Issues surrounding orphan disease and orphan drug policies in Europe

  title={Issues surrounding orphan disease and orphan drug policies in Europe},
  author={Alain Denis and Lut Mergaert and Christel Fostier and Irina Cleemput and Steven R A Simoens},
  journal={Applied Health Economics and Health Policy},
An orphan disease is a disease with a very low prevalence. Although there are 5000–7000 orphan diseases, only 50 orphan drugs (i.e. drugs developed to treat orphan diseases) were marketed in the EU by the end of 2008. In 2000, the EU implemented policies specifically designed to stimulate the development of orphan drugs. While decisions on orphan designation and the marketing authorization of orphan drugs are made at the EU level, decisions on drug reimbursement are made at the member state… 

Orphan Drugs for Rare Diseases

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Expenditure trends of orphan drugs in Serbia: 8-Year analysis of orphan drug market in Serbia

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  • 2012
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Spending on orphan drugs in five European countries was acceptable in 2007 in terms of the percentage of these countries’ overall drug expenditure, with the mean annual costs per patient varied widely, with costs being related to the prevalence of the disease for which the product is indicated.

Orphan drugs revisited.

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    The Canadian journal of clinical pharmacology = Journal canadien de pharmacologie clinique
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A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: the Bosentan Patient Registry

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