Regulatory perspectives on multiplicity in adaptive design clinical trials throughout a drug development program

Journal of Biopharmaceutical Statistics

Volumn 21, Issue 4, 2011, Pages 846-859

  Wang, Sue Jane ^a   James Hung, H M ^a   O'Neill, Robert ^a  

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

Author keywords

Confirmatory; Exploratory; Learn and confirm; Probability of correct decision; Probability of correct selection; Probability of success

Indexed keywords

ANALYTICAL ERROR; CLINICAL RESEARCH; CLINICAL TRIAL (TOPIC); COMPUTERIZED ADAPTIVE TESTING; CONTROL GROUP; DECISION MAKING; DOSE RESPONSE; DRUG ACTIVITY; DRUG DEVELOPMENT; DRUG EFFICACY; DRUG TOLERABILITY; DRUG TOXICITY; EFFECT SIZE; FOOD AND DRUG ADMINISTRATION; GOVERNMENT REGULATION; HYPOTHESIS; INTENTION TO TREAT ANALYSIS; MAXIMUM TOLERATED DOSE; PATIENT SELECTION; PRIORITY JOURNAL; PROBABILITY; PROGRAM DEVELOPMENT; REVIEW; SAMPLE SIZE; SAMPLING; STUDY DESIGN;

CLINICAL TRIALS AS TOPIC; DRUG DISCOVERY; ENDPOINT DETERMINATION; PROBABILITY; RESEARCH DESIGN; SAMPLE SIZE;

EID: 79957528017     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2011.552878     Document Type: Review

References (42)

1. Bauer, P. (1989). Multistage testing with adaptive designs (with Discussion). Biometrie und Informatik in Medizin and Biologie 20:130-148.
2. Bauer, P., Kieser, M. (1999). Combining different phases in the development of medical treatments within a single trial. Statistics in Medicine 18:1833-1848. (Pubitemid 29364054)
3. Bauer, P., Koenig, F. (2006). The reassessment of trial perspectives from interim data-A critical view. Statistics in Medicine 25:23-36. (Pubitemid 43136947)
4. Bauer, P., Köhne, K. (1994). Evaluations of experiments with adaptive interim analyses. Biometrics 50:1029-1041.
5. Bechhofer, R. E., Kiefer, J., Sobel, M. (1968). Sequential Identification and Ranking Procedures. Chicago: University of Chicago Press.
6. Bretz, F., Schmidli, H., Konig, F., Racine, A., Maurer, W. (2006). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: General concepts. Biometrical Journal 4:623-634. (Pubitemid 44367905)
7. Bretz, F., Koenig, F., Brannath, W., Glimm, E., Posch, M. (2009). Adaptive designs for confirmatory clinical trials. Statistics in Medicine 28:1181-1217.
8. Chen, Y. H. J., DeMets, D. L., Lan, K. K. G. (2004). Increasing the sample size when the unblinded interim result is promising. Statistics in Medicine 23:1023-1038. (Pubitemid 38477065)
9. Cook, T. D., Benner, R. J., Fisher, M. R. (2006). The WIZARD trial as a case study of flexible clinical trial design. Drug Information Journal 40:345-353. (Pubitemid 44267162)
10. Cui, L., Hung, H. M. J., Wang, S. J. (1999). Modification of sample size in group sequential clinical trials. Biometrics 55:321-324.
11. European Medicines Agency. (2007). Reflection Paper on Methodological Issues in Confirmatory Clinical Trials Planned With an Adaptive Design. European Medicines Agency, EMEA CHMP/EWP/2459/02, October.
12. Food and Drug Administration. (2010). Draft Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics. ComplianceRegulatoryInformation/Guidances/UCM201790.pdf (2010). Released on February 25, 2010, for public comments.
13. Food and Drug Administration. (2002). 21CFR314.126. Health Human Services, Code of Federal Regulation. Washington, DC.
14. Gibbons, J. D., Olkin, I., Sobel, M. (1977). Selecting and Ordering Populations: A New Statistical Methodology. New York: Wiley.
15. Glim, E., Maurer, W., Bretz, F. (2010). Hierarchical testing of multiple endpoints in groupsequential trials. Statistics in Medicine 29:219-228.
16. Haybittle, J. L. (1971). Repeated assessments of results in clinical trials of cancer treatments. British Journal of Radiology 44:793-797.
17. Hung, H. M. J., Wang, S. J. (2010). Challenges to multiple testing in clinical trials. Biometrical Journal (special issue on multiplicity) 52(6):747-756. DOI: 10.1002/biomj.200900206.
18. Hung, H. M. J., Wang, S. J., O'Neill, R. T. (2007). Statistical considerations for testing multiple endpoints in group sequential or adaptive clinical trials. Journal of Biopharmaceutical Statistics 17:1201-1210. (Pubitemid 350135837)
19. Hung, H. M. J., Wang, S. J., O'Neill, R. (2011). Flexible designs in regulatory applications. Statistics in Medicine, February 2011. DOI: 10.1002/sim.4021.
20. Inoue, L. Y. T., Thall, P. F., Berry, D. A. (2002). Seamlessly expanding a randomized phase II trial to phase III. Biometrics 58:823-831. (Pubitemid 36133760)
21. Korn, E. L., Freidlin, B. (2011). Outcome-adaptive randomization: is it useful? Journal of Clinical Oncology 29:771-776.
22. Lan, K. K. G., DeMets, D. L. (1983). Discrete sequential boundaries for clinical trials. Biometrika 70:659-663.
23. Lehmacher, W., Wassmer, G. (1999). Adaptive sample size calculations in group sequential trials. Biometrics 55:1286-1290.
24. Levy, G., Kaufmann, P., Buchsbaum, R., Montes, J., Barsdorf, A., Arbing, R., Battista, V., Zhou, X., Mitsumoto, H., Levin, B., Thompson, J. L. P. (2006). A two-stage design for a phase II clinical trial of coenzyme Q10 in ALS. Neurology 66:660-663. (Pubitemid 43739818)
25. Mueller, H. H., Schaefer H. (2004). A general statistical principle for changing a design any time during the course of a trial. Statistics in Medicine 23:2497-2508.
26. O'Brien, P. C., Fleming, T. R. (1979). A multiple testing procedure for clinical trials. Biometrics 35:549-556. (Pubitemid 10242750)
27. O'Connor, C. M., Dunne, M. W., Pfeffer, M. A., Muhlestein, J. B., Yao, L., Cupta, S., Benner, R. J., Fisher, M. R., Cook, T. D. (2003). Azithromycin for the secondary prevention of coronary heart disease events. The WIZARD study: A randomized controlled trial. Journal of the American Medical Association 290:1459-1466. (Pubitemid 37430400)
28. Peto, R., Pile, M. C., Armitage, P., Breslow, N. E., Cox, D. R., Howard, S. V., Mantel, N., McPherson, C. K., Peto, J., Smith, P. G. (1976). Design and analysis of randomized clinical trials requiring prolonged observation of each patients. I. Introduction and design. British Journal of Cancer 34:585-612. (Pubitemid 8004970)
29. Posch, M., Maurer, W., and Bretz, F. (2010). Type I error rate control in adaptive designs for confirmatory clinical trials with treatment selection at interim. Pharmaceutical Statistics, April 2010. DOI: 10.1002/pst.413.
30. Schaid, D. J., Wieand, S., Therneau, T. M. (1990). Optimal two-stage screening designs for survival comparisons. Biometrika 77:507-513.
31. Schmidli, H., Bretz, F., Racine-Poon, A., Maurer, W. (2006). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: Applications and practical considerations. Biometrical Journal 48:635-643. (Pubitemid 44367906)
32. Simon, R., Wittes, R. E., Ellenberg, S. S. (1985). Randomized phase II clinical trials. Cancer Treatment Reports 69:1375-1381. (Pubitemid 16163075)
33. Tamhane, A. C., Mehta, C. R., Liu, L. (2011). Testing a primary and a secondary endpoint in a group sequential design. Biometrics 66:1174-1184.
34. Thall, P. F., Simon, R., Ellenberg, S. S. (1988). Two-stage selection and testing designs for comparative clinical trials. Biometrika 75:303-310.
35. Thall, P. F., Simon, R., Ellenberg, S. S. (1989). A two-stage design for choosing among several experimental treatments and a control in clinical trials. Biometrics 45:537-547. (Pubitemid 19165264)
36. Todd, S., Stellard, N. (2005). A new clinical trial design combining phases 2 and 3: Sequential designs with treatment selection and a change of endpoint. Drug Information Journal 39:109-118. (Pubitemid 40647117)
37. Wang, S. J. (2010). Perspectives on the use of adaptive designs in clinical trials. Part I. Statistical Considerations and Issues. Journal of Biopharmaceutical Statistics 20(6):1090-1097.
38. Wang, S. J., Bretz, F. (2010). From adaptive design to modern protocol design for drug development. Part I: Editorial and summary of "Adaptive designs session" at the Third FDA/DIA Statistics Forum. Drug Information Journal 44(3):325-331.
39. Wang, S. J., Hung, H. M. J., O'Neill, R. T. (2009). Adaptive patient enrichment designs in therapeutic trials. Biometrical Journal 51(2):358-374.
40. Wang, S. J., Hung, H. M. J., O'Neill, R. T. (2010). Impacts of Type I error rate with inappropriate use of learn for confirm in adaptive designs. Biometrical Journal (special issue on multiplicity) 52(6):798-810. DOI: 10.1002/bimj.200900207.
41. Wang, S. J., Hung, H. M. J., O'Neill, R. T. (2011). Adaptive design clinical trials and logistics models in CNS drug development. European Neuropsychopharmacology Journal 21(2):159-166.
42. Wang, S. K., Tsiatis, A. A. (1978). Approximately optimal one-parameter boundaries for group sequential trials. Biometrics 43:193-199.