Are flexible designs sound?

Biometrics

Volumn 62, Issue 3, 2006, Pages 664-669

  Burman, Carl Fredrik ^a   Sonesson, Christian ^a  

^a ASTRAZENECA R AND D   (Sweden)

Author keywords

Adaptive design; Inference principles; Sample size re estimation; Sufficiency

Indexed keywords

BIOMETRICS; STATISTICAL TESTS;

ADAPTIVE DESIGNS; DATA INFORMATIONS; DESIGN MODIFICATIONS; EXTERNAL INFORMATIONS; FLEXIBLE DESIGNS; INFERENCE PRINCIPLE; SAMPLE SIZE RE-ESTIMATION; SAMPLE SIZES; SUFFICIENCY; TYPE-I ERROR;

DESIGN;

EXPERIMENTAL STUDY; HYPOTHESIS TESTING; INFORMATION;

ARTICLE; BAYES THEOREM; CONFIDENCE INTERVAL; FISHER EXACT TEST; METHODOLOGY; NULL HYPOTHESIS; SAMPLE SIZE; STATISTICAL ANALYSIS; VALIDATION PROCESS;

EID: 33646260128     PISSN: 0006341X     EISSN: 15410420     Source Type: Journal    
DOI: 10.1111/j.1541-0420.2006.00626.x     Document Type: Article

References (30)

1. Barndorff-Nielsen, O. (1978). Information and Exponential Families in Statistical Theory. New York: Wiley.
2. Bauer, P. and Köhne, K. (1994). Evaluation of experiments with adaptive interim analyses. Biometrics 50, 1029-1041.
3. Brannath, W., Posch, M., and Bauer, P. (2002). Recursive combination tests. Journal of the American Statistical Association 97, 236-244.
4. Chen, Y. H. J., DeMets, D. L., and Lan, K. K. G. (2004). Increasing the sample size when the unblinded interim result is promising. Statistics in Medicine 23, 1023-1038.
5. Cox, D. R. and Hinkley, D. V. (1974). Theoretical Statistics. London: Chapman and Hall.
6. Cui, L., Hung, H. M. J., and Wang, S.-J. (1999). Modifications of sample size in group sequential clinical trials. Biometrics 55, 853-857.
7. Denne, J. S. (2001). Sample size recalculation using conditional power. Statistics in Medicine 20, 2645-2660.
8. European Medicines Agency. (2006). Draft reflection paper on methodological issues in confirmatory clinical trials with flexible design and analysis plan. Available from http://www.emea.eu.int/pdfs/human/ewp/245902en.pdf (accessed March 2006).
9. Fisher, L. D. (1998). Self-designing clinical trials. Statistics in Medicine 17, 1551-1562.
10. Gould, A. L. (2001). Sample size re-estimation: Recent developments and practical considerations. Statistics in Medicine 20, 2625-2643.
11. Jennison, C. and Turnbull, B. W. (2000). Group Sequential Methods with Applications to Clinical Trials. London: Chapman and Hall.
12. Jennison, C. and Turnbull, B. W. (2003). Mid-course sample size modification in clinical trials based on observed treatment effect. Statistics in Medicine 22, 971-993.
13. Lawrence, J. and Hung, H. M. J. (2003). Estimation and confidence intervals after adjusting the maximum information. Biometrical Journal 45, 143-152.
14. Lehmann, E. L. (1986). Testing Statistical Hypotheses. New York: Wiley.
15. Lemacher, W. and Wassmer, G. (1999). Adaptive sample size calculations in group sequential trials. Biometrics 55, 1286-1290.
16. 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.
17. Müller, H.-H. and Scḧafer, H. (2001). Adaptive group sequential designs for clinical trials: Combining the advantages of adaptive and of classical group sequential trials. Biometrics 57, 886-891.
18. O'Brien, P. C. and Fleming, T. R. (1979). A multiple testing procedure for clinical trials. Biometrics 35, 549-556.
19. Pocock, S. J. (1977). Group sequential methods in the design and analysis of clinical trials. Biometrika 64, 191-199.
20. Posch, M., Bauer, P., and Brannath, W. (2003). Issues in designing flexible trials. Statistics in Medicine 22, 953-969.
21. Proschan, M. A. and Hunsberger, S. A. (1995). Designed extension of studies based on conditional power. Biometrics 51, 1315-1324.
22. Proschan, M. A., Liu, Q., and Hunsberger, S. (2003). Practical midcourse sample size modification in clinical trials. Controlled Clinical Trials 24, 4-15.
23. Scherag, A., Müller, H.-H., Dempfle, A., Hebebrand, J., and Schäfer, H. (2003). Data adaptive interim modification of sample sizes for candidate-gene association studies. Human Heredity 56, 56-62.
24. Shun, Z., Yuan, W., Brady, W. E., and Hsu, H. (2001). Type 1 error in sample size re-estimation based on observed treatment difference. Statistics in Medicine 20, 497-513.
25. Stein, C. (1945). A two-sample test for a linear hypothesis whose power is independent of the variance. Annals of Mathematical Statistics 16, 243-258.
26. Thach, C. T. and Fisher, L. D. (2002). Self-designing two-stage trials to minimize expected costs. Biometrics 58, 432-438.
27. Tsiatis, A. A. and Mehta, C. (2003). On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 90, 367-378.
28. Wang, S.-J., Hung, H. M. J., Tsong, Y., and Cui, L. (2001). Group sequential test strategies for superiority and non-inferiority hypotheses in active controlled clinical trials. Statistics in Medicine 20, 1903-1912.
29. Wittes, J. and Brittain, E. (1990). The role of internal pilot studies in increasing the efficiency of clinical trials. Statistics in Medicine 9, 65-72.
30. + exchange inhibitor eniporide as an adjunct to early reperfusion therapy for acute myocardial infarction. Journal of the American College of Cardiology 38, 1644-1650.