Design and semiparametric analysis of non-inferiority trials with active and placebo control for censored time-to-event data

Statistics in Medicine

Volumn 32, Issue 18, 2013, Pages 3055-3066

  Kombrink, Karola ^a   Munk, Axel ^a   Friede, Tim ^b  

^a UNIVERSITY OF GÖTTINGEN   (Germany)

^b UNIVERSITY MEDICAL CENTER GÖTTINGEN   (Germany)

Author keywords

Cox proportional hazards model; Non inferiority; Retention of effect; Sample size allocation; Semiparametric; Three arm design

Indexed keywords

ACCURACY; ALGORITHM; ARTICLE; CENSORED TIME TO EVENT DATA; GOLD STANDARD; MAJOR DEPRESSION; MATHEMATICAL COMPUTING; NON INFERIORITY TRIAL; PLACEBO EFFECT; SAMPLE SIZE; SEMIPARAMETRIC ANALYSIS; SIMULATION; STATISTICAL ANALYSIS; STATISTICAL DISTRIBUTION; STATISTICAL MODEL; STUDY DESIGN; SURVIVAL TIME;

COX PROPORTIONAL HAZARDS MODEL; NON-INFERIORITY; RETENTION OF EFFECT; SAMPLE SIZE ALLOCATION; SEMIPARAMETRIC; THREE-ARM DESIGN;

ALGORITHMS; COMPUTER SIMULATION; DATA INTERPRETATION, STATISTICAL; DEPRESSIVE DISORDER, MAJOR; HUMANS; MODELS, STATISTICAL; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; SAMPLE SIZE;

EID: 84880040485     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.5769     Document Type: Article

References (27)

1. FDA (Food and Drug Administration). Guidance for industry on non-inferiority clinical trials, 2010.
2. CHMP (Committee for Medicinal Products for Human Use). Guideline on the choice of the non-inferiority margin. EMEA/CPMP/EWP/2158/99, 2005.
3. Brown D, Volkers P, Day S. An introductory note to CHMP guidelines: choice of the non-inferiority margin and data monitoring committees. Statistics in Medicine 2006; 25:1623-1627. DOI: 10.1002/sim.2561.
4. Koch A, Röhmel J. Hypothesis testing in the "gold standard" design for proving the efficacy of an experimental treatment relative to placebo and a reference. Journal of Biopharmaceutical Statistics 2004; 14:315-325. DOI: 10.1081/BIP-120037182.
5. Mielke M, Munk A, Schacht A. Planning and assessing non-inferiority in a gold standard design with censored, exponentially distributed endpoints. Statistics in Medicine 2008; 27:5093-5110. DOI: 10.1002/sim.3348.
6. Pigeot I, Schäfer J, Röhmel J, Hauschke D. Assessing non-inferiority of an new treatment in a three-arm clinical trial including a placebo. Statistics in Medicine 2003; 22:883-899. DOI: 10.1002/sim.1450.
7. Hasler M, Vonk R, Hothorn LA. Assessing non-inferiority of an new treatment in a three-arm trial in the presence of heteroscedasticity. Statistics in Medicine 2008; 27:490-503. DOI: 10.1002/sim.3052.
8. Kieser M, Friede T. Planning and analysis of three-arm non-inferiority trials with binary endpoints. Statistics in Medicine 2007; 26:253-273. DOI: 10.1002/sim.2543.
9. Mielke M, Munk A. The assessment and planning of non-inferiority trials for retention of effect hypotheses - towards a general approach, 2009. arXiv:0912.4169v1[stat.ME].
10. Munzel U. Nonparametric non-inferiority analyses in the three-arm design with active control and placebo. Statistics in Medicine 2009; 28:3643-3656. DOI: 10.1002/sim.3727.
11. Hamilton M. A rating scale for depression. Journal of Neurology Neurosurgey and Psychiatry 1960; 23:56-62.
12. Montgomery SA. Clinically relevant effect sizes in depression. European Neuropsychopharmacology 1994; 4:283-284.
13. Nardi A, Michael S. Comparing Cox and parametric models in clinical studies. Statistics in Medicine 2003; 22:3597-3610. DOI: 10.1002/sim.3727.
14. Cox DR. Regression models and life-tables. Journal of the Royal Statistical Society, Series B 1972; 34:187-220.
15. Therneau TM, Grambsch PM. Modeling Survival Data: Extending the Cox Model. Springer: New York, 2001.
16. Cox DR. Partial likelihood. Biometrica 1975; 62:269-276.
17. Andersen PK, Borgan O, Gill RD, Keiding N. Statistical Models Based on Counting Processes. Springer, 1993.
18. Bernardo MVP, Harrington DP. Sample size calculations for the two-sample problem using the multiplicative intensity model. Statistics in Medicine 2001; 20:557-579. DOI: 10.1002/sim.693.
19. Hauschke D, Pigeot I. Establishing efficacy of a new experimental treatment in the 'gold standard' design. Biometrical Journal 2005; 47:782-786. DOI: 10.1002/bimj.200510169.
20. Koch A. Discussion on "Establishing Efficacy of an New Experimental Treatment in the 'Gold Standard' Design". Biometrical Journal 2005; 47:792-793. DOI: 10.1002/bimj.200510181.
21. Lange S, Freitag G. Choice of delta: requirements and reality - results of a systematic review. Biometrical Journal 2005; 47:12-27. DOI: 10.1002/bimj.200410085.
22. Röhmel J, Pigeot I. A comparison of multiple testing procedures for the gold standard non-inferiority trial. Journal of Biopharmaceutical Statistics 2010; 20:911-926. DOI: 10.1080/10543401003618942.
23. Conn AR, Scheinberg K, Vicente LN. Introduction to Derivative-Free Optimization, MPS/SIAM Series on Optimization 8. Society for Industrial and Applied Mathematics (SIAM): Philadelphia, PA, 2009.
24. Rothmann M, Li N, Chen G, Chi GYH, Temple R, Tsou H-H. Design and analysis of non-inferiority mortality trials in oncology. Statistics in Medicine 2001; 22:239-264. DOI: 10.1002/sim.1400.
25. Hida E, Toshiro T. On the three-arm non-inferiority trial including a placebo with a prespecified margin. Statistics in Medicine 2011; 30:224-231. DOI: 10.1002/sim.4099.
26. Röhmel J, Pigeot I. Statistical strategies for the analysis of clinical trials with an experimental treatment, an active control and placebo, and a prespecified fixed non-inferiority margin for the difference in means. Statistics in Medicine 2011; 30:3162-3164. DOI: 10.1002/sim.4299.
27. Hida E, Toshiro T. On the three-arm non-inferiority trial including a placebo with a prespecified margin. Statistics in Medicine 2011; 30:224-231. DOI: 10.1002/sim.4313.