Edition of 07 February 2020

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Editorial

EURORDIS Black Pearl Awards 2020

The announcement of the recipients of the EURORDIS Black Pearl Awards was made on 11 December 2019. The EURORDIS awards will be presented during a gala dinner on 18 February in Brussels. The awardees are patient advocates, scientists, policy makers, organisations, companies and media, distinguished for their achievements and outstanding commitment in improving the life of people living with rare diseases. Hundreds of nominations from the rare disease community were received for this edition. The awardees include Jana Popova from Bulgaria (Young Patient Advocate Award), Dr. Vytenis Andriukaitis from Lithuania, (Policy Maker Award), Dr. Daria Julkowska from France and Poland (European Rare Disease Leadership Award), Prof. Annemieke Aarstma-Rus from the Netherlands (Scientific Award), Dr. Lisa Sanders from the United States of America (Written Media Award), Tobea, a Greece based company (Company Award for Health Technology), and Boehringer Ingelheim of Germany (Company Award for Patient Engagement.)

Jana Popova: Young patient Advocate Award

Using her skills and expertise in media and digital communications, Jana Popova, an expert of media and digital communications, raised awareness of issues faced by the Spinal muscular atrophy (SMA) community, and rare diseases in general, through, for example, the European Alliance of Neuromuscular Disorders Associations (EAMDA) Executive Committee, and the European Patients Forum (EPF) Youth Group. Jana is a volunteer for the Bulgarian Association for Neuromuscular Diseases and encouraged cooperation between different European patient organisations for neuromuscular disorders. This award recognises her boundless commitment to amplifying the voice of patients with neuromuscular diseases and other young patients, showing that together, patient advocates can have a huge impact.

Dr. Vytenis Andriukaitis: Policy Maker Award

The Policy Maker Award is attributed to Dr. Andriukaitis to recognise his outstanding work and support of the rare disease community in his role as the European Commissioner for Health and Food Safety, from 2014 to 2019. Dr. Andriukaitis accomplished during his tenure as Commissioner significant developments in European policy, including the development and launch of the European Reference Networks and European Cooperation on Health Technology Assessment during his mandate. Dr. Andriukaitis has been a tenacious supporter of the added value of European action in public health, and a strong defender of meaningful patient engagement in all areas that have an impact on patients’ lives.

Dr. Daria Julkowska: European Rare Disease Leadership Award

The European Rare Disease Leadership Award recognises the outstanding leadership and dedication. Dr. Julkowska has had a greatly positive impact on rare disease research and partnerships in Europe and beyond. It acknowledges her commitment and leadership to the E-Rare European Research Area (ERA-NET) programme, IRDiRC community, and notably as coordinator of the European Joint Programme on Rare Diseases. The ambitious yet game-changing research programme was successfully evaluated and funded, bringing together over 130 institutions in over 30 countries.

Photo credit : Eurordis

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ERN News

ERN Coordinators met with the European Commission to discuss expansion

From 13 to 15 November 2019, the 24 ERNs Coordinators met with the European Commission for three days in Brussels to discuss about the ways to expand their networks. In 2019, the geographical coverage of the ERNs in Europe was increased from 44% to 74%, following the inclusion of 247 Associated National Centres and 4 National Coordination Hubs, which created 311 links to the ERNs. Affiliated Partners represent now around 25% of the total of clinical units participating in the ERNs. This will allow healthcare providers in Europe to access the right level of expertise through inclusion in the current networks, especially in countries where networks are not yet represented by full members.

The participants discussed fine-tuning of the ERNs’ areas of competence and actions, and they addressed the expansion of the diseases coverage within the existing network. The Chair of the Coordinators, Dr. Irene Mathijssen, and Ana Rath, Director of Orphanet, highlighted the difficulty of clearly identifying the gaps within and overlaps between the ERNs’ activities. It was decided that this would be further brainstormed amongst the Coordinators themselves. Working groups shared their activities and experience, demonstrating impressive advances. In the field of education and training, the Coordinators and the Commission are working to set up common templates and layouts for all documents produced by the networks. A joint repository will eventually be accessible to the public via the European Commission webpage. The Commission will also support the ERNs in the field of clinical guidelines, to enable the production of tools to support decisions for the treatment of rare diseases.

Research was also an important focus of the work conducted by the Coordinators during the meeting. Results from a survey conducted amongst ERN members by the European Joint Programme on Rare Diseases (EJP RD) showed that awareness of the various existing research infrastructures is still low amongst researchers. Raising awareness about research infrastructures is therefore of critical importance. The services offered by research infrastructure should met the needs of users, so several ERN 'use cases' were presented which could be further tested with ERN members as users. Orphanet presented its work and the revision of classification that is currently being undertaken with some ERNs, in order to align the standards used on both sides. Orphanet also collaborates with several ERNs to produce information for Doctors and patients, and that information is published on its website. The workshop was successful in its aims of increasing awareness of research infrastructures and was the first step towards increased collaboration.

Editorial

EURORDIS Black Pearl Awards 2020

Spotlight

Ready, set, go for rare disease day 2020

Rare Disease Day 2020: preparation around the globe underway

Belgium

France

Australia

Unites States of America

ERN News

ERN Coordinators met with the European Commission to discuss expansion

ERN eUROGEN includes new affiliated partners

MetabERN experts save a baby suffering from Glycogen Storage Disease

ERN-EuroBloodNet and Eurordis to establish an European Network of Sickle Cell Disease Patients Organisations

ERN-EuroBloodNet and the NCP Cleiss joint efforts to assist an Erasmus student affected by a rare disease

VASCERN’s work on the patient pathways

Orphanet News

European Conference on Rare Diseases 2020: Call for posters

Summary of the edition of 07 February 2020

Editorial

Political news

Scientific news

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