5.15.6. References

ALCIMED (2004): Study on Orphan Drugs. Published 16/11/04. Available at:

http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/ (Last access 18.04.2008).

Clarke JT (2006): Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy. CMAJ. 2006 Jan 17;174(2):189-90.

Dear JW, Lilitkarntakul P, Webb DJ (2006): Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products. Br J Clin Pharmacol 2006, 62:3, 264-271.

Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J (2007): Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007 Winter;23(1):36-42

European Commission (2006): Inventory of Community and Member States’ Incentive Measures to Aid the Research, Marketing, Development and Availability of Orphan Medicinal Products, Revision 2005. Available at: http://ec.europa.eu/enterprise/pharmaceuticals/orphanmp/doc/inventory_2006_08.pdf, (accessed 12.05.2007).

European Commission (2007): Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004. Available at: http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2007:324:0121:01:EN:HTML (accessed 18.04.2008)

European networks of reference for rare diseases in Europe. Available At:

http://ec.europa.eu/health/ph_threats/non_com/rare_8_en.htm (accessed 12.05.2007)

Eurordis (2005): Rare diseases: understanding this public health priority”. Available at:

http://www.eurordis.org/article.php3?id_article=918 (accessed 12.05.2007)

Haffner ME (2006): Adopting orphan drugs -- two dozen years of treating rare diseases. N Engl J Med. 2006 Feb 2;354(5):445-7.

Holzman NA (1978): Rare diseases, common problems: recognition and management. Pediatrics, 1978;62(6):1056-1060.

Kesselman I, Elstein D, Israeli A, Chertkoff R, Zimran A (2006): National health budgets for expensive orphan drugs: Gaucher disease in Israel as a model. Blood Cells Mol Dis. 2006 Jul-Aug;37(1):46-9. Epub 2006 Jul 7.

McCabe C, Claxton K, Tsuchiya A (2005): Orphan drugs and the NHS: should we value rarity? BMJ. 2005 Oct 29;331(7523):1016-9.

McKusick VA (2007): Mendelian Inheritance in Man and its online version, OMIM. Am J Hum Genet, 80: 588-604, 2007

NORD database of rare diseases. Available at: http://www.rarediseases.org/search/rdblist.html (accessed 08.05.2007)

Orphanet database of rare diseases and orphan drugs. Available at: http://www.orpha.net

Van Weely S, Leufkens HGM (2004): Orphan Diseases in “Priority Medicines for Europe and the World: a public health approach to innovation”. Available at: http://mednet3.who.int/prioritymeds/report/index.htm (accessed 15.05.2007)

Wastfelt M, Fadeel B, Henter JI. A journey of hope: lessons learned from studies on rare diseases and orphan drugs. J Intern Med. 2006; 260(1):1-10.