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National Policy

EURORDIS represents the voice of people with rare diseases and their families and in this capacity plays a central role in the regulatory process. The contribution of EURORDIS has been key to the adoption of important rare disease and orphan medicine legislation at the European level, including the EU Regulation on Orphan Medicinal Products, the EU Regulation on Paediatric Drugs, the EU Regulation on Advanced Therapies, the Commission Communication “Rare Diseases: Europe’s challenges, the Council Recommendation on European Action in the field of Rare Diseases, the EU Directive on Patients’ Rights in Cross-border Healthcare, and others.

By partnering with rare disease national alliances, EURORDIS also contributes to national processes, and facilitates the adoption and implementation of national plans and strategies for rare diseases in European countries.

Thanks to the EU Commission Communication and Council Recommendation on an action in the field of rare diseases, national rare diseases policies and plans have gained momentum.

Why is a rare disease national plan or strategy needed in your country?

In previous decades, rare diseases were nearly invisible in national healthcare systems. Due to a lack of knowledge on these very rare and complex diseases, patients, families and carers were in the dark and faced extreme difficulties in accessing a diagnosis, appropriate care and treatments.

The ultimate goal of a national rare disease plan/strategy is to guarantee rare disease patients’ access to timely and adequate medical and social care.

Click here to know 5 reasons to adopt a national plan for rare diseases in your country
  1. It helps to recognise and address the specificities of rare diseases in a comprehensive manner by setting up a political and legal framework, involving all stakeholders, and coordinating all relevant actions at the national and regional levels in different areas (access to care, access to adapted social services, innovative research, patient registries, participation in European and international research and policymaking…)
  2. National plans are an excellent tool to map out medical expertise on rare diseases across the country. They enable the adoption of an official process to accreditate Centres of Expertise, in charge of providing adequate clinical research, diagnosis and care through multidisciplinary teams of experts.
  3. They make us identify and establish social services and programmes relevant to rare diseases (resource centres, disability programmes, specialised training for professionals involved in diagnosing and management of rare diseases), and bridge the gaps between different services through case management.
  4. They establish the framework to better integrate the European legislations and Policy Recommendations into the national healthcare and social systems (e.g. orphan drugs, paediatric drugs, advanced therapies, clinical trials, cross-border healthcare, European Pillar of Social Rights).
  5. The national plans highlight the added value of rare disease patients’ organisations and National Alliances as key partners and involve them in decision-making committees and programmes dedicated to rare diseases in the fields of research, healthcare and social care.

What is included in a rare disease national plan/ strategy?

A plan or strategy includes relevant national measures to improve care at every stage of the patient’s journey, from diagnosis to access to treatments and therapies, through long-term follow-up, social care and services. A national rare disease plan/strategy often includes measures to foster innovative research in the field of rare diseases. Each measure needs to be associated with a defined budget.

A national plan/ strategy requires solid governance composed of a multi-stakeholder governing committee (including patient representatives) that monitors the implementation of the national measures against agreed timelines and public health indicators.

Which strategic areas should be taken into account to build a national plan/strategy?

EUROPLAN National Conference

The EUROPLAN National Conferences (co-funded by the European Commission within the EUROPLAN Project [2008-2011], the EUCERD Joint Action [2012 – 2015] and RD-ACTION [2015 – 2018)]) are the tool designed to promote the adoption and implementation of national plans or strategies for rare diseases in European countries based on the Recommendation of the EU Council on an action in the field of rare diseases of 8 June 2009, as well as to facilitate the integration of EU rare disease policies and recommendations into the national health and social systems.

EUROPLAN National Conferences are jointly organised in each country by a National Alliance of rare disease patient organisations and EURORDIS.

Key features of a EUROPLAN national conference
  • Patient-led – they are organised by national rare disease alliances in conjunction with EURORDIS.
  • Multi-stakeholder – national authorities, patients, carers, healthcare professionals, academia, industry, social workers, and insurers all attend to exchange their experiences and perspectives.
  • Integrated between European and national levels – participants use the conferences as an opportunity to assess the integration of EU regulations, policies and recommendations into national health and social systems.
  • Exhaustive – Discuss all strategic areas of a national plan/ strategy, including governance and also rare disease healthcare and research.
Impact of EUROPLAN National Conferences

In 2009, only five EU Member States had adopted a national rare disease plan (Bulgaria, France, Greece, Portugal and Spain).

Between 2010 and 2018, altogether 59 EUROPLAN National Conferences took place in 25 EU Member States, as well as in Georgia, Macedonia, Russia, Serbia and Ukraine. Some National Alliances organised several EUROPLAN conferences in their country.

As a result, by the end of 2018, 25 EU Member States had put in place a national plan or strategy for rare diseases. Malta, Poland and Sweden are still discussing the adoption of a strategy or plan.

Rare disease plans and strategies in European countries

Click on the flag to find the country’s national plan or strategy for rare diseases, as well as the relevant EUROPLAN conference report.