Using Outcomes to Analyze Patients Rather than Patients to Analyze Outcomes: A Step Toward Pragmatism in Benefit:Risk Evaluation

References

• Boers, M., Brooks, P., Fries, J. F., Simon, L. S., Strand, V., and Tugwell, P. (2010), “A First Step to Assess Harm and Benefit in Clinical Trials in One Scale,” Journal of Clinical Epidemiology, 63, 627–632.
   Google Scholar
• Califf, R. (2016), Letter to FDA Colleagues, February 26, 2016.
   Google Scholar
• Chuang-Stein, C. (1994), “A New Proposal for Benefit-Less Risk Analysis in Clinical Trials,” Control Clinical Trials, 15, 30–43.
   Google Scholar
• Chuang-Stein, C., Mohberg, N. R., and Sinkula, M. S. (1991), “Three Measures for Simultaneously Evaluating Benefits and Risks Using Categorical Data from Clinical Trials,” Statistics in Medicine, 10, 1349–1359.
   Google Scholar
• Claggett, B., Tian, L., Castagno, D., and Wei, L. J. (2015), “Treatment Selections Using Risk–Benefit Profiles Based on Data From Comparative Randomized Clinical Trials with Multiple Endpoints,” Biostatistics, 16, 60–72.
   Google Scholar
• D'Agostino, R. B., Massaro, J. M., and Sullivan, L. M. (2003), “Non-Inferiority Trials: Design Concepts and Issues—The Encounters of Academic Consultants in Statistics,” Statistics in Medicine, 22, 169–186.
   Google Scholar
• DeMets, D. L., and Califf, R. M. (2011), “A Historical Perspective on Clinical Trials Innovation and Leadership: Where Have the Academics Gone?,” JAMA, 305, 713–714.
   Google Scholar
• Evans, S. R. (2009), “Noninferiority Clinical Trials,” CHANCE, 22, 53–58.
   Google Scholar
• ——— (2010), “Estudos Clinicos de Nao-Inferioridade,” Revista Brasileira de Medicina, 67, 7.
   Google Scholar
• Evans, S. R., and Follmann, D. (2015), “Fundamentals and Innovation in Antibiotic Trials,” Statistics in Biopharmaceutical Research, 7, 331–336,.
   Google Scholar
• Evans, S. R., Rubin, D., Follmann, D., Pennello, G., Huskins, W. C., Powers, J. H., Schoenfeld, D., Chuang-Stein, C., Cosgrove, S. E., Fowler, Jr. V. G., Lautenbach, E., and Chambers, H. F. (2015), “Desirability of Outcome Ranking (DOOR) and Response Adjusted for Duration of Antibiotic Risk (RADAR),” CID, 61, 800–806.
   Google Scholar
• Flagas, M. E., and Kasiakou, S. K. (2005), “Colistin: The Revival of Polymyxins for the Management of Multidrug-Resistant Gram-Negative Bacterial Infections,” Clinical Infectious Diseases, 40, 1333–1341.
   Google Scholar
• Fleming, T. R. (2008), “Current Issues in Non-Inferiority Trials,” Statistics in Medicine, 27, 317–332.
   Google Scholar
• Follman, D. (2002), “Regression Analysis Based on Pairwise Ordering of Patients' Clinical Histories,” Statistics in Medicine, 21, 3353–3367.
   Google Scholar
• Follmann, D., Wittes, J., and Cutler, J. (1992), “The Use of Subjective Ranking in Clinical Trials with Application to Cardiovascular Disease,” Statistics in Medicine, 11, 427–437.
   Google Scholar
• Gelber, R. D., Gelman, R. S., and Goldhirsch, A. (1989), “A Quality-of-Life Oriented Endpoint for Comparing Treatments,” Biometrics, 45, 781–795.
   Google Scholar
• Hamasaki, T., and Evans, S. R. (2013), “Noninferiority Clinical Trials: Issues in Design, Monitoring, Analyses, and Reporting,” Igaku no Ayumi, 244, 1212–1216.
   Google Scholar
• Hartzell, J. D. et al. (2009), “Nephrotoxicity Associated with Intravenous Colistin (colistimethate sodium) Treatment at a Tertiary Care Medical Center,” Clinical Infectious Diseases, 48, 1724–1728.
   Google Scholar
• Koch-Weser, J. K. et al. (1970), “Adverse Effects of Sodium Colistimethate: Manifestations and Specific Reaction Rates During 317 Courses of Therapy,” Annals of Internal Medicine, 72, 857–868.
   Google Scholar
• Mancini, G. B. J., and Schulzer, M. (1999), “Reporting Risks and Benefits of Therapy by use of the Concepts of Unqualified Success and Unmitigated Failure: Application to Highly Cited Trials in Cardiovascular Medicine,” Circulation, 99, 377–383.
   Google Scholar
• McCullagh, P., and Nelder, J. A. (1989), Generalized Linear Models, New York: Chapman and Hall.
   Google Scholar
• Neaton, J., Gerry, G., Zuckerman, B. D., and Konstam, M. A. (2005), “Key Issues in End Point Selection for Heart Failure Trials: Composite Endpoints,” Journal of Cardiac Failure, 11, 567–575.
   Google Scholar
• Norton, J. D. (2011), “Longitudinal Model and Graphic for Benefit-Risk Analysis, with Case Study,” Drug Information Journal, 45, 741–747.
   Google Scholar
• Paul, M., Bishara, J., Levcovich, A., Chowers, M., Goldberg, E., Singer, P., Lev, S., Leon, P., Raskin, M., Yahav, D., and Leibovici, L. (2010), “Effectiveness and Safety of Colistin: Prospective Comparative Cohort Study,” Journal of Antimicrobial Chemotherapy, 65, 1019–1027.
   Google Scholar
• Pocock, S. J., Ariti, C. A., Collier, T. J., and Wang, D. (2012), “The Win Ratio: A New Approach to the Analysis of Composite Endpoints in Clinical Trials Based on Clinical Priorities,” European Heart Journal, 33, 176–182.
   Google Scholar
• Snappin, S. M. (2000), “Noninferiority Trials,” Current Controlled Trials in Cardiovascular Medicine, 1, 19–21.
   Google Scholar
• Thall, P. F., Cook, J. D., and Estey, E. H. (2006), “Adaptive Dose Selection Using Efficacy-Toxicity Trade-offs: Illustrations and Practical Considerations,” Journal of Biopharmaceutical Statistics, 16, 623–638.
   Google Scholar
• Tudur Smith, C., Hickey, H., Clarke, M., Blazeby, J., and Williamson, P. (2014), “The Trials Methodological Research Agenda: Results from a Priority Setting Exercise,” Trials, 15, 32.
   Google Scholar
• Wittkowski, K., Lee, E., Nussbaum, R., Chamian, F., and Krueger, J., (2004), “Combining Several Ordinal Measures in Clinical Studies,” Statistics in Medicine, 23, 1579–1592.
   Google Scholar
• Wolinsky, E., and Hines, J. D. (1962), “Neurotoxic and Nephrotoxic Effects of Colistin in Patients with Renal Disease,” New England Journal of Medicine, 266, 759–762.
   Google Scholar