Our Most Important Discovery: The Question

References

• Bonetti, M., and Gelber, R. D. (2004), “Patterns of Treatment Effects in Subsets of Patients in Clinical Trials,” Biostatistics, 5, 465–481. DOI: 10.1093/biostatistics/kxh002.
   PubMed Web of Science ®Google Scholar
• Clinical Trials Transformation Initiative (2016), “CTTI Recommendations: Data Monitoring Committees,” available at www.ctti-clinicaltrials.org/files/recommendations/dmc-recommendations.pdf.
   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. DOI: 10.1001/jama.2011.175.
   PubMed Web of Science ®Google Scholar
• Doernberg, S. B., Tran, T. T., Tong, S. Y. C., Paul, M., Yahav, D., Davis, J. S., Leibovici, L., Boucher, H. W., Corey, G. R., Cosgrove, S. E., Chambers, H. F., Fowler, V. G., Evans, S. R., and Holland, T. L. (2019), “Good Studies Evaluate the Disease While Great Studies Evaluate the Patient: Development and Application of a Desirability of Outcome Ranking Endpoint for Staphylococcus aureus Bloodstream Infection,” Clinical Infectious Diseases, 68, 1691–698. DOI: 10.1093/cid/ciy766.
   PubMed Web of Science ®Google Scholar
• Evans S. R. (2020a), “Our Most Important Discovery: The Question. The Biopharmaceutical Section Regulatory Industry Statistics Workshop,” available at https://www.youtube.com/watch?v=u8JDMhD-x20&feature=youtu.be
   Google Scholar
• Evans S. R. (2020b), “Radical Thinking: Scientific Rigor and Pragmatism,” Statistics in Biopharmaceutical Research, available at https://collaboration.fda.gov/pqxxxxogesrm/
   Google Scholar
• Evans, S. R., and Follmann, D. (20), “Using Outcomes to Analyze Patients Rather than Patients to Analyze Outcomes: A Step toward Pragmatism in Benefit:risk Evaluation,” Statistics in Biopharmaceutical Research, 8, 386–393. DOI: 10.1080/19466315.2016.1207561.
   PubMed Web of Science ®Google Scholar
• Evans, S. R., Bigelow, R., Chuang-Stein, C., Ellenberg, S., Gallo, P., He, W., Jiang, Q., and Rockhold, F. (2020), “Presenting Risks and Benefits: Helping the Data Monitoring Committee Do Its Job,” Annals of Internal Medicine, 172, 119–125. DOI: 10.7326/M19-1491.
   PubMed Web of Science ®Google Scholar
• Evans, S. R., Knutsson, M., Amarenco, P., Albers, G. W., Bath, P. M., Denison, H., Ladenvall, P., Jonasson, J., Easton, J. D., Minematsu, K., Molina, C. A., Wang, Y., Wong, K. S. L., and Johnston, S. C. (2020), “Methodologies for Pragmatic and Efficient Assessment of Benefits and Harms: Application.
   Google Scholar
• Evans, S. R., Rubin, D. B., Powers, J. H., and Follmann, D. (2018), “Analysis Populations in Anti-Infective Clinical Trials: Whom to Analyze?” Statistical Communications in Infectious Diseases, 10, 2017002. DOI: 10.1515/scid-2017-0002.
   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),” Clinical Infectious Diseases, 61, 800–806. DOI: 10.1093/cid/civ495.
   PubMed Web of Science ®Google Scholar
• Evans, S., and Ting, N. (2016), Fundamental Concepts for New Clinical Trialists, Boca Raton, FL: CRC Press.
   Google Scholar
• Johnston, S. C., Amarenco, P., Albers, G. W., Denison, H., Easton, J. D., Evans, S. R., Held, P., Jonasson, J., Minematsu, K., Molina, C. A., Wang, Y., and Wong, L.; SOCRATES Steering Committee and Investigators. (2016), “Ticagrelor Versus Aspirin in Acute Stroke or Transient Ischemic Attack,” New England Journal of Medicine, 375, 35–43. DOI: 10.1056/NEJMoa1603060.
   PubMed Web of Science ®Google Scholar
• Johnston, S. C., Amarenco, P., Denison, H., Evans, S. R., Himmelmann, A., James, S., Knutsson, M., Ladenvall, P., Molina, C. A., and Wang, Y. (2020), “Ticagrelor and Aspirin Versus Aspirin in Acute Ischemic Stroke or TIA,” New England Journal of Medicine, 383, 207–217. DOI: 10.1056/NEJMoa1916870.
   PubMed Web of Science ®Google Scholar
• Kumar, S., Harrison, S., Cavo, M., de la Rubia, J., Popat, R., Gasparetto, C., Hungria, V., Salwender, H., Suzuki, K., Kim, I., Punnoose, E. A., Hong, W. J., Freise, K. J., Yang, X., Sood, A., Jalaluddin, M., Ross, J. A., Ward, J. E., Maciag, P. C., Moreau, P. (2020), “Venetoclax or Placebo in Combination with Bortezomib and Dexamethasone in Patients with Relapsed or Refractory Multiple Myeloma (BELLINI): A Randomised, Double-Blind, Multicentre, Phase 3 Trial,” Lancet Oncology, 21, 1630–1642. DOI: 10.1016/S1470-2045(20)30525-8.
   PubMed Web of Science ®Google Scholar
• Levitan B, DiSantostefano R, and Evans SR. (2018), “Benefit-Risk Assessments of Medical Treatments,” in Pharmacoepidemiology (6th ed.), eds. B. L. Strom, S. E. Kimmel, and S. Hennessy, Hoboken, NJ: Wiley.
   Google Scholar
• Lodise Jr, T. P., Rosenkranz, S. L., Finnemeyer, M., Evans, S. R., Sims, M., Zervos, M. J., Creech, C. B., Patel, P. C., Keefer, M., Riska, P., Silveira, F. P., Scheetz, M., Wunderink, R. G., Rodriguez, M., Schrank, J., Bleasdale, S. C., Schultz, S., Barron, M., Stapleton, A., Wray, D., Chambers, H. F., Fowler, Jr V. G., and Holland, T. L. (2020), “The Emperor’s New Clothes: Prospective Observational Evaluation of the Association between Initial Vancomycin Exposure and Failure Rates among Adult Hospitalized Patients with MRSA Bloodstream Infections (PROVIDE),” Clinical Infectious Diseases, 70, 1536–1545. DOI: 10.1093/cid/ciz460.
   PubMed Web of Science ®Google Scholar
• Van Duin, D., Lok, J. J., Earley, M., Cober, E., Richter, S. S., Perez, F., Salata, R. A., Kalayjian, R. C., Watkins, R. R., Doi, Y., Kaye, K. S., Fowler, Jr V. G., Paterson, D. L., Bonomo, R. A., and Evans, S. R. (2018), “Colistin vs. Ceftazidime-avibactam in the Treatment of Infections due to Carbapenem-Resistant Enterobacteriaceae,” Clinical Infectious Diseases, 66, 163–171. DOI: 10.1093/cid/cix783.
   PubMed Web of Science ®Google Scholar
• Yahav, D., Franceschini, E., Koppel, F., Turjeman, A., Babich, T., Bitterman, R., Neuberger, A., Ghanem-Zoubi, N., Santoro, A., Eliakim-Raz, N., Pertzov, B., Steinmetz, T., Stern, A., Dickstein, Y., Maroun, E., Zayyad, H., Bishara, J., Alon, D., Edel, Y., Goldberg, E., Venturelli, C., Mussini, C., Leibovici, L., Paul, M.; Bacteremia Duration Study Group. (2019), “Seven Versus Fourteen Days of Antibiotic Therapy for Uncomplicated Gram-Negative Bacteremia: A Noninferiority Randomized Controlled Trial,” Clinical Infectious Diseases, 69, 1091– 1098. DOI: 10.1093/cid/ciy1054.
   PubMed Web of Science ®Google Scholar