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Erschienen in: Applied Health Economics and Health Policy 1/2013

01.02.2013 | Editorial

Cost-Effectiveness Assessment of Orphan Drugs

A Scientific and Political Conundrum

verfasst von: Steven Simoens, Eline Picavet, Marc Dooms, David Cassiman, Thomas Morel

Erschienen in: Applied Health Economics and Health Policy | Ausgabe 1/2013

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Excerpt

In September 2012, the Dutch Healthcare Insurance Board issued advice not to reimburse orphan drugs that target lysosomal storage disorders: agalsidase alfa and agalsidase beta for Fabry disease and alglucosidase alfa for Pompe disease [1, 2]. …
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Zurück zum Zitat Gericke CA, Riesberg A, Busse R. Ethical issues in funding orphan drug research and development. J Med Ethics. 2005;31(3):164–8.PubMedCrossRef Gericke CA, Riesberg A, Busse R. Ethical issues in funding orphan drug research and development. J Med Ethics. 2005;31(3):164–8.PubMedCrossRef
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Zurück zum Zitat European Commission. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Off J Eur Commun. 2000; L18(1): 1–5. European Commission. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Off J Eur Commun. 2000; L18(1): 1–5.
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Zurück zum Zitat Simoens S, Dooms M. Market access of orphan drugs: one size fits all? Hosp Pharm Eur. 2012;62:59–63. Simoens S, Dooms M. Market access of orphan drugs: one size fits all? Hosp Pharm Eur. 2012;62:59–63.
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Zurück zum Zitat Hughes-Wilson W, Palma A, Schuurman A, et al. Paying for the orphan drug system: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7(1):74.PubMedCrossRef Hughes-Wilson W, Palma A, Schuurman A, et al. Paying for the orphan drug system: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7(1):74.PubMedCrossRef
Metadaten
Titel
Cost-Effectiveness Assessment of Orphan Drugs
A Scientific and Political Conundrum
verfasst von
Steven Simoens
Eline Picavet
Marc Dooms
David Cassiman
Thomas Morel
Publikationsdatum
01.02.2013
Verlag
Springer International Publishing AG
Erschienen in
Applied Health Economics and Health Policy / Ausgabe 1/2013
Print ISSN: 1175-5652
Elektronische ISSN: 1179-1896
DOI
https://doi.org/10.1007/s40258-012-0004-y

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