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April 2010

Concept paper: Improving Access to Orphan Drugs (11th ERTC Workshop)

It has been acknowledged over recent years that, while the EU Orphan Drugs Regulation 141/2000 has stimulated research and development of orphan medicinal products in the EU, equitable and timely access to approved Orphan Drugs for rare diseases patients remains an issue. As underlined by the final conclusions and recommendations on Pricing & Reimbursement of the EU High Level Pharmaceutical Forum, “Effective market access and utilisation vary strongly between and within Member States”.

To address this issue, several policy documents have recently called for an increased cooperation between EU level authorities and Member States in order to improve access to Orphan Drugs for people living with rare diseases.