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Suggested Citation:"Appendix A: Bibliography." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
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Appendix A

Bibliography
1

Anglemyer, A., H. T. Horvath, and L. Bero. 2014. Healthcare outcomes assessed with observational study designs compared with those assessed in randomized trials. Cochrane Database of Systematic Reviews (4).

Berndt, E. R., and I. M. Cockburn. 2014. Price indexes for clinical trial research: A feasibility study. Monthly Labor Review. http://www.bls.gov/opub/mlr/2014/article/price-indexesfor-clinical-trial-research-a-feasibility-study-1.htm (accessed November 30, 2016).

Carroll, J. D., F. H. Edwards, D. Marinac-Dabic, R. G. Brindis, F. L. Grover, E. D. Peterson, E. M. Tuzcu, D. M. Shahian, J. S. Rumsfeld, C. M. Shewan, K. Hewitt, D. R. Holmes, Jr., and M. J. Mack. 2013. The STS-ACC transcatheter valve therapy national registry: A new partnership and infrastructure for the introduction and surveillance of medical devices and therapies. Journal of the American College of Cardiology 62(11):1026-1034.

CMS (Centers for Medicare & Medicaid Services). 2014. Guidance for the public, industry, and CMS staff: Coverage with evidence development.https://www.cms.gov/medicarecoverage-database/details/medicare-coverage-document-details.aspx?MCDId=27 (accessed November 30, 2016).

FDA (U.S. Food and Drug Administration). 2012. Strengthening our national system for medical device post-market surveillance.http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDRH/CDRHReports/UCM301924.pdf (accessed November 30, 2016).

FDA. 2016. Use of real-world evidence to support regulatory decision-making for medical devices: Draft guidance for industry and Food and Drug Administration staff.http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm513027.pdf (accessed November 29, 2016).

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1 This bibliography contains resources provided during presentations by workshop speakers, but not necessarily cited in the Proceedings of a Workshop. These resources are included here as additional direction for readers interested in further exploration of the topics discussed at the workshop.

Suggested Citation:"Appendix A: Bibliography." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
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Galson, S. K., and G. Simon. 2016. Real-world evidence to guide the approval and use of new treatments. Washington, DC: The National Academy of Medicine. https://nam.edu/wp-content/uploads/2016/10/Real-World-Evidence-to-Guide-the-Approval-and-Use-ofNew-Treatments.pdf (accessed June 6, 2017).

Han, H., H. Chao, A. Guerra, A. Sosa, G. Christopoulos, G. E. Christakopoulos, B. V. Rangan, S. Maragkoudakis, H. Jneid, S. Banerjee, and E. S. Brilakis. 2015. Evolution of the American College of Cardiology/American Heart Association clinical guidelines. Journal of the American College of Cardiology 65(25):2726-2734.

Hripcsak, G., P. B. Ryan, J. D. Duke, N. H. Shah, R. W. Park, V. Huser, M. A. Suchard, M. J. Schuemie, F. J. DeFalco, A. Perotte, J. M. Banda, C. G. Reich, L. M. Schilling, M. E. Matheny, D. Meeker, N. Pratt, and D. Madigan. 2016. Characterizing treatment pathways at scale using the OHDSI network. Proceedings of the National Academy of Sciences of the United States of America 113(27):7329-7336.

IOM (Institute of Medicine). 2001. Crossing the quality chasm: A new health system for the 21st century. Washington, DC: National Academy Press.

IOM. 2013a. Best care at lower cost: The path to continuously learning health care in America. Washington, DC: The National Academies Press.

IOM. 2013b. Large simple trials and knowledge generation in a learning health system: Workshop summary. Washington, DC: The National Academies Press.

IOM. 2013c. Sharing clinical research data: Workshop summary. Washington, DC: The National Academies Press.

Longhurst, C. A., R. A. Harrington, and N. H. Shah. 2014. A “Green Button” for using aggregate patient data at the point of care. Health Affairs 33(7):1229-1235.

NASEM (National Academies of Sciences, Engineering, and Medicine). 2016. Advancing the discipline of regulatory science for medical product development: An update on progress and a forward-looking agenda: Workshop summary. Washington, DC: The National Academies Press.

ONC (Office of the National Coordinator for Health Information Technology). 2015. Connecting health and care for the nation: A shared nationwide interoperability roadmap version 1.0.https://www.healthit.gov/sites/default/files/hie-interoperability/nationwideinteroperability-roadmap-final-version-1.0.pdf (accessed November 30, 2016).

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, J. Shuren, R. Temple, J. Woodcock, L. Q. Yue, and R. M. Califf. 2016. Real-world evidence—what is it and what can it tell us? New England Journal of Medicine 375(23):2293-2297.

Tricoci, P., J. M. Allen, J. M. Kramer, R. M. Califf, and S. C. Smith, Jr. 2009. Scientific evidence underlying the ACC/AHA clinical practice guidelines. Journal of the American Medical Association 301(8):831-841.

Vashisht, R., K. Jung, and N. Shah. 2016 Learning effective treatment pathways for type 2 diabetes from a clinical data warehouse. In Proceedings of the AMIA Annual Symposium 2016, Chicago, IL, May 12-16. Pp. 2036-2042.

Vestbo, J., D. Leather, N. Diar Bakerly, J. New, J. M. Gibson, S. McCorkindale, S. Collier, J. Crawford, L. Frith, C. Harvey, H. Svedsater, A. Woodcock, and Salford Lung Study Investigators. 2016. Effectiveness of fluticasone furoate-vilanterol for COPD in clinical practice. New England Journal of Medicine 375(13):1253-1260.

Suggested Citation:"Appendix A: Bibliography." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
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Page 51
Suggested Citation:"Appendix A: Bibliography." National Academies of Sciences, Engineering, and Medicine. 2017. Real-World Evidence Generation and Evaluation of Therapeutics: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24685.
×
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The volume and complexity of information about individual patients is greatly increasing with use of electronic records and personal devices. Potential effects on medical product development in the context of this wealth of real-world data could be numerous and varied, ranging from the ability to determine both large-scale and patient-specific effects of treatments to the ability to assess how therapeutics affect patients’ lives through measurement of lifestyle changes.

In October 2016, the National Academies of Sciences, Engineering, and Medicine held a workshop to facilitate dialogue among stakeholders about the opportunities and challenges for incorporating real-world evidence into all stages in the process for the generation and evaluation of therapeutics. Participants explored unmet stakeholder needs and opportunities to generate new kinds of evidence that meet those needs. This publication summarizes the presentations and discussions from the workshop.

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