Statistical issues on objective, design, and analysis of noninferiority active-controlled clinical trial

Journal of Biopharmaceutical Statistics

Volumn 13, Issue 1, 2003, Pages 29-41

  Tsong, Yi ^a   Wang, Sue Jane ^a   Hung, H M James ^a   Cui, Lu ^b  

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

^b SANOFI   (France)

Author keywords

Constancy assumption; Cross study comparison; Historical control; Unbiased estimate

Indexed keywords

NEW DRUG; PLACEBO;

ARTICLE; BIOSTATISTICS; CLINICAL RESEARCH; CLINICAL TRIAL; DRUG EFFICACY; DRUG RESEARCH; DRUG SCREENING; HYPOTHESIS; INTERMETHOD COMPARISON; METHODOLOGY; PRIORITY JOURNAL; SAMPLE SIZE; STATISTICAL ANALYSIS; STATISTICAL PARAMETERS;

CONTROLLED CLINICAL TRIALS; DATA INTERPRETATION, STATISTICAL; EPIDEMIOLOGIC RESEARCH DESIGN; MODELS, STATISTICAL; PLACEBOS;

EID: 0037285074     PISSN: 10543406     EISSN: None     Source Type: Journal    
DOI: 10.1081/BIP-120017724     Document Type: Article

References (36)

1. Blackwelder, W. C. (1982). Proving the null hypothesis in clinical trials. Control. Clin. Trials 3:345-353.
2. Blackwelder, W. C. (2002). Commentary-showing a treatment is good because it is not bad: when does "non-inferiority" imply effectiveness? Control. Clin. Trials 23:52-54.
3. Bucher, H. C., Guyatt, G. H., Griffith, L. E., et al. (1997). The results of direct and indirect treatment comparisons in meta-analysis of randomized controlled trials. J. Clin. Epidemiol. 50:683-691.
4. CBER/FDA Memorandum (June 1999). Summary of CBER considerations on selected aspects of active controlled trial design and analysis for the evaluation of thrombolytics in acute MI.
5. Durrleman, S., Simon, R. (1990). Planning and monitoring of equivalence studies. Biometrics 46:329-336.
6. Ebbutt, A. F., Frith, L. (1998). Practical issues in equivalence trials. Stat. Med. 17:1691-1701.
7. FDA (1992). Guidance on Statistical Procedure for Bioequivalence Studies Using Two-Treatment Crossover Design. Rockville, MD: Division of Bioequivalence, Office of Generic Drugs, Center for Drug Evaluation and Research, Food and Drug Administration.
8. Fisher, L. D. (1998). Active control trials: what about a placebo? A method illustrated with clopidogrel, aspirin and placebo. J. Am. Coll. Cardiol. 31:49A (abstract).
9. Fisher, L. D., Gent, M., Bηller, 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.
10. Fleming, T. R. (1987). Treatment evaluation in active control studies. Cancer Treat. Rep. 71(11):1061-1065.
11. Fleming, T. R. (1990). Evaluation of active control trials in AIDS. J. Acquir. Immune. Defic. Syndr. 3(Suppl 2):S82-S87.
12. Fleming, T. R. (2000). Design and interpretation of equivalence trials. Am. Heart J. 139:S171-S176.
13. Hasselblad, V., Kong, D. F. (2001). Statistical methods for comparison to placebo in active-control trials. Drug Inf. J. 35:435-449.
14. Hauck, W. W., Anderson, S. (1999). Some issues in the design and analysis of equivalence trials. Drug Inf. J. 33:109-118.
15. 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(4):651-659.
16. Hung, H. M. J., Wang, S. J., Tsong, Y., Lawrence, J., O'Neill, R. T. (2001). Some fundamental issues for non-inferiority testing in active controlled trials. Proceedings of Biopharmaceutical Section of American Statistical Association.
17. ICH Harmonised Tripartite Guidance (1999). Statistical principles for clinical trials, international conference on harmonisation of technical requirements for registration of pharmaceuticals for human use. Stat. Med. 18:1905-1942.
18. International Conference on Harmonisation (1998). Statistical principles for clinical trials (ICH E-9), Food and Drug Administration, DHHS.
19. 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.
20. Li, N., Rothman, M., Chen, G., Chi, G. (2001). Application of non-inferiority analysis in oncology NDA. Proceedings of Biopharmaceutical Section of American Statistical Association.
21. Mau, J. (1988). A statistical assessment of clinical equivalence. Stat. Med. 7:1267-1277.
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. Rohmel, J. (1998). Therapeutic equivalence investigations: statistical considerations. Stat. Med. 17:1703-1714.
24. Rothman, M., Li, N., Chen, G., Chi, G., Tsou, H. H. (2001). Non-inferiority methods for mortality trials. Proceedings of Biopharmaceutical Section of American Statistical Association.
25. Schuirmann, D. J. (1987). A comparison of the two one-sided tests procedure and the power approach for assessing the equivalence of average bioavailability. J. Pharm. Biopharm. 15:657-680.
26. Siegel, J. P. (2000). Equivalence and noninferiority trials. Am. Heart J. 139:S 166-S170.
27. Snappin, S. (2001). Alternatives for discounting historical data in the analysis of non-inferiority trials. International Chinese Statistical Association Bulletin. pp. 29-30.
28. Temple, R. (1983). Difficulties in Evaluating Positive Control Trials. Proceedings of the Biopharmaceutical Section, American Statistical Association. pp. 1-7.
29. Temple, R. (1996). Problems in interpreting active control equivalence trials. Accountability Res. 4:267-275.
30. 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. Ann. Intern. Med. 133:455-463.
31. Tsong, Y., Higgins, K., Wang, S. J., Hung, H. M. J. (1999). An overview of equivalence testing - CDER reviewers' perspective. Proceedings of the Biopharmaceutical Section of American Statistical Association. pp. 214-219.
32. Tsong, Y., Wang, S. J. Hung, H. M. J., Cui, L. (2001). Objectives, designs and analysis of non-inferiority active controlled clinical trial. Proceedings of Biopharmaceutical Section of American Statistical Association.
33. Wang, S. J., Hung, H. M. J., Tsong, Y. (2002). Utility and pitfall of some statistical methods in active controlled clinical trials. Control. Clin. Trials 23:15-28.
34. Wang, S. J., Hung, H. M. J., Tsong, Y., Cui, L., Nuri, W. A. (2001). Group sequential test strategies for superiority and non-inferiority hypotheses in active controlled clinical trials. Stat. Med. 20:1903-1912.
35. Wiens, B. L. (2002). Choosing an equivalence limit for noninferiority or equivalence studies. Control. Clin. Trials 23:2-14.
36. Wittes, J. (2001). Active-control trials: a linguistic problem. International Statistical Association Bulletin. pp. 39-40.