Multiplicity considerations in the design and analysis of clinical trials

Journal of the Royal Statistical Society. Series A: Statistics in Society

Volumn 159, Issue 1, 1996, Pages 93-110

  Cook, Richard J ^a   Farewell, Vern T ^b  

^a UNIVERSITY OF WATERLOO   (Canada)

^b NONE

Author keywords

Multiple outcomes; Multiple treatment arms; p value; Power; Sequential tests; Size; Type I error rate

Indexed keywords

EID: 0038975320     PISSN: 09641998     EISSN: None     Source Type: Journal    
DOI: 10.2307/2983471     Document Type: Article

References (53)

1. Armitage, P., McPherson, C. K. and Rowe, B. C. (1969) Repeated significance tests on accumulating data. J. R. Statist. Soc. A, 132, 235-244.
2. Bauer, P. (1991) Multiple testing in clinical trials. Statist. Med., 10, 871-890.
3. Cook, R. J. (1994) Interim monitoring of bivariate response using repeated confidence intervals. Contr. Clin. Trials, 15, 187-200.
4. _ (1996) Coupled error spending functions for parallel bivariate sequential tests. Biometrics, to be published.
5. Cook, R. J. and Farewell, V. T. (1995) Guidelines for monitoring efficacy and toxicity responses in clinical trials. Biometrics, 50, 1146-1152.
6. Cox, D. R. (1965) A remark on multiple comparison methods. Technometrics, 7, 223-224.
7. _ (1977) The role of significance tests. Scand. J. Statist., 4, 49-70.
8. Cox, D. R., Fitzpatrick, R., Fletcher A. E., Gore, S. M., Spiegelhalter, D. J. and Jones, D. R. (1992) Quality-of-life assessment: can we keep it simple (with discussion)? J. R. Statist. Soc. A, 155, 353-393.
9. Cox, D. R. and Spjotvoll, E. (1982) On partitioning means into groups. Scand. J. Statist., 9, 147-152.
10. Diddle, A. W., O'Connor, K. A. and Pearce, R. L. (1953) Evaluation of bed rest in threatened abortion. Obstetr. Gyn., 2, 63-67.
11. Duncan, O. B. (1955) Multiple range and multiple F-tests. Biometrics, 11, 1-42.
12. Dunnett, C. (1955) A multiple comparisons procedure for comparing several treatments with a control. J. Am. Statist. Ass., 50, 1096-1121.
13. Farewell, V. T. and Cook, R. J. (1992) Comment: Evaluating therapeutic interventions: some issues and experiences. Statist. Sci., 7, 446-448.
14. Fisher, R. A. (1946) Statistical Methods for Research Workers. New York: Hafner.
15. _ (1955) Statistical methods and scientific induction. J. R. Statist. Soc. B, 17, 69-78.
16. _ (1960) Scientific thought and the refinement of human reason. J. Oper. Res. Soc. Jpn, 3, 1-10.
17. _ (1971) The Design of Experiments. New York: Hafner.
18. _ (1973) Statistical Methods for Scientific Inference. New York: Hafner.
19. Fitzpatrick, R., Bury, M., Frank, A. and Donnelly, T. (1987) Problems in the assessment of outcome in a back pain clinic. Int. Disblty Stud., 9, 161-165.
20. Fleming, T. R. (1990) Evaluation of active control trials in AIDS. J. Acq. Immune Def. Synd., 3, S82-S87.
21. Friend, P., Hale, G., Waldman, S., Gore, S., Thiru, S., Joysey, V., Evans, D. B. and Calne, R. Y. (1994) Campath-1 prophylactic use after kidney transplantation - a randomized controlled trial. To be published.
22. Gupta, S. S. (1963) Probability integrals of multivariate normal and multivariate t. Ann. Math. Statist., 34, 792-828.
23. Hettmansperge, T. P. and Norton, R. M. (1987) Tests for patterned alternatives in K-sample problems. J. Am. Statist. Ass., 82, 292-299.
24. Hochberg, Y. (1988) A sharper Bonferroni procedure for multiple tests of significance. Biometrika, 75, 800-802.
25. Hoge, C. W., Shlim, D. R., Ghimire, M., Rabold, J. G., Pandey, P., Walch, A., Rajah, R., Gaudio, P. and Echeverria, P. (1995) Lancet, 345, 691-693.
26. Holm, S. (1979) A simple sequentially rejective multiple test procedure. Scand. J. Statist., 6, 65-70.
27. Hoppe, F. M. (1993) Multiple Comparisons, Selection, and Applications in Biometry. New York: Dekker.
28. Hughes, M. H. (1993) Stopping guidelines for clinical trials with multiple treatments. Statist. Med., 12, 901-915.
29. Jennison, C. and Turnbull, B. W. (1989) Interim analyses: the repeated confidence interval approach (with discussion). J. R. Statist. Soc. B, 51, 305-361.
30. Keuls, M. (1952) The use of the studentized range in connection with an analysis of variance. Euphytica, 1, 112-122.
31. Lan, K. K. G. and DeMets, D. L. (1983) Discrete sequential boundaries for clinical trials. Biometrika, 70, 659-663.
32. _ (1989) Group sequential procedures: calendar versus information time. Statist. Med., 8, 1191-1198.
33. Lehman, E. (1993) The Fisher, Neyman-Pearson theories of testing hypotheses: one theory or two? J. Am. Statist. Ass., 88, 1242-1249.
34. Lin, D. Y. (1991) Nonparametric sequential testing in clinical trials with incomplete multivariate observations. Biometrika, 78, 123-131.
35. Lin, D. Y. and Liu, P. Y. (1992) Nonparametric sequential tests against ordered alternatives in multi-armed clinical trials. Biometrika, 79, 420-427.
36. Liu, P. Y., Green, S., Wolf, M. and Crowley, J. (1993) Testing against ordered alternatives for censored survival data. J. Am. Statist. Ass., 88, 153-160.
37. Mau, J. (1988) A generalization of a nonparametric test for stochastically ordered distributions to censored survival data. J. R. Statist. Soc. B, 50, 403-412.
38. Newman, D. (1939) The distribution of range in samples from a normal population, expressed as an independent estimate of standard deviation. Biometrika, 31, 20-30.
39. Neyman, J. and Pearson, E. S. (1928) On the use and interpretation of certain test criteria for purposes of statistical inference. Biometrika A, 20, 170-240, 263-294.
40. _ (1933) On the problem of the most efficient tests of statistical hypotheses. Phil. Trans. R. Soc. Lond. A, 231, 289-337.
41. North American Symptomatic Carotid Endarterectomy Trial Collaborators (1991) Beneficial effect of carotid endarterectomy in symptomatic patients with high-grade carotid stenosis. New Engl. J. Med., 325, 445-453.
42. O'Brien, P. C. (1984) Procedures for comparing samples with multiple endpoints. Biometrics, 40, 1079-1087.
43. O'Brien, P. C. and Fleming, T. R. (1979) A multiple testing procedure for clinical trials. Biometrics, 35, 549-556.
44. Pocock, S. J. (1977) Group sequential methods in the design and analysis of clinical trials. Biometrika, 64, 191-199.
45. Pocock, S. J., Geller, N. L. and Tsiatis, A. A. (1987) The analysis of multiple endpoints in clinical trials. Biometrics, 43, 487-498.
46. Prentice, R. L., Storb, R., Brown, K. S. and Mason, M. W. (1984) HLA and disease: relative risk regression methods and multiple testing considerations. Biometrics, 40, 653-662.
47. Proschan, M. A., Follmann, D. A. and Geller, N. L. (1994) Monitoring multi-armed trials. Statist. Med., 13, 1441-1452.
48. Scheffé, H. (1953) A method for judging all contrasts in the analysis of variance. Biometrika, 40, 87-104.
49. Simes, R. J. (1986) An improved Bonferroni procedure for multiple tests of significance. Biometrika, 73, 751-754.
50. Tang, D. I., Gnecco, C. and Geller, N. L. (1989) Design of group sequential clinical trials with multiple endpoints. J. Am. Statist. Ass., 84, 776-779.
51. Torrance, G. W. (1987) Utility approach to measuring health related quality of life. J. Chron. Dis., 40, 593-600.
52. Tukey, J. (1949) Comparing individual means in the analysis of variance. Biometrics, 5, 99-114.
53. Wei, L. J., Lin, D. Y. and Weissfeld, L. (1989) Regression analysis of multivariate incomplete failure time data by modelling marginal distributions. J. Am. Statist. Ass., 84, 1065-1073.