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."
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.
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:
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.
European Medicines Agency: http://www.ema.europa.eu/ema/
International Rare Disease Research Consortium: