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

01-02-2013 | Editorial

Cost-Effectiveness Assessment of Orphan Drugs

A Scientific and Political Conundrum

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

Published in: Applied Health Economics and Health Policy | Issue 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]. …
Literature
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Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6:42.PubMedCrossRef
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Sheldon T. Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs. BMJ. 2012;345:e5461.PubMedCrossRef
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Simoens S, Cassiman D, Dooms M, et al. Orphan drugs for rare diseases: is it time to revisit their special market access status? Drugs. 2012;72(11):1437–43.PubMedCrossRef
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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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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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Simoens S, Dooms M. Market access of orphan drugs: one size fits all? Hosp Pharm Eur. 2012;62:59–63.
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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
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Metadata
Title
Cost-Effectiveness Assessment of Orphan Drugs
A Scientific and Political Conundrum
Authors
Steven Simoens
Eline Picavet
Marc Dooms
David Cassiman
Thomas Morel
Publication date
01-02-2013
Publisher
Springer International Publishing AG
Published in
Applied Health Economics and Health Policy / Issue 1/2013
Print ISSN: 1175-5652
Electronic ISSN: 1179-1896
DOI
https://doi.org/10.1007/s40258-012-0004-y

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