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Suggested Citation:"References." National Academies of Sciences, Engineering, and Medicine. 2017. Making Medicines Affordable: A National Imperative. Washington, DC: The National Academies Press. doi: 10.17226/24946.
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References (All links were last accessed on November 13, 2017.) Chapter 1 ASPE (Office of the Assistant Secretary for Planning and Evaluation). 2016. Observations on trends in prescription drug spending. March 8, 2016. https://aspe.hhs.gov/system/files/pdf/187586/Drugspending.pdf. Bach, P. 2009. Limits on Medicare’s ability to control rising spending on cancer drugs. New England Journal of Medicine 360(6):626–633. Bach, P. 2015. Why drugs cost so much. The New York Times, January 14. www.nytimes.com/2015/01/15/opinion/why-drugs-cost-so-much.html?_r=0. Bach, P. 2016. A new way to define value in drug pricing. New England Journal of Medicine Catalyst. February 24. http://catalyst.nejm.org/a-new-way-to-define-value-in-drug- pricing. Bach, P. 2017. Price & value of cancer drug. https://www.mskcc.org/research-areas/programs- centers/health-policy-outcomes/cost-drugs. Beitz, C. 2005. Cosmopolitanism and global justice. Journal of Ethics 9(1):11–27. Bennette, C. S., C. Richards, S. D. Sullivan, and S. D. Ramsey. 2016. Steady increase in prices for oral anticancer drugs after market launch suggests a lack of competitive pressure. Health Affairs 35(5):805–812. Boulier, B. L., T. S. Datta, and R. S. Goldfarb. 2007. Vaccination externalities. B.E. Journal of Economic Analysis & Policy 7(1):1–27. Buzaglo, J. S., E. S. Charap, L. House, N. K. Gupta, and M. Longacre. 2016. Definitions of value among patients with breast cancer. Journal of Clinical Oncology 35(15):e18259. CMS (Centers for Medicare & Medicaid Services). 2015. National health expenditures: 2015 highlights. www.cms.gov/research-statistics-data-and-systems/statistics-trends-and- reports/nationalhealthexpenddata/downloads/highlights.pdf. Cohen, I. G., N. Daniels, and N. Eyal. 2015. Identified versus statistical lives: An interdisciplinary perspective. New York: Oxford University Press. Conti, R. M., W. V. Padula, and R. A. Larson. 2016. Changing the cost of care for chronic myeloid leukemia: The availability of generic imatinib in the USA and the EU. Annals of Hematology 94(2):249-257. Cutler, D. M., and R. J. Zeckhauser. 2000. The anatomy of health insurance. Handbook of Health Economics 1:563–643. Daniels, N. 2007. Just health: Meeting health needs fairly. New York: Cambridge University Press. Danzon, P. 2014. Pricing and reimbursement of biopharmaceuticals and medical devices in the USA. Encyclopedia of Health Economics 3:127–135. De George, R. T. 2005. Intellectual property and pharmaceutical drugs: An ethical analysis. Business Ethics Quarterly 15(04):549–575. 125 PREPUBLICATION COPY: UNCORRECTED PROOFS

MAKING MEDICINES AFFORDABLE 126 Dunfee, T. W. 2006. Do firms with unique competencies for rescuing victims of human catastrophes have special obligations? Corporate responsibility and the AIDS catastrophe in sub-Saharan Africa. Business Ethics Quarterly 16(2):185–210. Dusetzina, S. B. 2016. Drug pricing trends for orally administered anticancer medications reimbursed by commercial health plans, 2000–2014. JAMA Oncology 2(7):960–961. Dusetzina, S. B., and N. L. Keating. 2015. Mind the gap: Why closing the doughnut hole is insufficient for increasing Medicare beneficiary access to oral chemotherapy. Journal of Clinical Oncology 34(4):375–380. Elegido, J. M. 2015. The just price as the price obtainable in an open market. Journal of Business Ethics 130(3):557–572. Elhauge, E. 2005. Sacrificing corporate profits in the public interest. New York University Law Review 80:733. Express Scripts. 2017. The Express Scripts 2016 drug trend report. http://lab.express- scripts.com/lab/drug-trend-report. Frederick, S., G. Loewenstein, and T. O’Donoghue. 2002. Time discounting and time preference: A critical review. Journal of Economic Literature 40(2):351–401. Fried, I., A. L. Beam, I. S. Kohane, and N. P. Palmer. 2017. Utilization, cost, and outcome of branded vs compounded 17-alpha hydroxyprogesterone caproate in prevention of preterm birth. JAMA Internal Medicine. https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2655241. Friedman, M. 2009. Capitalism and freedom. Chicago: University of Chicago Press (Reprint edition). Hale, V. G., K. Woo, and H. L. Lipton. 2005. Oxymoron no more: The potential of nonprofit drug companies to deliver on the promise of medicines for the developing world. Health Affairs 24(4):1057–1063. Hartman, M., A. B. Martin, D. Lassman, A. Catlin, and National Health Expenditure Accounts Team. 2015. National health spending in 2013: Growth slows, remains in step with the overall economy. Health Affairs 34(1):150–160. Heath, J. 2014. Morality, competition, and the firm: The market failures approach to business ethics. New York: Oxford University Press. Hsieh, N.-h. 2009. Corporate social responsibility and the priority of shareholders. Journal of Business Ethics 88:553–560. IOM (Institute of Medicine). 2009. The U.S. commitment to global health: Recommendations for the public and private sectors. Washington, DC: The National Academies Press. Kamal-Yanni, M. 2015. Hepatitis C drug affordability. The Lancet Global Health 3(2):e73–e74. Kanavos, P., A. Ferrario, S. Vandoros, and G. F. Anderson. 2013. Higher U.S. branded drug prices and spending compared to other countries may stem partly from quick uptake of new drugs. Health Affairs 32(4):753–761. Kantor, E. D., C. D. Rehm, J. S. Haas, A. T. Chan, and E. L. Giovannucci. 2015. Trends in prescription drug use among adults in the United States from 1999–2012. JAMA 314(17):1818–1830. Keehan, S. P., D. A. Stone, J. A. Poisal, G. A. Cuckler, A. M. Sisko, S. D. Smith, A. J. Madison, C. J. Wolfe, and J. M. Lizonitz. 2017. National health expenditure projections, 2016-25: Price increases, aging push sector to 20 percent of economy. Health Affairs 36(3):553– 563. PREPUBLICATION COPY: UNCORRECTED PROOFS

MAKING MEDICINES AFFORDABLE 127 Kesselheim, A. S., J. Avorn, and A. Sarpatwari. 2016. The high cost of prescription drugs in the United States: Origins and prospects for reform. JAMA 316(8):858–871. KFF (Kaiser Family Foundation). 2017a. Kaiser health tracking poll: Health care priorities for 2017. http://www.kff.org/health-costs/poll-finding/kaiser-health-tracking-poll-health- care-priorities-for-2017. KFF. 2017b. Public opinion on prescription drugs and their prices. http://www.kff.org/slideshow/public-opinion-on-prescription-drugs-and-their-prices. Lynch, J., and S. E. Gollust. 2010. Playing fair: Fairness beliefs and health policy preferences in the United States. Journal of Health Politics, Policy and Law 35(6):849–887. Mackey, T. K., B. A. Liang, R. Cuomo, R. Hafen, K. C. Brouwer, and D. E. Lee. 2014. Emerging and reemerging neglected tropical diseases: A review of key characteristics, risk factors, and the policy and innovation environment. Clinical Microbiology Reviews 27(4):949–979. Maitland, I. 2002. Priceless goods: How should life-saving drugs be priced? Business Ethics Quarterly 12(04):451–480. Nagel, T. 2005. The problem of global justice. Philosophy & Public Affairs 33(2):113–147. NCI (National Cancer Institute). 2017. PDQ financial toxicity and cancer treatment. https://www.cancer.gov/about-cancer/managing-care/track-care-costs/financial-toxicity- hp-pdq. Newhouse, J. P. 1988. Has the erosion of the medical marketplace ended? Journal of Health Politics, Policy and Law 13(2):263–278. Phelps, C. 2018. Health economics. 6th Edition. New York: Routledge Press. Politico. 2017. Americans’ top priorities for Congress through the end of the year. https://www.politico.com/f/?id=0000015e-7bce-d079-a3fe-7bce31540000. QuintilesIMS. 2016. Medicines use and spending in the U.S.: A review of 2015 and outlook to 2020. Parsippany, NJ: QuintilesIMS Institute. QuintilesIMS. 2017. Medicines use and spending in the U.S.: A review of 2016 and outlook to 2021. Parsippany, NJ: QuintilesIMS Institute. Ramsey, S., D. Blough, A. Kirchhoff, K. Kreizenbeck, C. Fedorenko, K. Snell, P. Newcomb, W. Hollingworth, and K. Overstreet. 2013. Washington State cancer patients found to be at greater risk for bankruptcy than people without a cancer diagnosis. Health Affairs 32(6):1143–1152. Rawls, J. 1971. A theory of justice. Cambridge, MA: Harvard University Press. Reuters. 2014. Sanofi rolls out large-scale supply of synthetic malaria drug. http://www.reuters.com/article/health-malaria-sanofi/sanofi-rolls-out-large-scale-supply- of-synthetic-malaria-drug-idUSL6N0QI1B020140812. Rosenblatt, M. 2017. The large pharmaceutical company perspective. New England Journal of Medicine 376(1):52–60. Rosenblatt, M., and H. Termeer. 2017. Reframing the conversation on drug pricing, New England Journal of Medicine Catalyst, October 19. https://catalyst.nejm.org/reframing- conversation-drug-pricing. Scherer, F. M. 2000. The pharmaceutical industry. Handbook of health economics 1:1297–1336. Taurel, S. 2005. The campaign against innovation. In Ethics and the pharmaceutical industry. Cambridge, UK: Cambridge University Press. Pp. 326–335. Vagelos, P. R. 1991. Are prescription drug prices high? Science 252(5009):1080–1084. PREPUBLICATION COPY: UNCORRECTED PROOFS

MAKING MEDICINES AFFORDABLE 128 Vagelos, P. R., and L. Galambos. 2004. Medicine, Science and Merck. Cambridge, UK: Cambridge University Press. Valdman, M. 2009. A theory of wrongful exploitation. Philosophers’ Imprint 9(6):1–14. Chapter 2 AACR (American Association for Cancer Research). 2011. AACR cancer progress report 2011. Clinical Cancer Research 22(19 Suppl):S1–S137. AISHealth. 2016. Profiles of the eight largest health insurers: 2016. https://aishealth.com/sites/all/files/bp82016.pdf. Anderson, J. L., P. A. Heidenreich, P. G. Barnett, M. A. Creager, G. C. Fonarow, R. J. Gibbons, J. L. Halperin, M. A. Hlatky, A. K. Jacobs, and D. B. Mark. 2014. ACC/AHA statement on cost/value methodology in clinical practice guidelines and performance measures. Circulation 129(22):2329–2345. Arrowsmith, J. 2011. Trial watch: Phase III and submission failures: 2007–2010. Nature Reviews Drug Discovery 10(2):87. Arrowsmith, J., and P. Miller. 2013. Trial watch: Phase II and phase III attrition rates 2011– 2012. Nature Reviews Drug Discovery 12(8):569. Balto, D. 2014. Testimony of David A. Balto before the Employee Benefits Security Administration Advisory Council on Employee Welfare and Pension Benefit Plans, U.S. Department of Labor. Hearing on section 408(b)(2) regulation: PBM compensation and fee disclosure. June 19. Balto, D. 2015. Testimony of David A. Balto before the House Judiciary Subcommittee on Regulatory Reform, Commercial and Antitrust Law. Hearing on The State of Competition in the Pharmacy Benefits Manager and Pharmacy Marketplaces. November 17. Balto, D. 2017. Testimony of David A. Balto before the California Senate Committee on Business, Profession and Economic Development. Hearing Section on Pharmacy Benefit Managers 101. March 20. Barclays Bank PLC. 2017. The equity research report—U.S. health care distribution & technology: How do PBMs make money? March 7. New York: Barclays Capital Inc. Barlas, S. 2016. Health plans and drug companies dip their toes into value-based pricing: The pressure is on P&T committees to monitor utilization. Pharmacy and Therapeutics 41(1):39–53. BLS (U.S. Bureau of Labor Statistics). 2016. Distribution of private sector employment by firm size class: 1993/Q1 through 2016/Q1, not seasonally adjusted. www.bls.gov/web/cewbd/table_f.txt. Boumil, M. M., and G. D. Curfman. 2013. On access and accountability—Two Supreme Court rulings on generic drugs. New England Journal of Medicine 369(8):696–697. Brill, A. 2014. Lost prescription drug savings from use of REMS programs to delay generic market entry. Matrix Global Advisers. www.gphaonline.org/media/cms/REMS_Studyfinal_July2014.pdf. Carrier, M. A., and S. D. Shadowen. 2016. Product hopping: A new framework. Notre Dame Law Review 92:167. CBO (Congressional Budget Office). 2014. Competition and the cost of Medicare’s prescription drug program. Washington, DC: Congressional Budget Office. PREPUBLICATION COPY: UNCORRECTED PROOFS

MAKING MEDICINES AFFORDABLE 129 Channick, S. A. 2006. The Medicare Prescription Drug, Improvement, and Modernization Act of 2003: Will it be good medicine for US health policy. Elder Law Journal 14:237. Chen, L. 2015. The most profitable industries in 2016. https://www.forbes.com/sites/liyanchen/2015/12/21/the-most-profitable-industries-in- 2016/#3c3ba5d15716. CMS (Centers for Medicare & Medicaid Services). 2015. CMS releases 2014 national health expenditures. https://www.cms.gov/Newsroom/MediaReleaseDatabase/Press- releases/2015-Press-releases-items/2015-12-02.html. CMS. 2016. 2016 annual report of the boards of trustees of the Federal Hospital Insurance and Federal Supplementary Medical Insurance trust funds. https://www.cms.gov/Research- Statistics-Data-and-Systems/Statistics-Trends-and- Reports/ReportsTrustFunds/downloads/tr2016.pdf. CMS. 2017. Medicare Part D – Direct and Indirect Remuneration (DIR). https://www.cms.gov/Newsroom/MediaReleaseDatabase/Fact-sheets/2017-Fact-Sheet- items/2017-01-19-2.html. Cohen, S., and W. Yu. 2012. The concentration and persistence in the level of health expenditures over time: Estimates for the U.S. population, 2008–2009. Statistical brief #354. January.meps.ahrq.gov/data_files/publications/st354/stat354.pdf. Cook, A., T. Kornfield, and M. Gold. 2000. The role of PBMs in managing drug costs: Implications for a Medicare drug benefit: Menlo Park, CA: Henry J. Kaiser Family Foundation. Credit Suisse. 2017. Equity research. Global pharmaceuticals & biotechnology report. April 18. https://research-doc.credit- suisse.com/docView?language=ENG&format=PDF&sourceid=em&document_id=10737 63651&serialid=%2FoS2%2F%2Buhfn1K9jHaHOw40qPqJMxew63e%2F6kudOybyhM %3D. Deloitte, L. L. P. 2016. Measuring the return from pharmaceutical innovation, 2016. www2.deloitte.com/uk/en/pages/life-sciences-and-healthcare/articles/measuring-return- from-pharmaceutical-innovation.html. DeMartino, J. K., and J. K. Larsen. 2012. Equity in cancer care: Pathways, protocols, and guidelines. Journal of the National Comprehensive Cancer Network 10(Suppl 1):S-1–S- 9. Duggan, M., and F. Scott Morton. 2010. The effect of Medicare Part D on pharmaceutical prices and utilization. American Economic Review 100(1):590–607. Dusetzina, S. B., and N. L. Keating. 2015. Mind the gap: Why closing the doughnut hole is insufficient for increasing Medicare beneficiary access to oral chemotherapy. Journal of Clinical Oncology 34(4):375–380. Dusetzina, S. B., R. M. Conti, L. Y. Nancy, and P. B. Bach. 2017. Association of prescription drug price rebates in Medicare Part D with patient out-of-pocket and federal spending. JAMA Internal Medicine 177(8):1185–1188. Dwivedi, G., S. Hallihosur, and L. Rangan. 2010. Evergreening: A deceptive device in patent rights. Technology in Society 32(4):324–330. EBRI (Employee Benefit Research Institute). 2016. Self-insured health plans: Recent trends by firm size, 1996–2015. www.ebri.org/pdf/notespdf/EBRI_Notes_07-no7-July16.Self- Ins.pdf. PREPUBLICATION COPY: UNCORRECTED PROOFS

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MAKING MEDICINES AFFORDABLE 131 Fung, A., M. Graham, D. Weil. 2007. Full disclosure: The perils and promise of transparency. Cambridge, UK: Cambridge University Press Gabriel, J. M. 2014. Medical monopoly: Intellectual property rights and the origins of the modern pharmaceutical industry. Chicago: University of Chicago Press. Gagne, J. J., and N. K. Choudhry. 2011. How many “me-too” drugs is too many? JAMA 305(7):711–712. GAO (Government Accountability Office). 2007. Prescription drugs: An overview of approaches to negotiate drug prices used by other countries and U.S. private payers and federal programs. Washington, DC: Government Accountability Office. Garber, A. M., and M. B. McClellan. 2007. Satisfaction guaranteed—“Payment by results” for biologic agents. New England Journal of Medicine 357(16):1575–1577. Garber, A. M., and C. E. Phelps. 1997. Economic foundations of cost-effectiveness analysis. Journal of Health Economics 16(1):1–31. Gesme, D. H., and M. Wiseman. 2011. Strategic use of clinical pathways. Journal of Oncology Practice 7(1):54–56. Gibson, T. B., R. Maclean, M. E. Chernew, A. M. Fendrick, and C. Baigel. 2015. Value-based insurance design: Benefits beyond cost and utilization. The American Journal of Managed Care 21(1):32–35. Goldberg, R. 2017. Reduce drug prices by eliminating PBM rebates. The Hill, February 14. http://thehill.com/blogs/congress-blog/healthcare/319479-reduce-drug-prices-by- eliminating-pbm-rebates. Goodman, S., and K. Dickersin. 2011. Metabias: A challenge for comparative effectiveness research. Annals of Internal Medicine 155(1):61–62. Grabowski, H., C. Brain, A. Taub, and R. Guha. 2017. Pharmaceutical patent challenges: Company strategies and litigation outcomes. American Journal of Health Economics 3(1):33–59. Happe, L. E., D. Clark, E. Holliday, and T. Young. 2014. A systematic literature review assessing the directional impact of managed care formulary restrictions on medication adherence, clinical outcomes, economic outcomes, and health care resource utilization. Journal of Managed Care Pharmacy 20(7):677–684. Helland, E., and S. A. Seabury. 2016. Are settlements in patent litigation collusive? Evidence from paragraph IV challenges. No. w22194. Cambridge, MA: National Bureau of Economic Research. Hemphill, C. S., and B. N. Sampat. 2012. Evergreening, patent challenges, and effective market life in pharmaceuticals. Journal of Health Economics 31(2):327–339. Hopkins, J., and Z. Tracer. 2017. Blame game over high drug prices escalates with new ad. Bloomberg, April 6. www.bloomberg.com/news/articles/2017-04-06/blame-game-over- high-drug-prices-gets-worse-with-lobby-s-new-ad. Howard, D. H., J. Kauh, and J. Lipscomb. 2010. The value of new chemotherapeutic agents for metastatic colorectal cancer. Archives of Internal Medicine 170(6):537–542. Huskamp, H. A., and N. L. Keating. 2005. The new Medicare drug benefit: Formularies and their potential effects on access to medications. Journal of General Internal Medicine 20(7):662–665. IOM (Institute of Medicine). 2010. A national cancer clinical trials system for the 21st century: Reinvigorating the NCI cooperative group program. Washington, DC: The National Academies Press. PREPUBLICATION COPY: UNCORRECTED PROOFS

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Next: Appendix A A Dissenting View »
Making Medicines Affordable: A National Imperative Get This Book
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Thanks to remarkable advances in modern health care attributable to science, engineering, and medicine, it is now possible to cure or manage illnesses that were long deemed untreatable. At the same time, however, the United States is facing the vexing challenge of a seemingly uncontrolled rise in the cost of health care. Total medical expenditures are rapidly approaching 20 percent of the gross domestic product and are crowding out other priorities of national importance. The use of increasingly expensive prescription drugs is a significant part of this problem, making the cost of biopharmaceuticals a serious national concern with broad political implications. Especially with the highly visible and very large price increases for prescription drugs that have occurred in recent years, finding a way to make prescription medicines—and health care at large—more affordable for everyone has become a socioeconomic imperative.

Affordability is a complex function of factors, including not just the prices of the drugs themselves, but also the details of an individual’s insurance coverage and the number of medical conditions that an individual or family confronts. Therefore, any solution to the affordability issue will require considering all of these factors together. The current high and increasing costs of prescription drugs—coupled with the broader trends in overall health care costs—is unsustainable to society as a whole.

Making Medicines Affordable examines patient access to affordable and effective therapies, with emphasis on drug pricing, inflation in the cost of drugs, and insurance design. This report explores structural and policy factors influencing drug pricing, drug access programs, the emerging role of comparative effectiveness assessments in payment policies, changing finances of medical practice with regard to drug costs and reimbursement, and measures to prevent drug shortages and foster continued innovation in drug development. It makes recommendations for policy actions that could address drug price trends, improve patient access to affordable and effective treatments, and encourage innovations that address significant needs in health care.

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