Medicinal products for human use
Orphan medicinal products
Orphan Medicinal Products
Latest Orphan Medicinal Products
Latest Orphan Designations
Patients suffering from rare diseases deserve access to the same quality of medicinal products as other patients within the European Union.
Since only a very small number of the population is affected by these diseases, the pharmaceutical industry has been reluctant in the past to invest in the research and development of medicinal products to treat them.
In response to this situation and in order to stimulate the research and development of orphan drugs, in 2000 the EU introduced new legislation with the aim of providing incentives for the development of orphan and other medicinal products for rare disorders.
The EU Regulation on orphan medicinal products (Regulation (EC) No 141/2000) establishes a centralised procedure for the designation of orphan medicinal products and puts in place incentives for the research, marketing and development of orphan medicinal products.
Today companies with an orphan designation for a medicinal product benefit from incentives such as fee waivers, a 10 year market exclusivity period post authorisation for designated products; scientific assistance for marketing authorisations, and the possibility of a Community marketing authorisation.
The Regulation also sets up a Committee for Orphan Medicinal Products (COMP), which is responsible for the scientific examination of applications leading to the designation of an Orphan Medicinal Product.
Since 2000, the Commission published a number of reports on orphan medicinal products:
- Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products - state of play 2015:
- Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products - state of play 2006.
- Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products - state of play 2002.
- 2006 report on the experience acquired as a result of the application of the new EU Orphan Regulation, taking account of the public health benefits acquired.
The reports revealed that the number of orphan medicinal products authorised has increased each year since the entry into force of the Regulation and is expected to increase in the years to come.
The complete listing of orphan medicinal products adopted in the EU can be found in the Community Register.
More detailed rules and guidelines are provided in:
- Commission Regulation (EC) No 847/2000 laying down implementing rules and setting out definitions essential for the application of the Regulation on orphan medicinal products.
- Commission Notice on the application of Articles 3, 5 and 7 of the Orphan Regulation (replacing the 2003 Commission Communication).
There are also a number of guidelines further specifying various aspects of EU orphan medicines legislation, in particular:
- The application of Article 8(2) of Regulation No 141/2000.
- The application of Article 8(1) and 8(3) of Regulation No 141/2000.
- 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 (March 2014).