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Orphan medicinal products

Patients suffering from rare diseases deserve the same quality of treatment as other patients within the European Union.

Given the small numbers of patients affected by rare diseases, the pharmaceutical industry has been reluctant in the past to invest in the research and development of medicinal products to treat them.

The EU introduced new legislation in 2000 with the aim of providing incentives for the development of medicines for rare diseases (so-called orphan medicinal products).

Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union.

To date, the European Commission has already authorised 174 orphan medicines for the benefit of patients suffering from rare diseases. The sponsors responsible for these medicines benefit from incentives such as fee waivers for the regulatory procedures and a 10 year market exclusivity.

Equally important, the European Commission has designated 1684 products as orphan medicinal products (this number refers to active orphan designations, not withdrawn or expired). The sponsors developing these products benefit from incentives such as protocol assistance. This assistance should facilitate the development and authorisation of innovative medicines for the benefit of the patients.

 

Read the whole article here: https://ec.europa.eu/health/human-use/orphan-medicines_en