Calculation of tolerance limits and sample size determination for clinical trials with dichotomous outcomes

Journal of Biopharmaceutical Statistics

Volumn 17, Issue 3, 2007, Pages 481-491

  Zaslavsky, Boris G ^a  

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

Author keywords

Binomial distribution; Clinical trial; Confidence probability; Dichotomous outcome; Negative binomial distribution; Tolerance limit

Indexed keywords

ALGORITHM; ARTICLE; BINOMIAL DISTRIBUTION; CLINICAL RESEARCH; CONFIDENCE INTERVAL; DATA ANALYSIS SOFTWARE; MATHEMATICAL COMPUTING; OUTCOME ASSESSMENT; PRIORITY JOURNAL; SAMPLE SIZE; SAMPLING; STATISTICS;

BINOMIAL DISTRIBUTION; CLINICAL TRIALS; CONFIDENCE INTERVALS; DATA INTERPRETATION, STATISTICAL; HUMANS; MODELS, STATISTICAL; SAMPLE SIZE; SOFTWARE; TREATMENT OUTCOME;

EID: 34247869623     PISSN: 10543406     EISSN: 15205711     Source Type: Journal    
DOI: 10.1080/10543400701199601     Document Type: Article

References (21)

1. Armitage, P., Colton, T. (1998). Encyclopedia of Biostatistics. New York: John Wiley & Sons.
2. Beyer, W. H. (1991). CRC Standard Probability and Statistics Tables and Formulae. Boca Raton, Ann Arbor, Boston: CRC Press Inc.
3. Brown, E. B., Iyer, H. K. (1997). Tolerance intervals for assessing individual bioequivalence. Stat. Med. 16:803-820.
4. Chow, S. C., Shao, J. (1999). Bioequivalence review for drug interchangeability. J. Biopharm. Stat. 9:485-497.
5. Clopper, C. J., Pearson E. S. (1934). The use of confidence or fiducial limits illustrated in the case of the binomial. Biometrika 26:404-413.
6. Cowan, E. P. (2002). Draft Guidance Document: Review Approval Process for Rapid HIV Tests at FDA (BPAC). Food and Drug Administration, Center for Biologics Evaluation and Research. http://www.cdc.gov/hiv/pubs/rt/B-Cowan.pdf
7. CYTEL Software Corporation. (2001). StatXact 5 for Windows User's Manual. Cambridge: MA.
8. Dumont, L. J., Dzik, W. H., Rebulla, P., Brandwein, H., and the members of the BEST Working Party of the ISBT. (1996). Practical guidelines for process validation and process control of white cell-reduced blood components: report of the Biomedical Excellence for Safer Transfusion (BEST) Working Party of the International Society of Blood Transfusion (ISBT). Transfusion 36:11-20.
9. Esinhart, J. D., Chinchilli, V. M. (1994a). Sample size considerations for assessing individual bioequivalence based on the method tolerance interval. Int. J. Clin. Pharm. Therapeutics 32:26-32.
10. Esinhart, J. D., Chinchilli, V. M. (1994b). Extension to the use of tolerance intervals for the assessment of individual bioequivalence. J. Biopharm. Stat. 4:39-52.
11. Food and Drug Administration, Center for Biologics Evaluation and Research. (2001). Guidance for Industry: Pre-Storage Leukocyte Reduction of Whole Blood and Blood Components Intended for Transfusion. Rockville, MD: www.fda.gov/cber/gdlns/preleuk.pdf
12. Gunkemeir, G. L., MacCulloch, D., Naftel, D. (1994). sample size requirements for evaluating heart valve with constant risk events. J. Heart Valve Dis. 4:53-58.
13. Hahn, G. J., Meeker, W. Q. (1991). Statistical Intervals. A Guide for Practitioners. New York, Chichester, Brisbane, Toronto, Singapore: John Wiley & Sons, Inc.
14. Hoffman, D., Kringle, R. (2005). Two-sided tolerance intervals for balanced and unbalanced random effects models. J. Biopharm. Stat. 15:283-293.
15. Hormuzd, A. K., Engels, E. A., Rosenberg, P. S. (2005). Assessing uncertainty in reference intervals via tolerance intervals: application to mixed model describing HIV infection. Stat. Med. 24:3185-3198.
16. Jennen-Steinmetz, C., Wellek, S. (2005). A new approach to sample size calculation for reference interval studies. Stat. Med. 20:3199-3212.
17. Shirke, D. T., Kumbhar, R. R., Kundu, D. (2005). Tolerance intervals for exponentiated scale family of distributions. J. Appl. Stat. 32:1067-1074.
18. Walter, W., Hauck, R. S. (2004). Modified two-sided normal tolerance intervals for batch acceptance of dose uniformity. Pharm. Stat. 3:89-97.
19. Westlake, W. J. (1979). Statistical aspects of comparative bioavailability trials. Biometrics 35:273-280.
20. Zacks, S. (1970). Uniformly most accurate upper tolerance limits for monotone likelihood ratio families of discrete distributions. JASA 65:307-316.
21. Zacks, S. (1971). The Theory of Statistical Inference. NY, London, Sydney, Toronto: John Wiley& Sons, Inc.