The EC Communication and Proposal for a Council Recommendation on Rare Diseases, which sets out an overall community strategy to diagnose, treat and care for the 30 million Europeans suffering from rare diseases, has inspired Member States like Ireland to accelerate the development of their own national plans for rare diseases.
As part of the events organised around Rare Disease Day, a conference took place on February 25th to highlight the need for a National Plan for rare diseases in Ireland. The objective of this event was to examine how the country can develop a successful national plan or framework to address rare diseases. 'The most important outcome on the day was the willingness on the part of the Irish Health Ministry to engage with stakeholders in this area to facilitate the development of a National Plan,' says Avril Daly, Chairman of the Genetic and Rare Disorders Organisation (GRDO).
Over 100 people from patient organisations, the industry, researchers and the Health Service Executive (HSE), met at the European Public Information Centre in Dublin to discuss ways in which they could turn ideas into reality. One of the guest speakers at the seminar was Christel Nourissier, Secretary General of EURORDIS. She spoke about National Plans in Europe and the lessons to be learnt from others further along the way, particularly France.
'There is certainly a great deal to learn from the first experience that took place in France from 2005 to 2008,' she argues. 'Obviously from its successes, particularly the designation and support of specialised centres; the development of the Orphanet database; of classification and coding for rare diseases; of a help line; availability of orphan drugs for patients; also funding for more research projects over longer periods. We can also learn from its failures: patient clinical data scattered or unavailable; no funding for databases; lack of training of professionals; no articulation between medical and social care, resulting in poor social services for patients; poor support to patient groups and poor monitoring of the French National Plan.'
Participants also heard about the national strategies currently being developed in Spain, Bulgaria, Hungary and Romania and about an EU-funded project called EuroPlan which aims at capitalising from these and other experiences. 'We can adapt models that have worked in other countries but also learn from mistakes made. Ultimately, Ireland's health system is unique and any National Plan must be tailored for us,' said Eibhlin Mulroe, CEO of the Irish Platform for Patients' Organisations, Science and Industry (IPPOSI).
The meeting in Dublin also highlighted the benefits for individual Member States of increased cooperation at the European level. 'In particular, it has raised awareness about the need for centres of expertise and European networks of reference, for the development of telemedicine, for disease management programmes and guidelines and for more consensus on screening,' explains Christel Nourissier. 'Clinicians and researchers need to network and use common tools: databases, and bio banks. These are all challenges that can be met only if all available expertise is gathered at EU level.'
'To better shape the future, the Rare Diseases Community is now waiting for the adoption of the Council Recommendation on Rare Diseases. This Recommendation, together with the Commission Communication on Rare Diseases adopted in November 2008, and its future EU Advisory Committee on Rare Diseases, the EuroPlan project, which capitalises on the experience of 24 Member States and the EURORDIS Council of 16 National Alliances on Rare Diseases will provide a general framework to promote and support the development of future National Plans,' said Yann Le Cam, CEO of EURORDIS.
After a successful Rare Disease Week, it appears that all stakeholders in Ireland are now ready to work together on a comprehensive and integrated strategy for rare diseases. Given their efforts it is reasonable to expect that the future National Plan will be based on the common principles and overarching values in Europe: access to high quality care, on the basis of equity and solidarity.
The seminar entitled “Focus on Rare Diseases in Ireland: What is the National Plan?” was a joint collaboration between the Genetic and Rare Disorders Organisation (GRDO), the Irish Platform for Patients' Organisations, Science and Industry (IPPOSI) and the Medical Research Charities Group (MRCG). It is the result of a long-standing series of events aimed at joining forces to create partnerships in Ireland. This process began at the EURORDIS Membership Meeting and Awareness Conference in Cork in 2004, and was followed by the annual IPPOSI Workshops and the Irish Medicines Board's Conference in 2008.
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This article was previously published in the May 2009 issue of our newsletter.
Author: Paloma Tejada
Photo credits: © GRDO