A comparison of multiple testing procedures for the gold standard non-inferiority trial

Journal of Biopharmaceutical Statistics

Volumn 20, Issue 5, 2010, Pages 911-926

  Rohmel, Joachim ^a   Pigeot, Iris ^b  

^a Universität Bremen ^*  (Germany)

^b UNIVERSITY OF BREMEN   (Germany)

Author keywords

3 Arm parallel group design; Assay sensitivity; Duality of test decisions and confidence bounds; Multiple type I error control

Indexed keywords

ARTICLE; COMPARATIVE STUDY; CONFIDENCE INTERVAL; MATHEMATICAL MODEL; NULL HYPOTHESIS; PRIORITY JOURNAL; SAMPLE SIZE; SIMULATION; STATISTICAL ANALYSIS;

ALGORITHMS; BIAS (EPIDEMIOLOGY); BIOSTATISTICS; COMPUTER SIMULATION; CONFIDENCE INTERVALS; CONTROLLED CLINICAL TRIALS AS TOPIC; HUMANS; RESEARCH DESIGN; SAMPLE SIZE; TREATMENT OUTCOME;

EID: 77955904711     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543401003618942     Document Type: Article

References (21)

1. Bauer, P., Kieser, M. (1996). A unifying approach for confidence intervals and testing of equivalence and difference. Biometrika 83:934-937.
2. Bauer, P., Röhmel, J., Maurer, W., Hothorn, L. A. (1998). Testing strategies in multi-dose experiments including active control. Stat. Med. 17:2133-2146.
3. Committee for Proprietary Medicinal Products. (2000). International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use. Choice of Control Group in Clinical Trials. CPMP/ICH/364/96. http://www. emea.europa.eu/pdfs/human/ich/036496en.pdf, accessed at September 29, 2008.
4. Committee for Proprietary Medicinal Products. (2001). Points to Consider on Application with 1. Meta-Analyses; 2. One Pivotal Trial. CPMP/EWP/2330/99. http://www.emea. europa.eu/pdfs/human/ewp/233099fen.pdf, accessed September 29, 2008.
5. Committee for Proprietary Medicinal Products. (2002). Note for Guidance on Clinical Investigation of Medicinal Products in the Treatment of Depression. CPMP/EWP/ 518/97 Revision 1 (CPMP adopted April 2002). http://www.emea.europa. eu/pdfs/ human/ewp/051897en.pdf, accessed September 29, 2008.
6. Dmitrienko, A., Tamhane, A. C., Wiens, B. L. (2008). General multistage gatekeeping procedures. Biometrical J. 50:667-677.
7. Farrington, C. P., Manning, G. (1990). Test statistics and sample size formulae for comparative binomial trials with null hypothesis of non-zero risk difference or nonunit relative risk. Stat. Med. 9:1447-1454.
8. Guilbaud, O. (2008). Simultaneous confidence regions corresponding to Holm's step-down procedure and other closed-testing procedures. Biometrical J. 50:678-692.
9. Hauschke, D., Pigeot, I. (2005a). Establishing efficacy of a new experimental treatment in the gold standard design. Biometrical J. 47:782-786.
10. Hauschke, D., Pigeot, I. (2005b). Rejoinder to establishing efficacy of a new experimental treatment in the gold standard design. Biometrical J. 47:797-798. (Pubitemid 43037330)
11. Hochberg, Y., Tamhane, A. C. (1987). Multiple Comparison Procedures. New York: John Wiley & Sons.
12. Hommel, G. A. (1988). Stagewise rejective multiple test procedure based on a modified Bonferroni test. Biometrika 75:383-386.
13. Hsu, J. C., Berger, R. (1999). Stepwise confidence intervals without multiplicity adjustment for dose response, multiple endpoints, and toxicity studies. J. Am. Stat. Assoc. 94:468-482.
14. Koch, A., Röhmel, J. (2004). Hypothesis testing in the "gold standard" design for proving the efficacy of an experimental treatment relative to placebo and a reference. J. Biopharm. Stat. 14:315-325.
15. Lewis, J. A., Jonsson, B., Kreutz, G., Sampaio, C., van Zwieten-Boot, B. (2002). Placebocontrolled trials and the Declaration of Helsinki. Lancet 359:1337-1340.
16. Maurer, W., Hothorn, L. A., Lehmacher, W. (1995). Multiple comparisons in drug clinical trials and pre-clinical assays: A-priori ordered hypotheses. In: Vollmar, J., ed. Biometrie in der Pharmazeutischen Industrie Band 6. Stuttgart: Fischer, pp. 3-18.
17. Pigeot, I., Schäfer, J., Röhmel, J., Hauschke, D. (2003). Assessing non-inferiority of a new treatment in a three-arm clinical trial including placebo. Stat. Med. 22:883-900.
18. Röhmel, J. (2005a). Discussion on establishing efficacy of a new experimental treatment in the gold standard design. Biometrical J. 47:790-791.
19. Röhmel, J. (2005b). On confidence bounds for the ratio of net differences in the gold standard design with reference, experimental, and placebo treatment. Biometrical J. 47:799-806.
20. Shaffer, J. P. (1986). Modified sequentially rejective multiple test procedures. J. Am. Stat. Assoc. 81:826-831.
21. Strassburger, K., Bretz, F. (2008). Compatible simultaneous lower confidence bounds for the Holm procedure and other Bonferroni-based closed tests. Stat. Med. 26:4914-4927.