Over Zeldzame Ziekten Beleid

What is the European policy in the field of rare diseases ?

A description of the European policy in the field of rare diseases can be found on the following website : ec.europa.eu/health-eu/health_problems/rare_diseases/index_en.htm

  • The programme of Community action in the field of public health (2003-2008)

    On September 2002, the European Parliament and the Council adopted a new Community action programme for public health. This programme runs for a 6-year period (from 1 January 2003 to 31 December 2008). The list of funded projects during this programme related to rare diseases can be found on the following website: http://ec.europa.eu/health/ph_programme/programme_en.htm
  • Programme for Community Action in the Field of Health (2007-2013)

    Rare diseases are now one of the priorities in the EU Public Health Programme 2007-2013. According to the DG SANCO Work Plans for the implementation of the Public Health Programme, the two main lines of action are the exchange of information via existing European information networks on rare diseases, and the development of strategies and mechanisms for information exchange and co-ordination at EU level to encourage continuity of work and trans-national co-operation.

    Furthermore, regarding rare diseases projects, DG SANCO prioritises generalist networks, which centralise information on as many rare diseases as possible - not just a specific group or a single disease - to improve information, monitoring and surveillance.

  • Rare Diseases Task Force

    In January 2004, the European Commission's Public Health Directorate set up the Rare Diseases Task Force (RDTF). Its aims are:
    • 'to advise and assist the European Commission Public Health Directorate in promoting the optimal prevention, diagnosis and treatment of rare diseases in Europe, in recognition of the unique added value to be gained for rare diseases through European co-ordination ;'
    • to provide a forum for discussion and exchange of views and experience on all issues related to rare diseases.

    The Rare Disease Task Force is led by Dr Ségolène Aymé, a medical geneticist and Director of Orphanet. The Deputy Leader is Professor Helen Dolk, director of the Eurocat programme on congenital disorders. Members are comprised of current and former project leaders of European research projects related to rare diseases, member state experts and representatives from relevant international organisations.

    The Task Force has its own website www.rdtf.org and produces a monthly newsletter on Rare Disease and Orphan Drug policy and research in Europe, OrphaNews Europe.

    The RDTF currently has three expert working groups providing advice and recommendations to the European Commission on:

    • European centres of expertise for rare diseases
    • Public Health Indicators for rare diseases
    • Coding and Classification of rare diseases

    Full details can be found on the RDTF website.

  • The Research Framework Programmes

    Rare diseases are one of the priorities of the calls for proposals for research funding launched by DG Research. DG Research recognises that, whilst rare diseases, by definition, affect far fewer people than common diseases, they can be life-threatening and chronically debilitating conditions and, to date, few drugs have been developed to treat them. The large variety of these diseases – many of which have a genetic basis – means that the research effort in Europe is fragmented. Greater focus should help to realise the benefits of pooling Europe's research resources to tackle such diseases.

    A list of projects funded by FP6 can be found on the following website: cordis.europa.eu/lifescihealth/major/rare-diseases-projects-1.htm

What are the National policies in the field of rare diseases ?

A few European countries have a national policy in the field of rare diseases or have taken initiatives in the field. A list of these initiatives can be found on the following website: ec.europa.eu/health/ph_threats/non_com/rare_9_en.htm

What is the US policy in the field of rare diseases ?

The Office of Rare Diseases (ORD) was established in 1993 within the Office of the Director of the National Institutes of Health (NIH). On November 6, 2002, the President established the Office in statute (Public Law 107-280, the Rare Diseases Act of 2002). More information on the activities of this office can be found on the following website: rarediseases.info.nih.gov

Last update: 06/03/13