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).

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

 Evaluation of the orphan regulation
 Impact Assessment

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:
 State of play 2015 & Commission Staff Working Document
 State of play 2006
 State of play 2002
 2006 report on the application of EU Orphan Regulation

Guidelines on EU orphan medicines legislation

 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