Rare is frequent and frequent is costly: rare diseases as a challenge for health care systems

European Journal of Health Economics

Volumn 16, Issue 2, 2015, Pages 113-118

  Graf von der Schulenburg, J Matthias ^a   Frank, Martin ^a  

^a LEIBNIZ UNIVERSITY HANNOVER   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

ECONOMIC ASPECT; EDITORIAL; HEALTH CARE; HEALTH CARE COST; HEALTH CARE SYSTEM; HUMAN; PRIORITY JOURNAL; RARE DISEASE; DRUG INDUSTRY; ECONOMICS; EUROPE; HEALTH CARE DELIVERY; HEALTH CARE PLANNING; ORGANIZATION AND MANAGEMENT; PATIENT PREFERENCE; RARE DISEASES;

DELIVERY OF HEALTH CARE; DRUG INDUSTRY; EUROPE; HEALTH PLANNING; HUMANS; PATIENT PREFERENCE; RARE DISEASES;

EID: 84925521817     PISSN: 16187598     EISSN: 16187601     Source Type: Journal    
DOI: 10.1007/s10198-014-0639-8     Document Type: Editorial

References (25)

1. Orphanet: about rare diseases. http://www.orpha.net/consor/cgi-bin/Education_AboutRareDiseases.php?lng=EN (2014). Accessed 2 May 2014
2. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. http://ec.europa.eu/health/files/eudralex/vol-1/reg_2000_141_cons-2009-07/reg_2000_141_cons-2009-07_en.pdf (2009). Accessed 2 May 2014
3. Orphanet: What is an orphan drug? http://www.orpha.net/consor/cgi-bin/Education_AboutOrphanDrugs.php?lng=EN&stapage=ST_EDUCATION_EDUCATION_ABOUTORPHANDRUGS (2014). Accessed 2 May 2014
4. Kaplan, W., Wirtz, V., Mantel-Teeuwisse, A., Stolk, P., Duthey, B., Laing, R.: Priority medicines for Europe and the World 2013 update. World Health Organization, Department of Essential Drugs and Medicines Policy. http://ec.europa.eu/enterprise/sectors/healthcare/files/docs/prioritymedicines_report_en.pdf (2013). Accessed 2 May 2014
5. Eidt, D., Frank, M., Reimann, A., Wagner, T.O.F., Mittendorf, T., Schulenburg, J.-M.: Maßnahmen zur Verbesserung der gesundheitlichen Situation von Menschen mit Seltenen Erkrankungen in Deutschland. A study for the German Federal Ministry of Health. http://www.gesundheitsforschung-bmbf.de/_media/Autorentexte_Massnahmen-seltene-Krankheiten_200908.pdf (2009). Accessed 2 May 2014
6. Musgrave, R.A.: The voluntary exchange theory of public economy. Q J Econ 53, 213–237 (1939)
7. Eidt, D., Mittendorf, T., Wagner, T.O.F., Reimann, A., Schulenburg, J.-M.: Cost analysis for ambulatory treatment of cystic fibrosis in Germany. Overview of the prospective study results. Med Klin 104, 529–535 (2009)
8. Eidt-Koch, D., Wagner, T.O.F., Mittendorf, T., Schulenburg, J.-M.: Outpatient medication costs of patients with cystic fibrosis in Germany. Appl Health Econ Health Policy 8, 11–118 (2010)
9. Article 12 of Directive 2011/24/EU of the European Parliament and of the Council of 16 March 2011 on the application of patientsʼ rights in cross-border healthcare: criteria of European Reference Networks (ERN) http://ec.europa.eu/health/cross_border_care/docs/cons_implementation_ern_concept_paper_en.pdf (2011). Accessed 2 May 2014
10. Van Dussen, L., Biegstraaten, M., Hollak, C.E., Dijkgraaf, M.G.: Cost-effectiveness of enzyme replacement therapy for type 1 Gaucher disease. Orphanet J Rare Dis 9, 51 (2014)
11. Rombach, S.M., Hollak, C.E.M., Linthorst, G.E., Dijkgraaf, M.G.W.: Cost-effectiveness of enzyme replacement therapy for Fabry disease. Orphanet J Rare Dis 8, 29 (2013)
12. Hutchings, A., Schey, C., Dutton, R., Achana, F., Antonov, K.: Estimating the budget impact of orphan drugs in Sweden and France 2013–2020. Orphanet J Rare Dis 9, 22 (2014)
13. McCabe, C., Claxton, K., Tsuchiya, A.: Orphan drugs and the NHS: should we value rarity? Br Med J 331, 1016–1019 (2005)
14. Drummond, M.F., Wilson, D.A., Kanavos, P., Ubel, P., Rovira, J.: Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care 23, 36–42 (2007)
15. European commission: register of designated orphan medicinal products http://ec.europa.eu/health/documents/community-register/html/alforphreg.htm (2014). Accessed 2 May 2014
16. Cutler, D.M.: Are we finally winning the war on cancer? J Econ Perspect 22, 3–26 (2008)
17. Glaeske, G., Höffken, K., Ludwig, W.-D., Schrappe, M., Weißbach, L., Wille, E.: Sicherstellung einer effizienten Arzneimittelversorgung in der Onkologie. A study for the German Federal Ministry of Health. http://www.bmg.bund.de/fileadmin/dateien/Downloads/O/Onkologie/Gutachten_Sicherstellung_einer_effizienten_Arzneimittelversorgung_in_der__Onkologie.pdf (2010). Accessed 2 May 2014
18. US Orphan Drug Act: http://www.fda.gov/regulatoryinformation/legislation/federalfooddrugandcosmeticactfdcact/significantamendmentstothefdcact/orphandrugact/default.htm (2014). Accessed 2 May 2014
19. Côte, A., Keating, B.: What is wrong with orphan drug policies? Value Health 15, 1185–1191 (2012)
20. Drummond, M.F., Towse, A.: Orphan drug policies: a suitable case for treatment. Eur J Health Econ 15, 335–340 (2014)
21. National action league for people with rare diseases: http://www.namse.de/english.html. Accessed 2 May 2014
22. National plan of action for people with rare diseases: action fields, recommendations, proposed actions. http://www.namse.de/images/stories/Dokumente/Aktionsplan/national%20plan%20of%20action.pdf. Accessed 2 May 2014
23. Zentrales Informationsportal über seltene Erkrankungen: http://www.portal-se.de/startseite.html. Accessed 2 May 2014
24. se-atlas—Kartierung von Versorgungsleistungen für Menschen mit Seltenen Erkrankungen: https://www.se-atlas.de/startseite/. Accessed 2 May 2014
25. BURQOL RD: http://www.burqol-rd.com/. Accessed 2 May 2014