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Drugs for rare diseases:
contrasting results in Europe

The EU Regulation on "orphan drugs" signals progress, but with strong limitations.

Pharmaceutical companies do not spontaneously develop drugs for rare diseases, deemed unprofitable. In 2000, the European Union adopted a Regulation designed to encourage the marketing of these so-called "orphan drugs," aimed at patients suffering from rare diseases.

Between April 2000 and April 2005, the EU status of "orphan drug" was granted to 268 substances, while 22 marketing authorisations were issued.

In only 5 of these 22 cases was there no existing treatment available. The clinical evaluation of these drugs is hampered by the small number of patients involved. However, some of the clinical dossiers are of decent quality, even if in other cases they could and should have been better (comparison with a drug already available for the same indication, evaluation according to clinical rather than laboratory criteria, etc.). In practice, there was not always evidence of a therapeutic advantage.

Access to such drugs, which are often extremely expensive (up to 300,000 euros a year), varies from one European country to another and is very unequal.

Overall, while there are more and better-evaluated drugs now available for patients who previously had no other alternative, too many of these drugs target the same diseases, are insufficiently evaluated, or are beyond the reach of patients in many countries.

©Prescrire November 2006

Source: "Médicaments pour des maladies rares : bilan contrasté en Europe" Rev Prescrire 2006 ; 26 (277) : 780-787.

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