An evidential approach to non-inferiority clinical trials

Pharmaceutical Statistics

Volumn 10, Issue 5, 2011, Pages 440-447

  Wang, Sue Jane ^a   Blume, Jeffrey D ^b  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^b VANDERBILT UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

constancy assumption; likelihood ratio; misleading evidence; non inferiority margin; strength of evidence

Indexed keywords

ACETYLSALICYLIC ACID; HEPARIN; LEPIRUDIN; PLACEBO;

ACUTE CORONARY SYNDROME; ARTICLE; CLINICAL TRIAL (TOPIC); CONCEPTUAL FRAMEWORK; EVIDENCE BASED MEDICINE; HEART INFARCTION; HUMAN; METHODOLOGY; PLACEBO EFFECT; REGRESSION ANALYSIS;

ACUTE CORONARY SYNDROME; ANTICOAGULANTS; ASPIRIN; CONTROL GROUPS; DRUGS, INVESTIGATIONAL; FIBRINOLYTIC AGENTS; HEPARIN; HIRUDINS; HUMANS; LIKELIHOOD FUNCTIONS; META-ANALYSIS AS TOPIC; MODELS, STATISTICAL; PLACEBOS; PLATELET AGGREGATION INHIBITORS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECOMBINANT PROTEINS; REGRESSION ANALYSIS; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 80053568926     PISSN: 15391604     EISSN: 15391612     Source Type: Journal    
DOI: 10.1002/pst.513     Document Type: Article

References (49)

1. Wang SJ, Hung HMJ, Tsong Y,. Non-inferiority analysis in active controlled clinical trials. Encyclopedia of Biopharmaceutical Statistics, Chapter 146, Volumes 1 and 2, 3rd ed. Marcel Dekker: New York, NY, 2010.
2. Wang SJ, Hung HMJ, Tsong Y,. Utility and pitfall of some statistical methods in active controlled clinical trials. Controlled Clinical Trials 2002; 23: 15-28. (Pubitemid 34164355)
3. Hung HMJ, Wang SJ, Tsong Y, Lawrence J, O'Neill RT,. Some fundamental issues with non-inferiority testing in active controlled clinical trials. Statistical in Medicine 2003; 22: 213-225. (Pubitemid 36163582)
4. Tsong Y, Wang SJ, Hung HMJ, Cui L,. Statistical issues on objective, design and analysis of non-inferiority active controlled clinical trial. Journal of Biopharmaceutical Statistics 2003; 13: 29-42.
5. D'Agostino RB, Massaro JM, Sullivan L,. Non-inferiority trials: design concepts and issues-The encounters of academic consultants in statistics. Statistics in Medicine 2003; 22: 169-186. (Pubitemid 36163579)
6. Hung HMJ, Wang SJ, O'Neill R,. A regulatory perspective on choice of margin and statistical inference issue in non-inferiority trials. Biometrical Journal 2005; 47: 28-36. (Pubitemid 40390436)
7. Hung HMJ, Wang SJ, O'Neil RT,. Issues with statistical risks for testing methods in noninferiority trial without a placebo arm. Journal of Biopharmaceutical Statistics 2007; 17: 201-213. (Pubitemid 46423595)
8. Hung HMJ, Wang SJ, O'Neill R,. Challenges and regulatory experiences with non-inferiority trial design without placebo arm. Biometrical Journal 2009; 51 (2): 324-334.
9. Royall RM,. Statistical evidence. Chapman & Hall: London, 1997.
10. Blume JD,. Likelihood methods for measuring statistical evidence. Statistics in Medicine 2002; 21 (17): 2563-2599. (Pubitemid 34960017)
11. Blume JD,. Likelihood and its Evidential Framework. In Handbook of The Philosophy of Science: Philosophy of Statistics, Gabbay DM, Woods J, (eds). San Diego: North Holland, 2011; pp. 493-511.
12. International Conference on Harmonization: Statistical principles for clinical trials (ICH E-9), Food and Drug Administration, DHHS, 1998.
13. International Conference on Harmonization: Guidance on choice of control group and related design and conduct issues in clinical trials (ICH E-10), Food and Drug Administration, DHHS, July 2000.
14. Holmgren EB,. Establishing equivalence by showing that a prespecified percentage of the effect of the active control over placebo is maintained. Journal of Biopharmaceutical Statistics 1999; 9: 651-659. (Pubitemid 29535953)
15. Snapinn SM,. Alternatives for discounting in the analysis of non-inferiority trials. Journal of Biopharmaceutical Statistics 2004; 14: 263-273. (Pubitemid 38715780)
16. Rothmann M, Li N, Chen G, Chi GYH, Temple RT, Tsou HH,. Non-inferiority methods for mortality trials. Statistics in Medicine 2003; 22: 239-264.
17. Wang SJ, Hung HMJ,. Assessment of treatment efficacy in non-inferiority trials. Controlled Clinical Trials 2003; 24: 147-155. (Pubitemid 36412014)
18. Hasselblad V, Kong DF,. Statistical methods for comparison to placebo in active-control trials. Drug Information Journal 2001; 35: 435-449. (Pubitemid 32524896)
19. Simon R,. Bayesian design and analysis of active control clinical trials. Biometrics 1999; 55: 484-487. (Pubitemid 29302573)
20. Temple R, Ellenberg SS,. Placebo-controlled trials and active-control trials in the evaluation of new treatments-Part 1: Ethical and scientific issues. Annals of Internal Medicine 2000; 133: 455-463.
21. Ellenberg SS, Temple R,. Placebo-controlled trials and active-control trials in the evaluation of new treatments-Part 2: Practical issues and specific cases. Annals of Internal Medicine 2000; 133: 464-470.
22. Julious SA, Wang SJ,. How biased are indirect comparisons, particularly when comparisons are made over time in controlled trials? Drug Information Journal 2008; 42: 625-633.
23. Chen YF, Wang SJ, Khin NA, Hung HMJ, Laughren TP,. Trial design issues and treatment effect modeling in multi-regional schizophrenia trials. Pharmaceutical Statistics 2010; 9 (3): 217-229.
24. OASIS-2 Investigators. Effects of recombinant hirudin (lepirudin) compared with heparin on death, myocardial infarction, refractory angina, and revascularization procedures in patients with acute myocardial ischemia without ST elevation: a randomized trial. Lancet 1999; 353 (9151): 429-438.
25. Oler A, Whooley MA, Oler J, Grady D,. Adding heparin to aspirin reduces the incidence of MI and death in patients with unstable angina: A meta-analysis. JAMA 1996; 276 (10): 811-815. (Pubitemid 26301581)
26. Blume JD,. How often Likelihood ratios are misleading in sequential trials. Communications in Statistics, Theory and Methods 2008; 37 (8): 1193-1206. (Pubitemid 351269018)
27. Goodman SN, Royall RM,. Evidence and scientific research. American Journal of Public Health 1988; 78 (12): 1568-1574.
28. Strug L, Hodge SE,. An alternative foundation for the planning and evaluation of linkage analysis: 1. Decoupling 'error probabilities' from 'measures of evidence'. Human Heredity 2006a; 61: 166-188. (Pubitemid 44385445)
29. Strug L, Hodge SE,. An alternative foundation for the planning and evaluation of linkage analysis: 1. Implications for multiple test adjustments. Human Heredity 2006b; 61: 200-209. (Pubitemid 44357217)
30. Van der Tweel I,. Repeated looks at accumulating data: to correct or not to correct? European Journal of Epidemiology 2005; 20: 205-211. (Pubitemid 40569167)
31. Birnbaum A,. On the foundations of statistical inference (with discussion). JASA 1962; 53: 259-326.
32. Barnard GA,. Statistical Inference. Journal of the Royal Statistical Society, Series B 1949; 11: 115-149.
33. Royall RM,. On the probability of observing misleading statistical evidence (with discussion). Journal of the American Statistical Association 2000; 95 (451): 760-767.
34. DerSimonian R, Laird R,. Meta-analysis in clinical trials. Controlled Clinical Trials 1986; 7: 177-188.
35. Whitehead A,. Meta-analysis of controlled clinical trials. John Wiley and Sons: New York, 2002; 153-154.
36. Liang KY, Zeger SL,. Longitudinal data analysis using GLMs. Biometrika 1986; 73: 12.
37. Laird NM, Ware JH,. Random-effects models for longitudinal data. Biometrics 1982; 38: 963-974.
38. Zeger SL, Liang KY,. Longitudinal data analysis for discrete and continuous outcomes. Biometrics 1986; 42: 121-130.
39. Zeger SL, Liang KY, Albert PS,. Models for longitudinal data: A GEE approach. Biometrics 1988; 44: 1049-1060.
40. Diggle PJ, Liang KY, Zeger SL,. Analysis of longitudinal data. Oxford Press: New York, 1994.
41. Blume JD, Su L, Acosta L, McGarvey S,. Statistical evidence for GLM parameters: a robust likelihood approach. Statistics in Medicine 2007; 26 (15): 2919-2936. (Pubitemid 46939419)
42. Eddings WD,. Topics in the philosophy of statistics: methods, data and theory. Unpublished Biostatistics PhD Thesis, Johns Hopkins University School of Public Health, 2003.
43. Edwards AWF,. Likelihood. Cambridge University Press: London, 1971.
44. Hacking I,. Logic of statistical evidence. Cambridge University Press: New York, 1965.
45. Wang SJ, Hung HMJ,. TACT Method for non-inferiority testing in active controlled trials. Statistics in Medicine 2003; 22 (2): 227-238. (Pubitemid 36163583)
46. Albert PS,. Longitudinal data analysis (repeated measures) in clinical trials. Statistics in Medicine 1999; 18: 1707-1732. (Pubitemid 29328979)
47. Blume JD,. How to choose a working model for measuring the statistical evidence about a regression parameter. International Statistical Review 2005; 73 (2): 351-363. (Pubitemid 43002838)
48. Royall RM, Tsou TS,. Interpreting statistical evidence using imperfect models: Robust adjusted likelihood functions. Journal of the Royal Statistical Society, Series B 2003; 65 (2): 391-404.
49. Tsou TS, Royall RM,. Robust likelihoods. JASA 1995; 90 (419): 316-320.