EURORDIS policy fact sheets for patient advocates

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

Patients are very effective participants in the policy making process. EURORDIS has made available this valuable data to rare disease patient organisations so that they can better advocate issues of specific importance with national and/or European policy decision makers.

How can patient advocates use these fact sheets?

These fact sheets are concise summaries of many of the policy issues that correspond to the EU Commission Communication and Council Recommendation. They are in no way exhaustive of the needs of each rare disease in each national context. Rather they are designed to serve as reference documents. Other documents, such as the “EURORDIS Declaration of common principles on centres of expertise and European reference networks for rare diseases”, can be used for the same purpose.

• Needs and Priorities for Rare Disease Research

  Rare disease research (RDR) represents a broad range of scientific investigations to establish knowledge on rare diseases (RD).

   

• Centres of Expertise

  Although no official or uniform definition of Centres of Expertise (CoE) exists across Europe, some European countries have established physical expert structures for the management and care of rare disease (RD) patients at the national level.
   

• European Reference Networks of Centres of Expertise

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

• Rare Disease Patient Registries

  Patient registries are on-going, exhaustive systems of data collection of patients with the same disease(s) from a geographically defined population over an extended period of time. By collecting patient data, patient registries constitute key instruments in supporting health service planning, increasing knowledge on rare diseases and support research.
   

• 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 (RD) help line (HL).
   

• Therapeutic Recreation Programmes 

  Therapeutic Recreation Programmes (TRP) are any organised recreation activity (summer camp, ad hoc trip) which gives people living with rare diseases (PLWRD) the possibility to take a break from focusing on their disease and treatment to concentrating on fun and leisure
   

• Respite Care Services

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

   

• Adapted Housing Services

  Adapted Housing Services (AHS) represent a particular type of service, often associated with multiple disabilities. Sometimes called ‘therapeutic apartments’, these services allow people living with rare diseases (PLWRD) to develop and enjoy some 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.
   

• Resource Centres

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

• "Declaration of common principles on centres of expertise and European reference networks for rare diseases"

  In 2008, EURORDIS adopted this declaration in order to improve patient care throughout Europe. Rare disease patients call upon national health authorities to endorse, publicise and implement the following declaration to contribute to the identification of centres of expertise and to support them financially.
   

• Memo on the Clinical Added Value on Orphan Drugs (CAVOD)

  How can cooperation at the EU level facilitate national decisions and improve access to orphan drugs? The summary of the main EURORDIS paper on the Clinical Added Value on Orphan Drugs (CAVOD) aims to illustrate this. The said cooperation will namely be based on the to-be-created working party on CAVOD, to be located ideally at the EMA.
   

• Frequently Asked Questions on Off-Label Use of Medicines

  Although all drugs 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 many patients as not all rare diseases benefit from an approved and well evaluated medicinal product.