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.

European Reference Networks

Information on European Reference Networks & the EURORDIS Patient Advocacy Groups

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EURORDIS and EPF call on payers to support access

Call on EU National Competent Authorities for Pricing & Reimbursement support

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EC Communication and Council Recommendation on Rare Diseases

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

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The European Commission Expert Group on Rare Diseases

The group’s mission is to support EU policy on rare diseases and it has adopted key recommendations.

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Why rare disease research matters

Read EURORDIS' Position Paper on rare disease research.

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EURORDIS Joint Statement on Data Protection Regulation proposals

EURORDIS Joint Statement urges policymakers to ensure that the new EU Data Protection Regulation will enhance health research while protecting personal data.

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EU Rare Disease Policies: An Overview

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

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EU Policy Framework

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

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Patients Access to Information

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

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Patients Access to Diagnosis & Care

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

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Patients Access to Treatment

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

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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.

Overview of National Rare Disease Policies

Find out about the political landscape of rare diseases across Europe and rare disease National Plans

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Guidance Documents for National Plans

Essential documents for establishing national plans.

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EUROPLAN – EURORDIS Tool Kit for National Conferences

Provides necessary information and guidance documents for organising a EUROPLAN National Conference.

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The EUROPLAN Project

Started in 2008, this EU project supports national strategies and plans for rare diseases in Member States and other European countries.

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EUROPLAN National Conferences 2012-2015

Find out where EUROPLAN National Conferences 2012-2015 are taking place in Europe

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Reports of EUROPLAN National Conferences 2012-2015

The EUROPLAN National Conference reports summarise  national discussions around rare disease policy in the context of European guidelines and identify bottlenecks and actions to be taken to improve care and services for patients

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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
 

 

Decide

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.

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Why get involved?

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

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How to participate?

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

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Discover the topics

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

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Flickr gallery

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

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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 diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect 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