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.

• Needs 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.

   

• Rare 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.
   

• Orphanet

  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.
   

• National 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.
   

• European 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.
   

• Centres 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.
   

• European 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.

   

• Newborn 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 Healthcare
  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 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.
   

• 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.
   

• 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.
   

• Respite 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.

   

• 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