EURORDIS 20th Anniversary membership meeting in Budapest

Budapest, May 19-20, 2017

 

 

Organized by EURORDIS, the Voice of Rare Disease Patients in Europe, with the partnership of HUFERDIS, Rare Diseases Hungary

The European Voice of persons with a rare disease, EURORDIS, held its 20th Anniversary meeting in Budapest. Much has been achieved: The Orphan Medicine Regulation, EU Commission and Council Recommendations, National Plans for Rare Diseases, and the Directive on Patient’s Right to Cross- Border Healthcare.

 

 

 

 

Still over 80% of the patients and families with a rare disease report a serious impact of the rare disease in daily lives. Access to diagnosis and treatment, care near home and IT infrastructure to support co-management of patients and their families and reduce time spend in coordination of their disease.

 

The recent established 24 European Reference Networks gave much room for discussion during the meeting. How healthcare providers in lower economy countries and non-EU countries are connected to an ERN? How is the work between the different ERN’s harmonized?

 

How and which data are collected? Who is the governance the collected data regulated when patients are considered as the owner of their own data?

 

 

 

The process of appointing expert centres to participate in the ERNs seems accidental and not fully transparent in many EU countries, as in Hungary –  added Gábor Pogány, the head of Rare Diseases Hungary. Why is none of the Hungarian metabolic screening laboratories and metabolic paediatric clinics assigned to the MetabERN? 60.000 samples of new-borns are screened for 26 rare diseases at the 1st Department of paediatrics, Semmelweis University per year; covering more than 60% of all nationwide assessments. Resulting in approximately 60 children diagnosed with a rare condition each year, accumulating to hundreds of children taken care by the metabolic clinic at the same hospital.

Mónika Héczei, mom of a 1,5-year-old boy with cystic fibrosis, also the member of Hungarian Cystic Fibrosis Association comments: “I am disappointed, that none of the Hungarian pulmonology institution has chosen to be included in the Pulmonology European Reference Network. I really hope this will change soon, as we have a very well equipped Cystic Fibrosis Centre in the National Korányi Institute of TB and Pulmonology.”

 

As it turns out, currently, only two Hungarian paediatric departments are assigned to any of the national 24 ERNs. Since most rare diseases present in childhood, and some of these children do not even live up to adulthood, it can be questioned how the selection procedure of expert centres will reach out equity to children in all EU countries.

 

All children have a right to the enjoyment of the highest attainable standard of health and to facilities for the treatment of illness and rehabilitation of health (the Convention on Rights of the Child, adopted by the United Nations). European Academy of Paediatrics endorses the principles of chronic care management for children and will support their harmonization and implementation.

 

These findings underline EAP’s motion to consider patient safety, especially for the vulnerable disabled children in Europe. The EAP would like to urge the European Commission to spread more information about the transparent, harmonized and evidence based criteria on how member states nominate Health Care Providers for different European Reference Networks.

http://eapaediatrics.eu/wp-content/uploads/2015/12/EAP-Statement-11-12-2011-.pdf

Reported by the observers:

Liesbeth Siderius, coordinator Rare Disease WG European Academy Paediatrics

Peter Altorjai, Association of Hungarian Primary Care Paediatricians, Budapest

 

Special acknowledgements to Lenneke Schrier for managing the official EAP Twitter channel.

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