Commentary: Limits on use of health economic assessments for rare diseases

QJM: An International Journal of Medicine

Volumn 107, Issue 3, 2014, Pages 241-245

  Hyry, H I ^a   Stern, A D ^b   Cox, T M ^a   Roos, J C P ^a  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^b HARVARD UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ORPHAN DRUG;

COST EFFECTIVENESS ANALYSIS; DRUG COST; HEALTH CARE PERSONNEL; HEALTH CARE SYSTEM; HEALTH ECONOMICS; HUMAN; NOTE; PRIORITY JOURNAL; RARE DISEASE; REIMBURSEMENT; SOCIETY; UNITED KINGDOM; ARTICLE; BUDGET; COST BENEFIT ANALYSIS; DRUG MANUFACTURE; ECONOMICS; EUROPEAN UNION; HEALTH CARE COST; NEEDS ASSESSMENT; QUALITY ADJUSTED LIFE YEAR; QUALITY OF LIFE;

BUDGETS; COST-BENEFIT ANALYSIS; DRUG COSTS; EUROPEAN UNION; HEALTH EXPENDITURES; HUMANS; NEEDS ASSESSMENT; ORPHAN DRUG PRODUCTION; QUALITY OF LIFE; QUALITY-ADJUSTED LIFE YEARS; RARE DISEASES;

EID: 84894321192     PISSN: 14602725     EISSN: 14602393     Source Type: Journal    
DOI: 10.1093/qjmed/hcu016     Document Type: Note

References (28)

1. McCabe C, Tsuchiya A, Claxton K. Orphan drugs revisited. Q J Med 2006;99:341-5.
2. Hughes DA, Tunnage B, Yeo ST. Orphan drugs revisited: author's response. Q J Med 2006;99:350-1.
3. Sheldon T. Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs. BMJ 2012;345:e5461.
4. Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA-OMP). Key Conclusions and Recommendations. http://ec.europa.eu/enterprise/sec tors/healthcare/files/docs/orphans_conlusions_en.pdf (6 February 2014, date last accessed).
5. Working Group on Mechanism of Coordinated Access to Orphan Medicinal Products. Transparent Value Framework. http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/ orphans_framework_en.pdf (6 February 2014, date last accessed).
6. NICE to Assess High Cost Drugs for Rare Conditions. 2012;http://www.nice.org.uk/newsroom/news/NICEToAssessHigh CostDrugsForRareConditions.jsp (6 February 2014, date last accessed).
7. Hughes-Wilson W, Palma A, Schuurman A, Simoens S. Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis 2012;7:74.
8. Simoens S, Cassiman D, Dooms M, Picavet E. Orphan drugs for rare diseases: is it time to revisit their special market access status? Drugs 2012;72:1437-43.
9. Hyry HI, Roos JCP, Manuel J, Cox TM. The legal imperative for treating rare disorders. Orphanet J Rare Dis 2013;8:135.
10. Sandel MJ. Justice: What's the Right Thing to Do? London, UK: Penguin Books, 2010.
11. Smith A. Qualms about QALY. Lancet 1987;1:1134-6.
12. Harris J. It's not NICE to discriminate. J Med Ethics 2005;31:373-5.
13. Rawlins MD. NICE work-providing guidance to the British National Health Service. N Engl J Med 2004;351:1383-5.
14. Steinbrook R. Saying no isn't NICE-the travails of Britain's National Institute for Health and Clinical Excellence. N Engl J Med 2008;359:1977-81.
15. Neumann PJ. Using Cost-Effectiveness Analysis to Improve Health Care: Opportunities and Barriers. Oxford, UK: Oxford University Press, 2005:157-8.
16. Hawkes N. Why is the press so nasty to NICE? BMJ 2008;337:a1906.
17. Davies JE, Neidle S, Taylor DG. Developing and paying for medicines for orphan indications in oncology: utilitarian regulation vs equitable care? Br J Cancer 2012;106:14-7.
18. Schlander M. The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process. J Med Ethics 2008;34:534-9.
19. WHO. Disability Weights, Discounting and Age Weighting of DALYs. http://www.who.int/healthinfo/global_burden_ disease/daly_disability_weight/en/index.html (6 February 2014, date last accessed).
20. Chandra A, Jena AB, Skinner JS. The pragmatist's guide to comparative effectiveness research. J Econ Perspect 2011;25:27-46.
21. Department of Health. Strengthening National Commissioning Consultation-Summary of Responses. Richmond UK: Her Majesty's Stationery Office, 2010.
22. Walshe JM. Management of rare diseases. Q J Med 2006;99:123-4.
23. Sandel MJ. What Money Can't Buy: The Moral Limits of Markets. London, UK: Penguin Books, 2012:110-3, 144.
24. Williams B. Moral Luck. Cambridge: Cambridge University Press, 1981:4.
25. Scheffler S. Rawls and utilitarianism. In: Freeman S, ed. The Cambridge Companion to Rawls. Cambridge, UK: CUP, 2003:443.
26. Connock M, Burls A, Frew E, Fry-Smith A, Juarez-Garcia A, McCabe C, et al. The clinical effectiveness and costeffectiveness of enzyme replacement therapy for Gaucher's disease: a systematic review. Health Technol Assess 2006;10:iii-iv, ix-136.
27. Council Recommendation of 8 June 2009 on an Action in the Field of Rare Diseases (2009/C 151/02). http://eur-lex. europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0 007:0010:EN:PDF (6 February 2014, date last accessed).
28. Roos J, Hyry H, Cox TM. Orphan drug pricing may warrant a competition law investigation. BMJ 2010;341: c6471.