Policy recommendations for rare disease centres of expertise |
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| Institution: | 1. European Organisation for Rare Diseases, 96 rue Didot, 75014 Paris, France;2. NHS England, London, UK;3. University of Southern Denmark, Unit for Health Promotion Research, Niels Bohrs Vej 9, DK-6700 Esbjerg, Denmark;1. Department of Pediatrics, Division of Allergy and Immunology, Morsani College of Medicine, University of South Florida, Tampa, Fla;2. Department of Pediatrics, Division of Allergy and Immunology, Children''s Hospital of Philadelphia, Philadelphia, Pa;3. Department of Pediatrics, Division of Allergy, Immunology and Rheumatology, David Geffen School of Medicine, University of California Los Angeles, Los Angeles, Calif;4. CSL Behring LLC, King of Prussia, Pa;1. Research Associate, University of Szeged, Faculty of Dentistry, Szeged, Hungary;2. Research Associate, Oral and Maxillo-Facial Surgery Department, University of Oulu, Oulu, Finland;3. Professor, Department of Cranio-Maxillo-Facial Surgery, European Face Centre, Vrije Universiteit Brussel, Brussels, Belgium;4. Professor, University of Szeged, Faculty of Dentistry, Szeged, Hungary;1. Institut Lumière Matière, UMR5306 Université Lyon 1-CNRS, Université de Lyon, 69622 Villeurbanne cedex, France;2. Institute of Condensed Matter Physics, EPFL, CH-1015 Lausanne, Switzerland;3. Synchrotron SOLEIL, L’Orme des Merisiers, Saint-Aubin BP 48, F-91192 Gif-sur-Yvette Cedex, France;4. European Synchrotron Radiation Facility, BP 220, F-38043 Grenoble Cedex, France |
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| Abstract: | AimRare diseases are a serious public health concern and are a priority in the EU. This study aims to develop policy recommendations for rare disease centres of expertise (CoEs) in order to improve standards and quality of care.Subject and methodsA modified 3-round Delphi technique was used. Participants included rare diseases patients, carers, patient representatives and healthcare professionals (HCPs) from CoEs in two countries—Denmark and the UK.ResultsThe results suggest the need to make improvements within current CoE environments, access to CoEs and the need for coordination and cooperation of services within and outside CoEs. It is recommended that CoEs are not overly ‘medicalised’, while at the same time they should be established as research facilities. The importance of including patient representatives in CoE performance management was also highlighted. Raising awareness and provision of appropriate training amongst non-specialist HCPs is seen as a priority for early and correct diagnosis and ensuring high quality care. Similarly, provision of targeted information about patients’ illness and care was considered essential along with access to social assistance within CoEs.ConclusionsPolicy recommendations were developed in areas previously recognised as having gaps. Their implementation is expected to strengthen and improve current care provision for rare disease patients. In member states where national plans and strategies are being developed, it is recommended to replicate the methodological approach used in this study as it has proven to be a helpful tool in rare disease centres of expertise policy development. |
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| Keywords: | Rare disease Delphi technique Health policy Centre of expertise European reference network |
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