Adaptive methods: Telling "the rest of the story"

Journal of Biopharmaceutical Statistics

Volumn 20, Issue 6, 2010, Pages 1150-1165

  Emerson, Scott S ^a   Fleming, Thomas R ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Confirmatory; Drug discovery; Enrichment; Exploratory; Prespecification; Seamless designs; Sequential clinical trials

Indexed keywords

ADAPTIVE DESIGN; CLINICAL RESEARCH; DRUG DEVELOPMENT; FOOD AND DRUG ADMINISTRATION; HUMAN; METHODOLOGY; PRIORITY JOURNAL; RANDOMIZATION; REVIEW; SAMPLE SIZE;

CLINICAL TRIALS AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; CLINICAL TRIALS, PHASE III AS TOPIC; DATA INTERPRETATION, STATISTICAL; DRUG APPROVAL; DRUG DISCOVERY; ENDPOINT DETERMINATION; EVIDENCE-BASED MEDICINE; GUIDELINES AS TOPIC; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; REPRODUCIBILITY OF RESULTS; RESEARCH DESIGN; SAMPLE SIZE; TREATMENT OUTCOME; UNITED STATES;

EID: 78249272973     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543406.2010.514457     Document Type: Review

References (36)

1. Armitage, P., McPherson, C. K., Rowe, B. C. (1969). Repeated significance tests on accumulating data. Journal of the Royal Statistical Society, Series A 132:235-244.
2. Bauer, P., Kohne, K. (1994). Evaluation of experiments with adaptive interim analyses. Biometrics 50:1029-1041.
3. Begg, C. B. (1989). Comment on investigating therapies of potentially great benefit: ECMO. Statistical Science 4:320-322.
4. Berry, D. A. (2004). Bayesian statistics and the efficiency and ethics of clinical trials. Statistical Science 19 (1):175-187.
5. Berry, D. A., Eick, S. G. (1994). Adaptive assignment versus balanced randomization in clinical trials: A decision analysis. Statistics in Medicine 14:231-246.
6. Berry, D. A., Müller, P., Grieve, A. P., Smith, M., Parke, T., Blazek, R., Mitchard, N., Krams, M. (2002). Adaptive Bayesian designs for dose-ranging drug trials. In: Gatsonis, C. L., Carlin, B., Carriquiry, A., eds. Case Studies in Bayesian Statistics V. Lecture Notes in Statist. 162. New York: Springer, pp. 99-181.
7. Bretz, F., Schmidli, H., Konig, F., Racine, A., Maurer, W. (2006). Confirmatory seamless phase II/III clinical trials with hypothesis selection at interim: General concepts. Biometrical Journal 48 (4):623-634. (Pubitemid 44367905)
8. Cytel Software Corp. (2010). East. Cambridge, MA: The Cytel Software Corp.
9. DeAngelo, D., O'Brien, S. M., Vey, N., Seiter, K., Stock, W., Cahill, A., Pigneux, A., Claxton, D., Stuart, R., Giles, F. J. (2008). A double blind placebo-controlled randomized phase III study of high dose continuous infusion cytosine arabinoside with or without VNP40101M in patients with first relapse of acute myeloid leukemia. Journal of Clinical Oncology 26 (May 20 suppl.; abstr. 7051).
10. Ellenberg, S. S., Fleming, T. R., DeMets, D. L. (2002). Data Monitoring Committees in Clinical Trials: A Practical Perspective. New York: John Wiley and Sons.
11. Emerson, S. S. (2006). Issues in the use of adaptive clinical trial designs. Statistics in Medicine 25:3270-3296.
12. Emerson, S. S., Kittelson, J. M., Gillen, D. L. (2007). Frequentist evaluation of group sequential designs. Statistics in Medicine 26 (28):5047-5080.
13. Fisher, L. D. (1998). Self-designing clinical trials. Statistics in Medicine 17:1551-1562.
14. Fleming, T. R. (2006). Standard vs. adaptive monitoring procedures: A commentary. Statistics in Medicine 25:3305-3312.
15. Fleming, T. R., Sharples, K., McCall, J., Moore, A., Rodgers, A., Stewart, R. (2008). Maintaining confidentiality of interim data to enhance trial integrity and credibility. Clinical Trials 5:157-167.
16. Food and Drug Administration. (1998). FDA Guidance Document: Guidance for Industry Providing Clinical Evidence of Effectiveness for Human Drugs and Biological Products. May. http://www.fda.gov/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/default.htm
17. Food and Drug Administration. (2010). Draft FDA Guidance Document: Guidance for Industry Adaptive Design Clinical Trials for Drugs and Biologics. February. http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/default.htm
18. Freidlin, B., Simon, R. (2005). Adaptive signature design: An adaptive clinical trial design for generating and prospectively testing a gene expression signature for sensitive patients. Clinical Cancer Research 11:7872-7878.
19. Inoue, L. Y., Thall, P. F., Berry, D. A. (2002). Seamlessly expanding a randomized phase II trial to phase III. Biometrics 58:823-831.
20. Jennison, C., Turnbull, B. W. (2001). Group Sequential Methods with Applications to Clinical Trials. Boca Raton, FL: CRC Press.
21. Jennison, C., Turnbull, B. W. (2006a). Adaptive and non-adaptive group sequential tests. Biometrika 93:1-21.
22. Jennison, C., Turnbull, B. W. (2006b). Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: Opportunities and limitations. Biometrical Journal 48:650-655.
23. Jiang, W., Freidlin, B., Simon, R. (2007). Biomarker adaptive threshold design: A procedure for evaluating treatment with possible biomarker-defined subset effect. Journal of the National Cancer Institute 99:1036-1043.
24. MPS Research Unit. (2008). PEST (Planning and Evaluation of Sequential Trials). MPS Research Unit, Lancaster University, Lancaster, UK.
25. Müller, H., Schäfer, H. (2001). Adaptive group sequential designs for clinical trials: Combining the 1968 advantages of adaptive and of classical group sequential approaches. Biometrics 57:886-891.
26. Proschan, M. A., Hunsberger, S. A. (1995). Designed extension of studies based on conditional power. Biometrics 51:1315-1324.
27. SAS Insitute, Inc. (2009). SAS PROC SEQDESIGN, PROC SEQTEST. Cary, NC: SAS Institute, Inc.
28. Shi, S. (2003). Estimation Following Self-Designing Clinical Trials. Unpublished M. S. thesis, Department of Biostatistics, University of Washington, Seattle.
29. Spiegelhalter, D. J., Abrams, K. R., Myles, J. P. (2004). Bayesian Approaches to Clinical Trials and Health-Care Evaluation. New York: Wiley.
30. TIBCO. (2002). S+SeqTrial. Seattle, WA: TIBCO.
31. Tsiatis, A. A., Mehta, C. R. (2003). On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 90:367-378.
32. UK Collaborative ECMO Trial Group. (1996). UK collaborative randomised trial of neonatal extracorporeal membrane oxygenation. Lancet 348:75-82.
33. Wald, A. (1947). Sequential Analysis. New York: Wiley.
34. Ware, J. H. (1989). Investigating therapies of potentially great benefit: ECMO. Statistical Science 4:306-316.
35. Whitehead, J. (1997). The Design and Analysis of Sequential Clinical Trials. New York: John Wiley & Sons.
36. Yao, Q., Wei, L. J. (1996). Play the winner for phase II/III clinical trials. Statistics in Medicine 15:2413-2423.