Monday, July 03, 2006

European Union gives boost to orphan drugs development

EU incentives for R&D on orphan drugs have proven successful, 22 new drugs have reached the market in five years. Patient organisations argue they cannot afford them.

More than 450 applications for orphan designation have been submitted between April 2000 and April 2005 and, of those, more than 260 have been designated and 22 have gone on to receive a marketing authorisation," concludes a Commission report on the impact of the first five years of the EU's orphan medicine regulation.

According to the Commission, the response to the orphan legislation, which provides for the pharma companies a 10-year market exclusivity for the orphan medicinal products, reduction of EMEA fees and support for R&D, has "far exceeded initial expectations".

Biotech industry has welcomed the report acknowledging the progress made, but states that "timely and equitable access for patients to the approved medicines remains an issue". It also claims that "additional economic incentives in most member states are still needed as some member states are delaying reimbursement of orphan drugs, thus limiting patient access to these drugs".

Eurodis, the umbrella patient organisation for rare disease patients in Europe, states that, often, when the products are available, patients cannot afford them, which is "not only inequitable because patient access to orphan drugs depends on the country where they live, but it is also totally unethical".


EU official documents

Commission Staff working document: on the experience acquired as a result of the application of Regulation (EC) No 141/2000 on orphan medicinal products and account of the public health benefits obtained (Document on the basis of Article 10 of Regulation (EC) No 141/2000) (27 June 2006)
Commission Staff working document: Outcome of the consultation (3 June 2006)
Commission press release: Fighting rare diseases: 22 new orphan drugs in five years (26 June 2006) [FR] [DE]
Commission: Rare diseases
European Medicines Agency (EMEA)
CORDIS News: Orphan medicine legislation spurs on research, innovation and growth, claims report (27 June 2006)

Industry Federations

EuropaBio - European Biopharmaceutical enterprises joint press release: Orphan Medicines Regulation for rare diseases on the road to success (27 June 2006)


European Organisation for Rare Diseases (Eurodis): Improving patient access to orphan drugs in Europe (April 2006)