PubMed

No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products--a pilot study.

Stolk P, Heemstra HE, Leufkens HG, Bloechl-Daum B, Heerdink ER.

Orphanet J Rare Dis. 2009 Dec 14;4:27.PMID: 20003427 [PubMed - indexed for MEDLINE]Free PMC ArticleFree textRelated citations

Drugs for rare diseases: mixed assessment in Europe.

[No authors listed]

Prescrire Int. 2007 Feb;16(87):36-42.PMID: 17323539 [PubMed - indexed for MEDLINE]Related citations

Predictors of orphan drug approval in the European Union.

Heemstra HE, de Vrueh RL, van Weely S, Büller HA, Leufkens HG.

Eur J Clin Pharmacol. 2008 May;64(5):545-52. Epub 2008 Jan 22.PMID: 18210097 [PubMed - indexed for MEDLINE]Free PMC ArticleFree textRelated citations

Orphan drug development across Europe: bottlenecks and opportunities.

Heemstra HE, de Vrueh RL, van Weely S, Büller HA, Leufkens HG.

Drug Discov Today. 2008 Aug;13(15-16):670-6. Epub 2008 Jun 24. Review.PMID: 18583178 [PubMed - indexed for MEDLINE]Related citations

Drugs for exceptionally rare diseases: do they deserve special status for funding?

Hughes DA, Tunnage B, Yeo ST.

QJM. 2005 Nov;98(11):829-36. Epub 2005 Oct 3. Review.PMID: 16203824 [PubMed - indexed for MEDLINE]Free ArticleRelated citations

Safety-related regulatory actions for orphan drugs in the US and EU: a cohort study.

Heemstra HE, Giezen TJ, Mantel-Teeuwisse AK, de Vrueh RL, Leufkens HG.

Drug Saf. 2010 Feb 1;33(2):127-37. doi: 10.2165/11319870-000000000-00000.PMID: 20082539 [PubMed - indexed for MEDLINE]Related citations

[Hope for patients with rare diseases--"orphan" drugs]

Kuzelová M, Kubácková K, Palágyi M, Smíd M.

Cas Lek Cesk. 2006;145(4):296-300. Review. Slovak. PMID: 16639930 [PubMed - indexed for MEDLINE]Related citations

[European ordinance on orphan drugs: changes and threats]

Linthorst GE, Hollak CE.

Ned Tijdschr Geneeskd. 2003 Jan 25;147(4):143-5. Dutch. PMID: 12635543 [PubMed - indexed for MEDLINE]Related citations

Medicines for children licensed by the European Agency for the Evaluation of Medicinal Products.

Ceci A, Felisi M, Catapano M, Baiardi P, Cipollina L, Ravera S, Bagnulo S, Reggio S, Rondini G.

Eur J Clin Pharmacol. 2002 Nov;58(8):495-500. Epub 2002 Sep 25.PMID: 12451425 [PubMed - indexed for MEDLINE]Related citations

Developing treatments for inborn errors: incentives available to the clinician.

Haffner ME.

Mol Genet Metab. 2004 Apr;81 Suppl 1:S63-6.PMID: 15050976 [PubMed - indexed for MEDLINE]Related citations

[Orphan drugs in France (2001-2005): access to information and characteristics]

Baumevieille M, Daveluy A, Aulois-Griot M, Haramburu F.

Therapie. 2007 Jan-Feb;62(1):9-16. Epub 2007 Mar 21. French. PMID: 17374342 [PubMed - indexed for MEDLINE]Related citations

[Orphan drugs--medications for patients with rare diseases]

Thielke D, Thyssen JP, Hansen BJ.

Ugeskr Laeger. 2006 Jun 5;168(23):2236-8. Danish. PMID: 16768975 [PubMed - indexed for MEDLINE]Related citations

A cross-national comparison of orphan drug policies: implications for the U.S. Orphan Drug Act.

Thamer M, Brennan N, Semansky R.

J Health Polit Policy Law. 1998 Apr;23(2):265-90.PMID: 9565894 [PubMed - indexed for MEDLINE]Related citations

[European incentives for orphan medicinal products]

Enzmann H, Lütz J.

Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2008 May;51(5):500-8. Review. German. PMID: 18696141 [PubMed - indexed for MEDLINE]Related citations

EU to review rare disease drugs market exclusivity.

Sheridan C.

Nat Biotechnol. 2004 Sep;22(9):1061. No abstract available. PMID: 15340455 [PubMed - indexed for MEDLINE]Related citations

Rare essentials: drugs for rare diseases as essential medicines.

Stolk P, Willemen MJ, Leufkens HG.

Bull World Health Organ. 2006 Sep;84(9):745-51.PMID: 17128345 [PubMed - indexed for MEDLINE]Free PMC ArticleFree textRelated citations

[Orphan diseases and orphan medicines: a Belgian and European study]

Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S.

J Pharm Belg. 2009 Dec;(4):131-7. Review. French. PMID: 20183989 [PubMed - indexed for MEDLINE]Related citations

The US Orphan Drug Act: rare disease research stimulator or commercial opportunity?

Wellman-Labadie O, Zhou Y.

Health Policy. 2010 May;95(2-3):216-28. Epub 2009 Dec 29.PMID: 20036435 [PubMed - indexed for MEDLINE]Related citations

Therapeutic profile of orphan medicines.

de Barros CM, Papoila AL.

Pharmacoepidemiol Drug Saf. 2007 Apr;16(4):435-40.PMID: 16958155 [PubMed - indexed for MEDLINE]Related citations

Medicines for children licensed by the European Medicines Agency (EMEA): the balance after 10 years.

Ceci A, Felisi M, Baiardi P, Bonifazi F, Catapano M, Giaquinto C, Nicolosi A, Sturkenboom M, Neubert A, Wong I.

Eur J Clin Pharmacol. 2006 Nov;62(11):947-52. Epub 2006 Oct 5.PMID: 17021892 [PubMed - indexed for MEDLINE]Related citations