References
- Aberegg, S. K., Hersh, A. M., and Samore, M. H. (2017), “Empirical Consequences of Current Recommendations for the Design and Interpretation of Noninferiority Trials,” Journal of General Internal Medicine, 33, 88–96. DOI: https://doi.org/10.1007/s11606-017-4161-4.
- Agresti, A. and Caffo, B. (2000), “Simple and Effective Confidence Intervals for Proportions and Differences of Proportions Result From Adding Two Successes and Two Failures,” The American Statistician, 54, 280–288. DOI: https://doi.org/10.1080/00031305.2000.10474560.
- Anderson, D. R., Dunbar, M., Murnaghan, J., Kahn, S. R., Gross, P., Forsythe, M., Pelet, S., Fisher, W., Belzile, E., Dolan, S., and Crowther, M. (2018), “Aspirin or Rivaroxaban for VTE Prophylaxis After Hip or Knee Arthroplasty,” New England Journal of Medicine, 378, 699–707. DOI: https://doi.org/10.1056/NEJMoa1712746.
- Blackwelder, W. C. (1982), “‘Proving the Null Hypothesis’ in Clinical Trials,” Controlled Clinical Trials, 3, 345–353. DOI: https://doi.org/10.1016/0197-2456(82)90024-1.
- Brown, L., and Li, X. (2005), “Confidence Intervals for Two Sample Binomial Distribution,” Journal of Statistical Planning and Inference, 130, 359–375. DOI: https://doi.org/10.1016/j.jspi.2003.09.039.
- Campbell, B. C., Mitchell, P. J., Churilov, L., Yassi, N., Kleinig, T. J., Dowling, R. J., Yan, B., Bush, S. J., Dewey, H. M., Thijs, V., and Scroop, R. (2018), “Tenecteplase Versus Alteplase Before Thrombectomy for Ischemic Stroke,” New England Journal of Medicine, 378, 1573–1582. DOI: https://doi.org/10.1056/NEJMoa1716405.
- Chan, I. S., and Zhang, Z. (1999), “Test-Based Exact Confidence Intervals for the Difference of Two Binomial Proportions,” Biometrics, 55, 1202–1209. DOI: https://doi.org/10.1111/j.0006-341x.1999.01202.x.
- Clopper, C. J., and Pearson, E. S. (1934), “The Use of Confidence or Fiducial Limits Illustrated in the Case of the Binomial,” Biometrika, 26, 404–413. DOI: https://doi.org/10.1093/biomet/26.4.404.
- Farrington, C. P., and 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. DOI: https://doi.org/10.1002/sim.4780091208.
- FDA (2016), Non-Inferiority Clinical Trials to Establish Effectiveness: Guidance for Industry.
- Gart, J. J. (1971), “The Comparison of Proportions: A Review of Significance Tests, Confidence Intervals and Adjustments for Stratification,” Revue de l’Institut International de Statistique, 39, 148–169. DOI: https://doi.org/10.2307/1402171.
- Hauck, W. W., and Anderson, S. (1986), “A Comparison of Large-Sample Confidence Interval Methods for the Difference of Two Binomial Probabilities,” The American Statistician, 40, 318–322. DOI: https://doi.org/10.2307/2684618.
- ICH (1998), “International Conference on Harmonisation. Statistical Principles for Clinical Trials E9,” available at https://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdfs.
- ICH (2000), “International Conference on Harmonisation. Choice of Control Group and Related Issues in Clinical Trials E10,” available at https://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E10/Step4/E10_Guideline.pdf.
- Julious, S. A., and Owen, R. J. (2011), “A Comparison of Methods for Sample Size Estimation for Non-Inferiority Studies With Binary Outcomes,” Statistical Methods in Medical Research, 20, 595–612. DOI: https://doi.org/10.1177/0962280210378945.
- Kannt, A., and Wieland, T. (2016), “Managing Risks in Drug Discovery: Reproducibility of Published Findings,” Naunyn-Schmiedeberg’s Archives of Pharmacology, 389, 353–360. DOI: https://doi.org/10.1007/s00210-016-1216-8.
- Kasivisvanathan, V., Rannikko, A. S., Borghi, M., Panebianco, V., Mynderse, L. A., Vaarala, M. H., Briganti, A., Budäus, L., Hellawell, G., Hindley, R. G., and Roobol, M. J. (2018), “MRI-Targeted or Standard Biopsy for Prostate-Cancer Diagnosis,” New England Journal of Medicine, 378, 1767–1777. DOI: https://doi.org/10.1056/NEJMoa1801993.
- Leek, J. T., and Peng, R. D. (2015), “Opinion: Reproducible Research Can Still Be Wrong: Adopting a Prevention Approach,” Proceedings of the National Academy of Sciences of the United States of America, 112, 1645–1646. DOI: https://doi.org/10.1073/pnas.1421412111.
- Little, R. J., D’Agostino, R., Cohen, M. L., Dickersin, K., Emerson, S. S., Farrar, J. T., Frangakis, C., Hogan, J. W., Molenberghs, G., Murphy, S. A., and Neaton, J. D. (2012), “The Prevention and Treatment of Missing Data in Clinical Trials,” New England Journal of Medicine, 367, 1355–1360. DOI: https://doi.org/10.1056/NEJMsr1203730.
- Little, R. J., and Rubin, D. B. (2014), Statistical Analysis With Missing Data (Vol. 333), New York: Wiley.
- Miettinen, O., and Nurminen, M. (1985), “Comparative Analysis of Two Rates,” Statistics in Medicine, 4, 213–226. DOI: https://doi.org/10.1002/sim.4780040211.
- Munos, B. (2009), “Lessons From 60 Years of Pharmaceutical Innovation,” Nature Reviews Drug Discovery, 8, 959. DOI: https://doi.org/10.1038/nrd2961.
- Murthy, V. L., Desai, N. R., Vora, A., and Bhatt, D. L. (2012), “Increasing Proportion of Clinical Trials Using Noninferiority End Points,” Clinical Cardiology, 35, 522–523. DOI: https://doi.org/10.1002/clc.22040.
- Newcombe, R. G. (1998), “Interval Estimation for the Difference Between Independent Proportions: Comparison of Eleven Methods,” Statistics in Medicine, 17, 873–890. DOI: https://doi.org/10.1002/(SICI)1097-0258(19980430)17:8<873::AID-SIM779>3.0.CO;2-I.
- NRC (2011), The Prevention and Treatment of Missing Data in Clinical Trials, Washington, DC: National Academies Press.
- Ogawa, C., Sato, Y., Suzuki, C., Mano, A., Tashiro, A., Niwa, T., Hamazaki, S., Tanahashi, Y., Suzumura, M., Hayano, S., and Hayakawa, M. (2018), “Treatment With Silver Nitrate Versus Topical Steroid Treatment for Umbilical Granuloma: A Non-Inferiority Randomized Control Trial,” PLoS One, 13, e0192688. DOI: https://doi.org/10.1371/journal.pone.0192688.
- Piaggio, G., Elbourne, D. R., Altman, D. G., Pocock, S. J., Evans, S. J., and Consort Group (2006), “Reporting of Noninferiority and Equivalence Randomized Trials: An Extension of the Consort Statement,” JAMA, 295, 1152–1160. DOI: https://doi.org/10.1001/jama.295.10.1152.
- Piaggio, G., Elbourne, D. R., Pocock, S. J., Evans, S. J., Altman, D. G., and Consort Group (2012), “Reporting of Noninferiority and Equivalence Randomized Trials: Extension of the Consort 2010 Statement,” JAMA, 308, 2594–2604. DOI: https://doi.org/10.1001/jama.2012.87802.
- Rabe, B. A., Day, S., Fiero, M. H., and Bell, M. L. (2018), “Missing Data Handling in Non-Inferiority and Equivalence Trials: A Systematic Review,” Pharmaceutical Statistics, 17, 477–488.
- Reeve, R. (2018), “Confidence Interval of Difference of Proportions in Logistic Regression in Presence of Covariates,” Statistical Methods in Medical Research, 27, 451–465. DOI: https://doi.org/10.1177/0962280216631583.
- Rehal, S., Morris, T. P., Fielding, K., Carpenter, J. R., and Phillips, P. P. (2016), “Non-Inferiority Trials: Are They Inferior? A Systematic Review of Reporting in Major Medical Journals,” BMJ Open, 6, e012594. DOI: https://doi.org/10.1136/bmjopen-2016-012594.
- Rubin, D. B. (1976), “Inference and Missing Data,” Biometrika, 63, 581–592. DOI: https://doi.org/10.1093/biomet/63.3.581.
- Sénat, M.-V., Affres, H., Letourneau, A., Coustols-Valat, M., Cazaubiel, M., Legardeur, H., Jacquier, J. F., Bourcigaux, N., Simon, E., Rod, A., and Héron, I. (2018), “Effect of Glyburide Vs Subcutaneous Insulin on Perinatal Complications Among Women With Gestational Diabetes: A Randomized Clinical Trial,” JAMA, 319, 1773–1780. DOI: https://doi.org/10.1001/jama.2018.4072.
- Sidi, Y., and Harel, O. (2018), “The Treatment of Incomplete Data: Reporting, Analysis, Reproducibility, and Replicability,” Social Science & Medicine, 209, 169–173. DOI: https://doi.org/10.1016/j.socscimed.2018.05.037.
- Suda, K. J., Hurley, A. M., McKibbin, T., and Moroney, S. E. M. (2011), “Publication of Noninferiority Clinical Trials: Changes Over a 20-Year Interval,” Pharmacotherapy, 31, 833–839. DOI: https://doi.org/10.1592/phco.31.9.833.
- Wald, A. (1943), “Tests of Statistical Hypotheses Concerning Several Parameters When the Number of Observations Is Large,” Transactions of the American Mathematical Society, 54, 426–482. DOI: https://doi.org/10.1090/S0002-9947-1943-0012401-3.
- Zha, R., and Harel, O. (2019), “Power Calculation in Multiply Imputed Data,” Statistical Papers, 1–27.