208
Views
0
CrossRef citations to date
0
Altmetric
Special Section: Selected Articles from the ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop 2020 - Lead and Impact: Turning Innovation into Practice

Historical Control Data Borrowing for Noninferiority Assessment

, , , &
Pages 463-472 | Received 17 Nov 2020, Accepted 11 Oct 2021, Published online: 01 Dec 2021

References

  • Austin, P. C. (2009), “Type I Error Rates, Coverage of Confidence Intervals, and Variance Estimation in Propensity-Score Matched Analyses,” The International Journal of Biostatistics, 5, Article 13. DOI: 10.2202/1557-4679.1146.
  • Chen, M. H., Ibrahim, J. G., Lam, P., Yu, A., and Zhang, Y. (2011), “Bayesian Design of Noninferiority Trials for Medical Devices Using Historical Data,” Biometrics, 67, 1163–1170. DOI: 10.1111/j.1541-0420.2011.01561.x.
  • Dejardin, D., Delmar, P., Warne, C., Patel, K., Rosmalen, J. V, and Lesaffre, E. (2018), “Use of a Historical Control Group in a Noninferiority Trial Assessing a New Antibacterial Treatment: A Case Study and Discussion of Practical Implementation Aspects,” Pharmaceutical Statistics, 17, 169–181. DOI: 10.1002/pst.1843.
  • Duan, Y., Ye, K., and Smith, E. P. (2006), “Evaluating Water Quality Using Power Priors to Incorporate Historical Information,” Environmetrics,17, 95–106. DOI: 10.1002/env.752.
  • Hauschke, D., and Pigeot, I. (2005), “Establishing Efficacy of a New Experimental Treatment in the Gold Standard Design” (with discussions), Biometerical Journal, 47, 782–798.
  • Hobbs, B. P., Carlin, B. P., Mandrekar, S. J., and Sargent, D. J. (2011), “Hierarchical Commensurate and Power Prior Models for Adaptive Incorporation of Historical Information in Clinical Trials,” Biometrics, 67, 1047–1056. DOI: 10.1111/j.1541-0420.2011.01564.x.
  • Hung, H. M., and Wang, S. J. (2013), “Statistical Considerations for Noninferiority Trial Designs Without Placebo,” Statistics Biopharmaceutical Research, 5, 239–247. DOI: 10.1080/19466315.2013.782821.
  • Hung, H. M., Wang, S. J., and O’Neill, R. (2007), “Issues With Statistical Risks for Testing Methods in Noninferiority Trial Without a Placebo Arm,” Journal of Biopharmaceutical Statistics, 17, 201–213. DOI: 10.1080/10543400601177343.
  • Hung, H. M., Wang, S. J., and O’Neill, R. (2009), “Challenges and Regulatory Experiences With Non-Inferiority Trial Design Without Placebo Arm,” Biometrical Journal, 51, 324–334. DOI: 10.1002/bimj.200800219.
  • Imai, K., and Ratkovic, M. (2014), “Covariate Balancing Propensity Score,” Journal of Royal Statistical Socety, Series B, 76, 243–263. DOI: 10.1111/rssb.12027.
  • Kang, S. H. (2016), “Avoiding Ambiguity With the Type I Error Rate in Noninferiority Trials,” Journal of Biopharmaceutical Statistics, 26, 452–465. DOI: 10.1080/10543406.2015.1052477.
  • Kang, S. H., and Ryu, Y. (2011), “The Adjustment of the Type I Error Rate in Noninferiority Trials With λ Margin Approach: Each of the Two Different New Drugs is Approved With Two Independent Trials With Same Active Control,” Journal of Biopharmaceutical Statistics, 21, 498–510. DOI: 10.1080/10543406.2010.482683.
  • Lee, W. S. (2006), “Evaluating the Effects of a Mandatory Government Program Using Matched Groups Within a Similar Geographic Location,” available at https://papers.ssrn.com/sol3/papers.cfm?abstract_id=936783.
  • Levenson, M. S., and Yue, L. Q. (2013), “Regulatory Issues of Propensity Score Methodology Application to Drug and Device Safety Studies,” Journal of Biopharmaceutical Statistics, 23, 110–121. DOI: 10.1080/10543406.2013.735778.
  • Pocock, S. (1976), “The Combination of Randomized and Historical Controls in Clinical Trials,” Journal of Chronic Diseases, 29, 175–188. DOI: 10.1016/0021-9681(76)90044-8.
  • Psioda, M. A., and Ibrahim, J. G. (2019), “Bayesian Clinical Trial Design Using Historical Data That Inform the Treatment Effect,” Biostatistics, 20, 400–415. DOI: 10.1093/biostatistics/kxy009.
  • Quan, H., Zhang, B., Lan, Y., Luo, X., and Chen, X. (2019), “Bayesian Hypothesis Testing With Frequentist Characteristics,” Contemporary Clinical Trials.105858. DOI: 10.1016/j.cct.2019.
  • Rosenbaum, P. R., and Rubin, D. B. (1983), “The Central Role of Propensity Score in Observational Studies for Causal Effects,” Biometrika, 70, 41–55. DOI: 10.1093/biomet/70.1.41.
  • US FDA (2020), “Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products: Guidance for Industry (December),” available at https://www.fda.gov/media/130897/download.
  • US FDA (2008), “Guidance for Industry Diabetes Mellitus—Evaluating Cardiovascular Risk in New Antidiabetic Therapies to Treat Type 2 Diabetes (December)”.
  • US FDA (2016), “Non-Inferiority Clinical Trials to Establish Effectiveness: Guidance for Industry (November),” available at https://www.fda.gov/media/78504/download.
  • Wang, F., and Gelfand, A. E. (2002), “A Simulation-Based Approach to Bayesian Sample Size Determination for Performance Under a Given Model and for Separating Models,” Statistical Science, 17, 193–208.
  • Wang, S. Y., and Kang, S. H. (2013), “Strength of Evidence of Noninferiority Trials With the Two Confidence Interval Method With Random Margin,” Journal of Biopharmaceutical Statistics, 23, 307–321. DOI: 10.1080/10543406.2011.616965.
  • Yue, L. Q. (2012), “Regulatory Considerations in the Design of Comparative Observational Studies Using Propensity Scores,” Journal of Biopharmaceutical Statistics, 22, 1272–1279. DOI: 10.1080/10543406.2012.715111.
  • Zhang, Z., Kim, H. J., Lonjon, G., Zhu, Y. (2019), written on behalf of AME Big-Data Clinical Trial Collaborative Group, “Balance Diagnostics After Propensity Score Matching,” Annals of Translational Medicine, 7, 16. DOI: 10.21037/atm.2018.12.10.

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.