On adaptive extensions of group sequential trials for clinical investigations

Journal of the American Statistical Association

Volumn 103, Issue 484, 2008, Pages 1621-1630

  Liu, Qing ^a   Anderson, Keaven M ^b  

^a JOHNSON AND JOHNSON PHARMACEUTICAL RESEARCH AND DEVELOPMENT   (United States)

^b MERCK RESEARCH LABORATORIES   (United States)

Author keywords

Adaptive design; Group sequential design; Sample size adjustment; Sequential confidence interval; Sequential p value

Indexed keywords

EID: 78249278606     PISSN: 01621459     EISSN: None     Source Type: Journal    
DOI: 10.1198/016214508000000986     Document Type: Article

References (30)

1. Armitage, P. (1975), Sequential Medical Trials (2nd ed.), New York: Wiley.
2. Canner, P. L. (1983), "Monitoring of the Data for Evidence of Adverse or Beneficial Treatment Effects," Controlled Clinical Trials, 4, 467-483.
3. CHMP (2007), "On Methodological Issues in Confirmatory Clinical Trials Planned With an Adaptive Design," CHMP/EWP/2459/02.
4. Cox, D. R., and Hinkley, D. V. (1974), Theoretical Statistics, London: Chapman & Hall.
5. Cui, L., Hung, H. M. J., and Wang, S. J. (1999), "Modification of Sample Size in Group Sequential Clinical Trials," Biometrics, 55, 853-857.
6. DeMets, D. L. (1984), "Stopping Guidelines vs. Stopping Rules: A Practitioner's Point of View," Communications in Statistics, Part A-Theory and Methods, 13, 2395-2418.
7. DeMets, D. L., and Ware, J. H. (1980), "Group SequentialMethods for Clinical Trials With a One-Sided Hypothesis," Biometrika, 67, 651-660.
8. - (1982), "Asymmetric Group Sequential Boundaries for Monitoring Clinical Trials," Biometrika, 69, 661-663.
9. Dupont, W. D. (1983), "Sequential Stopping Rules and Sequential Adjusted p Values: Does One Require the Other?" (with discussion), Controlled Clinical Trials, 4, 3-35.
10. Ellenberg, S. S., Fleming, T. R., and DeMets, D. L. (2002), Data Monitoring Committees in Clinical Trials: A Practical Perspective, West Sussex, U.K.: Wiley.
11. Fisher, L. (1998), "Self-Designing Clinical Trials," Statistics in Medicine, 17, 1551-1562.
12. Frisen, M. (2006), Discussion of "Are Flexible Designs Sound?" by Burnman, C. F., and Sonesson, C., Biometrics, 62, 678-680.
13. Hacking, I. (1965), Logic of Statistical Inference, Cambridge, U.K.: Cambridge University Press.
14. Hall, W. J., and Ding, K. (2008), "Sequential Tests and Estimates After Overrunning Based on p-Value Combination," IMS Collections, Pushing The Limits of Contemporary Statistics: Contributions in Honour of Jayanta K. Ghosh, 3, 33-45.
15. Hall,W. J., and Liu, A. (2002), "Sequential Tests and Estimators After Overrunning Based on Maximum-Likelihood Ordering," Biometrika, 89, 699-707.
16. ICH (1998), "Guidance on Statistical Principles for Clinical Trials," Federal Register, 63, 49583-49598.
17. Jennison, C., and Turnbull, B. W. (1989), "Interim Analysis: The Repeated Confidence Interval Approach" (with discussion), Journal of the Royal Statistical Society, Ser. B, 51, 305-361.
18. - (2000), Group Sequential Methods With Applications to Clinical Trials, Boca Raton, FL: Chapman & Hall.
19. Lan, K. K. G., and DeMets, D. L. (1983), "Discrete Sequential Boundaries for Clinical Trials," Biometrika, 70, 659-663.
20. Lan, K. K. G., and Wittes, J. (1994), "Data Monitoring in Complex Clinical Trials: Which Treatment Is Better?" Journal of Statistical Planning and Inference, 42, 241-255.
21. Lan, K. K. G., Lachin, J.M., and Bautista, O. (2003), "Over-Ruling a Group Sequential Boundary: A Stopping Rule vs. a Guideline," Statistics in Medicine, 22, 3347-3355.
22. Liu, Q., and Anderson, K. M. (2008), "Theory of Inference for Adaptively Extended Group Sequential DesignsWith Applications in Clinical Trials," available at http://www.amstat.org/publications/jasa/supplemental- materials.
23. Liu, Q., and Pledger, G. W. (2006), "On Design and Inference for Two-Stage Adaptive Clinical Trials With Dependent Data," Journal of Statistical Planning and Inference, 136, 1962-1984.
24. Liu, Q., Proschan,M. A., and Pledger, G.W. (2002), "A Unified Theory of Two-Stage Adaptive Designs," Journal of the American Statistical Association, 97, 1034-1041.
25. O'Hagan, A. (1989), Discussion of "Interim Analyses: The Repeated Confidence Interval Approach," by C. Jennison and B. W. Turnbull, Journal of the Royal Statistical Society, Ser. B, 51, 342.
26. Proschan, M. A. (1999), "Properties of Spending Function Boundaries," Biometrika, 86, 466-473.
27. Proschan, M. A., Lan, K. K. G., and Wittes, J. T. (2006), Statistical Monitoring of Clinical Trials, New York: Springer.
28. Royall, R. (1997), Statistical Evidence: A Likelihood Paradigm, London: Chapman & Hall.
29. Whitehead, J. (1992), "Overrunning and Underrunning in Sequential Clinical Trials," Controlled Clinical Trials, 13, 106-121.
30. - (1999), "On Being the Statistician on a Data and Safety Monitoring Board," Statistics in Medicine, 18, 3425-3434.