EURORDIS: Rare diseases are not yet recognised as an international public health priority

BRUSSELS, 18-3-2015 — /EuropaWire/ — The 22nd meeting of the EURORDIS Round Table of Companies recently took place in Brussels on the occasion of Rare Disease Day 2015 and focused on the theme ‘Rare Diseases: Going Global’.

This EURORDIS corporate-membership group meets twice a year to discuss topics relating to the development of and access to rare disease treatments. It brings together pharmaceutical, biotech and service companies whose work has an impact on people living with a rare disease and that share a common interest in rare diseases and orphan medicines development.

In the current climate, the development of innovative rare disease therapies is mostly global in nature due to the rarity of patients and experts, as well as to the fact that companies and investors have an increasingly international approach to the market.

Rare diseases are not yet recognised as an international public health priority. EURORDIS wants to change this in the next 5 to 10 years. In a world where more countries can provide better access to healthcare to at least part of their population, and where the Internet is changing how we access information and build communities, the serious situation that millions of people living with a rare disease find themselves in can be improved beyond OECD countries.

An international approach to research and health policy can make a major contribution to improving the lives of people living with a rare disease. This can be done by increasing access for a broader number of patients to information, an accurate diagnosis and quality comprehensive care, as well as by bringing them new, innovative therapies.

The meeting’s objective was to discuss the opportunities that ‘going international’ presents, as well as to look at possible approaches for a long-term, structured, progressive internationalisation that involves patients, patient organisations, researchers and industry.

Discussion focused on current strategic initiatives that have the potential to structure this internationalisation and how these initiatives can be synergised to optimise results. The following subjects were discussed:

• Awareness, which is currently achieved through Rare Disease Day, the annual international campaign aimed at raising awareness among policy makers, stakeholders and the public, initiated in 2008 and now involving over 80 countries from all continents.
• Patient communities such as RareConnect, a safe online space available in 5 languages where individuals affected by rare diseases can connect with each other and join conversations on their diseases or topics of common interest, initiated in 2010 and involving 40 countries.
• Research through groups such as the International Rare Diseases Research Consortium (IRDiRC), initiated in 2009-2010 and whose objectives are to have diagnostic tools available for most rare diseases and to help deliver 200 new rare disease therapies, both by 2020. Rare disease researchers have always collaborated internationally to ensure research excellence and the advancement of science, but in a fragmented way. IRDiRC creates a common policy framework, coordinates actions between funding bodies and organises international collaborations in critical areas.
• Visibility of rare diseases in healthcare systems through ongoing actions including: the incorporation of rare diseases into the revision of the international disease codification system ICD 11; the promotion and broader dissemination of Orpha code; and the indexation of Orpha code with codification systems other than ICD, such as SNOMED. Read more about codification of rare diseases.
• Multi-stakeholder policy conferences including: the European Conference on Rare Diseases & Orphan Products (ECRD) organised by EURORDIS and partners, which attracts a large number of participants beyond Europe; NORD’s Rare Diseases and Orphan Products Breakthrough Summit, which takes place in the US; and the International Conference on Rare Diseases & Orphan Drugs (ICORD), which promotes rare disease policy in new regions of the world.
• Regulatory agencies that are accelerating their framework of collaboration mostly between the EU, the US, Canada and Japan. During the meeting the need to strengthen collaboration in order to streamline regulatory requirements in support of global clinical trials was identified as a priority.
• Pharma and biotech companies that are not yet collaborating on an international level. Meeting participants established that this collaboration is now needed and identified potential pre-competitive initiatives that could be put into place to create a more favourable policy environment and partnership. Companies were also encouraged to join IRDiRC during the meeting.
• Patient organisations, which need to join together in an international network to enhance their capacities and to speak with one global voice, presented the EURORDIS initiative Rare Diseases International, which has been jointly founded with NORD-USA, CORD-Canada, JPA-Japan, CORD-China, IORD-India, ALIBER-Ibero-America and other groups.

Durhane Wong-Rieger, President of the Canadian Organization for Rare Disorders, attended the meeting and commented, “EURORDIS has a great track record of working internationally with different cultures and languages, inspiring beyond Europe. We need to make sure that patients around the world feel equally represented and that they have access to a real global community, not just through existing online tools but also through their own local communities. We need to think from the start about how to develop medicines from a global perspective, taking into account all developing countries.”

Stayed tuned for news on the launch of the new EURORDIS initiative Rare Diseases International.

Eva Bearryman, Junior Communications Manager, EURORDIS

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EURORDIS: Rare diseases are not yet recognised as an international public health priority