Impacts on type I error rate with inappropriate use of learn and confirm in confirmatory adaptive design trials

Biometrical Journal

Volumn 52, Issue 6, 2010, Pages 798-810

  Wang, Sue Jane ^a   James Hung, H M ^a   O'Neill, Robert T ^a  

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

Author keywords

Adaptive design; Learn and confirm; Learning free type I error rate; Studywise type I error rate

Indexed keywords

ERRORS;

ADAPTIVE DESIGNS; ADAPTIVE SELECTION; INTERIM ANALYSIS; LEARN AND CONFIRM; LEARN+; LEARNING DATA; LEARNING FREE TYPE I ERROR RATE; STUDYWISE TYPE I ERROR RATE; TYPE I ERROR RATE;

RISK ASSESSMENT;

EID: 78650100317     PISSN: 03233847     EISSN: 15214036     Source Type: Journal    
DOI: 10.1002/bimj.200900207     Document Type: Article

References (26)

1. Arnold, B. C., Balakrishnan, N. and Nagaraja, H. N. ( 1992). A First Course in Order Statistics. Wiley, New York, pp. 86-94.
2. Bauer, P., Brannath, W. and Posch, M. ( 2001). Flexible two stage designs: an overview. Methods of Information in Medicine 40, 117-121.
3. Bauer, P. and Kieser, M. ( 1999). Combining different phases in the development of medical treatments within a single trial. Statistics in Medicine 18, 1833-1848.
4. Bauer, P., Koenig, F., Brannath, W. and Posch, M. ( 2010). Selection and bias - two hostile brothers. Statistics in Medicine 29, 1-13.
5. Bowden, J. and Glimm, E. ( 2008). Unbiased estimation of selected treatment means in two-stage trials. Biometrical Journal 50, 515-527.
6. Brannath, W., Koenig, F. and Bauer, P. ( 2007). Multiplicity and flexibility in clinical trials. Pharmaceutical Statistics 6, 205-216.
7. Bretz, F., Koenig, F., Brannath, W., Glimm, E. and Posch, M. ( 2009a). Tutorial in biostatistics: adaptive designs for confirmatory clinical trials. Statistics in Medicine 28, 1181-1217.
8. Bretz, F., Maurer, W. and Gallo, P. ( 2009b). Discussion of "Some controversial multiple testing problems in regulatory applications" by H. M. J. Hung and S. -J. Wang. Journal of Biopharmaceutical Statistics 1, 25-34.
9. Bretz, F., Schmidli, H., Konig, F., Racine, A. and Maurer, W. (2006). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: general concepts. Biometrical Journal 48, 23-634.
10. Cui, L., Hung, H. M. J. and Wang, S. J. (1999). Modification of sample size in group sequential clinical trials. Biometrics 55, 853-857.
11. Draft Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics. Released on February 25, 2010 for public comments. Food and Drug Administration, Health Human Services, Code of Federal Regulation, 21CFR314.126. Available at .
12. International Conference on Harmonization: statistical principles for clinical trials (ICH E-9), Food and Drug Administration, DHHS, 1998.
13. Jennison, C. and Turnball, B. W. (2006). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: opportunities and limitations. Biometrical Journal 48, 650-655.
14. Kelly, P. J., Stallard, N. and Todd, S. ( 2005). An adaptive group sequential design for phase II/III clinical trials that select a single treatment from several treatments. Journal of Biopharmaceutical Statistics 15, 461-658.
15. Khan, A. and Schwarz, K. ( 2005). Study designs and outcomes in antidepressant clinical trials. Essential Psychopharmacology 6, 221-226.
16. Kimani, P. K., Stallard, N. and Hutton, J. L. ( 2009). Dose selection in seamless phase II/III clinical trials based on efficacy and safety. Statistics in Medicine 28, 917-936.
17. Kola, I. and Landis, J. ( 2004). Can the pharmaceutical industry reduce attrition rates ? Nature Reviews Drug Discovery 3, 711-715.
18. Muller, H.-H. and Schafer, H. (2001). Adaptive group sequential designs for clinical trials: combining the advantages of adaptive and of classical group sequential approaches. Biometrics 57, 886-891.
19. O'Brien, P. C. and Fleming, T. R. (1979). A multiple testing procedure for clinical trials. Biometrics 35, 549-556.
20. Pocock, S. J. (1979). Group sequential methods in the design and analysis of clinical trials. Biometrika 64, 191-199.
21. Schmidli, H., Bretz, F. and Racine-Poon, A. ( 2007). Bayesian predictive power for interim adaptation in seamless phase II/III trials where the endpoint is survival up to some specified timepoint. Statistics in Medicine 26, 4925-4938.
22. Schmidli, H., Bretz, F., Racine-Poon, A. and Maurer, W. (2006). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: applications and practical considerations. Biometrical Journal 48, 635-643.
23. Spiegelhalter, D. J., Freedman, L. S. and Blackburn, P. R. (1986). Monitoring clinical trials: conditional or predictive power? Controlled Clinical Trials 7, 8-17.
24. Thall, P. F. (2008). A review of phase 2-3 clinical trial designs. Lifetime Data Analysis 14, 37-53.
25. Wang, S. J., O'Neill, R. T. and Hung, H. M. J. ( 2007). Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharmaceutical Statistics 6, 227-244.
26. Yao, Q. and Wei, L. J. ( 1996). Play the winner for phase II/III clinical trials. Statistics in Medicine 15, 2413-2423.