Dependence, hyper-dependence and hypothesis testing in clinical trials

Contemporary Clinical Trials

Volumn 28, Issue 1, 2007, Pages 68-78

  Moyé, Lemuel A ^a   Baraniuk, Sarah ^a  

^a UNIVERSITY OF TEXAS SCHOOL OF PUBLIC HEALTH   (United States)

Author keywords

Clinical trials; Dependence; Endpoint analyses; Multiple endpoints; Multiplicity

Indexed keywords

ANALYTICAL ERROR; ARTICLE; CLINICAL TRIAL; EXPLORATORY RESEARCH; GEOGRAPHIC DISTRIBUTION; HYPOTHESIS; METHODOLOGY; OUTCOME ASSESSMENT; PROBABILITY; PROCESS DESIGN; PROSPECTIVE STUDY; STATISTICAL ANALYSIS;

CLINICAL TRIALS; HUMANS; RESEARCH DESIGN; STATISTICS;

EID: 33751196148     PISSN: 15517144     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.cct.2006.05.010     Document Type: Article

References (52)

1. Meinert C.L. Clinical trials: design, conduct, and analysis (1986), Oxford University Press, New York
2. Friedman L., Furberg C., and DeMets D. Fundamentals of clinical trials. 3rd edition (1986), Springer, New York
3. Yusuf S., Wittes J., Probstfield J., and Tyroler H.A. Analysis and interpretation of treatment effects in subgroups of patients in randomized clinical trials. J Am Med Assoc 266 (1991) 93-98
4. The European Agency for the Evaluation of Medicinal Products. ICH topic E9. Note for guidance on statistical principles in clinical trials. CMPM/ICH/363/96 (1998)
5. Moyé L. Multiple analyses in clinical trials: fundamentals for investigators (2003), Springer, New York
6. Sacks F.M., Pfeffer M.A., Moyé L.A., et al. The effect of pravastatin on coronary events after myocardial infarction in patients with average cholesterol levels. N Engl J Med 335 (1996) 1001-1009
7. The HOPE Study Investigators. The HOPE (Heart Outcomes Prevention Evaluation) Study: The design of a large, simple randomized trial of an angiotensin-converting enzyme inhibitor (ramipril) and vitamin E in patients at high risk of cardiovascular events. Can J Cardiol 12 (1996) 127-137
8. Miller R.G. Developments in multiple comparisons 1966-1976. J Am Stat Assoc 72 (1977) 779-788
9. Tukey J.W., Ciminera J.L., and Heyse J.F. Testing the statistical certainty of a response to increasing doses of a drug. Biometrics 41 (1985) 295-301
10. Dubey S.D. Adjustment of p-values for multiplicities of intercorrelating symptoms. Proceedings of the VIth international society for clinical biostatisticians, Germany (1985)
11. O'Brien P.C. Procedures for comparing samples with multiple endpoints. Biometrics 40 (1984) 1079-1089
12. Sankoh A.J., Huque M.F., and Dubey S.D. Some comments on frequently used multiple endpoint adjustment methods in clinical trials. Stat Med 16 (1997) 2229-2242
13. Hochberg Y., and Westfall P.H. On some multiplicity problems and multiple comparison procedures in biostatistics. In: Sen P.K., and Rao C.R. (Eds). Handbook of Statistics vol. 18 (2000), Elsevier Sciences B.B. 75-113
14. James S. Approximate multinomial probabilities applied to correlated multiple endpoints in clinical trials. Stat Med (1991) 1123-1135
15. Neuhauser M., Steinijans V.W., and Bretz F. The evaluation of multiple clinical endpoints with application to asthma. Drug Inf J 33 (1999) 471-477
16. Reitmeir P., and Wassmer G. One sided multiple endpoints testing in two-sample comparisons. Commun Stat Simul Comput 25 (1996) 99-117
17. Westfall P.H., Ho S.Y., and Prillaman B.A. Properties of multiple intersection-union tests for multiple endpoints in combination therapy trials. J Biopharm Stat 11 (2001) 125-138
18. Hochberg Y., and Liberman U. An extended Simes' test. Stat Probab Lett 21 (1994) 101-105
19. Zhang J., Qwuan H., Ng J., and Stepanavage M.E. Some statistical methods for multiple endpoints in clinical trials. Control Clin Trials 18 (1997) 204-221
20. Wright S.P. Adjusted P-values for simultaneous inference. Biometrics 48 (1992) 1005-1013
21. Simes R.J. An improved Bonferroni procedure for multiple tests of significance. Biometrika 73 (1986) 819-827
22. Holm S. A simple sequentially rejective multiple test procedures. Scand J Statist 6 (1979) 65-70
23. Hommel G. A stepwise rejective multiple test procedure based on a modified Bonferroni test. Biometrika 75 (1988) 383-386
24. Shaffer J.P. Modified sequentially rejective multiple test procedures. J Am Stat Assoc 81 (1986) 826-831
25. Department of Health and Human Services Food and Drug Administration Center for Drug Evaluation and Research. Transcript of the Oncologic Drug Advisory Committee Sixty-Ninth Meeting, December 6, 2001.
26. Questions to the FDA Oncology Drugs Advisory Committee. December 6, 2001.
27. Hochberg Y., and Tamhane A.C. Multiple comparison procedures (1987), Wiley, New York
28. Westfall P.H., and Young S.S. Resampling based multiple testing: examples and methods for P-value adjustment (1993), Wiley, New York
29. Moyé L.A. P-value interpretation in clinical trials. The case for discipline. Control Clin Trials 20 (1999) 40-49
30. Kalbfleisch J.D., and Prentice R.L. The statistical analysis of failure time data (1980), John Wiley, New York
31. Kleinbaum D.G., Kupper L.L., and Morgenstern H. Epidemdiologic research: principles and quantitative methods (1982), Van Nostrand Reinhold Company, New York
32. Cox D.R. Regression models and life-tables. JR Stat Soc B 34 (1972) 187-220
33. Grizzle J.E. The two-period change-over design and its use in clinical trials. Biometrics 21 (1965) 467-480
34. Armitage P., and Hills M. The two-period crossover trial. The Statistician 32 (1982) 119-131
35. Senn S. Cross-over trials in clinical research (1993), John Wiley and Sons, Chicester, New York
36. Lin Z. The number of centers in a multicenter clinical study: effects on statistical power. Drug Inf J 54 (2000) 379-396
37. Wiens B., and Dmitrienko A. The fallback procedure for evaluating a single family of hypotheses. J Biopharm Stat 15 (2005) 929-942
38. Moyé L.A. The perils of nonprospectively planned research. Part 1: drawing conclusions from sample-based research. Am Clin Lab: April 2001 (2001) 34-36
39. Westfall P.H., Young S.S., and Wright S.P. Adjusting p-values for multiplicity. Biometrics 49 (1993) 941-945
40. Westfall P.H., and Young S. P-value adjustments for multiple tests in multivariate binomial models. J Am Stat Assoc 84 (1989) 780-786
41. Westfall P.H., Krishnen A., and Young S.S. Using prior information to allocate significance levels for multiple endpoints. Stat Med 17 (1998) 2107-2119
42. Reitmeir P., and Wassmer G. Resampling-based methods for the analysis of multiple endpoints in clinical trials. Stat Med 18 (1999) 3453-3462
43. Dunnett C.W., and Tamhane A.C. Multiple testing to establish superiority/equivalence of a new treatment compared with k standard treatments. Stat Med 16 (1997) 2489-2506
44. Cheung S.H., and Holland B. Extension of Dunnett's multiple comparison procedure to the case of several groups. Biometrics 47 (1991) 21-32
45. Davis B.R., Cutler J.A., Gordon D.J., et al. Rationale and design for the Antihypertensive and Lipid Lowering Treatment to Prevent Heart Attack Trial (ALLHAT). Am J Hypertens 9 (1996) 342-360
46. The Clopidogrel in Unstable Angina to Prevent Recurrent Events Trial Investigators. Effects of clopidogrel in addition to aspirin in patients with acute coronary syndromes without st-segment elevation. N Engl J Med 345 (2001) 494-502
47. Y. Benjamini and Y. Hochberg. Controlling the False Discovery Rate: A Practical and Powerful Approach to Multiple Testing. J. Roy Stat Soc, Ser B. 57:289-300.
48. Y. Benjamini, D. Yekutieli. The Control of the false discovery rate in multiple testing under dependency. Tech Report, School of Mathematical Sciences, Tel Aviv University. April 27, 2001. Available at http://www.math.tau.ac.il/~benja/.
49. Lan K.K.G., and DeMets D.L. Discrete sequential boundaries for clinical trials. Biometrika 70 (1983) 659-663
50. Lan K.K.G., and DeMets D.L. Changing frequency of interim analysis in sequential monitoring. Biometrics 45 (1989) 1017-1020
51. Lan K.K.G., and DeMets D.L. Group sequential procedures. Calendar vs. information time. Stat Med 8 (1989) 1191-1198
52. Lan K.K., and Wittes J. The B-value. A tool for monitoring data. Biometrics 44 (1988) 579-585