eurordis.orgeurordis.org

EURORDIS – hvad går det ud på?EURORDIS – hvad går det ud på?

vv. Birthe Byskov Holm, formand. Birthe Byskov Holm, formandSjældne DiagnoserSjældne Diagnoser

1

eurordis.org 2

Breaking isolationBreaking isolationCreating a critical massCreating a critical mass

Learning from each otherLearning from each otherWorking together Working together

EURORDISEURORDIS

eurordis.org

Who are we?Who are we?

3

An international non-profit, non-governmental umbrella An international non-profit, non-governmental umbrella rare disease patients’ organisation representing rare disease patients’ organisation representing an estimated 30 million individuals in European estimated 30 million individuals in Europe

Our Mission:Our Mission:

To build a strong To build a strong pan-European communitypan-European community of of patient organisations and people living with rare patient organisations and people living with rare diseasesdiseases

To be To be their voicetheir voice at the European level at the European level To directly or indirectly fight against the impact of To directly or indirectly fight against the impact of

rare diseases on their livesrare diseases on their lives

eurordis.org

EURORDIS in briefEURORDIS in brief

4

Founded in 1997Founded in 1997

615 member patient organisations615 member patient organisations

58 countries (26 EU countries)58 countries (26 EU countries)

33 National Alliances of RD Patients Organisations33 National Alliances of RD Patients Organisations

40 European Federations of specific rare diseases40 European Federations of specific rare diseases

Outreach to over 1800 patient groupsOutreach to over 1800 patient groups

Over 4,000 rare diseases representedOver 4,000 rare diseases represented

29 staff members, offices in Paris, Brussels, London, Barcelona29 staff members, offices in Paris, Brussels, London, Barcelona

55 volunteer patient advocates and 205 volunteer moderators of 55 volunteer patient advocates and 205 volunteer moderators of online RD patient communitiesonline RD patient communities

eurordis.org

EURORDIS sources of fundingEURORDIS sources of funding

5

Revenues and Expenses Revenues and Expenses

eurordis.org

Community Building - overviewCommunity Building - overview

6

615 members in 58 countries615 members in 58 countries

Council of 33 National Alliances (including Canada, USA & LA)Council of 33 National Alliances (including Canada, USA & LA)→ Common Goals, National Plans, Rare Disease Day→ Common Goals, National Plans, Rare Disease Day

Council of 40 European Federations and NetworksCouncil of 40 European Federations and Networks→ Cross border health care, accelerated procedure for multi-centred clinical trials for → Cross border health care, accelerated procedure for multi-centred clinical trials for rare diseases, European Reference Networks of Centres of Expertise, Registries rare diseases, European Reference Networks of Centres of Expertise, Registries

Organisation of annual Membership Meetings Organisation of annual Membership Meetings → Barcelona 2002, Namur 2003, Cork 2004, Venice 2005, Berlin 2006, Paris 2007, → Barcelona 2002, Namur 2003, Cork 2004, Venice 2005, Berlin 2006, Paris 2007, Copenhagen 2008, Athens 2009, Krakow 2010, Amsterdam 2011, Brussels 2012, Copenhagen 2008, Athens 2009, Krakow 2010, Amsterdam 2011, Brussels 2012, Dubrovnik 2013, Berlin 2014Dubrovnik 2013, Berlin 2014

Organisation of the European Conference on Rare DiseasesOrganisation of the European Conference on Rare Diseases → Copenhagen 2001, Paris 2003, Luxembourg 2005, Lisbon 2007, Krakow 2010, → Copenhagen 2001, Paris 2003, Luxembourg 2005, Lisbon 2007, Krakow 2010, Brussels 2012, Berlin 2014Brussels 2012, Berlin 2014

eurordis.org

European Network of National AlliancesEuropean Network of National Alliances

7

• Composed of 33 National Alliances of Rare Disease Composed of 33 National Alliances of Rare Disease Patient Organisations – Switzerland: ProRaris!Patient Organisations – Switzerland: ProRaris!

• Governed by the Council of National AlliancesGoverned by the Council of National Alliances

• Fostering the visibility and recognition of National Fostering the visibility and recognition of National Alliances for Rare DiseasesAlliances for Rare Diseases

• Enhancing EURORDIS’ outreach to local RD patient Enhancing EURORDIS’ outreach to local RD patient groupsgroups

• Enhancing EURORDIS and National Alliance cooperation Enhancing EURORDIS and National Alliance cooperation in key policy areas: Common Goals, National Plans, Rare in key policy areas: Common Goals, National Plans, Rare Disease DayDisease Day

• Capacity building, Empowering and NetworkingCapacity building, Empowering and Networking

eurordis.org

Network of European RD FederationsNetwork of European RD Federations

8

Composed of 40 EURORDIS member European FederationsComposed of 40 EURORDIS member European Federations

Representing the disease-specific levelRepresenting the disease-specific level

Enhancing EURORDIS and the Federations capacity in Enhancing EURORDIS and the Federations capacity in priority policy areas: priority policy areas: European Reference Networks, European research projects, European Reference Networks, European research projects, therapy developmenttherapy development, web communities, information , web communities, information helplines, Cross-border Healthcarehelplines, Cross-border Healthcare

Complementing the Council of National Alliances by:Complementing the Council of National Alliances by:

Enhancing EURORDIS’ outreach to local RD patient Enhancing EURORDIS’ outreach to local RD patient groups to build a pan-European RD communitygroups to build a pan-European RD community

Capacity building, Empowering and NetworkingCapacity building, Empowering and Networking

eurordis.org

EURORDIS Membership MeetingEURORDIS Membership Meeting

9

A capacity building annual event for rare disease A capacity building annual event for rare disease advocates since 2002advocates since 2002

Past locations include: Barcelona (2002), Namur (2003), Past locations include: Barcelona (2002), Namur (2003), Cork (2004), Venice (2005), Berlin (2006), Paris (2007), Cork (2004), Venice (2005), Berlin (2006), Paris (2007), Copenhagen (2008), Athens (2009), Krakow (2010), Copenhagen (2008), Athens (2009), Krakow (2010), Amsterdam (2011), Brussels (2012), Dubrovnik (2013)Amsterdam (2011), Brussels (2012), Dubrovnik (2013)

EURORDIS Membership Meeting 2014, EURORDIS Membership Meeting 2014, Berlin 8 May, 2014Berlin 8 May, 2014

An opportunity for EURORDIS Members to:An opportunity for EURORDIS Members to:

Build capacities via targeted workshopsBuild capacities via targeted workshops Share experiencesShare experiences NetworkNetwork Be up-to-date in main policy and emerging issuesBe up-to-date in main policy and emerging issues

eurordis.org 10

6th European Conference on Rare Diseases & Orphan 6th European Conference on Rare Diseases & Orphan Products - Products - ECRD - ECRD - 23-25 May 2012, Brussels: 23-25 May 2012, Brussels:

@700 participants from 55 countries (1/2 patient reps + @700 participants from 55 countries (1/2 patient reps + doctors, researchers, policymakers and industry)doctors, researchers, policymakers and industry)

110 session chairs / speakers / panelists (25 from EUCERD)110 session chairs / speakers / panelists (25 from EUCERD) 38 sessions covering 7 Conference Themes (health policy, 38 sessions covering 7 Conference Themes (health policy,

research, drug development)research, drug development) 4 pre-conference tutorials + satellite meetings4 pre-conference tutorials + satellite meetings Posters accepted & presented: 166 Posters accepted & presented: 166 Simultaneous interpretation available: English, Dutch, Simultaneous interpretation available: English, Dutch,

French, German, Spanish and Russian French, German, Spanish and Russian www.rare-diseases.euwww.rare-diseases.eu

eurordis.org 11

7th European Conference on Rare Diseases & Orphan Products - 7th European Conference on Rare Diseases & Orphan Products - ECRD ECRD 2014 Berlin 2014 Berlin --

8–10 May 2014, Berlin, Germany 8–10 May 2014, Berlin, Germany

www.rare-diseases.euwww.rare-diseases.eu

eurordis.org 12

Shaping EU Rare Disease policiesShaping EU Rare Disease policies

eurordis.org

Major Advocacy Achievements - IMajor Advocacy Achievements - IContribution to EU drug regulationsContribution to EU drug regulations

13

• EU Regulation on Orphan Medicinal Products in 1999EU Regulation on Orphan Medicinal Products in 1999

Creation of the Creation of the COMP - Committee for Orphan Medicinal Products at COMP - Committee for Orphan Medicinal Products at the European Medicines Agency the European Medicines Agency (including 3 patients’ representatives (including 3 patients’ representatives for the first time)for the first time)

To date, over 1100 Orphan Drugs Designated + over 82 Products To date, over 1100 Orphan Drugs Designated + over 82 Products approved, benefiting approximately 3 million EU patientsapproved, benefiting approximately 3 million EU patients

• EU Regulation on Medicinal Products for Paediatric Use in 2006EU Regulation on Medicinal Products for Paediatric Use in 2006

Creation of the Creation of the PDCO – Paediatric Committee at the European PDCO – Paediatric Committee at the European Medicines Agency Medicines Agency (including 3 patients’ representatives and their (including 3 patients’ representatives and their alternates)alternates)

• EU Regulation on Advanced Therapy Medicinal Products in 2007EU Regulation on Advanced Therapy Medicinal Products in 2007 Creation of the Creation of the CAT - Committee for Advanced Therapies CAT - Committee for Advanced Therapies at the at the

European Medicines Agency (including 2 patients’ representatives and European Medicines Agency (including 2 patients’ representatives and their alternates)their alternates)

eurordis.org

Major Advocacy Achievements - IIMajor Advocacy Achievements - IIContribution to EU Directives/ RegulationsContribution to EU Directives/ Regulations

14

• EU Directive on Patients’ Rights in Cross-border Healthcare in EU Directive on Patients’ Rights in Cross-border Healthcare in 2011 2011

Provides the legal framework for European Reference Networks Provides the legal framework for European Reference Networks

Article 13 specifically on Rare DiseasesArticle 13 specifically on Rare Diseases

• Ongoing contribution from EURORDIS to:Ongoing contribution from EURORDIS to:

The ongoing revision of the EU Directive on Clinical Trials The ongoing revision of the EU Directive on Clinical Trials 2001/20/EC 2001/20/EC – Proposal from the EC for a EU Regulation on CTs – Proposal from the EC for a EU Regulation on CTs

The proposal from the EC for a EU Regulation on the protection of The proposal from the EC for a EU Regulation on the protection of individuals with regard to the processing of personal data individuals with regard to the processing of personal data – – revision of the proposal is currently under review by the Committee LIBE revision of the proposal is currently under review by the Committee LIBE of the European Parliamentof the European Parliament

eurordis.org

Major Advocacy Achievements - IIIMajor Advocacy Achievements - IIIShaping EU Rare Disease PolicyShaping EU Rare Disease Policy

15

• Communication from the European Commission Communication from the European Commission to the European to the European Parliament, the Council, the European Economic and Social Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions : « Committee and the Committee of the Regions : « Rare Diseases: Rare Diseases: Europe’s challenge », 11 November 2008Europe’s challenge », 11 November 2008

First comprehensive policy text addressing all issues faced by RD First comprehensive policy text addressing all issues faced by RD patients, from patients, from research on RDsresearch on RDs, to diagnosis, access to care and , to diagnosis, access to care and adapted services and development of training, education on RDsadapted services and development of training, education on RDs

• Council Recommendation on an Action in the field of Rare Council Recommendation on an Action in the field of Rare Diseases, 8 June 2009Diseases, 8 June 2009

Adopted by EU Ministers of Health Adopted by EU Ministers of Health

Promote the adoption of RD National Plan/Strategy in EU MSPromote the adoption of RD National Plan/Strategy in EU MS

Creation of the European Union Committee of Experts on Rare Creation of the European Union Committee of Experts on Rare Diseases (EUCERD) in November 2009Diseases (EUCERD) in November 2009

eurordis.org 16eurordis.org

Together we are stronger