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Public Health (31-08-2006)

22 new orphan drugs receive marketing authorisation in five years

More than 1 million patients suffering from life-threatening or chronically debilitating rare diseases in the EU could benefit from new treatments, thanks to European legislation introduced in 2000 that provides incentives for pharmaceutical companies to invest and research into rare illnesses.
A Commission Report into the impact of the Regulation on Orphan Medicinal Products six years on, reveals that since 2000, 22 new orphan medicines for the treatment of 20 different uncommon diseases have received marketing authorisation. In addition, some 270 further medicines have already been designated as orphan medicinal products, but are still undergoing clinical tests.
This Regulation (No 141/2000) sets out the criteria for orphan designation in the EU and describes the incentives to encourage research into them.
Orphan drugs are medicinal products intended for the diagnosis, prevention or treatment of life-threatening or very serious diseases. These drugs are called "orphans" because the pharmaceutical industry has little interest, under normal market conditions, in developing and marketing products intended for only a small number of patients suffering from very rare conditions.
For five years the European Commission, the European Agency for the Evaluation of Medicinal Products (EMEA) and Member States have provided incentives to the pharmaceutical industry for the research, development and marketing of such orphan medicinal products in the fields of cancer, metabolic disorders, immunology, and cardiovascular and respiratory disorders. It is unlikely that such medication would have been developed under normal market conditions.
For more information:
European Commission report on the impact of Regulation 141/2000:
European Commission web pages on rare diseases: