The Rare Disease Puzzle: Putting the Pieces Together
The European Conference on Rare Diseases and Orphan Products (ECRD 2014 Berlin)
PRESS RELEASE â€“ Berlin, 9 May, 2014
The 7th European Conference on Rare Diseases and Orphan Products (ECRD 2014 Berlin) opens today at Andelâ€™s Hotel in Berlin with over 750 participants from more than 40 countries in attendance. The two-day biennial event is Europeâ€™s foremost gathering of the rare disease community, offering a unique platform across all rare diseases and across all European countries to bring together all stakeholders â€“ patients, caregivers and patientsâ€™ representatives; academics, scientists, regulators, health care professionals, industry, policy makers, payers and representatives of the Member States
The Rare Disease Puzzle: Bringing the picture to life
Over the past two decades, different pieces of the complex puzzle that constitutes the needs of the rare disease community have been identified: research, information, treatment, diagnostics, expert care, social services, and the policies that will ensure these elements are developed, implemented and sustained in healthcare schemes. We now face the challenge of assembling all these pieces to create a complete, comprehensive picture.
ECRD 2014 Berlin comes at a key moment. Six overarching themes: Improving Healthcare Services; Knowledge Generation and Dissemination; Research; State-of-the-Art & Innovative Practices in Orphan Products; Emerging Concepts & Future Policies for Rare Disease Therapies; and Beyond Medical Care offer 36 exciting sessions - each representing a crucial piece of the rare disease puzzle â€“ which will drive the momentum geared toward bringing people with rare diseases the resources and healthcare they are entitled to receive.
Annette Widmann-Mauz, Parliamentary State Secretary, Federal Ministry of Health, Germany observes: â€œBefore we get a clear picture of a rare disease, there is a long and laborious process of trying, researching and learning. Therefore, networking and pooling of knowledge and potential is even more important. Through the ECRD 2014 Berlin conference, together we take a few steps further on the road to improving the quality of life and prospects for people living with a rare disease.â€
â€œThis is a pivotal moment,â€ states EURORDIS Chief Executive Officer Yann Le Cam, â€œAs regulatory and policy frameworks developed over the last years need now to be implemented thoroughly to address the needs of peopleliving with rare diseases in Europe. As more countries develop and implement dedicated strategies for rare diseases, as European-level agencies prioritise rare diseases in their funding strategies, as stakeholders work together to develop the resources these strategies embody, including the Centres of Expertise and European Reference Networks for rare diseases, as industry puts more rare disease treatments into the developmental pipeline, we are focusing on initiatives to ensure that patients can actually access rare disease treatments as they become available. Dedicated sessions on Progressive Patient Access Schemes, Patient Involvement in Benefit-Risk Assessment, Mechanisms of Coordinated Access and Transparent Value Framework, Managed Entry Agreements and other emerging ideas for sustaining access to orphan medicines are amongst the many, many highlights ECRD 2014 Berlin offersâ€.
Amongst the distinguished panel of the conference plenary Chairs and Speakers are Annette Widmann-Mauz, Parliamentary State Secretary, Federal Ministry of Health, Germany; Greet Musch, Director General of Pre-authorisation - Federal Agency for Medicinal and Health Products Belgium; John F. Ryan, Director of Public Health at the European Commissionâ€™s DG Health and Consumers; Stefan Schreck, Head of the Health Information Unit at DG Health and Consumers, European Commission; Irene Norstedt, Head of Unit, Personalised Medicine, DG Research, European Commission; Paul Lasko, Chair of the International Rare Disease Research Consortium (IRDiRC); and Jordi Llinares Garcia of the European Medicines Agency.
With simultaneous interpretation of key sessions into French, Spanish, German, Polish and Russian, including the Plenary Session, ECRD fosters a true international exchange of experiences and ideas.200 posters will be displayed on-site and dedicated poster sessions are planned, along with plenty of networking opportunities. A free App for iPhones and Android offers full access to the conference programme, the posters, and the means for participants to connect.
In the European Union, any disease affecting fewer than 5 people in 10 000 is considered rare. Rare diseases are chronically debilitating and frequently life-threatening. Over 6000 rare diseases have been identified, of which a majority affect children. Because information is scarce, research is insufficient, and resources are scattered, people living with a rare disease face common challenges such as a lack of diagnostics, information, expertise, medicines, treatments, and social services as well as economic challenges, psychological burden, and a lack of practical support in everyday life. There are 30 million people living with a rare disease in Europe.
The European Organisation for Rare Diseases (EURORDIS) represents 624 rare disease organisations in 58 countries, covering more than 4,000 rare diseases. EURORDIS is the voice of the estimated 30 million patients living with a rare disease throughout Europe and beyond.
EURORDIS is a non-governmental patient-driven alliance of patient organisations and individuals active in the field of rare diseases dedicated to improving the quality of life of all people living with rare diseases in Europe. It is supported by its members and by the French Muscular Dystrophy Association (AFM), the European Commission, corporate foundations and the health industry. EURORDIS was founded in 1997. More information on: www.eurordis.org