Market uptake of orphan drugs - A European analysis

Journal of Clinical Pharmacy and Therapeutics

Volumn 37, Issue 6, 2012, Pages 664-667

  Picavet, E ^a   Annemans, L ^b   Cleemput, I ^c   Cassiman, D ^d   Simoens, S ^a  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b GHENT UNIVERSITY   (Belgium)

^c BELGIAN HEALTH CARE KNOWLEDGE CENTRE KCE   (Belgium)

^d UNIVERSITY HOSPITALS LEUVEN   (Belgium)

Author keywords

market access; market uptake; orphan drugs

Indexed keywords

AMBRISENTAN; ARSENIC TRIOXIDE; AZACITIDINE; CLOFARABINE; DASATINIB; DEFERASIROX; EVEROLIMUS; LENALIDOMIDE; NELARABINE; NILOTINIB; NITISINONE; ORPHAN DRUG; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; SITAXSENTAN; SORAFENIB; SUNITINIB; TEMSIROLIMUS; TRABECTEDIN;

ARTICLE; BUDGET; COST EFFECTIVENESS ANALYSIS; DRUG COST; DRUG MARKETING; ECONOMIC ASPECT; EUROPE; GROSS NATIONAL PRODUCT; HEALTH CARE ACCESS; HEALTH TECHNOLOGY ASSESSMENT; HUMAN; REFERENCE DATABASE; REIMBURSEMENT;

BUDGETS; CLUSTER ANALYSIS; COMMERCE; COST-BENEFIT ANALYSIS; EUROPEAN UNION; GROSS DOMESTIC PRODUCT; HEALTH SERVICES ACCESSIBILITY; HEALTHCARE DISPARITIES; HUMANS; ORPHAN DRUG PRODUCTION; REIMBURSEMENT MECHANISMS; TECHNOLOGY ASSESSMENT, BIOMEDICAL;

EID: 84868378943     PISSN: 02694727     EISSN: 13652710     Source Type: Journal    
DOI: 10.1111/j.1365-2710.2012.01364.x     Document Type: Article

References (25)

1. European Commission. Regulation (EC) No 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products. Official J Eur Communities, 2000; L18: 1-5.
2. The European Parliament. Charter of fundamental rights of the European Union. Official J Eur Communities, 2000; 364: 1-22.
3. European Commission. Register of designated orphan medicinal products. 2012. Available at: http://ec.europa.eu/health/documents/community-register/ html/orphreg.htm (accessed 2 May 2012).
4. Schieppati A, Henter JI, Daina E, Aperia A,. Why rare diseases are an important medical and social issue. Lancet, 2008; 371: 2039-2041.
5. European Commission. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Official J Eur Communities, 2000; 18: 1-5.
6. Blankart CR, Stargardt T, Schreyogg J,. Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia. Pharmacoeconomics, 2011; 29: 63-82.
7. Kole A, Faurisson F,. Rare diseases social epidemiology: analysis of inequalities. Adv Exp Med Biol, 2010; 686: 223-250.
8. Eurordis. Eurordis survey on orphan drugs availability in Europe. 2007. Available at: http://www.eurordis.org/IMG/pdf/2007ODsurvey-eurordis.pdf (accessed 2 May 2012).
9. Orofino J, Soto J, Casado MA, Oyaguez I,. Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007. Appl Health Econ Health Policy, 2010; 8: 301-315.
10. Alcimed. Phase I overview of the conditions for marketing orphan drugs in Europe. 2010. Available at: http://ec.europa.eu/health/files/orphanmp/doc/ pricestudy/final-final-report-part-1-web-en.pdf
11. Kanavos P, Åmer S,. Providing access to modern treatments and influencing policy in orphan diseases: the international experience and evidence from the UK. The Londen School of Economics and Political Science, Report No: 20. 2005.
12. Heemstra HE,. Variations in access and use of orphan drugs among EU Member States. Eur J Hosp Pharm Pract, 2010; 16: 25-27.
13. Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J,. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care, 2007; 23: 36-42.
14. Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S,. A comparative study of European rare disease and orphan drug markets. Health Policy, 2010; 97: 173-179.
15. Belgian Health Care Knowledge Centre (KCE). Drug reimbursement systems: international comparison and policy recommendations. Report No.: 147C. 2010.
16. Simoens S, Dooms M,. How much is the life of a cancer patient worth? A pharmaco-economic perspective. J Clin Pharm Ther, 2011; 36: 249-256.
17. Executive Agency for Health and Consumers. Final CAVOD study report "Creation of process for the exchange of knowledge between Member States and European authorities on the scientific assessment of the clinical added value for orphan medicines". Report No.: EAHC/2010/Health/05. 2012.
18. Neumann PJ, Drummond MF, Jonsson B, Luce BR, Schwartz JS, Siebert U, Sullivan SD,. Are Key Principles for improved health technology assessment supported and used by health technology assessment organizations? Int J Technol Assess Health Care, 2010; 26: 71-78.
19. The Economist. The Economist Intelligence Unit. 2011. Available at: http://www.eiu.com/
20. XE. The World's Favorite Currency Site. 2011. Available at: http://www.xe.com/
21. U.S.Census Bureau. (2011) International Data Base. Available at: http://www.census.gov/ipc/www/idb/rank.php
22. Obradovic M, Mrhar A, Kos M,. Market uptake of biologic and small-molecule-targeted oncology drugs in Europe. Clin Ther, 2009; 31: 2940-2952.
23. Lettmeier B, Muhlberger N, Schwarzer R, Sroczynski G, Wright D, Zeuzem S, Siebert U,. Market uptake of new antiviral drugs for the treatment of hepatitis C. J Hepatol, 2008; 49: 528-536.
24. Denis A, Mergaert L, Fostier C, Cleemput I, Hulstaert F, Simoens S,. Critical assessment of belgian reimbursement dossiers of orphan drugs. Pharmacoeconomics, 2011; 29: 883-993.
25. Pharmaforum. Final conclusions and recommendations of the pharmaceutical forum-improving access to orphan medicines for all affected E.U. citizens. 2008.