2014 is a turning point for rare diseases in Europe and a busy agenda awaits EURORDIS!

2014 begins with the celebration of a milestone: EURORDIS now has over 600 member patient organisations (606), located throughout 56 different countries, including 526 full members in 26 EU countries, and representing over 4000 different conditions! We extend a warm welcome to our newest members. In addition, EURORDIS has adopted a membership re-assessment process to regularly review that its existing members still fulfill all criteria; its implementation starts in 2014. To learn more about the criteria for joining EURORDIS and about the re-assessment process, consult the EURORDIS Membership information section of our website section of our website, available in seven languages. As our movement grows, we look forward to standing shoulder-to-shoulder with all EURORDIS members in our mission to build a strong pan-European community able to speak with one voice, to act together and to engage with all stakeholders to create positive change for all people living with rare diseases.

We hope to see many members at the upcoming capacity-building Membership Meeting as well as the European Conference for Rare Diseases and Orphan Products-ECRD 2014 Berlin taking place in Germany 8-10 May, expected to be a milestone in 2014 by bringing to the surface the many achievements of the dynamic multistakeholder rare disease community in Europe while beating the drums of future policy priorities.

EURORDIS will expand its outreach in Central & Eastern Europe throughout 2014 including more patient groups, supporting efforts in the region to raise awareness, create national alliances, and promote rare disease policies. Also, we will continue to provide our website & eNews in Russian and offer Russian interpretation at ECRD 2014 Berlin.

Three strategic initiatives will strengthen rare disease community building:
- “Common Goals & Mutual Commitments between National Alliances in Europe & EURORDIS: An agenda between 2014 & 2020”: through this recently adopted initiative EURORDIS will foster the convergence and collaboration with and between the National Alliances and all its member organisations
- “Rare Diseases International”: will be launched as an informal global network of rare disease patient organisations able to speak with one global voice. Turning rare diseases into an international movement is a current priority for EURORDIS.
- “RareConnect”: with its 50 international moderated social forums, which are rapidly growing, EURORDIS will scale up this platform in 2014 and expand its utility, creating more online rare disease communities and topic groups, extending translations from 5 to 7 languages adding Portuguese and Russian, developing new functions, and exploring partnerships, in particular with academic researchers.

Advocacy

As declared last year by Herman van Rompuy, President of the European Council, “In the European Union, rare diseases are now one of the main priorities for public health and research policies. With the constructive support of EURORDIS, several policy initiatives have been established over the past decade”. After 15 years of relentless efforts to create this rare disease community and promote the cause of people living with rare diseases, 2014 is a turning point as we enter a new phase over the next 5 to 10 years, as national rare disease strategies will be enhanced and deepened while being increasingly integrated into European common approaches. European Reference Networks of Rare Diseases and European Research Infrastructures will provide new opportunities for translating research into innovative treatments and information technology into knowledge generation, toward better care. These goals can be achieved only through empowered patient advocates partnering with all stakeholders and reaching out across society at large.

A main and foremost key advocacy priority for EURORDIS is to continue supporting the development of national rare disease plans/strategies throughout Europe and beyond, a cornerstone to turn our vision into real change for patients and families. As these plans/strategies develop, the advocacy activities become more technical and require refocusing, while enhancing an exchange of best measures across Europe. EURORDIS’ active participation in the new European Commission Expert Group on Rare Diseases (succeeding the European Union Committee of Experts on Rare Diseases) with its EURORDIS Policy Action Group will be essential in reaching that goal. Monitoring the State of the Art of rare disease policies, developing Recommendations & the follow- up on their implementation are two 2014 challenges for: Centres of Expertise, European Reference Networks, Cross-border Healthcare, registries, access to orphan drugs, coding of rare diseases, social policy and services, as well as national plan indicators. Monitoring the implementation of the EU Directive on Patients’ Rights on Cross-border Healthcare, supporting the development of an EU Platform on Rare Disease Registries, participating in new long-term infrastructure projects on registries, biobanks, and –omics, and supporting clinical research are other areas EURORDIS will pursue.

Rare diseases are now a sustainable policy and budget priority in EU policy programmes through 2020 – particularly the 3rd EU Heath Programme (Health for Growth) and the 8th EU Research and Technology Framework Programme (Horizon 2020); stimulating an ambitious implementation is another key advocacy priority for EURORDIS in coming years.

EURORDIS will continue to be extremely vigilant on the future EU Regulation on Data Protection; essential issues are at stake for people living with rare diseases. As the policy debates and voting come to a close in 2014, we need to be certain that this EU legislation will protect citizens from abusive use of their health information while not being detrimental to health research; if not, EURORDIS will take initiatives to promote the right trade-off for a fair public health policy.

Access

The European Medicines Agency’s Committee for Orphan Medicines (COMP) held its 150th meeting in 2013. EURORDIS has actively participated in the designation of over 1200 orphan medicines to date, including protocol assistance, paediatric studies and overall clinical development up to marketing authorisation. New in 2014, EURORDIS is actively engaged in the International Rare Disease Research Consortium (IRDiRC) leading the Therapeutic Scientific Committee to reach the goal of 200 new orphan medicines by 2020, while co-leading the Stakeholder Forum of the European HTA Network and other related Working Groups on Patient Reported Outcomes in partnership with relevant learned societies.

As more products enter the developmental pipeline, it is crucial that mechanisms are in place to ensure equitable access across Europe.

Earlier, Wider, Equitable, Sustainable Access to Medicines are thus key words for 2014 and EURORDIS has forks in different fires for improving access to medicinal products and services, including plans to promote and participate in dialogue around Medicine Alternative Development Pathways, and contributing to pilots for Common HTA Assessment Reports as well as for the Mechanism of Coordinated Access (MoCA) to orphan medicines to promote Member State collaboration around a common value for new orphan medicines. Similarly, EURORDIS will promote European collaboration on pricing based on value, volume, and post-Marketing Authorisation data generation, also exploring Managed Entry Agreement and Equitable Pricing mechanisms for improving access

An Access Campaign will be launched in 2014 to assess the difficulties patients currently experience in getting medicines and medical care and to promote targeted solutions. EURORDIS is adopting a Working Paper addressing the impact of the economic crisis on people with rare diseases as countries across Europe tighten spending on health and other areas.

New and innovative EURORDIS website sections dedicated to medicines associated with position papers and capacity building sessions are now in place for 2014. The section on Pharmacovigilance & patient reported adverse events created in 2013 will be promoted through 2014. A new section on Compassionate Use exploring how patients with no adequate treatment might be eligible to access a medicine on a compassionate use basis will be available from February 2014. Shortages of Medicines and how to address the related challenges is another area of focus.

In parallel, the EURORDIS Summer School Session 2014 on Medicines Development, Clinical Trials, EU Regulatory Affairs and HTA will train an additional 40 patient advocates. The 250 alumni are regularly further supported by eLearning training modules, regular webinars on specific topics, monthly reports and exchanges.

It is time in 2014 to create a EURORDIS Panel of Experts. Respect and influence of EURORDIS opinions are growing, while the complexity of policy issues to be addressed demands greater input from a variety of experienced and committed leaders in the fields.

Rare Disease Day 2014

EURORDIS continues to increase resource allocation to coordinate Rare Disease Day and is looking forward to wide participation all around the world in 2014 to promote rare diseases as an international public health priority. The www.rarediseaseday.org website has been revamped to offer more information and be even more user-friendly. As always, EURORDIS is organising a Policy Event for the occasion. To be held on 25 February 2014 in Brussels, this multi-stakeholder gathering will reflect the Rare Disease Day 2014 theme of Care by putting the focus on Improving Access to Rare Disease Care: The Vision of Patients.

Rare Disease Day will also be celebrated by the 3rd EURORDIS “Black Pearl” Gala Dinner to promote the cause and support for rare diseases and featuring our first Rare Disease Day Ambassador, Sean Hepburn Ferrer. The impressive roster of 15 high level Honorary Patrons includes European Parliament President Martin Shultz, Her Highness Princess Anne de Ligne, the First Ladies of Croatia, Macedonia and Malta, Health Ministers and Ambassadors from the United Kingdom, Germany, France, Denmark, Ireland, Luxembourg, Monaco, and Canada; and the unique support of our Gala Committee and corporate partners. This event will be the occasion to recognise outstanding international achievements and commitment to rare diseases through the increasingly prestigious and significant EURORDIS Awards 2014.

Throughout 2014, EURORDIS will continue to provide up-to-date information for patient advocates and patients & their families. Our website and eNews will continue to provide reliable useful information and serve as a voice for the EURORDIS rare disease community to come together and share our news, our strength, our actions, and our commitment. EURORDIS TV and our Social Media on Facebook and Twitter will be highly supported to make our communication lively and interactive. Information that facilitates capacity-building and empowerment are especially emphasised. The recently launched EURORDIS InfoHub will be fine-tuned to ensure an optimal performance, allowing users to find the information they need.

Cross-Cutting support

The contribution of EURORDIS Volunteers is impressive in terms of high level competences, personal skills, intense and long term commitment, and diversity of diseases, countries and languages. As the number of Volunteers grows rapidly, EURORDIS will recognise this unique feature for a European Civil Society Organisation by adopting a Charter of EURORDIS Volunteers, at its General Assembly 2014, including mutual commitments and best practices to support volunteers; by launching a new section on the EURORDIS website; and by systematically including more volunteers in all activities.

The team of 30 staff, reorganised and strengthened in 2013, will remain stable in 2014 with the addition of a fund-raising manager. After the consolidation of our offices in Paris and Brussels in previous years, our office in Barcelona, dedicated to RareConnect & Social Media, will be consolidated in 2014.
The development priority in 2014 will be the elaboration and submission of new projects aligned with the above strategic priorities as well as new initiatives in fund raising to grow and diversify our private resources.

Thus, we look forward to sharing an active, productive 2014 together with all our friends in the rare disease community.

About Yann Le Cam

Yann Le Cam is a patients’ association advocate who has dedicated 25 years of professional and personal commitment to health and medical research non governmental organisations in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases. He has three daughters, the eldest of whom has cystic fibrosis. Yann is one of the founders of EURORDIS in 1996-1997 and its Chief Executive Officer since 2002. He is the Vice Chairman of the EU Committee of Experts on Rare Diseases since 2011. He was one of the first patient representatives appointed to the Committee for Orphan Medicinal Products (COMP) at the European Drug Agency (EMA) where he served 9 years and was its Vice Chairman for 6 years. He served on the Management Board and Executive Committee of the French HTA agency ANAES now called HAS for 5 years, on the DIA Advisory Committee Europe for 3 years, on the EPPOSI Board for 6 years. Yann is one of the founding members of the International Alliance of Patients Organisations (IAPO).

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