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
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 more than 200 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has designated around 2000 products as orphan medicinal products (this number refers to active orphan designations, not withdrawn or expired).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.
Latest orphan medicinal products
Latest orphan designations
EU action on orphan medicinal products
The EU Regulation on orphan medicinal products establishes a centralised procedure for the designation of orphan medicinal products and puts in place incentives for their research, development and marketing.
Pharmaceutical companies can benefit from incentives such as fee waivers, scientific assistance for marketing authorisation and the possibility of an EU marketing authorisation with a 10-year market exclusivity period.
The Committee for Orphan Medicinal Products (COMP) in the European Medicines Agency, is responsible for the scientific examination of applications for approval of orphan medicinal products.
Review of EU orphan legislation
Impact of EU orphan regulation
Since 2000, the European Commission has published a number of reports on orphan medicinal products. They reveal an annual increase in the number of orphan medicinal products being authorised, since the Regulation entered into force.
- Inventory of EU and national incentives to support research and development:
- 2006 report on the application of EU Orphan Regulation
Guidelines on EU orphan medicines legislation
- EU Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another 2022/C 440/02
- Commission Regulation (EC) No 847/2000 laying down implementing rules and definitions for the application of the Regulation
- Commission Notice on the application of Articles 3, 5 and 7 of the Orphan Regulation (replacing the 2003 Commission Communication)
- Article 8(2) of Regulation No 141/2000
- Article 8(1) and 8(3) of Regulation No 141/2000