An evaluation of inferential procedures for adaptive clinical trial designs with pre-specified rules for modifying the sample size

Biometrics

Volumn 70, Issue 3, 2014, Pages 556-567

  Levin, Gregory P ^a   Emerson, Sarah C ^b   Emerson, Scott S ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

^b OREGON STATE UNIVERSITY   (United States)

Author keywords

Adaptive designs; Clinical trials; Estimation; Group sequential tests; Inference; Sample size modification

Indexed keywords

MEAN SQUARE ERROR; MEDICAL APPLICATIONS;

ADAPTIVE DESIGNS; CLINICAL TRIAL; GROUP SEQUENTIAL TEST; INFERENCE; POINT ESTIMATE; SAMPLE SIZE MODIFICATION; SAMPLE SIZES; SEQUENTIAL TESTS; SIZE MODIFICATION; TREATMENT EFFECTS;

ERRORS;

ALGORITHM; CLINICAL TRIAL (TOPIC); COMPUTER SIMULATION; EVALUATION STUDY; LABORATORY DIAGNOSIS; METHODOLOGY; OUTCOME ASSESSMENT; PROCEDURES; REPRODUCIBILITY; SAMPLE SIZE; SENSITIVITY AND SPECIFICITY; STATISTICAL ANALYSIS; STATISTICAL MODEL;

ALGORITHMS; CLINICAL TRIALS AS TOPIC; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; FALSE POSITIVE REACTIONS; MODELS, STATISTICAL; OUTCOME ASSESSMENT (HEALTH CARE); REPRODUCIBILITY OF RESULTS; RESEARCH DESIGN; SAMPLE SIZE; SENSITIVITY AND SPECIFICITY;

EID: 84927697746     PISSN: 0006341X     EISSN: 15410420     Source Type: Journal    
DOI: 10.1111/biom.12168     Document Type: Article

References (37)

1. Armitage, P., McPherson, C., and Rowe, B. (1969). Repeated significance tests on accumulating data. Journal of the Royal Statistical Society 132, 235-244.
2. Bauer, P. and Kohne, K. (1994). Evaluation of experiments with adaptive interim analyses. Biometrics 50, 1029-1041.
3. Brannath, W., König, F., and Bauer, P. (2006). Estimation in flexible two stage designs. Statistics in Medicine 25, 3366-3381.
4. Brannath, W., Mehta, C. R., and Posch, M. (2009). Exact confidence bounds following adaptive group sequential tests. Biometrics 65, 539-546.
5. Chang, M. N. (1989). Confidence intervals for a normal mean following a group sequential test. Biometrics 45, 247-254.
6. Chang, M. N., Gould, A. L., and Snapinn, S. M. (1995). P-values for group sequential testing. Biometrika 82, 650-654.
7. Chang, M. N. and O'Brien, P. C. (1986). Confidence intervals following group sequential tests. Controlled Clinical Trials 7, 18-26.
8. Cui, L., Hung, H. M. J., and Wang, S.-J. (1999). Modification of sample size in group sequential clinical trials. Biometrics 55, 853-857.
9. Denne, J. S. and Jennison, C. (2000). A group sequential t-test with updating of sample size. Biometrika 87, 125-134.
10. Emerson, S. C., Rudser, K. D., and Emerson, S. S. (2011). Exploring the benefits of adaptive sequential designs in time-to-event endpoint settings. Statistics in Medicine 30, 1199-1217.
11. Emerson, S. S. (1988). Parameter estimation following group sequential hypothesis testing. PhD thesis, University of Washington.
12. Emerson, S. S. and Fleming, T. R. (1990). Parameter estimation following group sequential hypothesis testing. Biometrika 77, 875-892.
13. Fleming, T. R. (2006). Standard versus adaptive monitoring procedures: A commentary. Statistics in Medicine 25, 3305-3312.
14. Food and Drug Administration (2010). Guidance for industry: Adaptive design clinical trials for drugs and biologics. http://www.fda.gov/downloads/Drugs/Guidance ComplianceRegulatoryInformation/Guidances/ucm201790.pdf.
15. Gao, P., Liu, L., and Mehta, C. (2013). Exact inference for adaptive group sequential designs. Statistics in Medicine 32, 3991-4005.
16. Gillen, D. L. and Emerson, S. S. (2005). A note on p-values under group sequential testing and nonproportional hazards. Biometrics 61, 546-551.
17. Jennison, C. and Turnbull, B. W. (2000). Group Sequential Methods with Applications to Clinical Trials. Boca Raton: Chapman and Hall/CRC.
18. Jennison, C. and Turnbull, B. W. (2006a). Adaptive and nonadaptive group sequential tests. Biometrika 93, 1-21.
19. Jennison, C. and Turnbull, B. W. (2006b). Efficient group sequential designs when there are several effect sizes under consideration. Statistics in Medicine 25, 917-932.
20. Kittelson, J. M. and Emerson, S. S. (1999). A unifying family of group sequential test designs. Biometrics 55, 874-882.
21. Lehmacher, W. and Wassmer, G. (1999). Adaptive sample size calculations in group sequential trials. Biometrics 55, 1286-1290.
22. Lehmann, E. L. (1959). Testing Statistical Hypotheses. New York: Wiley.
23. Levin, G. P., Emerson, S. C., and Emerson, S. S. (2013a). Adaptive clinical trial designs with pre-specified rules for modifying the sample size: Understanding efficient types of adaptation. Statistics in Medicine 32, 1259-1275.
24. Levin, G. P., Emerson, S. C., and Emerson, S. S. (2013b). An evaluation of inferential procedures for adaptive clinical trial designs with pre-specified rules for modifying the sample size. UW Biostatistics Working Paper Series. Working Paper 388 http://biostats.bepress.com/uwbiostat/paper388.
25. Liu, Q. and Anderson, K. M. (2008). On adaptive extensions of group sequential trials for clinical investigations. Journal of the American Statistical Association 103, 1621-1630.
26. Mehta, C., Posch, M., Bauer, P., and Brannath, W. (2007). Repeated confidence intervals for adaptive group sequential trials. Statistics in Medicine 26, 5422-5433.
27. Mehta, C. R. and Pocock, S. J. (2011). Adaptive increase in sample size when interim results are promising: A practical guide with examples. Statistics in Medicine 30, 3267-3284.
28. Müller, H.-H. and Schäfer, H. (2001). Adaptive group sequential designs for clinical trials: Combining the advantages of adaptive and of classical group sequential approaches. International Biometric Society 57, 886-891.
29. O'Brien, P. C. and Fleming, T. R. (1979). A multiple testing procedure for clinical trials. Biometrics 35, 549-556.
30. Pocock, S. J. (1977). Group sequential methods in the design and analysis of clinical trials. Biometrika 64, 191-199.
31. Posch, M., Bauer, P., and Brannath, W. (2003). Issues in designing flexible trials. Statistics in Medicine 22, 953-969.
32. Proschan, M. A. and Hunsberger, S. A. (1995). Designed extension of studies based on conditional power. Biometrics 51, 1315-1324.
33. S+SeqTrial (2002). Insightful Corporation. Seattle, Washington.
34. Tsiatis, A. A., Rosner, G. L., and Mehta, C. R. (1984). Exact confidence intervals following a group sequential test. Biometrics 40, 797-803.
35. Wang, S. K. and Tsiatis, A. A. (1987). Approximately optimal one-parameter boundaries for group sequential trials. Biometrics 43, 193-199.
36. Whitehead, J. (1986). On the bias of maximum likelihood estimation following a sequential test. Biometrika 73, 573-581.
37. Whitehead, J. (1992). Overrunning and underrunning in sequential clinical trials. Controlled Clinical Trials 13, 106-121.