The European Medicines Agency welcomes the 1000th orphan designation for a
medicine intended for use in patients with a rare disease, issued today.
Orphan designations are issued by the European Commission on the
recommendation of the Agency's Committee for Orphan
Medicinal Products (COMP). They allow companies
developing medicines intended for use in small numbers of patients in the
European Union (EU) to benefit from a range of incentives. These include
assistance with development, reduced fees for regulatory procedures and
protection from competition once the medicine is on the market.
These measures were introduced in 1999 in order to stimulate the
development of diseases that might otherwise be neglected by the pharmaceutical
industry.
About 30 million people living in the EU suffer from a rare disease,
defined as a life-threatening or chronically debilitating condition that
affects no more than 5 in 10,000 people in EU. This is equivalent to around
250,000 people or less for each disease.
To date, the 1000 orphan designations have led to the authorisation of 70 orphan
medicines for use in patients across the EU.
The Agency maintains a searchable list of opinions on applications
for rare disease (orphan) designations and
information on how to apply for orphan
designation.
The full list of orphan designations granted by the European Commission is
available in the Community
register of orphan medicinal products for human use
.
.