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Rare disease patients, academic researchers, NGOs and industry united their voices in a call to EU governments to take action this Autumn to address the issues facing patients affected by rare diseases in Europe.
Patients, Academia & Industry
Call on EU Leaders to Improve Access to Orphan Medicines for All EU Patients
Paris, 17 October 2008 – Rare disease patients, academic researchers, NGOs and industry united their voices in a call to EU governments to take action this Autumn to address the issues facing patients affected by rare diseases in Europe. The need is urgent because, for some patients, time is short.
At a workshop hosted at the French National Assembly under the French EU Presidency, the European Platform for Patients’ Organisations, Science & Industry (EPPOSI) brought together a multi-stakeholder group of 150 European experts to discuss proposals for EU-wide action on the effective access to innovative medicines to treat rare diseases in the Union. The overwhelming conclusions were that EU leaders need to act on a series of concrete measures to address the shortfalls in effective diagnosis, treatment and care for rare disease patients in the EU.
EU policy initiatives such as the Commission Communication on Rare Diseases scheduled for adoption in early November 2008 will provide the ideal opportunity to act. Workshop attendees urged national governments to adopt the related Council Recommendation on Rare Diseases immediately – ideally at the Council of Ministers (Health) on 16 December 2008. In addition, they highlighted six key recommendations:
1. The creation of an expert Working Group to evaluate the clinical added value of each new rare disease treatment. A scientific European common assessment report on the clinical added value of each orphan medicine – as well as any other rare disease treatment – should not prolong the approval process but help Member States in their reimbursement decisions by pooling scarce available expertise about the clinical value of a new treatment. This Committee at the European Medicines Agency (EMEA) should comprise members of the Committee for Orphan Medicinal Products (COMP) and of the Committee for Human Products (CHMP) as well as Member State health technology assessment experts and patients’ representatives.
2. New treatments for rare diseases should be granted conditional pricing and reimbursement immediately following Marketing Authorisation on the basis of this clinical added value assessment. Evidence on clinical effectiveness should be gathered on an on-going basis about the real-life contribution of the treatment to the therapeutic strategy for the targeted disease in order to allow confirmation or revision of the decision to reimburse, based on a robust data-set.
3. Population screening, including new-born screening and “situational” screening should be explored at a European level, to improve early and accurate diagnosis. European guidelines should be developed on new-born and situational screening, advised by a multi-stakeholder EU advisory committee. More research should be done to improve the speed, quality and accuracy, and range of tests, which will allow parents and patients to have a firm diagnosis wherever possible.
4. Centres of Expertise and European Reference Networks for rare diseases need to be established and sustainably funded. It is not realistic to expect every physician to be able to recognise and treat each of the 5,000+ known rare diseases, many of which are highly complex and heterogeneous. The creation of centres of expertise and European network will allow the pooling of information and expertise. New technologies can facilitate this sharing and could help overcome some of the challenges posed by factors such as location, distance and language.
5. Treatment for rare diseases must be funded at a national level – not at the regional, local or hospital level – to avoid “penalising” physicians or hospitals that diagnose or care for rare disease patients.
6. The rare disease community should work together to increase awareness amongst health professionals – including during their education – as well as policy-makers and the public at large.
National governments already adopted a joint position on rare diseases at the EU’s High Level Pharmaceutical Forum on 2 October 2008 and the workshop participants strongly endorsed the Guiding Principles contained in the “Improving access to orphan medicines for all affected EU citizens.” However, they called on Member States to turn these principles into reality by adopting the European Commission’s upcoming series of measures proposed to address the shortfalls in effective diagnosis, treatment and care for rare disease patients in the EU.
The European Union unanimously approved legislation in 1999 to ensure that Rare Disease patients receive the same quality of treatment as other patients. But 10 years on, many patients in the EU are being denied correct diagnosis and treatment. This Autumn will provide the opportunity to correct this inequitable and unjust situation.
Closing the workshop, Roselyne Bachelot-Narquin, French Minister of Health, pledged France’s commitment to treating rare disease patients as a high priority for the French EU Presidency. She commented: “This Communication is a priority of the French EU Presidency in the area of health” and continued: “The European level is the appropriate level to address the disparities in access to treatment.”
Workshop participants welcomed the French government’s commitment and leadership and called on all EU national governments to grasp this rare opportunity to help rare disease patients, their families and their carers.
“Europe’s Rare Disease communities are not likely to have another such opportunity to focus political actions to address their needs for another 10 years,” said Yann Le Cam, head of the European rare disease patients’ organisation EURORDIS and co-chair of the EPPOSI workshop. “The eyes of the patients and their families, researchers, scientific community, treating physicians and medical community, as well as industry and other sponsors that have dedicated themselves to bringing treatments to market will be firmly fixed on our leaders during the next months. We urge them to take these recommendations into account.”