The regulation of orphan medicines in the EU: Objectives reached and main challenges when facing the future

Pharmaceuticals Policy and Law

Volumn 9, Issue 3-4, 2007, Pages 327-342

  Westermark, Kerstin ^a,b  

^a EUROPEAN MEDICINES AGENCY   (United Kingdom)

^b MEDICAL PRODUCTS AGENCY   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85013580697     PISSN: 13892827     EISSN: 2210495X     Source Type: Journal    
DOI: None     Document Type: Article

References (18)

1. M. Haffner, Adopting Orphan Drugs-Two Dozen Years of Treating Rare Diseases, N ENGL J MED 354 (2006), 445-447.
2. C. Ashbury, The Orphan Drug Act, The First 7 Years, JAMA 265 (1991), 893-897.
3. J. Torrent-Farnell and R. Morros, The EU challenges in the designation of orphan medicinal products, Pharmaceuticals Policy and LAW 3 (2001), 19-30.
4. M. Drummond, D. Wilson, P. Kanavos, P. Ubel and J. Rovira, Assessing the economic challenges posed by orphan drugs, Int J Tecnol Assess Health Care 23 (2007), 36-42.
5. General information-European Commission website on orphan medicinal products: http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/.
6. The EU legislation on orphan drug designation (Regulation (EC) No 141/2000): http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/141 2000/141 2000en.pdf.
7. Commission Regulation (EC) N0 847/2000): http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/rev1/en1.pdf.
8. The register of orphan medicinal products: http://ec.europa.eu/enterprise/pharmaceuticals/register/index.htm.
9. General report on the experience acquired with the application of Regulation (EC)No 141/2000 on orphan medicinal products during the first five years of publication: http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/orphan en 06-2006.pdf.
10. Inventory of Community and member States’ incentive measures to aid research, marketing, development and availability of orphan medicinal products. Revision 5: http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/inventory 2006 08.pdf.
11. EU Community Research Programmes: http://cordis.europa.eu/fp7/cooperation/health en.html.
12. EMEA website on orphan drug applications: www.emea.europa.eu/htms/human/comp/orphapp.htm
13. Guideline on format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to the other: www.emea.europa.eu/pdfs/human/comp/628300en.pdf.
14. Points to consider on the calculation and reporting of the prevalence of a condition for orphan designation: www.emea.europa.eu/pdfs/human/comp/043601.pdf.
15. Guideline on the elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation: www.emea.europa.eu/pdfs/human/comp/6697204en.pdf.
16. Guideline on clinical trials in small populations: www.emea.europa.eu/pdfs/human/chmp/ewp/8356105en.pdf.
17. EMEA SME office website: www.emea.europa.eu/SME/SMEoverview.htm.
18. Network for Health Technology Assessment (HTA): www.eunethta.org.