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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
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Appendix A

References

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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

Falk, M. J., L. Shen, M. Gonzalez, J. Leipzig, M. T. Lott, A. P. Stassen, M. A. Diroma, D. Navarro-Gomez, P. Yeske, R. Bai, R. G. Boles, V. Brilhante, D. Ralph, J. T. DaRe, R. Shelton, S. F. Terry, Z. Zhang, W. C. Copeland, M. van Oven, H. Prokisch, D. C. Wallace, M. Attimonelli, D. Krotoski, S. Zuchner, X. Gai, and the MSeqDR Consortium Participants. 2015. Mitochondrial Disease Sequence Data Resource (MSeqDR): A global grassroots consortium to facilitate deposition, curation, annotation, and integrated analysis of genomic data for the mitochondrial disease clinical and research communities. Molecular Genetics and Metabolism 114(3):388–396.

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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

Khera, A. V., H. H. Won, G. M. Peloso, C. O’Dushlaine, D. Liu, N. O. Stitziel, P. Natarajan, A. Nomura, C. A. Emdin, N. Gupta, I. B. Borecki, R. Asselta, S. Duga, P. A. Merlini, A. Correa, T. Kessler, J. G. Wilson, M. J. Bown, A. S. Hall, P. S. Braund, D. J. Carey, M. F. Murray, H. L. Kirchner, J. B. Leader, D. R. Lavage, J. N. Manus, D. N. Hartzel, N. J. Samani, H. Schunkert, J. Marrugat, R. Elosua, R. McPherson, M. Farrall, H. Watkins, E. S. Lander, D. J. Rader, J. Danesh, D. Ardissino, S. Gabriel, C. Willer, G. R. Abecasis, D. Saleheen, F. E. Dewey, S. Kathiresan, and the Myocardial Infarction Genetics Consortium, DiscovEHR Study Group, CARDIoGRAM Exome Consortium, and Global Lipids Genetics Consortium. 2017. Association of rare and common variation in the lipoprotein lipase gene with coronary artery Disease. JAMA 317(9):937–946.

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Mallal, S., E. Phillips, G. Carosi, J. M. Molina, C. Workman, J. Tomazic, E. Jägel-Guedes, S. Rugina, O. Kozyrev, J. F. Cid, P. Hay, D. Nolan, S. Hughes, A. Hughes, S. Ryan, N. Fitch, D. Thorborn, A. Benbow, and the PREDICT-1 Study Team. 2008. HLA-B5701 screening for hypersensitivity to abacavir. New England Journal of Medicine 358(6):568–579.

Manrai, A. K., B. H. Funke, H. L. Rehm, M. S. Olesen, B. A. Maron, P. Szolovits, D. M. Margulies, J. Loscalzo, and I. S. Kohane. 2016. Genetic misdiagnoses and the potential for health disparities. New England Journal of Medicine 375(7):655–665.

Martin, M. A., J. M. Hoffman, R. R. Freimuth, T. E. Klein, B. J. Dong, M. Pirmohamed, J. K. Hicks, M. R. Wilkinson, D. W. Haas, D. L. Kroetz, and the Clinical Pharmacogenetics Implementation Consortium. 2014. Clinical Pharmacogenetics Implementation Consortium guidelines for HLA-B genotype and abacavir dosing: 2014 update. Clinical Pharmacology & Therapeutics 95(5):499–500.

Mega, J. L., T. Simon, J. P. Collet, J. L. Anderson, E. M. Antman, K. Bliden, C. P. Cannon, N. Danchin, B. Giusti, P. Gurbel, B. D. Horne, J. S. Hulot, A. Kastrati, G. Montalescot, F. J. Neumann, L. Shen, D. Sibbing, P. G. Steg, D. Trenk, S. D. Wiviott, and M. S. Sabatine. 2010. Reduced-function CYP2C19 genotype and risk of adverse clinical outcomes among patients treated with clopidogrel predominantly for PCI: A meta-analysis. JAMA 304(16):1821–1830.

Mosteller, M., L. Hosking, K. Murphy, J. Shen, K. Song, M. Nelson, and S. Ghosh. 2017. No evidence of large genetic effects on steroid response in asthma patients. Journal of Allergy and Clinical Immunology 139(3):797–803, e7.

Mu, T. W., D. M. Fowler, and J. W. Kelly. 2008. Partial restoration of mutant enzyme homeostasis in three distinct lysosomal storage disease cell lines by altering calcium homeostasis. PLOS Biology 6(2):e26.

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NASEM. 2016c. Biomarker tests for molecularly targeted therapies: Key to unlocking precision medicine: Report in brief. http://nationalacademies.org/hmd/~/media/Files/Report%20Files/2016/Biomarkers/Biomarkers-RiB.pdf (accessed April 26, 2017).

Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×

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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
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Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 94
Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 95
Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 96
Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 97
Suggested Citation:"Appendix A: References." National Academies of Sciences, Engineering, and Medicine. 2017. Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/24829.
×
Page 98
Next: Appendix B: Statement of Task and Workshop Agenda »
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Those involved in the drug development process face challenges of efficiency and overall sustainability due in part to high research costs, lengthy development timelines, and late-stage drug failures. Novel clinical trial designs that enroll participants based on their genetics represent a potentially disruptive change that could improve patient outcomes, reduce costs associated with drug development, and further realize the goals of precision medicine.

On March 8, 2017, the Forum on Drug Discovery, Development, and Translation and the Roundtable on Genomics and Precision Health of the National Academies of Sciences, Engineering, and Medicine hosted the workshop Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development. Participants examined successes, challenges, and possible best practices for effectively using genetic information in the design and implementation of clinical trials to support the development of precision medicines, including exploring the potential advantages and disadvantages of such trials across a variety of disease areas. This publication summarizes the presentations and discussions from the workshop.

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