section, Joint Stat. Meetings, Am. Stat. Assoc., pp. 1577-1581, ASA.
16. Jones, B., Jarvis, P., Lewis, J. A., Ebbutt, A. F. (1996). Trials to assess equivalence: the importance of rigorous methods. Br. Med. J. 313:36-39.
17. Li, H., Tsong, Y. (2003). Adaptive group sequential methods for clinical trials with high rate of sample size loss. Proceedings of the Biopharm. Section, Joinst Stat. Meetings, Am. Stat. Assoc. CD version, ASA.
18. Mau, J. (1988). A statistical assessment of clinical equivalence. Stat. Med. 7:1267-1277.
19. Morikawa, T., Yoshida, M. (1995). A useful testing strategy in phase III trials: combined test of superiority and test of equivalence. J. Biopharm. Stat. 5:297-306.
20. Phillips, K. F. (2004). A new test of noninferiority for anti-infective trials. Stat. Med. 22:201-212.
21. Pigeot, I., Schäfer, J., Röhmel, J., Hauschke, D. (2003). Assessing noninferiority of a new treatment in a three-arm clinical trial including a placebo. Stat. Med. 22:883-899.
22. Pledger, G., Hall, D. B. (1990). Active control equivalence studies: do they address the efficacy issue? Statistical Issues in Drug Research and Development. New York: Marcel Dekker, pp. 226-238.
23. Quan, H., Shih, W. (2002). Treatment by stratum interaction test for equivalence and noninferiority trials. Proceedings of the Biopharm. Section, Joint Stat. Meetings, Am. Stat. Assoc., CD Version, ASA.
24. Quan, H., Bolognese, J., Yuan, W. (2001). Assessment of equivalence on multiple endpoints. Stat. Med. 20:159-3173.
25. Quan, H., Zhang, J. (2003). Estimate of standard deviation of a log-transformed variable using arithmatic means and variations. Stat. Med. 22:2723-2736.
26. Rothman, M. D., Tsou, H.-H. (2003). On noninferiority analysis based on delta-method confidence intervals. J. Biopharm. Stat. 13:565-583.
27. Rothman, M., Li, N., Chen, G., Chi, G., Tsou, H.-H. (2003). Design and analysis of noninferiority mortality trials in oncology. Stat. Med. 22:239-264.
28. Satterthwaite, F. E. (1946). An approximate distribution of estimates of variance components. Biometric. Bull. 2:110-114.
29. Shih, W. J., Quan, H., Li, G. (2004). Two-stage adaptive strategy for superiority and noninferiority hypotheses in active controlled clinical trials. Stat. Med. 23:2781-2798.
30. Snapinn, S. M. (2004). Alternatives of discounting in the analysis of noninferiority trials. J. Biopharm. Stat. 14:263-273.
31. Temple, R. (1996). Problems in interpreting active control equivalence trials. Accountability in Research 4:267-275.
32. Temple, R., Ellenberg, S. S. (2000). Placebo-controlled trials and active-control trials in the evaluation of new treatments - Part 1: Ethical and Scientific Issues, Part 2: ethical and scientific issues. Annals Intern. Med. 133:455-463.
33. Tsong, Y. (2007). The utility of active-controlled noninferiority/ equivalence trials in drug development. Int. J. Pharm. Med. (accepted)
34. Tsong, Y., Zhang, J. J. (2005). Testing superiority and noninferiority hypotheses in active controlled clinical trials. Biometric. J. 47(1):62-74.
35. Tsong, Y., Zhang, J. J. (2006). Simultaneous test for superiority and noninferiority hypotheses in active controlled clinical trials. J. Biopharm. Stat. 17(2):309-327.
36. Tsong, Y., Wang, S.-J., Hung, H.-M. J. (2003). Statistical issues in objectives, designs and analysis of noninferiority test active controlled clinical trials. J. Biopharm. Stat. 13:29-41.
37. Tsong, Y., Zhang, J. J., Wang, S.-J. (2004). Group sequential design and analysis of clinical equivalence assessment for generic non-systemic drug products. J. Biopharm. Stat. 14:359-373.
38. Tsong, Y., Zhang, J., Levenson, M. (2007a). Choice of δ and the dependence of noninferiority active controlled clinical trials. J. Biopharm. Stat. 17(2):279-288.
39. Tsong, Y., Levenson, M., Zhang, J. (2007b). Choice of and the dependence of noninferiority active controlled clinical trials. Stat. in Med. (accepted).
40. Tsong, Y., Higgins, K., Wang, S.-J., Hung, H.-M. J. (1999). An overview of equivalence testing - CDER reviewers' perspective. Proceedings of the Biopharm. Section, Joint Stat. Meetings, Am. Stat. Assoc., pp. 214-219.
41. Wang, S.-J., Hung, H.-M. J., Tsong Y. (2002). Utility and pitfall of some statistical methods in active controlled clinical trials. Cont. Clin. Trials 23:15-28.
42. Wang, S.-J., Hung, H.-M. J., Tsong, Y., Cui, L., Nuri, W. A. (2001). Group sequential test strategies for superiority and noninferiority hypotheses in active controlled clinical trials. Stat. Med. 20:1903-1912.
43. Wiens, B. L., Fiola, M. (2002). Equivalence of multiple treatments with a control: design and analysis issues. Proceedings of the Biopharm. Section, Am. Stat. Assoc. pp. 3709-3711.
44. Wiens, B. L., Heyes, J. F. (2003), Testing for interactions in studies of noninferiority. J. Biopharm. Stat. 13:103-115.