The utility of active-controlled noninferiority/equivalence trials in drug development

International Journal of Pharmaceutical Medicine

Volumn 21, Issue 3, 2007, Pages 225-233

  Tsong, Yi ^a,b  

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

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

Author keywords

[No Author keywords available]

Indexed keywords

GENERIC DRUG; PLACEBO;

BIOASSAY; BIOEQUIVALENCE; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; DRUG MANUFACTURE; DRUG SCREENING; FOOD AND DRUG ADMINISTRATION; PRIORITY JOURNAL; REVIEW; SAMPLE SIZE;

EID: 33947256217     PISSN: 13649027     EISSN: 14735679     Source Type: Journal    
DOI: 10.2165/00124363-200721030-00005     Document Type: Review

References (20)

1. Schuirmann DJ. A comparison of the two one-sided tests procedure and the power approach for assessing the equivalence of average bioavailability. J Pharmacokinet Biopharm 1987; 15: 657-80
2. Tsong Y, Zhang JJ, Wang S-J. Group sequential design and analysis of clinical equivalence assessment for generic nonsystemic drug products. J Biopharm Stat 2004; 14: 359-73
3. Ellenberg SS, Temple R. Placebo-controlled trials and active-controlled trials in the evaluation of new treatment. Part 2: practical issues and scientific cases. Ann Intern Med 2000; 133 (6): 464-70
4. Temple R, Ellenberg SS. Placebo-controlled trials and active-control trials in the evaluation of new treatments. Part 1: ethical and scientific issues. Ann Intern Med 2000; 133 (6): 455-63
5. Hauschke D, Kieser M, Diletti E, et al. Sample size determination for proving equivalence based on the ratio of two means for normally distributed data. Stat Med 1999; 18: 93-105
6. Pigeot I, Schäfer J, Röhmel J, et al. Assessing non-inferiority of a new treatment in a three-arm clinical trial including a placebo. Stat Med 2003; 22: 883-99
7. Committee for Medicine Products for Human (CHMP). Guidance on the choice of the non-inferiority margin. CPMP/EWP/2158/99 [online]. Available from URL: http://www.emea.eu.int/pdfs/human/ewp/215899en.pdf [Accessed 2007 Jan 26]
8. Tsong Y, Wang S-J, Hung H-M, et al. Statistical issues on objective, design and analysis of noninferiority active-controlled clinical trial. J Biopharm Stat 2003; 13: 29-41
9. Hung H-MJ, Wang S-J, Tsong Y, et al. Some fundamental issues for noninferiority testing in active controlled trials. Stat Med 2003; 22: 213-25
10. ICH harmonised tripartite guideline. Statistical principles for clinical trials: E9 [online]. Available from URL: http://www.ich.org/LOB/media/MEDIA485. pdf [Accessed 2007 Jan 31]
11. ICH harmonised tripartite guideline. Choice of control group and related issues in clinical trials: E10 [online]. Available from URL: http://www.ich.org/LOB/media/MEDIA486.pdf [Accessed 2007 Jan 31]
12. Tsong Y, Higgins K, Wang S-J, et al. An overview of equivalence testing: CDER reviewers' perspective. Proceedings of the Biopharmaceutical Section of the Joint Statistics Meetings of the American Statistical Association. 1999, 219
13. Holmgren EB. Establishing equivalence by showing that a specified percentage of the effect of the active control over placebo is maintained. J Biopharm Stat 1999; 9: 651-9
14. Snapinn SM. Alternatives of discounting in the analysis of noninferiority trials. J Biopharm Stat 2004; 14: 263-73
15. 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. Stat Med 1990; 9: 1447-54
16. Tsong Y, Levenson M, Zhang, J. Choice of λ and the dependence of non-inferiority active controlled clinical trials. Stat Med. In press
17. Tsong Y, Zhang J, Levenson M. Choice of δ and the dependence of non-inferiority active controlled clinical trials. J Biopharm Stat 2007: 17 (2): 279-88
18. Morikawa T, Yoshida M. A useful testing strategy in phase III trials: combined test of superiority and test of equivalence. J Biopharm Stat 1995; 5: 297-306
19. Tsong Y, Zhang JJ, et al. Testing superiority and non-inferiority hypotheses in active controlled clinical trials. Biometrical J 2005; 47 (1): 62-74
20. Tsong, Y, Zhang JJ. Simultaneous test for superiority and non-inferiority hypotheses in active controlled clinical trials. J Biopharm Stat 2007: 17 (2): 247-57