The EU rare diseases policy framework: the context for ERNs

Transcription

1 The EU rare diseases policy framework: the context for ERNs Jaroslaw Waligora DG Health and Food Safety, European Commission Unit C.1, Programme Management and Diseases

2 Report from an expert group of the Rare Diseases Task Force to the High Level Group on Health Services and Medical Care September Rationale for establishing a European collaboration for the delivery of health care and medical services in the field of rare diseases The RDTF recommends exploring all possible forms of cooperation between Member States in the field of health services and medical care for rare diseases.

3 Policy framework Commission Communication on Rare Diseases: Europe's Challenge COMM(2008)679 Council Recommendation on action in the field of rare diseases 2009/C 151/02 Implementation report on the Commission Communication and Council Recommendation on Rare Diseases COM(2014) 548

4 Plans and strategies in the field of rare diseases Adequate definition, codification and inventorying of rare diseases Research on rare diseases Centers of expertise and European reference networks Gathering the expertise on rare diseases at European level Empowerment of patient organisations Sustainability

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6 Expert group on rare diseases recommendations EUCERD Recommendations on Quality Criteria for Centres of Expertise for Rare Diseases in Member States (24 October 2011) EUCERD Recommendations on European Reference Networks for Rare Diseases (January 2013) Addendum (June 2015)

7 Rare immunological and auto-inflammatory diseases Rare bone diseases Rare cancers* and tumours Rare cardiac diseases Rare connective tissue and musculoskeletal diseases Rare malformations and developmental anomalies and rare intellectual disabilities Rare endocrine diseases Rare eye diseases Rare gastrointestinal diseases Rare gynaecological and obstetric diseases Rare haematological diseases Rare craniofacial anomalies and ENT (ear, nose and throat) disorders Rare hepatic diseases Rare hereditary metabolic disorders Rare multi-systemic vascular diseases Rare neurological diseases Rare neuromuscular diseases Rare pulmonary diseases Rare renal diseases Rare skin disorders Rare urogenital diseases European Patient Advocacy Groups (epag)

8 Recommendations to support the incorporation of rare diseases into social services and policies 3. European Reference Networks for RD have a key role in facilitating integrated care provision in line with the EUCERD recommendations on European Reference Networks for Rare Diseases (10) and the Directive on patients rights in cross-border healthcare (Article 12, 4-ii): Rare Disease European Reference Networks (RD ERNs) need to collaborate with each other, as well as with patient groups, health and social care providers; RD ERNs follow a multi-disciplinary approach; RD ERNs could function as a platform to share experiences and promote cooperation between MS, to develop precise descriptions of the services required and elaborate common guidelines.

9 RD-ACTION Objectives To continue implementation of the policy priorities, and to support the work of the Commission Expert Group on Rare Diseases by gathering expertise and producing data necessary to its action. Management structure JA-MB Contribute to solutions to ensure an appropriate codification of rare diseases in health information systems Support the further development and sustainability of the Orphanet database on rare diseases

10 EU Platform on Rare Diseases Registration Objectives: Transfer and hosting the European-level coordinating activities of the European surveillance networks EUROCAT and SCPE to the JRC To support interoperability of existing and rare diseases registries

11 Joint Action on rare cancers

12 Thank you!