ARCHIVE PAGE OF WWW.EUCERD.EU, WEBSITE OF THE FORMER EUROPEAN EXPERT GROUPS ON RARE DISEASES & EUCERD JOINT ACTION

 

INTRODUCTION.. 1

HISTORY OF EUROPEAN-LEVEL RARE DISEASE MULTSTAKEHOLDER GROUPS. 2

RECOMMENDATIONS. 4

REPORTS. 6

MEETING REPORTS. 10

EXPERT WORKSHOPS. 11

EUCERD JOINT ACTION.. 13

Outputs & deliverables. 15

Final deliverables. 15

Outcomes. 15

 

 

INTRODUCTION

 

To date, three groups of stakeholders in the field of rare diseases have been successively brought together by the European Commission to provide expert advice in this area to their services: The Rare Diseases Task Force (2004-2009), The European Union Committee of Experts on Rare Diseases (2010-2013) and The EC Expert Group on Rare Diseases (2013-2016). From 2018 issues related to rare diseases are discussed at the European level by the Steering Group on Health Promotion, Disease Prevention and Management of Non-Communicable Diseases, whilst questions related to European Reference Networks (ERNs) for Rare Diseases are dealt with by the Board of Member States of the ERNs.

This page is an archive of the EUCERD and ECEGRD�s activities and information concerning the work of the EUCERD Joint Action (N� 2011 22 01, co-funded by the EU Health Programme).

The final meeting of the EUCERD Joint Action, presenting the outcomes and future perspectives was held on 15th September 2015 in Luxembourg and the Joint Action ended at the end of 2015. The deliverables and outcomes are available on this page.

The support to the elaboration of policies in the field of rare diseases and improvement of the codification of rare diseases provided by the EUCERD Joint Action continued in the scope of RD-Action, a Joint Action co-funded via the 3rd EU Health Programme for the years 2015-2018.

 

  

HISTORY OF EUROPEAN-LEVEL RARE DISEASE MULTSTAKEHOLDER GROUPS

 

To date, three groups of stakeholders in the field of rare diseases have been brought together successively by the European Commission to provide expert advice in this area to their services: The Rare Diseases Task Force (2004-2009), The European Union Committee of Experts on Rare Diseases (2010-2013) and The EC Expert Group on Rare Diseases (2013-2016).

 

The Rare Diseases Task Force (2004-2009)

The Rare Diseases Task Force was established in January 2004 via Commission Decision 2004/192/EC of 25 February 2004 on the programme of Community action in the field of public health (2003 to 2008). The RDTF was replaced by the European Union Committee of Experts on Rare Diseases.

The members of the RDTF included current and former rare disease research project leaders, elected experts from Member States, and representatives from relevant international organisations (DG Research, DG Enterprise, EuroStat, EMA, WHO, OECD). Orphanet director S�gol�ne Aym� was the chair person.

Over 6 years, the RDTF played a pivotal role instigating key collaborative rare diseases initiatives in Europe. The RDTF met 12 times over the course of its mandate, and many key topics were brought forward for discussion in relation to rare disease research, policy and actions. The scientific activity of the RDTF was funded from 2005 onwards, which allowed the publication of OrphaNews Europe, the official newsletter of the Rare Diseases Task Force. Published every two weeks, this newsletter has served as a vital communication tool, bringing news on policy developments, medical and scientific findings, orphan drugs, research projects, patient activities, events and new relevant publications to the global rare disease community � including patients, healthcare professionals, researchers, industry professionals and health policy makers.

Various working groups were identified and constituted to meet the specific objectives of the RDTF: this included the Standards of Care working group, the Public Health Indicators working group, and the Coding, Classification and Data Confidentiality Group. These working groups produced various reports, recommendations and scoping papers.

One of the most notable contributions of the RDTF was its pivotal role between June and October 2007 in drafting the Communication Rare Diseases: Europe�s Challenges, in close collaboration with the European Commission. The process ultimately culminated in the adoption of the European Council Recommendation on an Action in the Field of Rare Diseases in June 2009.

The European Union Committee of Experts on Rare Diseases replaced the European Commission�s Rare Diseases Task Force (RDTF) in 2010.

The European Union Committee of Experts on Rare Diseases (2010-2013)

The European Union Committee of Experts on Rare Diseases was formally established via the European Commission Decision of 30 November 2009 (2009/872/EC). This committee was charged with aiding the European Commission with the preparation and implementation of Community activities in the field of rare diseases, in cooperation and consultation with the specialised bodies in Member States, the relevant European authorities in the fields of research and public health action and other relevant stakeholders acting in the field.

The 51-member committee with representatives from all Member States and stakeholder groups was chaired by S�gol�ne Aym� and co-chaired by Kate Bushby, Yann Le Cam and Helena K��ri�inen. The EUCERD met 3 times a year and organised a number of workshops supported by the EUCERD Joint Action.

Over the 3-year mandate of the EUCERD, 5 recommendations were adopted in the field of rare diseases concentrating on centres of expertise, European Reference Networks, the clinical added value of orphan medicinal products information flow, registries & data collection, and indicators for national plans. An Opinion on potential areas of collaboration at European level in the field of new born screening was also issued.

The EUCERD was a highly successful forum for exchange of experience and cooperation between stakeholders appreciated greatly by the rare disease community. The EUCERD was replaced at the end of its mandate by the European Commission Expert Group on Rare Diseases from 2014 onwards.

An editorial documenting the achievements of the EUCERD entitled �The European Union Committee of Experts on Rare Diseases: three productive years at the service of the rare disease community� was published by the Orphanet Journal of Rare Diseases in February 2014 to mark Rare Disease Day.

The European Commission Expert Group on Rare Diseases (2014 onwards)

The European Commission Expert Group on Rare Diseases, established via Commission Decision 2013/C 219/04 of 30 July 2013, was active until 2016.

This group included representatives from all the Member States plus nominated experts, representatives of patient organisations, patients� organisations in the field of rare diseases, European associations of producers of products or service providers relevant for patients affected by rare diseases, and European professional associations or scientific societies acting in the field of rare diseases.

After the Expert Group

From 2018 issues related to rare diseases are discussed at the European level by the Steering Group on Health Promotion, Disease Prevention and Management of Non-Communicable Diseases, whilst questions related to European Reference Networks (ERNs) for Rare Diseases are dealt with by the Board of Member States of the ERNs.

 

RECOMMENDATIONS

 

European Committee of Experts on Rare Diseases (EUCERD)

The EUCERD issued the following 5 recommendations on centres of expertise, European Reference Networks, registries, indicators for national plans and improving informed decisions based on the clinical added value of orphan medicinal products:

EUCERD Recommendations on Rare Disease Patient Registration and Data Collection (5 June 2013)

�       Executive Summary

�       Powerpoint presentation of the Recommendation (June 2013)

EUCERD Recommendations on Core Indicators for Rare Disease National Plans/ Strategies (6 June 2013)

�       Executive Summary

�       Powerpoint presentation of the Recommendation (January 2014)

EUCERD Recommendations on European Reference Networks for Rare Diseases (31 January 2013) and Addendum (10 June 2015)

�       Executive Summary

�       Powerpoint presentation of the Recommendation (January 2013)

Recommendations of the EUCERD to the European Commission and Member States on Improving Informed Decisions Based on the Clinical Added Value of Orphan Medicinal Products (CAVOMP) Information Flow (September 2012)

�       Executive Summary

�       Powerpoint presentation of the Recommendation (September 2012)

 EUCERD Recommendations on Quality Criteria for Centres of Expertise for Rare Diseases in Member States (24 October 2011)

�       Translations: BG � Bulgarian, CS � Czech, DA � Danish, DE � German, EL � Greek, ES � Spanish, ET � Estonian, FI � Finnish, FR � French, HU � Hungarian, IT � Italian, LT � Lithuanian, LV � Latvian, MT � Maltese, NL � Dutch, PL � Polish, PT � Portuguese, RO � Romanian, SK � Slovakian, SL � Slovenian, SV � Swedish

�       Executive Summary

�       Powerpoint presentation of the Recommendations (October 2011)

Opinions

The EUCERD also issued the following Opinion.
EUCERD Opinion on Potential Areas of European Collaboration in the field of New Born Screening � July 2013

 EC Expert Group on Rare Diseases

The EUCERD issued the following recommendation:

 

REPORTS

 

The European Union Committee of Experts on Rare Diseases and EC Rare Disease Task Force, and the EUCERD Joint Action (N� 2011 22 01), the have published the reports on the following subjects:

Annual reports on the state of the art of rare diseases activities in Europe

2014 Edition � arising from the EUCERD Joint Action N�2011 22 01

An article based on this work has been published: Charlotte Rodwell, S�gol�ne Aym� Rare disease policies to improve care for patients in Europe, Biochim Biophys Acta. 2015 Feb 25. pii: S0925-4439(15)00059-9. (Open Access)

2013 Edition � arising from the EUCERD Joint Action N�2011 22 01

2012 Edition � arising from the EUCERD Joint Action N�2011 22 01

2011 Edition

2010 Edition

National Plans/Strategies for Rare Diseases

� arising from the EUCERD Joint Action N�2011 22 01

Centres of expertise & European Reference Networks

Registries and Health Indicators

�        RDTF Report: Patient Registries for Rare Diseases � April 2009, Update June 2011

Rare diseases in international nomenclatures

Genetic Testing

Orphan Medicinal Products

Specialised Social Services

 

MEETING REPORTS

EUCERD MEETING REPORTS

The public summary reports of previous EUCERD meetings can be accessed here:

RDTF MEETING REPORTS

The Rare Diseases Task Force held two meetings per year at the European Commission in Luxembourg from 2004 to 2009.

�        Report on the 1st Rare Diseases Task Force meeting � 20/01/2004

�        Report on the 2nd Rare Diseases Task Force meeting � 14/10/2004

�        Report on the 3rd Rare Diseases Task Force meeting � 20/06/2005

�        Report on the 4th Rare Diseases Task Force meeting � 14/12/2005

�        Report on the 5th Rare Diseases Task Force meeting � 08/06/2006

�        Report on the 6th Rare Diseases Task Force meeting � 14/12/2006

�        Report on the 7th Rare Diseases Task Force meeting � 20/06/2007

�        Report on the 8th Rare Diseases Task Force meeting � 23/10/2007

�        Report on the 9th Rare Diseases Task Force meeting � 28/02/2008

�        Report on the 10th Rare Diseases Task Force meeting � 13/11/2008

�        Report on the 11th Rare Diseases Task Force meeting � 30/04/2009

�        Report on the 12th Rare Diseases Task Force meeting � 23/10/2009

 

EXPERT WORKSHOPS

 

The EUCERD and CEGRD organised, with the support of two successive Joint Actions, a number of expert workshops on the following themes:

National plans/strategies for rare diseases

International nomenclatures

Centres of expertise and European Reference Networks

Registries

Health indicators

Social Services for Rare Diseases

Others

�        EUCERD Joint Action Workshop: Cross-Border Genetic Testing in the European Union 15-16 December 2014, Newcastle �organised through the EUCERD Joint Action N�2011 22 01

�        Workshop: The genetic testing offer in the EUEuropean workshop on the genetic testing offer in Europe, Ispra, 19-20 November 2012 � organised by the EC Joint Research Centre

�        EUCERD/Eurobiomed Event � Rare2011 Conference European Day, 4 November 2011, Montpellier, France � organised through the RDTF/EUCERD Scientific Secretariat Joint Action N� 2008 22 91 with Eurobiomed

�        Workshop on monitoring and evaluation of health projects in the field of rare diseases � 27 November 2014, Lisbon � organised through the EUCERD Joint Action N�2011 22 01

 

 

 EUCERD JOINT ACTION

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The EUCERD Joint Action: Working for Rare Diseases (N� 2011 22 01) was co-funded by the European Commission (Executive Agency for Health and Consumers, now CHAFEA) in the context of the European Union�s Second Programme of Community Action in the field of Health. It started on 1 March 2012 and ended in 2015, supporting the activities and mandate of the European Union Committee of Experts on Rare Diseases until the end of 2013 when the EUCERD ended its mandate. From 2014 it supported the activities of the European Commission Expert Group on Rare Diseases which replaced the EUCERD.

This Joint Action was led by Prof. Kate Bushby, former Vice-Chair of the EUCERD and member of the Expert Group.

The EUCERD was mandated to assist the EC in formulating and implementing the Community�s activities in the field of rare diseases, to foster exchanges of relevant experience, policies and practices between the Member States and stakeholders. Rare diseases are a priority area for action in the Public Health Programme (2008-2013). These activities have been defined in the Communication of the European Commission, entitled �Rare Diseases: Europe�s challenge� (11 November 2008) and the Council Recommendation on an action in the field of rare diseases (8 June 2009).

Specifically, this Joint Action addressed the following priority areas of the Council Recommendation:

To achieve its aims, the Joint Action built on the achievements of previous European initiatives in the field, such as the EC Rare Disease Task Force, Orphanet, the Europlan project, and the outputs of and the several rare disease networks that have received EU funding over the past years.

This Joint Action comprised five main areas of work:

This last area of work aimed, with the input of the EUCERD, to propose a model for sustainable action.

The expected outcome was integrated strategy for the implementation of rare disease policies through the exchange of experience between Member State health authorities already involved in rare disease policy definition and implementation and via a series of recommendations from the EUCERD and clear communication of these recommendations to national policy makers, patient organisations and learned societies.

Specific expected outcomes were the following:

Outputs & deliverables

The following outputs and public final deliverables of the EUCERD Joint Action (N�2011 22 01) are currently available. A meeting presenting the outcomes of the Joint Action was organised on 15 September 2015 in Luxembourg.

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Final deliverables

 

Outcomes

National plans and strategies

International RD nomenclatures

Specialised social services

Quality of care/centres of expertise

Integration of RD activities

Registries

European Reference Networks

Genetic Testing

Reports on the State of the Art of RD Activities

o   2014 Edition � arising from the EUCERD Joint Action N�2011 22 01

o   An article based on this work has been published: Charlotte Rodwell, S�gol�ne Aym� Rare disease policies to improve care for patients in Europe, Biochim Biophys Acta. 2015 Feb 25. pii: S0925-4439(15)00059-9. (Open Access)

o   2013 Edition � arising from the EUCERD Joint Action N�2011 22 01

o   2012 Edition � arising from the EUCERD Joint Action N�2011 22 01