We are breaking the isolation and despair of children and adults amongst the most vulnerable citizens in our society today.
“Join Together for Better Care” is the precise and strong common message launched by patient groups and stakeholders from 83 countries and regions on Rare Disease Day 2014!
In hundreds of cities, thousands of patient groups and hundreds of thousands of volunteers all mobilised to successfully generate massive media attention and policy-makers engagement.
From Rare Disease Day 2014, focusing on partnering to improve care for all, six key messages have emerged to shape our future actions:
1/ Now is the right time to launch “The International Voice of Rare Disease Patients” embedded in the Rare Diseases International network.
By bringing together all 6000 different rare diseases across Europe, EURORDIS – Rare Diseases Europe has created a critical mass of 30 million people with rare diseases who can no longer be ignored. We help identify and address the common issues patients and their families face. We call for public attention and social justice. We help shape a favourable environment to generate positive actions.
Most of the patients groups throughout the world we invited to answer our survey responded with enthusiasm; and 96% of the 100+ respondents are willing to join in this international initiative. By bringing together rare disease national alliances, rare disease specific international federations or networks and isolated patient groups from six continents through the Rare Disease Day annual awareness campaign and the RareConnect multi-language platform of online patient communities at global level, EURORDIS already forges de facto an international movement for rare diseases.
This movement will be enhanced with the upcoming launch of EURORDIS – Rare Diseases International, the network of rare disease patient advocates, to build our capacities through information and experience exchange and through collaboration, in order to speak with one voice at the global level, on behalf of an even greater critical mass of millions of People with Rare Diseases.
Our determination is as strong as our long-term ambition to turn rare diseases into a public health priority at national, European and global levels.
The National Organization for Rare Disorders (NORD) motto changed from its pioneering times: “Out of the Darkness into light” to “Alone we are Rare. Together we are Strong” while the EURORDIS motto is “The Voice of Rare Disease Patients in Europe”. All three of these slogans are valid to describe the rise of rare disease patients around the world from ignorance to recognition, from isolation to unity, from silence to voice.
As the situation of rare disease patients improves over the next years and decades, we cannot create fortresses of access to care from which the poorest patients in rich countries would be excluded, nor from which patients from emerging countries or developing countries would remain excluded. The life of one person has the same value as the life of any other – in any part of the world. Solidarity does not end at our borders. Solidarity should not be merely a concept displayed on the screen of our computers when making information available worldwide, while real care is inaccessible to many, as if it were a precious object in the locked window of a jeweller’s.
2/ The European approach for a comprehensive, integrated and long term strategy stimulates similar approaches in more countries.
In Europe, the 2008 Commission Communication Rare Diseases: Europe’s Challenges followed by the 2009 Council Recommendation on an action in the field of rare diseases, and the 2011 Directive on Patients’ Rights to Cross-Border Healthcare completed the 1999 EU Regulation on Orphan Medicinal Products, establishing a comprehensive and integrated EU strategy for research, diagnostics, treatment and care.
This structured strategic approach to the specific research and public health challenges of rare diseases is inspiring other regions in the world: Canada, Brazil, Mexico, Russia, Australia, New Zealand are amongst countries joining those who are already engaged: the USA, Taiwan, Japan, Singapore, and South Korea.
3/ The year 2014 is a turning point to initiate and deepen more structured actions for the next 10 years.
EU Member States’ plans or strategies on rare diseases will be consolidated while being increasingly integrated into European common approaches, with European Reference Networks and European Research Infrastructures providing new opportunities for translating important scientific breakthroughs into innovative treatments, and advances in information technology into knowledge generation. All of these efforts are aimed at better care. The two EU Programmes Health for Growth (2014-2020) and Horizon 2020 are expected to support long term actions to reach these objectives, while the Innovative Medicines Initiatives – IMI 2 will provide new opportunities for public-private partnership in pre-competitive areas for the health industry. At the same time, the International Rare Diseases Research Consortium (IRDiRC) is federating the strategies of all major funders of rare disease research across the world with the aim to create diagnostic tools for most rare diseases and 200 new approved rare disease therapies by 2020.
4/ Medical Care and Social Care need to be brought closer.
Each rare disease affecting patients is complex and requires multidisciplinary high level medical and paramedical competences. Each Person Living with a Rare Disease has both medical and social challenges to face. Patients need to be placed at the centre of a holistic approach. People with Rare Diseases are not a kaleidoscope with sharply-cut facets in different colours: the geometry of fragmented public policies separating prevention and primary care from hospital care; medical diagnostics from social diagnostics; medical care from social support and services; social services and support from compensation for incapacities; with layers of responsibilities between local, regional and national authorities. This is simply non-sense: Non-human sense! Non-Policy efficiency sense! Non-Cost Effectiveness sense! Care is much more than a diagnostic tool or an innovative treatment. Care comprises all these different aspects to put patients at the centre with respect and kindness; to empower patients to master their life and engage to the maximum of their possible decisions and actions.
5/ The financial and economic crisis is not a reason to stop acting in favour of People with Rare Diseases; to the contrary!
In tough times, the principle of social justice calls to increase action in support of the most vulnerable members of our society, including the elderly, people affected by neurodegenerative disorders, people with learning and behavioural disorders, poor and isolated people, and people living with rare diseases.
There are numerous policies and actions which do not cost much in terms of new resources at regional and national levels and yet which can make a huge difference. Quality information and orientation, engaging therapeutic education, efficient organisation of multidisciplinary centres of expertise, good practices of diagnostics and care, mutualisation of expertise at national / European / International levels, and much more. The economic challenges oblige all stakeholders to be smarter than ever in the optimal use of rare resources. These low-cost actions need to be performed first and permanently. Rare diseases, at the margin of mainstream public health or healthcare systems, offer a goldmine of innovation and new paradigms transferable to all health policies and sectors.
Solidarity and creativity in caring about each other contribute to making our society a better place to live for everybody.
6/ Overall, and this may be the main message to take from this Rare Disease Day 2014 dedicated to “Care”: These goals can be achieved only through empowered patient advocates partnering with all stakeholders and policy makers, reaching out across society at large, to defend the core values in which our civil society actions are rooted. Rare Disease Day is an essential action which simultaneously offers the aim, the method and the tools toward achieving empowered rare disease patient groups around the world!