Rare Disease Blogs

No doubt, the EU Cross-Border Health Care Directive is a new important legislation to improve quality and access to health services for patients!
This policy lays down the basis of the future organisation of healthcare services in the European Union and grant new rights to patients to take advantage of being European citizens. When we will look back, five or ten years from now, this legislation will also appear as a building block for rare diseases. However be aware that the limit of its full impact for people living with rare diseases may emerge from the potential gaps between the principles set forth in this EU legislation and the national legislations to transpose it in Member states.

While the Directive applies broadly to health and all diseases in general, it recognise rare diseases for its specificities (rarity of expertise, rarity of patients, limited knowledge and therapeutic interventions) requiring specific measures to organise and disseminate this expertise at the European level – through the European Reference Network – and to support easier patient mobility across EU to access quality diagnosis and care, when needed. These new avenues are extremely important for the coming years and for the long term.
In addition, Member States have now an official role in orientating patients to adequate services. When wanting to go in another Member States, in particular to access diagnostic expertise, this access could not be anymore denied and at minimum the Member States will have to justify why. These provisions are shifting the rights to the patient side, creating better transparency in the case by case decision making process for access to and reimbursement of health services – even if turning this policy into a day to day reality for patients will take years. Also, this create a new situation which will put pressure on the Member States, hence inviting them to engage them more into taking measures to raise the level of their health services at national and regional levels where the patients live and want to be treated.

After the EU Regulations on Orphan Drugs (1999), on Paediatric Use of Medicines (2006), on Advanced Therapies (2007) and more recently the Commission Communication (2008) and the Council Recommendation on Rare Diseases (2009), this EU Directive on Cross Border Health Care consacres rare diseases as a public health priority in its Article 13. In itself, this is a higher recognition for people living with rare diseases: an EU Directive is at the highest legislative level, right below an EU Regulation but right above a Council Recommendation, hence it is more binding for Member States.

Last six years, EURORDIS has navigated through and contributed regularly all along the very long and complex decision making process of this EU Directive. Two past versions have been rejected by the Council mostly because some Member States were afraid that this legislation will stimulate an important flow of patients seeking better or quicker diagnosis and care in another Member States at risk of higher costs for the healthcare payers in their country of origin. This latest version of the Directive has been negotiated carefully, through high level political procedures involving the Commission, the European Parliament and the Council.
We can reassure you, that in this process we have been involved regularly and appropriately, so that the voice of people living with rare diseases has beeen heard. Some time advocacy requires speaking with a strong voice and media attention, in case like this one, it requires fine jewellery art for which the raw material is legitimacy and credibility. When needed, we worked closely with the relevant national rare disease alliances and that was a very rewarding experience.
As always, it is not our “dream text”, hardly good, but a realistic compromise for new gains. The European Parliament adopted it on January 19. The Council should adopt it in April.
Through that long process with several failures, EURORDIS and all supporting policy makers have taken advantage the political situation to make surface the specific case of rare diseases which concentrate all the questions of the added value of a European approach to the organisation of scarce expertise to provide high level health services requiring mobility both of expertise and patients.

However, a the next challenge is already ahead of us: patients and families living with rare diseases will really take advantage of its full potential only if all stakeholders and policy makers are taking a firm grasp on the national policy process during the upcoming 30 months for the transposition in the national legislation, health policy and organisation. And this is when this Directive requires a good understanding and appropriate actions by the national alliances, patient groups, medical experts, hospital policy makers and managers, health care budget policy makers and managers. EURORDIS will support that process and we will engage the EU Committee of Experts on Rare Diseases as well as the future Joint Actions on Rare Diseases, Joint Action on HTA and (possibly) Joint Action on Cross Border Health Care so to make concrete and detailed recommendations on how to transpose this Directive in national legislations and policies as well as how to monitor its implementation to measure its impact.