313
Views
1
CrossRef citations to date
0
Altmetric
Articles

Avoiding ambiguity with the Type I error rate in noninferiority trials

Pages 452-465 | Received 12 May 2014, Accepted 24 Mar 2015, Published online: 08 Jan 2016

References

  • Brittain, E., Hu, Z. (2009). Noninferiority trial design and analysis with an ordered three-level categorical endpoint. Journal of Biopharmaceutical Statistics 19:685–699.
  • Carroll, K. J. (2013). Statistical issues and controversies in active-controlled, “non-inferiority” trials. Statistics in Biopharmaceutical Research 5(3):229–238.
  • Chow, S. C., Shao, J., Wang, H. (2003). Sample Size Calculations in Clinical Research. Boca Raton, FL: CRC Press.
  • Chow, S. C., Shao, J. (2006). On non-inferiority margin and statistical tests in active control trials. Statistics in Medicine 25:1101–1113.
  • Chow, S. C., Hsieh, T. C., Chi, E., Yang, J. (2010). A comparison of moment-based and probability-based criteria for assessment of follow-on biologics. Journal of Biopharmaceutical Statistics 20:31–45.
  • Chow, S. C., Wang, J., Endrenyi, L., Lachenbruch P. A. (2013a). Scientific considerations for assessing biosimilar products. Statistics in Medicine 32(3):370–381.
  • Chow, S. C., Yang, L. Y., Starr, A., Chiu, S. T. (2013b). Statistical methods for assessing interchangeability of biosimilars. Statistics in Medicine 32:442–448.
  • D’Agostino, R. B., Massaro, J. M., Sullivan, L. M. (2003). Non-inferiority trials: Design concepts and issues – the encounters of academic consultants in statistics. Statistics in Medicine 22:169–186.
  • Dilba, G., Bretz, F., Hothorn, L. A., Guiard, V. (2006). Power and sample size computations in simultaneous tests for non-inferiority based on relative margins. Statistics in Medicine 25:1131–1147.
  • Fleming, T. R. (2008). Current issues in non-inferiority trials. Statistics in Medicine 27:317–332.
  • Hilton, J. F. (2010). Non-inferiority trial designs for odds ratios and risk differences. Statistics in Medicine 29:982–993.
  • Hsieh, T. C., Chow, S. C., Yang, L. Y., Chi E. (2013). The evaluation of biosimilarity index based on reproducibility probability for assessing follow-on biologics. Statistics in Medicine 32(3):406–414.
  • Hung, H.-M., Wang, S.-J., Tsong, Y., Lawrence, J., O’Neill, R. (2003). Some fundamental issues for noninferiority testing in active controlled trials. Statistics in Medicine 22:213–225.
  • Hung, H.-M., Wang, S.-J., O’Neill, R. (2005). A regulatory perspoective on choice of margin and statistical inference issue in non-inferiority trials. Biometrical Journal 47(1):28–36.
  • Hung, H.-M., Wang, S.-J., O’Neill, R. (2007). Issues with statistical risks for testing methods in non-inferiority trial without a placebo arm. Journal of Biopharmaceutical Statistics 17:201–213.
  • Hung, H.-M., Wang, S.-J., O’Neill, R. (2009). Challenges and regulatory experiences with non-inferiority trial design without placebo arm. Biometrical Journal 51(2):324–334.
  • Hung, H.-M., Wang, S.-J. (2013). Statistical considerations for noninferiority trial designs without placebo. Statistics in Biopharmaceutical Research 5(3):239–247
  • Kang, S. H., Chow, S. C. (2013). Statistical assessment of biosimilarity based on relative distance between follow-on biologics. Statistics in Medicine 32(3):382–392.
  • Kang, S. H., Kim, Y. (2014). Sample size calculations for the development of biosimilar products. Journal of Biopharmaceutical Statistics 24:1215–1224.
  • Kang, S. H., Ryu, Y. (2011). The adjustment of the type I error rate in non-inferiority trials with -margin approach: each of two different new drugs is approved with two independent trials with the same active control. Journal of Biopharmaceutical Statistics 21:498–510.
  • Kang, S. H., Tsong, Y. (2010). Strength of evidence of non-inferiority trials – The adjustment of the type I error rate in non-inferiority trials with the synthesis method. Statistics in Medicine 29:1477–1487.
  • Odem-Davis, K., Fleming, T. R. (2013). Adjusting for unknown bias in noninferiority clinical trials. Statistics in Biopharmaceutical Research 5(3):248–258.
  • Rothman, M. D., Wiens, B. L., Chan, I. S. F. (2012). Design and Analysis of Non-Inferiority Trials. Boca Raton, FL: Chapman & Hall/CRC.
  • Sanchez, M. M., Chen, X. (2006). Choosing the analysis population in non-inferiority studies: Per protocol or intent-to-treat. Statistics in Medicine 25:1169–1181.
  • Sankoh, A. (2008). A note on the conservativeness of the confidence interval approach for the selection of non-inferiority margin in the two-arm active-control trial. Statistics in Medicine 27:3732–3742.
  • Sheng, D., Kim, M. Y. (2006). The effects of non-compliance on intent-to-treat analysis of equivalence trials. Statistics in Medicine 25:1183–1199.
  • Snapinn, S., Jiang, Q. (2008). Controlling the type I error rate in non-inferiority trials. Statistics in Medicine 27:371–381.
  • Soon, G., Zhang, Z., Tsong, Y., Nie, L. (2013). Assessing overall evidence from noninferiority trials with shared historical data. Statistics in Medicine 32(14):2349–2363.
  • Tsong, Y., Wang, S.-J., and Hung, H.-M. (2003). Statistical issues on objective, design and analysis of noninferiority active-controlled clinical trial. Journal of Biopharmaceutical Statistics 13:29–41.
  • US FDA. (2010). Draft Guidance for Industry: Non-Inferiority Clinical Trials. Rockville, MD: FDA.
  • US FDA. (2012). Guidance for Industry: Scientific Considerations in Demonstrating Biosimilarity to a Reference Product. Rockville, MD: FDA.
  • Wang, S.-J., Hung, H.-M., and Tsong, Y. (2002). Utility and pitfalls of some statistical methods in active controlled clinical trials. Controlled Clinical Trials 23:15–28.
  • Wang, S.-J., Hung, H.-M. (2003). Assessing treatment efficacy in non-inferiority trials. Controlled Clinical Trials 24:147–155.
  • Wang, S., Kang, S. H. (2013). Strength of evidence of non-inferiority trials with the two confidence interval method with random margin. Journal of Biopharmaceutical Statistics 23:307–321.
  • World Health Organization. (2009). Guidelines on Evaluation of Similar Biotherapeutic Products. Geneva, Switzerland: WHO.
  • Zhang, N., Yang, J., Chow, S. C., Endrenyi, L., Chi, E. (2013). Impact of variability on the choice of biosimilarity limits in assessing follow-on biologics. Statistics in Medicine 32(3):424–433.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.