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Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
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Page 139
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
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Page 140
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
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Page 141
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 142
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
×
Page 143
Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2019. Examining the Impact of Real-World Evidence on Medical Product Development: Proceedings of a Workshop Series. Washington, DC: The National Academies Press. doi: 10.17226/25352.
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Page 144

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References Alphs, L. D. and C. A. Bossie. 2016. ASPECT-R—A Tool to Rate the Pragmatic and Explanatory Characteristics of a Clinical Trial Design. Innovations in Clinical Neuroscience 13(1-2):15. Alphs, L., C. Benson, K. Cheshire-Kinney, J. P. Lindenmayer, L. Mao, S. C. Rodriguez, and H. L. Starr. 2015. Real-world outcomes of paliperidone palmitate compared to daily oral antipsychotic therapy in schizophrenia: A randomized, open-label, review board-blinded 15- month study. Journal of Clinical Psychiatry 76(5):554-561. Avalos, L. A., H. Chen, and D. K. Li. 2015. Antidepressant medication use, depression, and the risk of preeclampsia. CNS Spectrums 20(1):39-47. Bai, Y., H. Deng, A. Shantsila, and G. Y. Lip. 2017. Rivaroxaban versus Dabigatran or warfarin in real-world studies of stroke prevention in atrial fibrillation: Systematic review and meta- analysis. Stroke 48(4):970-976. Bakerly, N. D., A. Woodcock, J. P. New, J. M. Gibson, W. Wu, D. Leather, and J. Vestbo. 2015. The Salford Lung Study protocol: A pragmatic randomised phase III real-world effectiveness trial in chronic obstructive pulmonary disease. Respiratory Research 16:101. Berger, M. L., H. Sox, R. J. Willke, D. L. Brixner, H. G. Eichler, W. Goettsch, D. Madigan, A. Makady, S. Schneeweiss, R. Tarricone, S. V. Wang, J. Watkins, and C. D. Mullins. 2017. Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the Joint ISPOR_ISPE Special Task Force on Real-World Evidence in Health Care Decision Making. Value in Health 20(8):1003-1008. Chrischilles, E. A., J. J. Gagne, B. Fireman, J. Nelson, S. Toh, A. Shoaibi, M. E. Reichman, S. Wang, M. Nguyen, R. Zhang, R. Izem, M. R. Goulding, M. R. Southworth, D. J. Graham, C. Fuller, H. Katcoff, T. Woodworth, C. Rogers, R. Saliga, N. D. Lin, C. N. McMahill- Walraven, V. P. Nair, K. Haynes, and R. M. Carnahan. 2018. Prospective surveillance pilot of rivaroxaban safety within the US Food and Drug Administration Sentinel System. Pharmacoepidemiology and Drug Safety 27(3):263-271. Cocoros N. M., S. D. Pokorney, K. Haynes, C. Garcia, H. R. Al-Khalidi, S. M. Al-Khatib, P. Archdeacon, J. C. Goldsack, T. Harkins, N. D. Lin, D. Martin, D. McCall, V. Nair, L. Parlett, R. Temple, C. McMahill-Walraven, C. B. Granger, and R. Platt. 2018. FDA-Catalyst-Using FDA’s Sentinel Initiative for large-scale pragmatic randomized trials: Approach and lessons learned during the planning phase of the first trial. Clin Trials 16(1):90-97. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS 139

140 EXAMINING THE IMPACT OF RWE ON MEDICAL PRODUCT DEVELOPMENT Connolly, S. J., M. D. Ezekowitz, S. Yusuf, J. Eikelboom, J. Oldgren, A. Parekh, J. Pogue, P. A. Reilly, E. Themeles, J. Varrone, and S. Wang. 2009. Dabigatran versus warfarin in patients with atrial fibrillation. New England Journal of Medicine 361(12):1139-1151. Cornfield, J., W. Haenszel, E. C. Hammond, A. M. Lilienfeld, M. B. Shimkin, and E. L. Wynder. 2009. Smoking and lung cancer: Recent evidence and a discussion of some questions. Journal of the National Cancer Institute 22(1):173-203. CTTI (Clinical Trials Transformation Initiative). 2018a. QBD (Quality by Design) Toolkit. http://www.ctti-clinicaltrials.org/toolkit/QbD (accessed November 2, 2018.). CTTI. Mobile Clinical Trials (MCTs). 2018b. https://www.ctti- clinicaltrials.org/programs/mobile-clinical-trials (accessed December 17, 2018). Daniel, G. W., M. Menis, G. Sridhar, D. Scott, A. E. Wallace, M. V. Ovanesov, B. Golding, S. A. Anderson, J. Epstein, D. Martin, and R. Ball. 2012. Immune globulins and thrombotic adverse events as recorded in a large administrative database in 2008 through 2010. Transfusion 52(10):2113-2121. DiMasi, J. A., H. G. Grabowski, and R. W. Hansen. 2016. Innovation in the pharmaceutical industry: New estimates of R&D costs. Journal of Health Economics 47:20-33. Duke-Margolis Center for Health Policy. 2017. A framework for regulatory use of real-world evidence. https://healthpolicy.duke.edu/sites/default/files/atoms/files/rwe_white_paper_2017.09.06.pdf (accessed November 6, 2018). Duke-Margolis Center for Health Policy. 2018. Characterizing RWD quality and relevancy for regulatory purposes. https://healthpolicy.duke.edu/sites/default/files/atoms/files/characterizing_rwd.pdf (accessed December 20, 2018). Evidation & Novartis. 2018. Real-world use of wearable devices in a large multiple sclerosis cohort. Poster presentation at the American Academy of Neurology Annual Meeting, Los Angeles, CA. FDA (U.S. Food and Drug Administration). 2018a. FDA’s MyStudies Application (App). https://www.fda.gov/Drugs/ScienceResearch/ucm624785.htm (accessed December 5, 2018). FDA. 2018b. Real-world evidence. https://www.fda.gov/scienceresearch/specialtopics/realworldevidence/default.htm (accessed December 17, 2018). FDA. 2013. Best practices for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. Guidance for industry and FDA staff. https://www.fda.gov/downloads/drugs/guidances/ucm243537.pdf (accessed December 17, 2018). FDA. 2017a. OneSource Checklists: Tools for integrating care and research and catalyzing a sea change in the practice of medicine. https://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/ucm574079.htm (accessed December 21, 2018). FDA. 2017b. Use of real-world evidence to support regulatory decision-making for medical devices. Guidance for industry and Food and Drug Administration staff. https://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocu ments/ucm513027.pdf (accessed November 6, 2018). FDA. 2018. The FDA MyStudies App: Patient centered outcomes research trust fund enabler for distributed clinical trials and real world evidence studies. Collection of patient-provided P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

REFERENCES 141 information through a mobile device application for use in comparative effectiveness and drug safety research. https://www.fda.gov/downloads/Drugs/ScienceResearch/UCM625206.pdf (accessed November 6, 2018.) Fergusson, D. A., P. C. Hébert, C. D. Mazer, S. Fremes, C. MacAdams, J. M. Murkin, K. Teoh, P. C. Duke, R. Arellano, M. A. Blajchman, and J. S. Bussières. 2008. A comparison of aprotinin and lysine analogues in high-risk cardiac surgery. New England Journal of Medicine 358(22):2319-2331. Fralick, M., S. Schneeweiss, and E. Patorno. 2017. Risk of diabetic ketoacidosis after initiation of an SGLT2 inhibitor. New England Journal of Medicine 376(23):2300-2302. Franklin, J. M. and S. Schneeweiss. 2017. When and how can real world data analyses substitute for randomized controlled trials? Clinical Pharmacology & Therapeutics 102(6):924-933. Galson, S. and G. Simon. 2016. Real-World Evidence to Guide the Approval and Use of New Treatments. NAM Perspectives. Discussion Paper, National Academy of Medicine, Washington, DC. doi: 10.31478/201610b. Gambhir, S. S., T. J. Ge, O. Vermesh, and R. Spittler. 2018. Toward achieving precision health. Science Translational Medicine 10(430):eaao3612. Giles, J. T., N. Sattar, S. E. Gabriel, P. M. Ridker, S. Gay, C. Warne, D. Musselman, L. Brockwell, E. Shittu, M. Klearman, and T. Fleming. 2016. Comparative cardiovascular safety of tocilizumab vs etanercept in rheumatoid arthritis: Results of a randomized, parallel- group, multicenter, noninferiority, phase 4 clinical trial. Arthritis & Rheumatology 68(suppl 10). Gill, J., P. Kanavos, B. Avouac, R. Duncombe, J. Hutton, K. Jahnz- Spandonaro, and M. Thomas. 2016. The use of real world evidence in the European context: An analysis of key expert opinion. http://www.lse.ac.uk/business-and- consultancy/consulting/assets/documents/the-use-of-real-world-evidence-in-the-european- context.pdf (accessed December 21, 2018). Giugliano, R. P., C. T. Ruff, E. Braunwald, S. A. Murphy, S. D. Wiviott, J. L. Halperin, A. L. Waldo, M. D. Ezekowitz, J. I. Weitz, J. Špinar, and W. Ruzyllo. 2013. Edoxaban versus warfarin in patients with atrial fibrillation. New England Journal of Medicine 369(22):2093- 2104. Go, A. S., E. M. Hylek, L. H. Borowsky, K. A. Phillips, J. V. Selby, and D. E. Singer. 1999. Warfarin use among ambulatory patients with nonvalvular atrial fibrillation: The AnTicoagulation and Risk Factors in Atrial Fibrillation (ATRIA) study. Annals of Internal Medicine 131(12):927-934. Goettsch, W. and A. Makady. 2015. Glossary of definitions of common terms. IMI GetReal. https://www.imi-getreal.eu/Portals/1/Documents/01%20deliverables/D1.3%20- %20GetReal%20Glossary%20of%20Definitions%20of%20Common%20Terms%20%28Incl uding%20Comments%20%26%20Replies%20from%20Consultation%20Rounds%29.pdf (accessed December 17, 2018). Gökbuget, N., M. Kelsh, V. Chia, A. Advani, R. Bassan, H. Dombret, M. Doubek, A. K. Fielding, S. Giebel, V. Haddad, D. Hoelzer, C. Holland, N. Ifrah, A. Katz, T. Maniar, G. Martinelli, M. Morgades, S. O'Brien, J. M. Ribera, J. M. Rowe, A. Stein, M. Topp, M. Wadleigh, and H. Kantarjian. 2016. Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia. Blood Cancer Journal 6:e473. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

142 EXAMINING THE IMPACT OF RWE ON MEDICAL PRODUCT DEVELOPMENT Granger, C. B., J. H. Alexander, J. J. McMurray, R. D. Lopes, E. M. Hylek, M. Hanna, H. R. Al- Khalidi, J. Ansell, D. Atar, A. Avezum, and M.C. Bahit. 2011. Apixaban versus warfarin in patients with atrial fibrillation. New England Journal of Medicine 365(11):981-992. Greene, S. M., R. J. Reid, and E. B. Larson. 2012. Implementing the learning health system: From concept to action. Annals of Internal Medicine, 157(3):207-210. Hammond, E. C., and D. Horn. 1954. The relationship between human smoking habits and death rates: A follow-up study of 187,766 men. Journal of the American Medical Association 155(15):1316-1328. Huang, G., X. Sun, D. Liu, Y. Zhang, B. Zhang, G. Xiao, X. Li, X. Gao, C. Hu, M. Wang, H. Ren, and S. Qin. 2018. The efficacy and safety of anti-PD-1/PD-L1 antibody therapy versus docetaxel for pretreated advanced NSCLC: A meta-analysis. Oncotarget 9(3):4239-4248. Izurieta, H. S., M. Wernecke, J. Kelman, S. Wong, R. Forshee, D. Pratt, Y. Lu, Q. Sun, C. Jankosky, P. Krause, and C. Worrall. 2017. Effectiveness and duration of protection provided by the live-attenuated herpes zoster vaccine in the Medicare population ages 65 years and older. Clinical Infectious Diseases 64(6):785-793. Kim, S. C., D. H. Solomon, J. R. Rogers, S. Gale, M. Klearman, K. Sarsour, and S. Schneeweiss. 2017. Cardiovascular safety of tocilizumab versus tumor necrosis factor inhibitors in patients with rheumatoid arthritis: A multi-database cohort study. Arthritis & Rheumatology 69(6):1154-1164. Kimmelman, J., and A. J. London. 2015. The structure of clinical translation. Hastings Center Report 45(2): 27-39. London, A. J. 2018. Learning health systems, clinical equipoise and the ethics of response adaptive randomization. Journal of Medical Ethics 44:409-415. Madjid, M., C. C. Miller, V. V. Zarubaev, I. G. Marinich, O. I. Kiselev, Y.V. Lobzin, A. E. Filippov, and S.W. Casscells III. 2007. Influenza epidemics and acute respiratory disease activity are associated with a surge in autopsy-confirmed coronary heart disease death: Results from 8 years of autopsies in 34,892 subjects. European Heart Journal 28(10):1205- 1210. Medical Device Registry Task Force & the Medical Devices Epidemiology Network. 2015. Recommendations for a national medical device evaluation system. https://mdepinet.org/wp- content/uploads/Recommendations-for-a-National-Medical-Device-Evaluation-System_24- Aug-2015.pdf (accessed December 17, 2018). Moseley, J. B., K. O’Malley, N. J. Petersen, T. J. Menke, B. A. Brody, D. H. Kuykendall, J. C. Hollingsworth, C. M. Ashton, and N. P. Wray. 2002. A controlled trial of arthroscopic surgery for osteoarthritis of the knee. New England Journal of Medicine 347(2):81-88. Neal, B., V. Perkovic, K. W. Mahaffey, D. De Zeeuw, G. Fulcher, N. Erondu, W. Shaw, G. Law, M. Desai, and D. R. Matthews. 2017. Canagliflozin and cardiovascular and renal events in type 2 diabetes. New England Journal of Medicine 377(7):644-657. Palmsten, K., K. F. Huybrechts, K. B. Michels, P. L. Williams, H. Mogun, S. Setoguchi, and S. Hernández-Díaz. 2013. Antidepressant use and risk for preeclampsia. Epidemiology (Cambridge, Mass.) 24(5):682. Patel, M. R., K. W. Mahaffey, J. Garg, G. Pan, D. E. Singer, W. Hacke, B. Breithardt, J. L. Halperin, G. J. Hankey, J. P. Piccini, R. C. Becker, C. C. Nessel, J. F. Paolini, S. D. Berkowitz, K. A. Fox, R. M. Califf, for the ROCKET AF Investigators. 2011. Rivaroxaban versus warfarin in nonvalvular atrial fibrillation. New England Journal of Medicine 365(10):883-891. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

REFERENCES 143 Patorno, E., A. B. Goldfine, S. Schneeweiss, B. M. Everett, R. J. Glynn, J. Liu, and S. C. Kim. 2018. Cardiovascular outcomes associated with canagliflozin versus other non-gliflozin antidiabetic drugs: Population based cohort study. BMJ 360:k119. Pew Charitable Trusts. 2018. Mobile fact sheet. http://www.pewinternet.org/fact-sheet/mobile/ (accessed December 21, 2018). The Physicians Foundation. 2018. 2018 Survey of America’s Physicians Practice Patterns and Perspectives. https://physiciansfoundation.org/wp-content/uploads/2018/09/physicians- survey-results-final-2018.pdf (accessed December 21, 2018). Rosenbaum, P. R. 2010. Observational studies, 2nd ed. New York, NY: Springer. Rubin, D. and N. Thomas. 2000. Combining propensity score matching with additional adjustments for prognostic covariates. Journal of the American Statistical Association 95(450):573-585. Ruff, C. T., R. P. Giugliano, E. Braunwald, E. B. Hoffman, N. Deenadayalu, M. D. Ezekowitz, A. J. Camm, J. I. Weitz, B. S. Lewis, A. Parkhomenko, and T. Yamashita. 2014. Comparison of the efficacy and safety of new oral anticoagulants with warfarin in patients with atrial fibrillation: A meta-analysis of randomised trials. The Lancet 383(9921):955-962. Schneeweiss, S., J. D. Seeger, J. Landon, and A. M. Walker. 2008. Aprotinin during coronary- artery bypass grafting and risk of death. New England Journal of Medicine 358(8):771-783. Seeger, J. D., K. Bykov, D. B. Bartels, K. Huybrechts, K. Zint, and S. Schneeweiss. 2015. Safety and effectiveness of dabigatran and warfarin in routine care of patients with atrial fibrillation. Thrombosis and Haemostasis 114(06):1277-1289. Sentinel. 2017. ICPE presentation: Developing a mother–infant cohort in Sentinel’s PRISM program as a resource to monitor the safety of vaccine use during pregnancy. https://www.sentinelinitiative.org/communications/publications/2017-icpe-presentation- developing-mother-infant-cohort-sentinels-prism (accessed December 17, 2018). Sentinel. 2018. Sentinel common data model. https://www.sentinelinitiative.org/sentinel/data/distributed-database-common-data- model/sentinel-common-data-model (accessed December 17, 2018). Sherman, R. E., S. A. Anderson, G. J. Dal Pan, G. W. Gray, T. Gross, N. L. Hunter, L. LaVange, D. Marinac-Dabic, P. W. Marks, M. A. Robb, and J. Shuren. 2016. Real-world evidence— what is it and what can it tell us. New England Journal of Medicie 375(23):2293-2297. Thompson, W. W., D. K. Shay, E. Weintraub, L. Brammer, N. Cox, L. J. Anderson, and K. Fukuda. 2003. Mortality associated with influenza and respiratory syncytial virus in the United States. JAMA 289(2):179-186. Thompson, W. W., D. K. Shay, E. Weintraub, L. Brammer, C. B. Bridges, N. J. Cox, and K. Fukuda. 2004. Influenza-associated hospitalizations in the United States. JAMA 292(11):1333-1340. Tseng, H. F., N. Smith, R. Harpaz, S. R. Bialek, L. S. Sy, and S. J. Jacobsen. 2011. Herpes zoster vaccine in older adults and the risk of subsequent herpes zoster disease. JAMA 305(2):160- 166. Udell, J. A., R. Zawi, D. L. Bhatt, M. Keshtkar-Jahromi, F. Gaughran, A. Phrommintikul, A. Ciszewski, H. Vakili, E. B. Hoffman, M. E. Farkouh, and C. P. Cannon. 2013. Association between influenza vaccination and cardiovascular outcomes in high-risk patients: A meta- analysis. JAMA 310(16):1711-1720. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

144 EXAMINING THE IMPACT OF RWE ON MEDICAL PRODUCT DEVELOPMENT Van Ermen, A., M. P. Hermanson, J. M. Moran, N. K. Sweitzer, M. R. Johnson, and O. Vardeny. 2013. Double dose vs. standard dose influenza vaccination in patients with heart failure: A pilot study. European Journal of Heart Failure 15(5):560-564. Vardeny, O., N. K. Sweitzer, M. A. Detry, J. M. Moran, M. R. Johnson, and M. S. Hayney. 2009. Decreased immune responses to influenza vaccination in patients with heart failure. Journal of Cardiac Failure 15(4):368-373. Waldo, A. L., R. C. Becker, V. F. Tapson, K. J. Colgan, and NABOR Steering Committee. 2005. Hospitalized patients with atrial fibrillation and a high risk of stroke are not being provided with adequate anticoagulation. Journal of the American College of Cardiology 46(9):1729- 1736. Yih, W. K., T. A. Lieu, M. Kulldorff, D. Martin, C. N. McMahill-Walraven, R. Platt, N. Selvam, M. Selvan, G. M. Lee, and M. Nguyen. 2014. Intussusception risk after rotavirus vaccination in U.S. infants. New England. Journal of Medicine 370:503-512. Zinman, B., C. Wanner, J. M. Lachin, D. Fitchett, E. Bluhmki, S. Hantel, M. Mattheus, T. Devins, O. E. Johansen, H. J. Woerle, and U. C. Broedl. 2015. Empagliflozin, cardiovascular outcomes, and mortality in type 2 diabetes. New England Journal of Medicine 373(22):2117- 2128. P REP UBLI CATI ON COP Y: UNCORR ECTED P ROOFS

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Randomized controlled trials (RCTs) have traditionally served as the gold standard for generating evidence about medical interventions. However, RCTs have inherent limitations and may not reflect the use of medical products in the real world. Additionally, RCTs are expensive, time consuming, and cannot answer all questions about a product or intervention. Evidence generated from real-world use, such as real-world evidence (RWE) may provide valuable information, alongside RCTs, to inform medical product decision making.

To explore the potential for using RWE in medical product decision making, the National Academies of Sciences, Engineering, and Medicine planned a three-part workshop series. The series was designed to examine the current system of evidence generation and its limitations, to identify when and why RWE may be an appropriate type of evidence on which to base decisions, to learn from successful initiatives that have incorporated RWE, and to describe barriers that prevent RWE from being used to its full potential. This publication summarizes the discussions from the entire workshop series.

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