Brussels, 26/06/2006 (Agence Europe) - In a report published on Monday 26 June, the European Commission welcomed the five years of progress made in the application of a regulation on Orphan medicinal products. Between April 2000 and April 2005, the centralised authorisation procedure for marketing the European Agency for the Evaluation of Medicinal Products (EMEA) in London and the incentive programme, triggered more than 450 applications for orphan designation. Some 270 further medicines have already been designated as orphan medicinal products, but are still undergoing clinical tests.
Almost a million patents are suffering from a rare disease, in some cases, like Fabry disease, there are only a few thousand sufferers in the whole of the EU but have, in this way been able to receive treatment. Other medicines authorised involve the treatment of particularly rare forms of leukaemia, pulmonary arterial hypertension, gastro-intestinal tumours, acromegaly, Gaucher's disease (neuro-lipidiose), hyperammoniaemia, familial adenomatous polyposi or treatment of high-grade dysplasia in Barrett's Esophagus. There are two main reasons for establishing an orphan medicinal product: either the condition affects less than 5 in 10,000 persons in the EU (“prevalence criterion”) or it is unlikely that the marketing of the medicinal product would generate sufficient return (“insufficient return on investment criterion”).
The regulation has also had a positive economic impact. A significant number of start-ups have been created and many existing companies have begun research on rare diseases. All the companies surveyed have increased their total number of employees between 2000 and 2004 (43% increase on average). Moreover, R&D expenditure on rare diseases has grown faster than general medicinal R&D investment (more than two-fold on average). Regarding the “innovativeness”, the orphan initiative thus appears to have already made a positive impact on research into rare diseases. An EMEA review underlines, that novel/innovative products constitute 53% of all applications for orphan designation. Approximately 21% of the products that have been the subject of a designation application over the same period are biotechnology products (e.g. monoclonal antibodies or recombinant enzymes) and emerging therapies such as anti-sense, gene therapy and cell therapy. This figure is expected to further increase as more and more innovative biotech products are presented for orphan designation. The five year report and more information: (http: //ec.europa.eu/enterprise/pharmaceuticals/orphanmp/index.htm).