EURORDIS advocating to improve patient access to orphan drugs in Europe Rare disease patients do not have equitable and timely access to the approved orphan drugs they need. This is often because the national authorities in their countries take too long to decide on the pricing and reimbursement of these drugs, thus delaying their arrival on the market. EURORDIS advocates for immediate action to promote cooperation at the European level in order to facilitate national decisions on pricing and reimbursement, thus reducing these delays and improving patients’ effective access to orphan drugs. This advocacy action is taken to implement the policy framework adopted at EU level in the last 12 months and is based on a broad consultation of concerned stakeholders.
Rare Disease Day 2010: Coming up! With only three months to go, the organisation of the third edition of Rare Disease Day is well on track. This year the rare disease community will drum up around the motto “Patients & Researchers: Partners for Life”. Thousands of patient organisations will be organising activities on and around 28th February to raise awareness of rare diseases and the impact that they have on patients’ lives. Read on to find out what’s happening and how you can get involved too!
Bridging Patients and Researchers: The case of DEBRA International How can patients help advance research into their disease? One answer comes from a young woman suffering from EB (Epidermolysis Bullosa), who recently accepted to be among the first people to try a new skin-cell therapy that has the potential to greatly improve EB patients’ quality of life. Read on to find out how Melissa Smith, and the patient organisation to which she belongs, is helping to translate painstaking research into treatment.
Noonan Syndrome Angels: An Italian patient organisation is fighting for them Antonella Esposito, the President of the Italian Association of Noonan Syndrome patients, shares her experience as a multi-tasking woman: psychotherapist, militant and mother of three children, one with Noonan Syndrome.
Swine flu (influenza H1N1) and rare diseases A considerable amount of information about swine flu is currently featured in the media and on the internet and ranges from the denial of any risk to absolute alarmism. We therefore consider it useful for Eurordis to publish a message that is independent of both national policies and commercial interests.
Many rare diseases carry risk factors with regard to flu. It is therefore important that patients and the persons who frequently provide daily care can benefit from suitable protection measures and, if necessary, effective early treatment.
Patient associations have shown for years that they form an independent channel of validated scientific information consulted by a broad public.
The report gives the 2008 highlights of Eurordis’ work in the areas of networking & communications; access to information, diagnosis, treatment & care; advocacy & policy development; and therapeutic development & research, among other topics.