The document discusses the Dutch model for building centers of expertise for rare diseases. It outlines the Dutch healthcare landscape and the mission of the Dutch Steering Committee to establish high-level care centers for rare diseases. Timelines are provided for establishing criteria for the expertise centers from 2008-2011, with the committee advising the health minister in 2011 on organizing the centers and financing multidisciplinary rare disease care. The general approach focuses on improving quality of life, long-term and multidisciplinary care, data collection, and involvement in European networks. The Dutch model involves expertise centers conducting research and providing care through multidisciplinary teams connected to local treatment centers and international networks. Main criteria for expertise centers include offering long-term multidisciplinary

1. Building Centres of Expertise The Dutch Model Prof dr FJM Gabre ë ls Dutch Steering Committee on Rare Diseases and Orphan Drugs Krakow, May 14, 2010

2. The Dutch Landscape eight university hospitals twenty-six large teaching hospitals, providing highly specialised medical care approximately 6-8% of the European population is affected by a rare disease in the course of life: in the Netherlands about one million people

3. Mission Dutch Steering Committee the Steering Committee is architect and booster of the policy on developing expertise centres for rare diseases work plan 2008-2011: to establish - high level care for rare diseases - development of health care pathways - improvement of information a consultation document made by the Committee will be discussed with the stakeholders

4. Timelines (1) 2008-2009 top-down consultation document with criteria for expertise centres and expert teams, derived from criteria in European documents 2010-2011 bottom-up consultation round with different stakeholders (a.o. professionals, treatment centres, patients) to gain support for the plans financial paragraph in consultation with relevant authorities

5. Timelines (2) 2011 The Dutch Steering Committee will advice the Minister of Health about the organisation of expertise centres a model for financing the multidisciplinary high-level care for rare diseases a proposal on monitoring the quality of expertise centres in the future

6. General starting points improvement of quality of life long term care (transition from child to adult care and secondary prevention) multidisciplinary teams central registration of data to improve research, care and treatment development of health care pathways participation in European networks implementation of new techniques (e-health)

7. Dutch model expertise centre combination of fundamental research, professional education, cure and care by an multidisciplinary team with international networks (some may get the status of European expertise centre) expert team local treatment centre offering high-level care and clinical research in association with expertise centre Not every rare disease will have an expertise centre in the Netherlands (small country / few patients)

8. Main criteria Expertise centre multidisciplinary team offering long term care, i.c. secondary prevention and transition of care (child to adult) minimal number of patients setting up databases consultation centre for professionals and patients development of health care pathways performing fundamental research (international) translation of research into new treatments and care training and education of professionals

9. Conclusions Expertise centres and expert teams for rare diseases are important to improve - longitudinal treatment and care - knowledge of rare disorders - stimulation of research - quality of life of these patients National supervising organ will indicate expertise centres and audit the quality of centres in the Netherlands