Abstract
We develop a Bayesian approach for the design of noninferiority clinical trials with co-primary endpoints and multiple dose comparison. The Bayesian approach has the potential of power increase and hence sample size reduction due to the incorporation of the historical data and the correlation structure among multiple co-primary endpoints while it still maintains the family-wise type I error control without additional multiplicity adjustment. In this chapter, we compare the Bayesian method to the conventional frequentist method with or without Bonferroni multiplicity adjustment resulting from the multiple dose comparison. The proposed method is also applied to the design of a clinical trial, in which the study drug at a low dose level and at a high dose level is compared with the active control in terms of the bivariate co-primary endpoints.
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References
Chen M-H, Ibrahim JG, Lam P, Yu A, Zhang Y (2011) Bayesian design of non-inferiority trials for medical devices using historical data. Biometrics 67:1163–1170
Chuang-Stein C, Stryszak P, Dmitrienko A, Offen W (2007) Challenge of multiple co-primary endpoints: a new approach. Stat Med 26:1181–1192
CPMP (2000) Points to consider on switching between superiority and non-inferiority. http://www.emea.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003658.pdf
CHMP (2005) Guideline on the choice of the non-inferiority margin.http://www.emea.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003636.pdf
CPMP Working Party on Efficacy of Medicinal Products Note for Guidance III/3630/92-EN (1995) Biostatistical methodology in clinical trials in applications for marketing authorizations for medicinal products. Stat Med 14:1659–1682
Dmitrienko A, Offen W, Wang O, Xiao D (2006) Gatekeeping procedures in dose-response clinical trials based on the Dunnett test. Pharm Stat 5:19–28
Dmitrienko A, Tamhane AC, Bretz F (2010) Multiple testing problems in pharmaceutical statistics. Chapman & Hall, Boca Raton
Eaton ML, Muirhead RJ (2007) On a multiple endpoints testing problem. J Stat Plan Inference 137:3416–3429
FDA Guidance for industry (2010) Non-inferiority clinical trials. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM202140.pdf
Gonen M, Westfall PH, Johnson WO (2003) Bayesian multiple testing for two-sample multivariate endpoints. Biometrics 59:76–82
Hung HJ, Wang SJ (2004) Multiple testing of noninferiority hypotheses in active controlled trials. J Biopharm Stat 14:327–335
Ibrahim JG, Chen M-H (2000) Power prior distributions for regression models. Stat Sci 15:46–60
ICH Harmonised tripartite guideline (1998) Statistical principles for clinical trials (E9). http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf
ICH Harmonized tripartite guideline (2000). Choice of control group and related issues in clinical trials (E10). http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E10/Step4/E10_Guideline.pdf
Kong L, Kohberger RC, Koch GG (2004) Type I error and power in noninferiority/equivalence trials with correlated multiple endpoints: an example from vaccine development trials. J Biopharm Stat 14:893–907
Laska NS, Tang D, Meisner MJ (1992) Testing hypothesis about an identified treatment when there are multiple endpoints. J Am Stat Assoc 87:825–831
Liu KJ, Chang KC (2011) Test non-inferiority and sample size determination based on the odds ratio under a cluster randomized trial with noncompliance. J Biopharm Stat 21:94–110
Liu Y, Hsu J, Ruberg S (2007) Partition testing in dose-response studies with multiple endpoints. Pharm Stat 6:181–192
Narayan P, Ashutosh Tewari A, Members Of United States 93-01 Study Group (1998) A second phase i11 multicenter placebo controlled study of 2 dosages of modified release Tamsulosin in patients with symptoms of benign prostatic hyperplasia. J Urol 160:1701–1706
Ng TH (2003) Issues of simultaneous tests for noninferiority and superiority. J Biopharm Stat 13:629–639
Röhmel J, Pigeot I (2010) A comparison of multiple testing procedures for the gold standard non-inferiority trial. J Biopharm Stat 20:911–926
Scott JG, Berger JO (2006) An exploration of aspects of Bayesian multiple testing. J Stat Plan Inference 136:2144–2162
Sugimoto T, Sozu T, Hamasaki T (2012) A convenient formula for sample size calculations in clinical trials with multiple co-primary continuous endpoints. Pharm Stat 11:118–128
Tamimi NAM, Mincik I, Haughie S, Lamb J, Crossland A, Peter Ellis P (2010) A placebo-controlled study investigating the efficacy and safety of the phosphodiesterase type 5 inhibitor UK-369,003 for the treatment of men with lower urinary tract symptoms associated with clinical benign prostatic hyperplasia. BJU Int 106:674–680
Tsong Y, Zhang J (2007) Simultaneous test for superiority and noninferiority hypotheses in active controlled clinical trials. J Biopharm Stat 17:247–257
Welch BL (1947) The generalization of student’s problem when several different population variance are involved. Biometrika 34:28–35
Xu H, Nuamah I, Liu J, Lim P, Sampson A (2009) A Dunnett-Bonferroni-based parallel gatekeeping procedure for dose-response clinical trials with multiple endpoints. Pharm Stat 8:301–316
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Li, W., Chen, MH., Tan, H., Dey, D. (2015). Bayesian Design of Noninferiority Clinical Trials with Co-primary Endpoints and Multiple Dose Comparison. In: Chen, Z., Liu, A., Qu, Y., Tang, L., Ting, N., Tsong, Y. (eds) Applied Statistics in Biomedicine and Clinical Trials Design. ICSA Book Series in Statistics. Springer, Cham. https://doi.org/10.1007/978-3-319-12694-4_2
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DOI: https://doi.org/10.1007/978-3-319-12694-4_2
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