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Research Article

Risk difference, relative risk, and odds ratio for non-inferiority clinical trials with risk rate endpoint

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Pages 15-30 | Received 26 Sep 2021, Accepted 07 Apr 2022, Published online: 06 Jul 2022

References

  • Aberegg, S. K., A. M. Hersh, and M. H. Samore. 2018. Empirical consequences of current recommendations for the design and interpretation of noninferiority trials. Journal of General Internal Medicine 33 (1):88–96. doi:10.1007/s11606-017-4161-4.
  • Agresti, A., and B. A. Coull. 1998. Approximate is better than “exact” for interval estimation of binomial proportions. The American Statistician 52 (2):119–126.
  • Altman, D., D. Machin, T. Bryant, and M. Gardner. 2000. Statistics with confidence: Confidence intervals and statistical guidelines. Hoboken, New Jersey: John Wiley & Sons.
  • Aversano, T., C. C. Lemmon, and L. Liu. 2012. Outcomes of PCI at hospitals with or without on-site cardiac surgery. New England Journal of Medicine 366 (19):1792–1802. doi:10.1056/NEJMoa1114540.
  • Bikdeli, B., J. W. Welsh, Y. Akram, N. Punnanithinont, I. Lee, N. R. Desai, S. Kaul, G. W. Stone, J. S. Ross, and H. M. Krumholz. 2019. Noninferiority designed cardiovascular trials in highest-impact journals: Main findings, methodological quality, and time trends. Circulation 140 (5):379–389. doi:10.1161/CIRCULATIONAHA.119.040214.
  • Bilimoria, K. Y., J. W. Chung, L. V. Hedges, A. R. Dahlke, R. Love, M. E. Cohen, D. B. Hoyt, A. D. Yang, J. L. Tarpley, J. D. Mellinger, et al. 2016. National cluster-randomized trial of duty-hour flexibility in surgical training. New England Journal of Medicine. 374(8):713–727. doi:10.1056/NEJMoa1515724.
  • Brown, L. D., T. Tony Cai, and A. DasGupta. 2001. Interval estimation for a binomial proportion. Statistical Science 16 (2):101–117. doi:10.1214/ss/1009213286.
  • Chen, J. J., Y. Tsong, and S.-H. Kang. 2000. Tests for equivalence or noninferiority between two proportions. Drug Information Journal 34 (2):569–578. doi:10.1177/009286150003400225.
  • Chow, S.-C., H. Wang, and J. Shao. 2017. Sample size calculations in clinical research. Boca Raton, Florida: Chapman and Hall/CRC.
  • Chowdhury, S., R. C. Tiwari, and S. Ghosh. 2019. Non-inferiority testing for risk ratio, odds ratio and number needed to treat in three-arm trial. Computational Statistics & Data Analysis 132:70–83. doi:10.1016/j.csda.2018.08.018.
  • D’Agostino Sr, R. B., J. M. Massaro, and L. M. Sullivan. 2003. Non-inferiority trials: Design concepts and issues–the encounters of academic consultants in statistics. Statistics in Medicine 22 (2):169–186. doi:10.1002/sim.1425.
  • Donner, A., and G. Y. Zou. 2012. Closed-form confidence intervals for functions of the normal mean and standard deviation. Statistical Methods in Medical Research 21 (4):347–359. doi:10.1177/0962280210383082.
  • EMA. 2005. “Guideline on the Choice of the Noninferiority Margin.” Accessed 2 February 2022. https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-choice-non-inferiority-margin_en.pdf.
  • Fagerland, M. W., and R. G. Newcombe. 2013. Confidence intervals for odds ratio and relative risk based on the inverse hyperbolic sine transformation. Statistics in Medicine 32 (16):2823–2836. doi:10.1002/sim.5714.
  • Fagerland, M. W., S. Lydersen, and P. Laake. 2015. Recommended confidence intervals for two independent binomial proportions. Statistical Methods in Medical Research 24 (2):224–254. doi:10.1177/0962280211415469.
  • FDA. 2007. “Statistical guidance on reporting results from studies evaluating diagnostic tests: Guidance for industry and FDA staff.” Accessed 20 January 2022. https://www.fda.gov/media/71147/download.
  • FDA. 2016. “Non-inferiority clinical trials to establish effectiveness: guidance for industry.” Accessed 16 December 2021. https://www.fda.gov/media/78504/download.
  • Head, S. J., S. Kaul, A. J. J. C. Bogers, and A. P. Kappetein. 2012. Non-inferiority study design: Lessons to be learned from cardiovascular trials. European Heart Journal 33 (11):1318–1324. doi:10.1093/eurheartj/ehs099.
  • ICH. 2001. “Guidance for industry. E10 Choice of control groups and related issues in a clinical trial.” Accessed 2 February 2022. https://database.ich.org/sites/default/files/E10_Guideline.pdf.
  • James Hung, H. M., S.-J. Wang, Y. Tsong, J. Lawrence, and R. T. O’Neil. 2003. Some fundamental issues with non-inferiority testing in active controlled trials. Statistics in Medicine 22 (2):213–225. doi:10.1002/sim.1315.
  • Kaul, S., and G. A. Diamond. 2006. Good enough: A primer on the analysis and interpretation of noninferiority trials. Annals of Internal Medicine 145 (1):62–69. doi:10.7326/0003-4819-145-1-200607040-00011.
  • Lavizzari, A., M. Colnaghi, F. Ciuffini, C. Veneroni, S. Musumeci, I. Cortinovis, and F. Mosca. 2016. Heated, humidified high-flow nasal cannula vs nasal continuous positive airway pressure for respiratory distress syndrome of prematurity: A randomized clinical noninferiority trial. JAMA Pediatrics 170 (12):1228–1234. doi:10.1001/jamapediatrics.2016.3743.
  • Leung, J. T., S. L. Barnes, S. T. Lo, and D. Y. Leung. 2020. Non-inferiority trials in cardiology: What clinicians need to know. Heart 106 (2):99–104. doi:10.1136/heartjnl-2019-315772.
  • Macaya, F., N. Ryan, P. Salinas, and S. J. Pocock. 2017. Challenges in the design and interpretation of noninferiority trials: Insights from recent stent trials. Journal of the American College of Cardiology 70 (7):894–903. doi:10.1016/j.jacc.2017.06.039.
  • Manley, B. J., L. S. Owen, L. W. Doyle, C. C. Andersen, D. W. Cartwright, M. A. Pritchard, S. M. Donath, and P. G. Davis. 2013. High-flow nasal cannulae in very preterm infants after extubation. New England Journal of Medicine 369 (15):1425–1433. doi:10.1056/NEJMoa1300071.
  • Mol, G. C., M. A. Van de Ree, F. A. Klok, M. J. A. M. Tegelberg, F. B. M. Sanders, S. Koppen, O. De Weerdt, T. Koster, M. M. C. Hovens, H. A. H. Kaasjager, et al. 2016. One versus two years of elastic compression stockings for prevention of post-thrombotic syndrome (OCTAVIA study): Randomised controlled trial. British Medical Journal 353.
  • Newcombe, R. G. 1998. Interval estimation for the difference between independent proportions: Comparison of eleven methods. Statistics in Medicine 17 (8):873–890. doi:10.1002/(SICI)1097-0258(19980430)17:8<873::AID-SIM779>3.0.CO;2-I.
  • Niraula, S., and I. F. Tannock. 2016. Harms of intermittent vs continuous androgen-deprivation therapy for prostate cancer. JAMA Oncology 2 (4):461–462. doi:10.1001/jamaoncol.2015.4682.
  • Rothman, K. J., and K. B. Michels. 1994. The continuing unethical use of placebo controls. New England Journal of Medicine 331 (6):394–398. doi:10.1056/NEJM199408113310611.
  • Rothmann, M. D., B. L. Wiens, and I. S. F. Chan. 2011. Design and analysis of non-inferiority trials. Boca Raton, Florida: CRC Press.
  • Serruys, P. W., S. Silber, S. Garg, R. Jan Van Geuns, G. Richardt, P. E. Buszman, H. Kelbæk, A. J. van Boven, S. H. Hofma, A. Linke, et al. 2010. Comparison of zotarolimus-eluting and everolimus-eluting coronary stents. New England Journal of Medicine. 363(2):136–146. doi:10.1056/NEJMoa1004130.
  • Wallis, S. 2013. Binomial confidence intervals and contingency tests: Mathematical fundamentals and the evaluation of alternative methods. Journal of Quantitative Linguistics 20 (3):178–208. doi:10.1080/09296174.2013.799918.
  • Weir, I. R., and L. Trinquart. 2018. Design of non-inferiority randomized trials using the difference in restricted mean survival times. Clinical Trials 15 (5):499–508. doi:10.1177/1740774518792259.
  • Widmer, M., G. Piaggio, T. M. H. Nguyen, A. Osoti, O. O. Owa, S. Misra, A. Coomarasamy, H. Abdel-Aleem, A. A. Mallapur, Z. Qureshi, et al. 2018. Heat-stable carbetocin versus oxytocin to prevent hemorrhage after vaginal birth. New England Journal of Medicine. 379(8):743–752. doi:10.1056/NEJMoa1805489.
  • Windecker, S., P. W. Serruys, S. Wandel, P. Buszman, S. Trznadel, A. Linke, K. Lenk, T. Ischinger, V. Klauss, F. Eberli, et al. 2008. Biolimus-eluting stent with biodegradable polymer versus sirolimus-eluting stent with durable polymer for coronary revascularisation (LEADERS): A randomised non-inferiority trial. The Lancet. 372(9644):1163–1173. doi:10.1016/S0140-6736(08)61244-1.
  • Zou, G. Y., and A. Donner. 2008. Construction of confidence limits about effect measures: A general approach. Statistics in Medicine 27 (10):1693–1702. doi:10.1002/sim.3095.

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