Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases

Hastings Center Report

Volumn 42, Issue 1, 2012, Pages 27-34

  Largent, Emily A ^a   Pearson, Steven D ^b  

^a NIH Department of Bioethics   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; COST BENEFIT ANALYSIS; DRUG MANUFACTURE; ECONOMICS; ETHICS; HEALTH CARE DELIVERY; HEALTH CARE ORGANIZATION; HEALTH INSURANCE; HUMAN; INSURANCE; PATIENT ADVOCACY; RARE DISEASE; RESOURCE ALLOCATION; UNITED STATES;

COST-BENEFIT ANALYSIS; DELIVERY OF HEALTH CARE; HEALTH CARE RATIONING; HUMANS; INSURANCE COVERAGE; INSURANCE, HEALTH; ORPHAN DRUG PRODUCTION; PATIENT ADVOCACY; RARE DISEASES; RESOURCE ALLOCATION; UNITED STATES;

EID: 84861928513     PISSN: 00930334     EISSN: 1552146X     Source Type: Journal    
DOI: 10.1002/hast.12     Document Type: Article

References (31)

1. A. Jonsen, "Bentham in a Box: Technology Assessment and Health Care Allocation, " Law, Medicine, and Health Care 14, nos. 3-4 (1986): 172-74.
2. U.S. Food and Drug Administration, The Orphan Drug Act (as amended), http://www.fda.gov/orphan/oda.htm.
3. ORPHANET, "About Rare Diseases, " available from http://www.orpha.net/con-sor/cgi-bin/Education_AboutRareDiseases.php?lng=EN, accessed April 21, 2009.
4. G. Roosendaal and F. Lafeber, "Prophylactic Treatment for Prevention of Joint Disease in Hemophilia-Cost versus Beneft, " New England Journal of Medicine 357 (2007): 603-5.
5. R.C. Griggs et al., "Clinical Research for Rare Disease: Opportunities, Challenges, and Solutions, " Molecular Genetics and Metabolism 96, no. 1 (2009): 20-26.
6. A. Meyers, "History of the American Orphan Drug Act, " presentation to the International Conference of Rare Diseases and Orphan Drugs, Spain, February 18, 2000, http://www.rarediseases.org/docs/policy/HistoryoftheAmericanOrphanDru-gAct.pdf.
7. T. Cote et al., "Orphan Products: An Emerging Trend in Drug Approvals, " Nature Reviews Drug Discovery 9, no. 1 (2010): 84; E. Seoane-Vazquez et al., "Incentives for Orphan Drug Research and Development in the United States, " Orphanet Journal of Rare Diseases 3, no. 1 (2008): 33.
8. G. Anand, "How Drugs For Rare Diseases Became Lifeline for Companies, " Wall Street Journal, November 15, 2005.
9. C. Adams and V. Brantner, "Spending on New Drug Development, " Health Economics 19, no. 2 (2010): 130-41.
10. Seoane-Vazquez et al., "Incentives for Orphan Drug Research and Development in the United States."
11. DA. Hughes, B. Tunnage, and ST. Yeo, "Drugs for Exceptionally Rare Diseases: Do They Deserve Special Status for Funding?" QJM: An International Journal of Medicine 98, no. 11 (2005): 829-36; M. Schlander and M. Beck, "Expensive Drugs for Rare Disorders: To Treat or Not to Treat? The Case of Enzyme Replacement Therapy for Mucopolysaccharidosis VI, " Current Medical Research and Opinion 25, no. 5 (2009): 1285-93; K. Miles, C. Packer, and A. Stevens, "Quantifying Emerging Drugs for Very Rare Conditions, " QJM: An International Journal of Medicine 100 (2007): 291-95.
12. E. Nord et al., "Who Cares about Cost? Does Economic Analysis Impose or Refect Social Values?" Health Policy 34, no. 2 (1995): 79-94; M. Osborne and T.W. Evans, "Allocation of Resources in Intensive Care: A Transatlantic Perspective, " Lancet 343 (1994): 778-80.
13. C. Fried, An Anatomy of Values: Problems of Personal and Social Choice (Cambridge, Mass.: Harvard University Press, 1970).
14. T Beauchamp and J. Childress, Principles of Biomedical Ethics, 5th ed. (Oxford, U.K.: Oxford University Press, 2001).
15. P Dolan et al., "QALY Maximisation and People's Preferences: A Methodological Review of the Literature, " Health Economics 14, no. 2 (2005): 197-208;
16. NICE Citizens Council, "NICE Citizens Council Report: Rule of Rescue, " January 2006, http://www.nice.org.uk/niceMedia/pdf/board-meeting/brdjul06item6a.pdf.
17. PA. Ubel and G. Loewenstein, "Distributing Scarce Livers: The Moral Reasoning of the General Public, " Social Science and Medicine 42, no. 7 (1996): 1049-55.
18. Fried, An Anatomy of Values.
19. J. Rawls, A Theory of Justice, rev. ed. (1971; Cambridge, Mass.: Belknap Press, 1999).
20. Beauchamp and Childress, Principles of Biomedical Ethics.
21. R. Cookson, C. McCabe, and A. Tsuchiya, "Public Healthcare Resource Allocation and the Rule of Rescue, " Journal of Medical Ethics 34, no. 7 (2008): 540-44.
22. D. Parft, "Equality and Priority, " Ratio 10 (1997): 202-221.
23. W.A. Gahl, J.Z. Balog, and R. Kleta, "Nephropathic Cystinosis in Adults: Natural History and Effects of Oral Cysteamine Therapy, " Annals of Internal Medicine 147, no. 4 (2007): 242-50.
24. A.S. Meyers, "Insurance Reimbursement for Orphan Drugs, " National Organization for Rare Disorders Position Paper, August 6, 2000, http://www.rarediseases.org/docs/policy/InsuranceReimbursement-forOrphanDrugs.pdf
25. C. McCabe et al., "Orphan Drugs Revisited, " QJM: An International Journal of Medicine 99, no. 5 (2006): 341-45.
26. Anand, "How Drugs For Rare Diseases Became Lifeline for Companies."
27. T. Fojo and C. Grady, "How Much Is Life Worth: Cetuximab, Non-Small Cell Lung Cancer, and the $440 Billion Question, " Journal of the National Cancer Institute 101 (2009): 1044-48.
28. M.G. Kris et al., "Efficacy of Gef-tinib, an Inhibitor of the Epidermal Growth Factor Receptor Tyrosine Kinase, in Symptomatic Patients with Non-Small Cell Lung Cancer: A Randomized Trial, " Journal of the American Medical Association 290 (2003): 2149-58.
29. E. Nord, "Concerns for the Worse Off: Fair Innings versus Severity, " Social Sci-ence and Medicine 60, no. 2 (2005): 257-63.
30. J.A. Sollano et al., "The Economics of Drug Discovery and the Ultimate Valuation of Pharmacotherapies in the Marketplace, " Clinical Pharmacology and Therapeutics 84, no. 2 (2008): 263-66.
31. N. Daniels, "Accountability for Reasonableness, " British Medical Journal 321 (2000): 1300-1301.