2,535
Views
13
CrossRef citations to date
0
Altmetric
Research Report

Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe

, &

References

  • Hutchings A, Schey C, Dutton R, et al. Estimating the budget impact of orphan drugs in Sweden and France 2013-2020. Orphanet J Rare Dis 2014;9:22
  • Schlander M, Beck M. Expensive drugs for rare disorders: to treat or not to treat? The case of enzyme replacement therapy for mucopolysaccharidosis VI. Curr Med Res Opin 2009;25:1285-93
  • Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis 2011;6:42
  • McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: should we value rarity? Br Med J 2005;331:1016-19
  • Williams S. The five most expensive drugs in the world. The Motley Fool. 2013. Available from: www.fool.com/investing/general/2013/06/29/the-5-most-expensive-drugs-in-the-world.aspx [Last accessed 21 June 2014]
  • Mauskopf JA, Sullivan SD, Annemans L, et al. Principles of good practice for budget impact analysis: report of the ISPOR Task Force on good research practices--budget impact analysis. Value Health 2007;10(5):336-47
  • Schey C, Milanova T, Hutchings A. Estimating the budget impact of orphan medicines in Europe: 2010 - 2020. Orphanet J Rare Dis 2011;6:62
  • Kobayashi K, DeLab RJ. FDA review practices and priorities for drugs used in cancer treatment. Ann N Y Acad Sci 2001;949:341-4
  • Orphanet. Available from: www.orpha.net/ [Last accessed on 20 December 2013]
  • Orphanet Report Series. Prevalence of rare diseases: bibliographic data. 2012. Available from: www.orpha.net
  • EvaluatePharma. Orphan Drug Report. 2013
  • Seoane-Vazquez E, Rodriguez-Monguio R, Szeinbach SL, Visaria J. Incentives for orphan drug research and development in the United States. Orphanet J Rare Dis 2008;3:33
  • EU Competition Commission Pharmaceutical Sector Inquiry, Fact Sheet 1. Prices, time to generic entry and consumer savings. Available from: http://ec.europa.eu/competition/sectors/pharmaceuticals/inquiry/fact_sheet_1.pdf [Last accessed on 19 December 2013]
  • Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nature Rev Drug Dev 2010;9(12):921-9
  • Orfali M, Feldman L, Bhattachariee V, et al. Raising orphans: how clinical development programs of drugs for rare and common diseases are different. Clin Pharmacol Ther 2012;92(2):262-4
  • IMS Health Market Prognosis, June 2013. Available from: www.imshealth.com/deployedfiles/imshealth/Global/Content/Corporate/Press%20Room/Total_World_Pharma_Market_Topline_metrics_2012-17_regions.pdf [Last accessed on 30 January 2014]
  • OECD. Health at a glance: Europe 2012, OECD Publishing. 2012. Available from: http://dx.doi.org/10.1787/9789264183896-en [Last accessed on 20 December 2013]
  • Sullivan SD, Mauskopf JA, Augustovski F, et al. Budget impact analysis—principles of good practice: report of the ISPOR. 2012 Budget Impact Analysis Good Practice II Task Force. Value Health 2014;17(1):5-14
  • European Commission. Guideline on aspects of the application of Article 8(1) and (3) of Regulation (EC) No 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivity. Official Journal of the European Union. 2008
  • Adang EM. Economic evaluation of innovative technologies in health care should include a short-run perspective. Eur J Health Econ 2008;9(4):381-4
  • A.D.A.M. Editorial Board. Familial lipoprotein lipase deficiency. In: Dugdale DC, Zieve D, editors. A.D.A.M. Medical Encyclopedia (National Center for Biotechnology Information). 2013
  • Jacobsohn DA, Vogelsang GB. Acute graft versus host disease. Orphanet J Rare Dis 2007;2:35
  • Genetics Home Reference. National Center for Biotechnology Information, Bethesda, Maryland, Available from: http://ghr.nlm.nih.gov/ [ Last accessed on 19 December 2013]
  • Lobo C, Angulo IL, Aparicio LR, RELATH Investigators. Retrospective epidemiological study of Latin American patients with transfusional hemosiderosis: the first Latin American epidemiological study in iron overload--the RELATH study. Hematology (Am Soc Hematol Educ Program) 2011;16(5):265-73
  • Zimmerhackl LB, Besbas N, Jungraithmayr T, European Study Group for Haemolytic Uraemic Syndromes and Related Disorders. Epidemiology, clinical presentation, and pathophysiology of atypical and recurrent hemolytic uremic syndrome. Semin Thromb Hemost 2006;32(2):113-20
  • Cystic Fibrosis Foundation. Cystic fibrosis foundation patient registry: annual data report 2011. Bethesda, MD: 2011
  • Health Grades. Homozygous familial hypercholesterolemia. Available from: www.rightdiagnosis.com/h/homozygous_familial_hypercholesterolemia/basics.htm [Last accessed on 20 December 2013]
  • Hoffman HM, Yasothan U, Kirkpatrick P. Fresh from the pipeline: rilonacept. Nat Rev Drug Discov 2008;7(5):385-6
  • Coelho T, Ericzon BG, Falk R, et al. A physician’s guide to transthyretin amyloidosis. 2008. Available from: www.amyloidosis.org/pdf/TTR%2008.pdf [Last accessed on 20 December 2013]
  • Centers for Disease Control and Prevention. Anthrax (2009). Available from: www.cdc.gov/nczved/divisions/dfbmd/diseases/anthrax/technical.html [Last accessed on 20 December 2013]
  • Fraser D. Hypophosphatasia. Am J Med 1957;22(5):730-46
  • Berentsen S, Ulvestad E, Langholm R, et al. Primary chronic cold agglutinin disease: a population based clinical study of 86 patients. Haematologica 2006;91(4):460-6
  • Organ Procurement and Transplantation Network. Available from: http://optn.transplant.hrsa.gov/ [Last accessed on 3 January 2014]
  • United States Renal Data System. Annual data report: atlas of chronic kidney disease and end-stage renal disease in the United States. Bethesda, MD: National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases. 2011
  • World Health Organization. GKT1 Activity and Practices. Available from: www.who.int/transplantation/gkt/statistics/en/index.html [Last accessed on 20 December 2013]
  • Orens JB, Garrity ERJr. General overview of lung transplantation and review of organ allocation. Proc Am Thorac Soc 2009;6(1):13-19
  • Hoofnagle JH, Carithers RLJr, Shapiro C, Ascher N. Fulminant hepatic failure: summary of a workshop. Hepatology 1995;21(1):240-52
  • Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA 1999;20;281(3):249-54

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.