Statistical Considerations for NonInferiority/Equivalence Trials in Vaccine Development

REFERENCES

• Agresti , A. , Min , Y. ( 2001 ). On small-sample confidence intervals for parameters in discrete distributions . Biometrics 57 : 963 – 971 [INFOTRIEVE] [CSA]
   PubMed Web of Science ®Google Scholar
• Blackwelder , W. C. ( 1982 ). Proving the null hypothesis in clinical trials . Controlled Clinical Trials 3 : 345 – 353 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMedGoogle Scholar
• Blackwelder , W. C. ( 1995 ). Similarity/equivalence trials for combination vaccine . Williams , J. C. ; Goldenthal , K. L. , Burns , D. L. , Lewis , B. P. eds. Combined Vaccines and Simultaneous Administration . Ann. New York Acad. Sciences , 754 : 321 – 328 .
   Google Scholar
• Chan , I. S. F. ( 1998 ). Exact tests of equivalence and efficacy with a non-zero lower bound for comparative studies . Statistics in Medicine 17 : 1403 – 1413 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Chan , I. S. F. ( 2002 ). Power and sample size determination for noninferiority trials using an exact method . Journal of Biopharmaceutical Statistics 12 ( 4 ): 457 – 469 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMedGoogle Scholar
• Chan , I. S. F. ( 2003 ). Proving noninferiority or equivalence of two treatments with dichotomous endpoints using exact methods , Statistical Methods in Medical Research 12 : 37 – 58 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Chan , I. S. F. , Mehrotra , D. V. ( 2003 ). Confidence intervals and hypothesis testing . Encyclopedia of Biopharmaceutical Statistics, , 2nd ed. New York : Marcel Dekker , pp. 231 – 234 .
   Google Scholar
• Chan , I. S. F. , Wang , W. W. B. , Heyse , J. F. ( 2003 ). Vaccine clinical trials . Encyclopedia of Biopharmaceutical Statistics. , 2nd ed. Chow , S. C. eds. New York : Marcel Dekker, Inc. pp. 1005 – 1022 .
   Google Scholar
• Chan , I. S. F. , Zhang , Z. X. ( 1999 ). Test-based exact confidence intervals for the difference of two binomial proportions . Biometrics 55 : 1202 – 1209 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Dunnett , C. W. , Gent , M. (1977). Significance testing to establish equivalence between treatments with special reference to data in the form of 2 × 2 tables. Biometrics 33:593–602 [INFOTRIEVE] [CSA]
   PubMed Web of Science ®Google Scholar
• Ebbutt , A. F. , Frith , L. ( 1999 ). Practical issues in equivalence trials . Stat. in Med. 1691 – 1701 [CSA]
   PubMed Web of Science ®Google Scholar
• European Medicines Agency (EMEA) . ( 2005 ). Committee for Medicinal Products for Human Uses (CHMP) , Guideline on the choice of noninferiority margin . July .
   Google Scholar
• Farrington , C. P. , Manning , G. ( 1990 ). Test statistics and sample size formulae for comparative binomial trials with null hypothesis of non-zero risk difference or non-unity relative risk . Statistics in Medicine 9 : 1447 – 1454 [INFOTRIEVE] [CSA]
   PubMed Web of Science ®Google Scholar
• FDA . ( 1997 ). Guidance for industry for the evaluation of combination vaccines for preventable diseases: production, testing and clinical studies . Center for Biologics Evaluation and Research, Food and Drug Administration [CSA]
   Google Scholar
• et al. . ( 1999 ). Interference of antibody production to hepatitis B surface antigen in a combination hepatitis A/hepatitis B vaccine . J. Infect. Dis. 180 ( 6 ): 2018 – 2022 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Gail , M. , Simon , R. ( 1985 ). Testing for qualitative interactions between treatment effects and patient subsets . Biometrics 41 : 361 – 372 [INFOTRIEVE] [CSA]
   PubMed Web of Science ®Google Scholar
• Gail , M. H. , Mark , S. D. , Carroll , R. J. , Green , S. B. , Pee , D. ( 1996 ). On design considerations and randomization-based inference for community intervention trials . Statistics in Medicine 15 : 1069 – 1092 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Ganju , J. , Mehrotra , D. V. ( 2003 ). Stratified experiments re-examined with emphasis on multicenter clinical trials . Controlled Clinical Trials 24 : 167 – 181 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMedGoogle Scholar
• Horne , A. D. ( 1995 ). The statistical analysis of immunogenicity data in vaccine trials: a review of methodologies and issues . Ann. N Y Acad. Sci. 754 : 329 – 46 [INFOTRIEVE] [CSA]
   PubMed Web of Science ®Google Scholar
• Horne , A. D. , Lachenbruch , P. A. , Getson , P. R. , Hsu , H. ( 2001 ). Analysis of studies to evaluate immune response to combination vaccines . Clinical Infectious Diseases 33 ( Suppl 4 ): S306 – 11 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMedGoogle Scholar
• ICH E9 Expert Working Group . ( 1999 ). Statistical principles for clinical trials: ICH harmonized tripartite guidelines . Statistics in Medicine 18 : 1905 – 1942 [CSA]
   PubMed Web of Science ®Google Scholar
• ICH E10 Guideline . ( 2000 ). Choice of control group and related issues in clinical trials . International Conference on Harmonization (ICH) , July .
   Google Scholar
• Kong , L. , Kohberger , R. C. , Koch , G. G. ( 2004 ). Type I error and power in noninferiority/equivalence trials with correlated endpoints: an example from vaccine development trials . J. of Biopharmaceutical Statistics 14 : 893 – 907 [CROSSREF] [CSA]
   PubMedGoogle Scholar
• Li , X. , Mehrotra , D. V. , Barnard , J. ( in press ). Analysis of incomplete longitudinal binary data using multiple imputation . Statistics in Medicine [CSA]
   Web of Science ®Google Scholar
• Liang , K. , Zeger , S. ( 2000 ). Longitudinal data analysis of continuous and discrete response for pre-post designs . Sankhya 62 : 134 – 138 [CSA]
   Google Scholar
• Mehrotra , D. V. ( 2001 ). Stratification issues with binary endpoints . Drug Information Journal 35 ( 4 ): 1343 – 1350 [CSA]
   Google Scholar
• Mehrotra , D. V. ( 2002 ) Stratified comparative clinical trials—analysis and interpretation issues . Proceeding of International Biometrics Conference [CSA]
   Google Scholar
• Mehrotra , D. V. Vaccine clinical trials – A statistical primer . Journal of Biopharmaceutical Statistics ( in press ) [CSA]
   Web of Science ®Google Scholar
• Mehrotra , D. V. , Chan , I. S. F. ( 2000 ). Testing for treatment by stratum interaction in stratified comparative binomial trial . Joint Statistical Meetings . August [CSA]
   Google Scholar
• Mehrotra , D. V. , Chan , I. S. F. , Berger , R. L. ( 2003 ). A cautionary note on exact unconditional inference for a difference between two independent binomial proportions . Biometrics 59 : 441 – 450 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Mehrotra , D. V. , Railkar , R. ( 2000 ). Minimum risk weights for comparing treatments in stratified binomial trials . Statistics in Medicine 19 : 811 – 825 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Miettinen , O , Nurminen , M. ( 1985 ). Comparative analysis of two rates . Statistics in Medicine 4 : 213 – 226 [INFOTRIEVE] [CSA]
   PubMed Web of Science ®Google Scholar
• Pan , G. H. , Wolfe , D. A. (1997). Test for qualitative interaction of clinical significance. Statistics in Medicine 16:1645–1652 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Plikaytis , B. D. , Carlone , P. M. ( 2004 ). Statistical considerations for vaccine immunogenicity trials: Part 1 and Part 2 , Vaccine 23 ( 13 ): 1596 – 1614 [CROSSREF] [CSA]
   Google Scholar
• Schuirmann , D. J. ( 1987 ). A comparison of the two one-sided tests procedure and the power approach for assessing the equivalence of average bioavailability . Journal of Pharmacokinetics and Biopharmaceutics 15 : 657 – 680 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMedGoogle Scholar
• Stine , R. A. , Heyse , J. F. ( 2001 ). Nonparametric measures of overlap . Stat in Med 20 : 215 – 236 [CROSSREF] [CSA]
   PubMed Web of Science ®Google Scholar
• Temple , R. ( 1996 ). Problems in interpreting active control equivalence trials . Accountability in Research 4 : 267 – 275 [INFOTRIEVE] [CSA]
   PubMedGoogle Scholar
• Wang , W. W. B , Li , D. , Liu , F. , Chan , I. S. F. ( 2003 ). Analysis of immune responses in vaccine clinical trials with a pre-post design . Joint Statistical Meetings . San Francisco, August [CSA]
   Google Scholar
• Wiens , B. L. Heyse , J. F. ( 2003 ). Testing for interaction in studies of noninferiority . Journal of Biopharmaceutical Statistics 13 : 103 – 115 [INFOTRIEVE] [CROSSREF] [CSA]
   PubMedGoogle Scholar
• Wiens , B. L. , Heyse , J. F. , Matthews , H. ( 1996 ). Similarity of three treatments, with application to vaccine development . Proceedings of the Biopharmaceutical Section, Joint Statistical Meeting 203 – 206 [CSA]
   Google Scholar