A two-stage sample size recalculation procedure for placebo- and active-controlled non-inferiority trials

Statistics in Medicine

Volumn 25, Issue 19, 2006, Pages 3396-3406

  Schwartz, Todd A ^a   Denne, Jonathan S ^b  

^a IL   (United States)

^b ELI LILLY AND COMPANY   (United States)

Author keywords

Internal pilot; Non inferiority; Sample size recalculation; Two stage design

Indexed keywords

ARTICLE; CALCULATION; CLINICAL RESEARCH; DATA ANALYSIS; METHODOLOGY; PATIENT SELECTION; PROBABILITY; RANDOMIZATION; SAMPLE SIZE; SENSITIVITY AND SPECIFICITY; STATISTICAL ANALYSIS; SYSTEMATIC ERROR;

COMPUTER SIMULATION; CONTROLLED CLINICAL TRIALS; CONTROLLED CLINICAL TRIALS AS TOPIC; DRUG EVALUATION; HUMANS; PLACEBOS; RANDOM ALLOCATION; RESEARCH DESIGN; SAMPLE SIZE;

EID: 33749332490     PISSN: 02776715     EISSN: 10970258     Source Type: Journal    
DOI: 10.1002/sim.2651     Document Type: Article

References (11)

1. Temple R, Ellenberg SS. Placebo-controlled trials and active-control trials in the evaluation of new treatments (Part I: ethical and scientific issues). Annals of Internal Medicine 2000; 133:455-463.
2. Röhmel J. Therapeutic equivalence investigations: statistical considerations. Statistics in Medicine 1998; 17:1703-1714.
3. Koch GG, Tangen CM. Nonparametric analysis of covariance and its role in noninferiority clinical trials. Drug Information Journal 1999; 33:1145-1159.
4. Pigeot I, Schäfer J, Röhmel J, Hauschke D. Assessing non-inferiority of a new treatment in a three-arm clinical trial including placebo. Statistics in Medicine 2003; 22:883-899.
5. Fieller EC. The distribution of the index in a normal bivariate population. Biometrika 1932; 24:428-440.
6. Denne JS. Designing an active control non-inferiority trial with a placebo arm. Presentation at FDA/PhRMA Workshop Non-Inferiority Trials: Challenges and Opportunities, Bethesda, MD, 2002.
7. Wittes J, Brittain E. The role of internal pilot studies in increasing the efficiency of clinical trials. Statistics in Medicine 1990; 9:65-72.
8. Proschan MA, Hunsberger SA. Designed extension of studies based on conditional power. Biometrics 1995; 51:1315-1324.
9. SAS Institute Inc. The SAS System, Version 8. Cary, NC, SAS Institute Inc., 1999.
10. Hadgu A, Koch G. Application of generalized estimating equations to a dental randomized trial. Journal of Biopharmaceutical Statistics 1999; 9:161-178.
11. Jennison C, Turnbull BW. Group Sequential Methods with Applications to Clinical Trials. Chapman & Hall/CRC: London, Boca Raton, FL, 2000.