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Links from PubMed

Items: 16

1.

The many vs. the few.

Loewenstein G.

Hastings Cent Rep. 2012 Sep-Oct;42(5):7-8; author reply 8-9. No abstract available.

PMID:
23050293
2.

[Shift of focus in the financing of Hungarian drugs. Reimbursement for orphan drugs for treating rare diseases: financing of enzyme replacement therapy in Hungary].

Szegedi M, Molnár MJ, Boncz I, Kosztolányi G.

Orv Hetil. 2014 Nov 2;155(44):1735-41. doi: 10.1556/OH.2014.30031. Review. Hungarian.

PMID:
25344850
3.

Commissioning for rare diseases: view from the frontline.

Burls A, Austin D, Moore D.

BMJ. 2005 Oct 29;331(7523):1019-21. Review. No abstract available.

4.

Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Tambuyzer E.

Nat Rev Drug Discov. 2010 Dec;9(12):921-9. doi: 10.1038/nrd3275. Epub 2010 Nov 9. Review.

PMID:
21060315
5.

[Authorization and reimbursement of orphan drugs in an international comparison].

Roll K, Stargardt T, Schreyögg J.

Gesundheitswesen. 2011 Aug;73(8-9):504-14. doi: 10.1055/s-0030-1262864. Epub 2010 Sep 16. Review. German.

PMID:
20848380
6.

[European incentives for orphan medicinal products].

Enzmann H, Lütz J.

Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2008 May;51(5):500-8. doi: 10.1007/s00103-008-0523-8. Review. German.

PMID:
18696141
7.

Economic considerations in the provision of treatments for rare diseases.

McCabe C, Edlin R, Round J.

Adv Exp Med Biol. 2010;686:211-22. doi: 10.1007/978-90-481-9485-8_13. Review.

PMID:
20824448
8.

Fair pricing of "old" orphan drugs: considerations for Canada's orphan drug policy.

Roberts EA, Herder M, Hollis A.

CMAJ. 2015 Apr 7;187(6):422-5. doi: 10.1503/cmaj.140308. Epub 2015 Feb 23. Review. No abstract available.

9.

[Orphan drugs : New opportunities for the treatment of rare diseases].

Beck M.

Internist (Berl). 2016 Nov;57(11):1132-1138. Review. German.

PMID:
27527064
10.

Proposed 'grant-and-access' program with price caps could stimulate development of drugs for very rare diseases.

Valverde AM, Reed SD, Schulman KA.

Health Aff (Millwood). 2012 Nov;31(11):2528-35. doi: 10.1377/hlthaff.2012.0235. Review.

PMID:
23129684
11.

[Rare diseases and their patient organization: the Hungarian Federation of People with Rare and Congenital Diseases].

Pogány G.

Orv Hetil. 2014 Mar 2;155(9):329-33. doi: 10.1556/OH.2014.29819. Review. Hungarian.

PMID:
24566696
12.

The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.

Kumar Kakkar A, Dahiya N.

Drug Dev Res. 2014 Jun;75(4):231-4. doi: 10.1002/ddr.21176. Epub 2014 May 14. Review.

PMID:
24829189
13.

Financing drug discovery for orphan diseases.

Fagnan DE, Gromatzky AA, Stein RM, Fernandez JM, Lo AW.

Drug Discov Today. 2014 May;19(5):533-8. doi: 10.1016/j.drudis.2013.11.009. Epub 2013 Nov 20. Review.

14.

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
15.

[High cost drugs for rare diseases in Brazil: the case of lysosomal storage disorders].

de Souza MV, Krug BC, Picon PD, Schwartz IV.

Cien Saude Colet. 2010 Nov;15 Suppl 3:3443-54. Review. Portuguese.

16.

Orphan drugs and the NHS: should we value rarity?

McCabe C, Claxton K, Tsuchiya A.

BMJ. 2005 Oct 29;331(7523):1016-9. Review. No abstract available.

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