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EURORDIS Rare Disease Day 2014 Policy Event seeks to Improve Access to Rare Disease Care

March 2014

EURORDIS Rare Disease Day 2014 Policy EventOn the occasion of Rare Disease Day 2014 and in line with this year’s theme of Care, EURORDIS hosted a Policy Event in Brussels on 25 February 2014 that explored the over-arching topic of Improving Access to Rare Disease Care: The Vision of Patients.

As 2014 marks the 15th anniversary of the adoption of the European Regulation on Orphan Medicinal Products, the EURORDIS Policy Event focused on 15 key questions relating to improving care for people with rare diseases. An innovative format presented the Vision of Patients on three key topics followed by discussions based on the questions presented to panels of stakeholders of patient representatives, national authorities, European policy makers, researchers, health professionals, and industry.

The first topic: Empowering People with Rare Diseases & Their Families began with the Vision of Patients presented by Avril Daly (Fighting Blindness Ireland), who evoked the hope people with rare diseases feel that the Who … what … where and when” questions patients experience will become increasingly clear in the near future and that patients and families will be better able to navigate the “perfect storm” of their rare disease diagnosis. Discussions centred on how new information & communication technologies (ICT) are empowering patients and families – as well as health professionals, researchers and the entire rare disease community – to better understand and manage rare diseases. The Specialised Social Services and social policies, as well as the training of professionals and patients, are another key area for empowering patients and families, improving autonomy and quality of life. View the Panel Discussion on Topic 1

The Vision of Patients presented by Nick Sireau (AKU Society) for the second topic: Addressing Unmet Medical Needs emphasised the need for a collaborative system that identifies different groups to work together with a focus on patients’ needs, and less bureaucracy, with more straightforward funding mechanisms at all levels. In discussions, an emphasis was put on treating rare diseases earlier – before irreversible symptoms appear, whenever possible. Early dialogue between payers, Health Technology Assessment bodies, industry, the EMA – and involving patients – is needed to improve access, which is more driven today by financial issues than by marketing authorisation. View the Panel Discussion on Topic 2

The third topic: Improving Patients’ Access to Quality Care and Innovative Treatments emphasised the pivotal role of the European Reference Networks (ERNs) for improving access to quality care. Much work has been done to make these structures optimal – now it is crucial that adequate funding is made available. Cost-effectiveness studies are also necessary to determine the most efficient ways forward. Common, interoperable tools for ERNs are also vital. Henk-Willem Laan (Marshall-Smith Syndrome Research Foundation), who presented the Vision of Patients, underlined the point that patients and families need one established place for care – rather than going from hospital to hospital in search of solutions and care. This would truly be cost effective. View the Panel Discussion on Topic 3

Rare Disease Day 2014 has the slogan Join Together for Better Care and that is exactly what the EURORDIS Rare Disease Day 2014 Brussels Policy Event allowed participants to do: join forces as a diverse group of stakeholders, coming together to find ways to improve access to the care rare disease patients and families are entitled to receive. EURORDIS thanks all who took part in this event and urges eNews readers to take the time to watch the videotaped discussions

Louise Taylor, Communications and Development Writer, EURORDIS