Rare Disease Policy

EURORDIS represents the voice of patients 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 legislations 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 a 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.


An overview of the key pillars of EU Rare Disease policy.

Rare 2030 Foresight Study

Rare2030 is a foresight study that gathers the input of a large group of patients, practitioners and key opinion leaders to propose policy recommendations that will lead us to a better future for people living with a rare disease in Europe.


Two milestones in EU Rare Disease Policy

The Commission Communication and Council Recommendation have established a strategy for meeting the challenge of rare diseases.


Major Recommendations shaping RD policy

The EU Committee of Experts on Rare Diseases (EUCERD, 2010-2013) and the European Commission Expert Group on Rare Diseases (CEG-RD, 2013-2016) were established to support EU policy on rare diseases


Rare cancers

Rare cancers are rare occurrences of a common disease affecting less than 6 per 100,000 individuals a year, or one in five new patients diagnosed with cancer.


The Network of Parliamentary Advocates for Rare Diseases

A network of European and national members of parliament advocating to improve the lives of people living with a rare disease.


The European Conference on Rare Diseases & Orphan Products (ECRD)

The leading conference gathering all rare disease stakeholders in Europe


European Reference Networks

Information on European Reference Networks & the European Patient Advocacy Groups


Patients Access to Diagnosis & Care

EU level initiatives are improving access to rare disease diagnostics and care across Europe.


Patients Access to Treatment

EU level legislation and initiatives encourage the development of rare disease medicines and foster equitable access across Europe.


Breaking the Access Deadlock to Leave No One Behind

A contribution by EURORDIS and its members on possibilities for patients' full and equitable access to rare disease therapies in Europe This position paper is a contribution from EURORDIS and its members that offers a synthesis of their analysis, reflections and perspectives on the issue of access to rare disease therapies....


The EURORDIS Access Campaign

The EURORDIS Access Campaign pushes for improved access to, and the development of rare disease medicines, diagnostic tests and medical devices.


Social policy & integrated care

EURORDIS promotes the integration of rare diseases into social policies and also integrated care for rare disease patients and their carers.


Patients Access to Information

Key EU-level policies and initiatives foster patient and professional access to information on rare diseases.


EU Rare Disease Policies: An Overview

More than half of Member State legislation stem from EU policies and strategies, including directives, regulations and recommendations. 


EU Policy Framework

Key legislative texts, expert committees and funding bodies contribute to the EU rare disease policy framework.


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?

Six reasons to adopt a national plan for rare diseases in your country


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

A plan/ 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.


Documents necessary for the design and elaboration of a national plan/ strategy for rare diseases

The European Recommendations and policies listed have been adopted by consensus by EU Member States and stakeholders (academia, industry, patients).


EUROPLAN National Conferences

The EUROPLAN National Conferences are the tool designed to promote the adoption and implementation of national plans or strategies for rare diseases in European countries.


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.


Rare Diseases International

RDI is a network of rare disease patient organisations and advocates interested in building the global alliance of rare disease patients and families across the world


NGO Committee for Rare Diseases

The NGO Committee for Rare Diseases engages at the UN level to elevate rare diseases to a priority within global public health.



EURORDIS policy fact sheets for patient advocates

Empowering the voice of patients in policy making at national and EU levels

Patients are active participants in the policy making process. EURORDIS has created specific Policy Fact Sheets to help rare disease patients and organisations better advocate issues of importance with national and/or European policy decision makers.

How can patient advocates use these Policy Fact Sheets?

The Policy Fact Sheets provide concise summaries of the key policy issues delineated in the European Commission's Communication on Rare Diseases: Europe's Challenges and the Council Recommendation on an action in the field of rare diseases. They are designed to serve as reference documents. Other EURORDIS documents, including Position papers, Concept papers, and Statements, are also useful for patient advocacy purposes.


  • Eurordis Policy Fact Sheet on Needs and Priorities for Rare Disease ResearchNeeds and Priorities for Rare Disease Research

    Rare disease research represents a broad range of scientific investigations to establish knowledge and find treatments for rare diseases.


  • Eurordis Policy Fact Sheet on Rare Disease Patient RegistriesRare Disease Patient Registries

    Rare Disease Patient Registries represent a fundamental research effort upon which a number of critical activities are based. They constitute key instruments for increasing knowledge on Rare Diseases by pooling data for fundamental and clinical research, epidemiological research, and real-life post-marketing observational studies.

  • Eurordis Policy Fact Sheet on Centres of ExpertiseOrphanet

    Orphanet is the reference portal for information and documentation on rare diseases and orphan drugs. Orphanet provides freely available, user-friendly Web-based access to medically validated, comprehensive information, thus improving knowledge and contributing to the accurate, diagnosis and appropriate care and treatment of patients with rare diseases.

  • Eurordis Policy Fact Sheet on National Help Lines for Rare DiseasesNational Help Lines for Rare Diseases

    Any rare disease patient organisation that offers 1) information about a given rare disease or rare disease related topic or 2) psychological support to the patient, can be classified as a rare disease help line.

  • Eurordis Policy Fact Sheet on National Help Lines for Rare DiseasesEuropean Network of Rare Disease Help Lines

    The European Network of Rare Disease Help Lines was created by EURORDIS to provide support and share experience and information between national or disease specific help lines across Europe. EURORDIS, on behalf of the European Network of Rare Diseases Help Lines, has asked the European Commission DG Connect to reserve a unique six digit 116 phone number for help lines for rare diseases across Europe.

  • Eurordis Policy Fact Sheet on Centres of ExpertiseCentres of Expertise

    Centres of Expertise are a core element of all National Plans on Rare Diseases that the EU Member States are encouraged to adopt by the end of 2013 under the Council Recommendation of 8 June 2009 on an action in the field of rare diseases.

  • Eurordis Policy Fact Sheet on European Reference Networks of Centres of ExpertiseEuropean Reference Networks of Centres of Expertise

    A European Reference Network of Centres of Expertise is the physical or virtual networking of knowledge and expertise of national Centres of Expertise in more than one European country.


  • Eurordis Policy Fact Sheet on Needs and Priorities for Rare Disease ResearchNewborn screening

    Newborn screening is the process of systematically testing newborns just after birth for certain treatable diseases. Ideally, this practice is part of a larger programme that includes confirmatory diagnosis, immediate care, treatment and follow-up. Recent and continued developments of screening techniques, as well as the increase of possibilities for treatment, have led to the expansion of NBS to include potentially detectable rare diseases.

  • Q&A for the transposition of the Directive on Cross-Border HealthcareFrequently Asked Questions on Off-Label Use of Medicines
    This document is intended to respond to some of the main questions patients’ representatives may have to best understand the new legislation and to advocate at national level in the best interest of patients for the transposition in national law.

  • Frequently Asked Questions on Off-Label Use of MedicinesFrequently Asked Questions on Off-Label Use of Medicines

    Although all medicines need approval for specific indications before they can be sold, the practice of prescribing pharmaceuticals for an unapproved indication is very common with up to one-fifth of all drugs being prescribed as “off label”. Off-label use of medicines can help patients, as not many rare diseases benefit from an approved and well evaluated medicinal product.

  • Eurordis Policy Fact Sheet on Therapeutic Recreation Programmes Resource Centres

    Actions performed by Resource Centres are generally specifically targeted to people living with rare diseases. These centres often function in partnership or cooperation with Centres of Expertise or constitute part of a Centre of Expertise themselves.

  • Eurordis Policy Fact Sheet on Therapeutic Recreation Programmes Adapted Housing Services

    Adapted Housing Services represent a particular type of service, often associated with multiple disabilities. Sometimes called ‘therapeutic apartments’, these services allow people living with rare diseases to develop and enjoy a level of autonomy by living within the comfort of their own home, alone or with peers, assisted by supportive staff, rather than being placed in an institution.

  • Eurordis Policy Fact Sheet on Respite Care ServicesRespite Care Services

    Respite care is provided, on a short term basis, for people living with rare diseases so that their carers can have some relief from care giving.


  • Eurordis Policy Fact Sheet on Therapeutic Recreation Programmes Therapeutic Recreation Programmes 

    Therapeutic Recreation Programmes are any organised recreation activity  that gives people living with rare diseases the possibility to take a break from focusing on their disease and treatment to concentrate instead on fun and leisure


























Page created: 13/09/2012
Page last updated: 07/11/2014



Launched in May 2009 within the POLKA project, the Decide rare disease discussion kits are designed to empower patients and their representatives to become advocates for their cause. The Decide sessions have helped numerous rare disease patients to voice their opinions, to feel more confident and to have, at last, a say in decision making.


About rare disease Decide

Learn about the Decide method and how it’s being used by the rare disease community.


Why get involved?

Learn why thousands of people have become involved so far and why you should too.


How to participate?

Learn how to organise your own Decide session and start giving your input into rare disease issues.


Discover the topics

Six Decide discussion topics were created in 22 European languages to help empower patients and patient representatives.


Flickr gallery

Barcelona, Krakow, Brussels…just a few of the places that Decide sessions have been held. Visit the Decide gallery on Flickr.



EURORDIS Rare Barometer Programme

Rare Barometer programme

At the heart of the EURORDIS Rare Barometer Programme is the idea that the advocacy work of EURORDIS and its members should continue to be increasingly based on patient perspectives.

At EURORDIS, requests for patient perspectives in health, research and social policy-making are on the rise as the benefits associated with evidence-based programmes or policies are being increasingly recognised and required by all stakeholders. To best respond to our growing advocacy role and achieve a high quality evidence-base from people living with a rare disease, the EURORDIS Rare Barometer Programme will rely heavily on patient engagement to inform sound policy from the patient perspective by carrying out surveys, focus groups, individual face-to-face interviews and other opinion-gathering methods to gain firsthand feedback from patients. 

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The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseasesRare Disease International Bringing together patients, families and experts to share experiences in a moderated multi-language forum. RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases