Issues with statistical risks for testing methods in noninferiority trial without a placebo arm

Journal of Biopharmaceutical Statistics

Volumn 17, Issue 2, 2007, Pages 201-213

  Hung, H M James ^a,c   Wang, Sue Jane ^b   O'Neill, Robert ^b  

^a Office of Biostatistics   (United States)

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

^c Division of Biometrics I   (United States)

Author keywords

Across trial or unconditional Type I error rate; Active controlled; Fixed margin method; Noninferiority margin; Placebo controlled; Synthesis method; Within trial or conditional Type I error rate

Indexed keywords

PLACEBO;

ARTICLE; META ANALYSIS; METHODOLOGY; NONINFERIORITY TRIAL DESIGN; PRIORITY JOURNAL; RISK; TECHNIQUE;

CLINICAL TRIALS; CONTROL GROUPS; CONTROLLED CLINICAL TRIALS; DATA INTERPRETATION, STATISTICAL; HUMANS; META-ANALYSIS; MODELS, STATISTICAL; PLACEBOS; RESEARCH DESIGN;

EID: 33947221619     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543400601177343     Document Type: Article

References (44)

1. Blackwelder, W. C. (1982). Proving the null hypothesis in clinical trials. Contr. Clinic. Trials 3:345-353.
2. Blackwelder, W. C. (2002). Showing a treatment is good because it is not bad: when does noninferiority imply effectiveness? Contr. Clinic. Trials 23:52-54.
3. Bucher, H. C., Guyatt, G. H., Griffith, L. E., Walter, S. D. (1997). The results of direct and indirect treatment comparisons in meta-analysis of randomized controlled trials. J. Clinic. Epidemiol. 50:683-691.
4. Chow, S. C., Shao, J. (2006). On noninferiority margin and statistical tests in active control trial. Statistics Med. 25:1101-1113.
5. Committee for Medicinal Products for Human Use (CHMP). (2006). Guideline on the choice of the noninferiority margin. Statistics Med. 25:1628-1638.
6. Committee for Proprietary Medicinal Products (CPMP). (2000). Points to consider on switching between superiority and noninferiority 2000. (Available at http://www.eudra.org/emea.html).
7. D'Agostino, R. B., Massaro, J. M., Sullivan, L. (2003). Non-inferiority trials: design concepts and issues - the encounters of academic consultants in statistics. Statistics Med. 22: 169-186.
8. Department of Health and Human Services, Food and Drug Administration [Docket No. 99D-3082]. (1999). International Conference on Harmonisation: Choice of Control Group in Clinical Trials (E10). Federal Register 64(185):51767-51780.
9. Dunnett, C. W., Tamhane, A. C. (1997). Multiple testing to establish superiority/equivalence of a new treatment compared with kappa standard treatments. Statistics Med. 16: 2489-2506.
10. Ebbutt, A. F., Frith, L. (1998). Practical issues in equivalence trials. Statistics Med. 17: 1691-1701.
11. Ellenberg, S. S., Temple, R. (2000). Placebo-controlled trials and active-control trials in the evaluation of new treatments - Part 2: Practical issues and specific cases. Ann. Inter. Med. 133:464-470.
12. Fleming, T. R. (1987). Treatment evaluation in active control studies. Cancer Treat. Rep. 71:1061-1064.
13. Fleming, T. R. (2000). Design and interpretation of equivalence trials. Am. Heart J. 139: S171-S176.
14. Gould, A. L. (1991). Another view of active-controlled trials. Contr. Clinic. Trials 12:474-485.
15. Hasselblad, V., Kong, D. F. (2001). Statistical methods for comparison to placebo in active-control trials. Drug Inf. J. 35:435-449.
16. Hauschke, D. (2005). Choice of delta: a special case. Drug Inf. J. 35:875-879.
17. Hauschke, D., Pigeot, I. (2005). Establishing efficacy of a new experimental treatment in the Gold Standard design (with discussions). Biomet. J. 47:782-798.
18. 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.
19. Hung, H. M. J., Wang, S. J. (2004). Multiple testing of noninferiority hypotheses in active controlled trials. J. Biopharm. Stat. 14:327-335.
20. Hung, H. M. J., Wang, S. J., Tsong, Y., Lawrence, J., O'Neill, R. T. (2003). Some fundamental issues with noninferiority testing in active controlled clinical trials. Statistical Med. 22:213-225.
21. Hung, H. M. J., Wang, S. J., O'Neill, R. A. (2005). Regulatory perspective on choice of margin and statistical inference issue in noninferiority trials. Biomet. J. 47:28-36.
22. International Conference on Harmonization: guidance on choice of control group and related design and conduct issues in clinical trials (ICH E-10), Food and Drug Administration, DHHS, July 2000.
23. Jones, B., Jarvis, P., Lewis, J. A., Ebbutt, A. F. (1996). Trials to assess equivalence: the importance of rigorous methods. British Med. J. 313:36-39.
24. Laster, L. L., Johnson, M. F. (2003). Non-inferiority trials: the at least as good as criterion. Statistics Med. 22:187-200.
25. Laster, L. L., Johnson, M. F., Kotler, M. L. (2006). Non-inferiority trials: the at least as good as criterion with dichotomous data. Statistics Med. 25:1115-1130.
26. Lawrence, J. (2005). Some remarks about the analysis of active control studies. Biomet. J. 47:616-622.
27. Morikawa, T., Yoshida, M. A (1995). Useful testing strategy in phase III trials: combined test of superiority and test of equivalence. J. Biopharm. Stat. 5:297-306.
28. Ng, T. H. (2001). Choice of delta in equivalence testing. Drug Inf. J. 35:1517-1527.
29. 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.
30. Rohmel, J. (1998). Therapeutic equivalence investigations: statistical considerations. Statistics Med. 17:1703-1714.
31. Rothmann, M., Li, N., Chen, G., Chi, G. Y. H., Temple, R. T., Tsou, H. H. (2003). Noninferiority methods for mortality trials. Statistics Med. 22:239-264.
32. Simon, R. (1999). Bayesian design and analysis of active control clinical trials. Biometrics 55:484-487.
33. Snapinn, S. M. (2004). Alternatives for discounting in the analysis of noninferiority trials. J. Biopharm. Stat. 14:263-273.
34. Temple, R. (1987). Difficulties in evaluating positive control trials. Proceedings of the Biopharmaceutical Section of American Statistical Association. 1-7.
35. Temple, R. (1996). Problems in interpreting active control equivalence trails. Account. Res. 4:267-275.
36. 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. Ann. Internal Med. 133:455-463.
37. Tsong, Y., Wang, S. J., Hung, H. M. J., Cui, L. (2003). Statistical issues on objective, design and analysis of noninferiority active controlled clinical trial. J. Biopharm. Stat. 13:29-42.
38. Wang, S. J., Hung, H. M. J. (2003). Assessment of treatment efficacy in noninferiority trials. Contr. Clinic. Trials 24:147-155.
39. Wang, S. J., Hung, H. M. J., Tsong, Y. (2002). Utility and pitfall of some statistical methods in active controlled clinical trials. Contr. Clinic. Trials 23:15-28.
40. Wang, S. J., Hung, H. M. J., Tsong, Y. (2003). Noninferiority analysis in active controlled clinical trials. Encyclopedia of Biopharmaceutical Statistics. 2nd ed. New York: Marcel Dekker, pp. 674-677.
41. Wang, Y. C., Chen, G., Chi, G. (2006). A ratio test in active control noninferiority trials with a time-to-event endpoint. J. Biopharm. Stat. 16:151-164.
42. Wang, S. J., Hung, H. M. J., Tsong, Y., Cui, L., Nuri, W. (1997). Changing the study Objective in clinical trials. In: Proceedings of the Biopharmaceutical Section of American Statistical Association, pp. 64-69.
43. Wiens, B. (2002). Choosing an equivalence limit for noninferiority or equivalence studies. Contr. Clinic. Trials 23:2-14.
44. World Medical Association Declaration of Helsinki (1997). Recommendations guiding physicians in biomedical research involving human subject. J. Am. Med. Assoc. 277:925-926.