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Commission takes measures to help rare disease patients

A new EU regulation on 'Orphan medicinal products', has been adopted by the European Commission. It will allow pharmaceutical companies to apply to the European Agency for the Evaluation of Medicinal products (EMEA) to designate candidate medicines as 'orphan medicinal product...
A new EU regulation on 'Orphan medicinal products', has been adopted by the European Commission. It will allow pharmaceutical companies to apply to the European Agency for the Evaluation of Medicinal products (EMEA) to designate candidate medicines as 'orphan medicinal products'.

Rare diseases are classified as those affecting fewer than 5 patients in every 10,000 of the EU population. Companies are normally reluctant to develop 'orphan drugs' for these diseases because of their limited market. The new regulation aims to combat this problem by providing pharmaceutical companies with incentives to invest in orphan drugs.

'These medicines could benefit patients not only in Europe but also in developing countries, as diseases such as malaria or sleeping sickness could also be eligible,' says the Commission.

The regulation sets out a series of incentives, including market exclusivity and the possibility for fee exemptions for marketing authorisation activities. Although it came into force in January, the measures could not be implemented until the Commission adopted the necessary definitions and implementing criteria.

The regulation also creates the Committee for Orphan Medicinal Products - the first European scientific committee where patients' representatives participate fully in decisions on whether a potential medicine meets the 5 in 10,000 criterion, or is unlikely to be profitable enough to justify the investment needed. The committee held its first meeting on 17 April 2000.

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