EUROPEAN COMMISSION
PRESS RELEASE
Brussels, 7 June 2012
1,000th designation of an orphan medicinal product:
improving the lives of patients
Over 30 million European citizens suffer from a rare disease. The small numbers of
patients affected by a single disease and the fragmentation of knowledge about rare
diseases translates into difficulties for people to get the right diagnosis, the right medical
advise and the right medicine. In many cases, the medicine they need has not yet been
developed. Today's 1000th designation of an orphan medicinal product marks a
tremendous success in improving this situation.
Orphan medicines relate to diseases which affect not more than 5 persons in 10.000 in the
EU. As a consequence, the economic potential of such medicines is very limited which
hampers the development of new medicines.
In response, the EU adopted dedicated legislation in 1999 which provides strong incentives
for medicines which have been designated as orphan medicines. They can obtain, under
clearly defined conditions, market exclusivity of up to 10 years once they are authorised.
The record number reached today of 1000 designations demonstrates that the legal
framework for orphan product designations lives up to its potential: not only does it
support research, it also helps identify promising products and bolster their development
with the goal of market authorisation. Indeed, these 1000 designations have already led
to the authorisation of around 70 orphan medicinal products for the treatment of 62
orphan diseases.
European Commissioner in charge of Health and Consumer Policy, John Dalli, said: "I am
committed to helping people with rare diseases obtain appropriate and timely diagnosis,
information and care. The EU's legislation on orphan medicinal products has been a great
success in providing strong incentives for the development of medicinal products for the
diagnosis, prevention and treatment of rare diseases. These incentives are key to develop
medicines that can make a difference ; the difference between a life of suffering with no
adequate medication, or a better life with appropriate treatment."
Background:
1. What are rare diseases?
Rare diseases are life-threatening or chronically debilitating diseases of such a low
prevalence (affecting not more than 5 persons in 10 000 in the EU) that specific R&D
efforts are needed to address them. The diseases can have very different causes,
treatments, curability and expected evolution.
IP/12/573
2. What is the role of the Regulation on orphan medicinal
products?
The Regulation on Orphan Medicinal Products was adopted in 1999 and entered into force
in 2000. It addresses the need of incentives for the development and placing on the
market of medicinal products for the diagnosis, prevention or treatment of mainly rare
conditions. Without such incentives, it is unlikely that medicinal products would be
developed for rare diseases as the cost of developing and marketing products for these
disorders would not be recovered by sales. The incentives contained in the legislation aim
to assist sponsors receiving orphan designations in the development of medicinal products
with the ultimate goal of providing medicinal products for the patients.
3. What is the role of support for research and innovation ?
The EU support of research and innovation in the field of rare diseases is important. Rare
diseases research projects have been supported for more than two decades through the
European Community Framework Programmes for Research and Technological
Development.
The EU's investments on rare diseases-related collaborative research is expected to
exceed €430 million under the 7th Framework Programme in areas such as natural history,
pathophysiology and the development of preventive, diagnostic and therapeutic
interventions.
4. What is the role of the European Medicines Agency?
Prior to obtaining Commission’s designation as an orphan medicinal product, applicants
(e.g. a pharmaceutical company, a research centre or a university) must submit their
application file to the European Medicines Agency (EMA). The agency, through its
Committee for Orphan Medicinal Products, conducts a thorough scientific assessment of
the products, to determine whether they qualify as orphan medicines, meant to primarily
address rare diseases. The Committee’s opinions are subsequently sent to the
Commission, which takes the final decision about granting the designation and its related
incentives. The latter mainly consist in fee reductions or scientific advice from EMA, to help
successful applicants pursue their R&D efforts, in view of achieving market authorisation.
For more information:
Regulation on Orphan medicines designations:
http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF
List of orphan designations:
The full list of orphan designations granted by the European Commission as well as of the
orphan medicinal products which it has authorised is available in the Community register
of orphan medicinal products held by the European Commission.
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European Medicines Agency: http://www.ema.europa.eu/ema/
International Rare Disease Research Consortium:
http://ec.europa.eu/research/health/medical-research/rare-diseases/irdirc_en.html
Contacts :
Frédéric Vincent (+32 2 298 71 66)
Aikaterini Apostola (+32 2 298 76 24)
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