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The tremendous promise

The tremendous promise evident in today’s biopharmaceutical pipeline represents not only the ever-growing knowledge of the genetic and biological basis of disease, but also the dedication of researchers to translate this incredible science into new and potentially life-saving medicines for patients. Innovative approaches target a broad range of diseases, and this chapter highlights just a few examples of how researchers are striving to transform the lives of patients and the trajectory of today’s most complex and challenging diseases. REFERENCES 1 Adis R&D Insight Database. Accessed March 2016. 2 Analysis Group, Inc. Innovation in the biopharmaceutical pipeline. http://phrma.org/sites/default/files/pdf/2013innovationinthebiopharma ceuticalpipeline-analysisgroupfinal.pdf. Published January 2013. Accessed March 2016. 3 Tufts Center for the Study of Drug Development. Personalized medicine gains traction but still faces multiple challenges. Impact Report. 2015 May/June; 17(3). 4 Pharmaceutical Research and Manufacturers of America. A decade of innovation in chronic diseases: 2006-2016. http://phrma.org/sites/ default/files/pdf/decade-of-innovation-chronic-disease.pdf. Published 2016. Accessed March 2016. 5 Pharmaceutical Research and Manufacturers of America. A decade of innovation in chronic diseases: 2006-2016. http://phrma.org/sites/ default/files/pdf/decade-of-innovation-chronic-disease.pdf. Published 2016. Accessed March 2016. 6 Ledford H. Drug that boosts nerve signals offers hope for multiple sclerosis. http://www.nature.com/news/drug-that-boosts-nervesignals-offers-hope-for-multiple-sclerosis-1.17367. Published April 22, 2015. Accessed March 2016. 7 The Global Genes Project. RARE diseases: Facts and statistics. http://globalgenes.org/rare-diseases-facts-statistics/. Published 2015. Accessed March 2016. 8 Pariser A, Yao L. Rare diseases and orphan drugs. In: Mulberg AE, Murphy D, Dunne J, Mathis LL, eds. Pediatric Drug Development: Concepts and Applications. Hoboken, NJ: John Wiley & Sons;2013:130-148. http://onlinelibrary.wiley.com/book/10.1002/9781118312087. Accessed March 2016. 9 The Global Genes Project. RARE diseases: Facts and statistics. http://globalgenes.org/rare-diseases-facts-statistics/. Published 2015. Accessed March 2016. 10 The Global Genes Project. RARE diseases: Facts and statistics. http://globalgenes.org/rare-diseases-facts-statistics/. Published 2015. Accessed March 2016. 11 Pharmaceutical Research and Manufacturers of America. Rare diseases: A report on orphan drugs in the pipeline. http://phrma.org/ sites/default/files/pdf/Rare_Diseases_2013.pdf. Published 2013. Accessed March 2016. 12 American Chemical Society. Chemical & Engineering News Supplement. The 2015 top 20 drugs in the pipeline. http://cen.acs.org/ content/dam/cen/supplements/censup09282015.pdf. Published September 2015. Accessed March 2016. 13 Pharmaceutical Research and Manufacturers of America. Medicines in development for leukemia and lymphoma. http://www.phrma. org/sites/default/files/pdf/leukemia-lyphoma-2015.pdf. Accessed March 2016. 14 Pharmaceutical Research and Manufacturers of America. Medicines in development for leukemia and lymphoma. http://www.phrma. org/sites/default/files/pdf/leukemia-lyphoma-2015.pdf. Accessed March 2016. 15 American Chemical Society. Chemical & Engineering News Supplement. The 2015 top 20 drugs in the pipeline. http://cen.acs.org/ content/dam/cen/supplements/censup09282015.pdf. Published September 2015. Accessed March 2016. 16 American Chemical Society. Chemical & Engineering News Supplement. The 2015 top 20 drugs in the pipeline. http://cen.acs.org/ content/dam/cen/supplements/censup09282015.pdf. Published September 2015. Accessed March 2016. 66 The Promise of the Pipeline

Looking Ahead CONCLUSION

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