Rare Diseases

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Vlasta Zmazek, Board of Directors
Ljubljana, 5th November 2014
eurordis.org
eurordis.org
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What is a rare disease?
A rare disease in Europe is a disease affecting
less than 1 in 2,000 citizens
Many are of genetic origin
Over 50% affect children
Over 6,000 distinct rare diseases
6 to 8% of the population over lifespan
30 million people living with a rare disease in Europe
Main groups: metabolic disorders, neuro-muscular,
bleeding disorders, cardiovascular and respiratory,
autoimmune diseases, skin diseases, rare cancers…
“Rare Diseases: Understanding this Public Health Priority”, available in
English, French, German, Spanish, Hungarian, Turkish (www.eurordis.org)
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Characteristics of Rare Diseases
Chronic, progressive, degenerative,
disabling and frequently life-threatening
Patients are few and geographically
scattered
Experts are few and geographically
scattered
Research is fragmented
Reliable information is scarce
Resources are limited
Sustainability is an issue
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Find solutions to common problems
Speak with one voice
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Who are we?
An international non-profit, non-governmental umbrella
rare disease patients’ organisations representing
an estimated 30 million individuals in Europe
Our Mission
To build a strong pan-European community of patient
organisations and people living with rare diseases
To be their voice at the European level
To directly or indirectly fight against the impact of rare
diseases on their lives
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EURORDIS in brief
Founded in 1997
633 member patient organisations
59 countries (26 EU countries)
33 National Alliances of RD Patients Organisations
44 European Federations of specific rare diseases
Outreach to over 1800 patient groups
Over 4,000 rare diseases represented
30 staff
Offices in Paris, Brussels, London, Barcelona, Geneva
260 Volunteers: 55 patient advocates and 205 moderators
4 Million € Budget
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EURORDIS: Revenues and Expenses
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EURORDIS GOVERNANCE CHART 2014
MEMBERS
GENERAL ASSEMBLY
Financial Audit Deloitte
BOARD OF DIRECTORS
European Public
Affairs Committee
BOARD OF OFFICERS
EURORDIS Panel of
Experts
CHIEF EXECUTIVE OFFICER
President
Vice President
General Secretary
Treasurer
Officer
STAFF
EURORDIS Action Groups &
Task Forces
Therapeutic
Action Group (EMA)
Policy Action Group
(Commission Expert
Group on RDs
Policy Action Group –
Rare Cancers
(Commission Expert
Group on Cancer Control)
DITA Task Force
(Drug, Information,
Transparency & Access)
EURORDIS STANDING
COMMITTEES & COUNCILS
European Public
Affairs Committee
Council of
National Alliances on
Rare Diseases
Council of European
Federations on
Rare Diseases
Council of Rare Diseases
International
EURORDIS COMMITTEES & WORKING GROUPS
Health Policy:
Communication:
- Social Policy WG
- Editorial Committee
- EUROPLAN Advisors
- Rare Disease Day Working Group
- RareConnect Steering Committee - Gala Dinner Committee
- RareConnect Advisory Committee - EYRD 2019 Steering Committee
Research & Therapies:
Cross-Cutting
- RARE-Bestpractices WG
- Operating Grant
- Registries & Biobank WG
- RD-Connect Joint Patient Advisory Council
- Summer School Faculty
EURORDIS
CONFERENCE
PROGRAMME
COMMITTEE
European Conference
on Rare Diseases and
Orphan Products 2014,
Berlin
EURORDIS
Membership Meeting
2015, Madrid
EURORDIS Board of Directors
EURORDIS Staff
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Overcoming fragmentation
Creating a critical mass
Learning from each other
Working together
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Community Building
633 member organisations in 59 countries
Council of 33 National Alliances (including Canada & USA)
→ Common Goals & Mutual Commitments, National Plans, Rare Disease Day
Council of 44 European Federations and Networks
→ Cross border health care, clinical trials for rare diseases, European
Reference Networks of Centres of Expertise, registries, access to medicines
Membership Meetings
→ Barcelona 2002, Namur 2003, Cork 2004, Venice 2005, Berlin 2006, Paris
2007, Copenhagen 2008, Athens 2009, Krakow 2010, Amsterdam 2011,
Brussels 2012, Dubrovnik 2013, Berlin 2014, Madrid 2015
European Conference on Rare Diseases & Orphan Products
(ECRD)
→ Copenhagen 2001, Paris 2003, Luxembourg 2005, Lisbon 2007, Krakow
2010, Brussels 2012, Berlin 2014, Edinburgh 2016
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European Network of National Alliances
Composed of 33 National Alliances of Rare Disease Patient
Organisations
Governed by the Council of National Alliances (CNA)
Common Goals and Mutual Commitments 2014-2020
Fostering the visibility and recognition of National Alliances for Rare
Diseases
Enhancing EURORDIS and National Alliance cooperation in key
policy areas based on Common Goals: Rare Disease Day and
National Plans (Research, Centres of Expertise, Registries & Data
Collection, Best Practices, Information, Access to treatments, Social)
Enhancing EURORDIS’ outreach to local RD patient groups
Capacity building, Empowering and Networking
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Network of European RD Federations
Composed of over 40 EURORDIS member European Federations
Representing the disease-specific level
Governed by the Council of European Federations (CEF)
Enhancing EURORDIS and the Federations capacity in priority
policy areas:
European Reference Networks, registries, European research
infrastructures, European research projects, therapy development,
access to medicines, web communities, information helplines,
Cross-border Healthcare
Enhancing EURORDIS’ outreach to local RD patient groups
Capacity building, Empowering and Networking
Rare!Together: sharing good practices to develop European or
International Networks & Federations
Financial support to activities of European RD Federations
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EURORDIS Membership Meeting
A capacity building annual event for rare disease advocates since
2002
Around Europe: Barcelona (2002), Namur (2003), Cork (2004),
Venice (2005), Berlin (2006), Paris (2007), Copenhagen (2008),
Athens (2009), Krakow (2010), Amsterdam (2011), Brussels (2012),
Dubrovnik (2013), Berlin (2014)
EURORDIS Membership Meeting 2015, Madrid, 28-30 May
An opportunity for EURORDIS Members:
Joining Capacity Building Workshops in priority policy areas
Learning From Each Other Forum to share experiences
Networking
Being up-to-date in main policy and emerging issues
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EURORDIS Member Organisations
2000 - June 2014
700
634
606
600
543
492
500
447
402
400
359
309
273
300
236
216
200
187
194
210
218
100
0
2000
2001
2002
2003
2004
2005
2006
2007
2008
2009
2010
2011
2012
2013
2014
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7th European Conference on Rare
Diseases & Orphan Products - ECRD
8-10 May 2014, Berlin, Germany
www.rare-diseases.eu
+800 participants from 41 countries
138 session chairs / speakers / panellists
Themes: Research; knowledge Generation; Innovative
Practices in Orphan Products; Future Policies for Rare
Disease Therapies; Beyond Medical Care
Translations: French, German, Polish, Russian and Spanish
4 pre-conference tutorials + satellite meetings
200 selected Posters presented
Growing role of social media, e.g., +2000 Tweets posted
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8th European Conference on Rare Diseases &
Orphan Products - ECRD
26-28 May 2016, Edinburgh, Scotland
www.rare-diseases.eu
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Patient Advocates Empowerment
Information & Training
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Patient Advocates Empowerment
Enables patients, families, and patient advocacy groups to:
Become aware
Gain trust
Gain strength in their capacity to act, make choices and
transform choices into actions
Gain more power to take control of their life, their rights, and
their social, economic and political conditions
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Information & Networking
Website in 7 languages: English, French, German,
Italian, Portuguese, Spanish, Russian
Weekly eNews in 7 languages
EURORDIS TV www.eurordis.org/tv
EURORDIS InfoHub www.eurordis.org/tv
Blog www.rarediseaseblogs.net
Fact Sheets, Position Papers, Publications
Rare Disease Day www.rarediseaseday.org
Online Patient Conversations www.RareConnect.org
European Network of Rare Disease Help Lines
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EURORDIS Website
www.eurordis.org
+30,000 visits per month. Visitors from over 190 countries
Information in 7 languages:
rare diseases - patient organisations
orphan medicinal products
patient experience and testimonials
EU policies - national plans for rare diseases
research - genetic testing - newborn screening
Centres of Expertise - European Reference Networks –
registries
specialised social services - help lines
online patient communities, videos, conferences and other
events
EURORDIS activities, and much more
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Rare Disease Day
Held on the last day of February each year
Created & coordinated annually by EURORDIS since 2008
Participation in over 80 countries and regions in 2014
An occasion to raise public and policy-maker awareness
for rare diseases
Annual Policy Event in Brussels
Rare Disease Day 2015 Theme and Slogan:
Living with a Rare Disease: Day-by-day, hand-in-hand
www.rarediseaseday.org
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Campaign for a European
Year for Rare Diseases 2019
What and why?
A European Year, officially designated by the EU, is an occasion to
raise public and political awareness about all rare diseases
When?
2019 is an important year for the rare disease community:
20 year anniversary of the adoption of the EU Regulation on OMPs
10 year anniversary of the Commission Communication and
Council Recommendation on rare diseases
How can I get involved?
Sign-up to support the campaign for a European Year for Rare
Diseases in 2019: www.eurordis.org/eyrd2019
Tweet using the hashtag #EYRD2019
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Rare Connect
Launched in 2010
Free-access online rare disease patient communities
Over 65 communities involving over 564 patient
associations
Supported by 3 full-time community managers and 242
volunteer moderators
Translators offer free translations between 5 languages
(English, French, German, Italian, Spanish)
www.rareconnect.org
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EURORDIS Summer School
Since 2008
For Patient advocates
5-day annual training on:
Clinical Trials & Medicines Development
EU Regulatory Processes & EMA
HTA, Reimbursement, Patient Access
Translational & International Research
Location: Barcelona, Spain
New Master Class format & peer-to-peer real case studies
227 patient advocates trained from 34 countries, over 70 diseases
Alumni involved in regulatory processes at the EMA and/or in
collaboration with sponsors and/or as EURORDIS’ Volunteers
EURORDIS also continues its involvement with the European Patients’
Academy on Therapeutic Innovation project (EUPATI)
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EURORDIS Round Table of Companies
Sharing knowledge of orphan medicinal products &
rare disease therapies development
Since 2004, 10 years
45 companies
2 workshops per year
bring together 80-100
representatives of
industry, regulatory
agencies, patient groups,
clinicians and academics
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EURORDIS Round Table of Companies –
ERTC
2014 Workshops
20th ERTC workshop - 26 February 2014, Brussels:
"Unlocking Europe's Potential in Rare Disease Therapies”
Celebrating 10 years of dialogue & collaborative efforts
between patient advocates, industry, regulatory agencies &
national competent authorities to forward the development of
treatments for rare diseases.
87 participants from 12 countries
21st ERTC Workshop – 30 September 2014, Barcelona:
“Sharing Rare Disease Patient Data: Translating Principles
into Action ”
80 participants from 14 countries
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Patients & Industry: Partnering in
Innovation
Engagement of patient advocates
all along the value chain of research & development
of RD therapies
as well as in placing on the market & providing good
medical practices, help save time & money, create
additional value for products, generate value to
shareholders & society
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Voicing
Rare Disease patients’ expectations
Shaping EU Rare Disease policies
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Major Advocacy Achievements
Contribution to EU medicines regulations
EU Regulation on Orphan Medicinal Products in 1999
Creation of the COMP - Committee for Orphan Medicinal Products at
the European Medicines Agency (including 3 patients’ representatives
for the first time)
As of Sept 2014: over 1300 Orphan Drugs Designated & 93 Products
approved, benefiting approximately 3 million EU patients
EU Regulation on Medicinal Products for Paediatric Use in 2006
Creation of the PDCO – Paediatric Committee at the European
Medicines Agency (including 3 patients’ representatives and their
alternates)
EU Regulation on Advanced Therapy Medicinal Products in 2007
Creation of the CAT - Committee for Advanced Therapies at the
European Medicines Agency (including 2 patients’ representatives and
their alternates)
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Major Advocacy Achievements
Shaping EU Rare Disease Policy
Communication from the European Commission to the European
Parliament, the Council, the European Economic and Social
Committee and the Committee of the Regions : « Rare Diseases:
Europe’s Challenge » 11 November 2008
First comprehensive policy text addressing issues faced by RD patients,
from research on RDs, to diagnosis, access to care and adapted services
and development of training, education, awareness on RDs
Council Recommendation on an Action in the field of Rare
Diseases, 8 June 2009
Adopted by EU Ministers of Health
Promote the adoption of RD National Plan/Strategy in EU MS
Creation of the European Union Committee of Experts on Rare Diseases
(EUCERD) in November 2009
Creation of the European Commission Expert Group on Rare Diseases in
July 2013
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Recent Advocacy Achievements
Contribution to EU Directives/ Regulations
EU Directive on Patients’ Rights in Cross-border
Healthcare in 2011
Article 13 specifically on Rare Diseases
Provides the legal framework for European Reference Networks
Provides the legal framework for the mobility of patients across EU
Most recent contributions from EURORDIS to:
The revision of the EU Directive on Clinical Trials 2001/20/EC: The
Directive has become a EU Regulation (adopted by the Council of the
European Union in May 2014)
The proposal from the EC for a EU Regulation on the protection of
individuals with regard to the processing of personal data –
proposal is currently undergoing the legislative process
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Voicing
Rare Disease patients’ expectations
Positioned at the heart of relevant
decision-making committees
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EURORDIS’ REPRESENTATION
IN EXTERNAL NETWORKS, ORGANISATIONS AND INSTITUTIONS IN 2014
EMA
European Medicines Agency
European Commission
COMP
Committee for Orphan
Medicinal Products
PDCO
Paediatric Committee
CAT
Committee for Advanced
Therapies
European and International
Not-for-Profit Organisations
DIA: Drug Information Association
Commission Expert
Group on
Rare Diseases
Commission Expert
Group on
Cancers
EU Health Policy Forum
SAWP
Scientific Advice Working
Party
CHMP
Committee for Medicinal
Products for Human Use
E-Rare
EUROPABIO Patients Advisory Group
ECRIN
EPF: European Patients’ Forum
BBMRI
Stakeholders Forum
IAPO: International Alliance of
Patients’ Organizations
Treat NMD
= Non-Governmental Organisations
EuroBioBank
RD-Connect
Learned Societies:
ICORD: International Conference on
Rare Diseases and Orphan Drugs
Rare Cancer Europe
PBSA: Pan-European Blood Safety
Alliance
Maladies Rares Info Service (French
Helpline for RDs)
Rare Disease Platform in Paris
= Governmental Institutions
EUnetHTA 2
Stakeholders Forum
EFPIA Think Tank: European Federation
of Pharmaceutical Industries and
Associations
IRDiRC: International Rare Disease
Research Consortium
PCWP
Patients’ & Consumers’
Working Party
European Networks:
International Society for
Pharmaco-economics and
Outcomes Research
(ISPOR)
European Federation of
Internal Medicine (EFIM)
European Hospital &
Healthcare Federation
(HOPE) - tbc
International Federation
of Social Workers (IFSW)
-tbc
Involved in EMA Committees
COMP: Committee for Orphan Medicinal
Products
2 EURORDIS representatives (Vice-Chair +
1 full member) + 1 Observer
PDCO: Paediatric Committee
2 EURORDIS representatives (full member & alternate)
CAT: Committee for Advanced Therapies
2 EURORDIS representatives (full member & alternate)
PCWP: Patients' and Consumers' Working Party
2 EURORDIS representatives (full members)
Scientific Advice & Protocol Assistance
CHMP: Committee for Human Medicinal Products
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Involved in Commission Expert Group on
Rare Diseases (former EUCERD)
Brings together European main decision-makers in the field of Rare
Diseases
Objective: Assist the Commission in the drawing up of legal
instruments and policy documents, including guidelines and
recommendations, in the field of rare diseases
Representation: 28 EU Member States + Iceland, Norway,
Switzerland + EC, EMA COMP + industry + academia + individual
experts + patients’ representatives
All 8 patients’ representatives
members of EURORDIS.
They cover main rare disease patient support groups and
different European regions. They coordinate advocacy work
through regular contact.
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Contribution to the 5 Recommendations
EUCERD (Nov. 2009 – July 2013)
Quality Criteria for Centres of Expertise for Rare Diseases
in Member States, October 2011
Rare Disease European Reference Networks (RD ERNs),
January 2013
Clinical Added Value of Orphan Medicinal Products
(CAVOMP) Information Flow, September 2012
Recommendations on Rare Disease Patient Registration
and Data Collection, June 2013
Core Indicators for Rare Disease National Plans/ Strategies,
June 2013
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Involved in Commission Expert Group on
Cancer Control (1st meeting - 23-24 Sept 2014)
• Brings together European main decision-makers in the field of
Cancers
• Objective: Assist the Commission in drawing up legal instruments
and policy documents, including guidelines and recommendations, in
the field of cancers, including rare cancers
• Representation: 28 EU Member States + Iceland, Norway,
Switzerland + EC + industry + academia / cancer prevention +
patients’ representatives
• 6 patients’ representatives
2 members of EURORDIS
they represent rare cancer patient support groups & different
European regions. They coordinate advocacy work together
with the EURORDIS group of volunteers on rare cancers.
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EURORDIS published its position
on Rare Disease Research
2010: Why Research on
Rare Diseases?
2011: Patients’ Priorities
and Needs for Rare
Disease Research 20142020
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Involved in IRDiRC – International Rare
Diseases Research Consortium
200 new therapies
for rare diseases
by 2020
Means to diagnose most rare
diseases by 2020
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Involved in IRDiRC – International Rare
Disease Research Consortium
EURORDIS is a partner of the Consortium
IRDIRC Executive Committee: 1 EURORDIS representative
Therapies Scientific Committee: Yann Le Cam, Elected Chair
Therapies Scientific Committee Working Groups (WG) include
patients’ representatives/ members of EURORDIS
WG on Chemically-derived products including repurposing
WG on Biotechnology-derived products including cell- &
gene-based therapies
WG on Orphan drug development and regulatory processes
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Launch of Rare Diseases International
What is Rare Diseases International (RDI)?
An initiative aiming to create an informal network of rare disease
patient organisations to form a global alliance representing patients &
families of all nationalities across all rare diseases.
Who is involved?
RDI is a EURORDIS initiative with National Alliances around the world
with whom we have signed partnership agreements (MoUs).
Why Rare Diseases International?
To enhance capacities of Rare Diseases International members through
information, exchange, networking, mutual support, joint actions
To represent its members & people living with rare diseases internationally
To promote RDs as an International Public Health & Research priority
through public awareness and policy (+280 million people living with rare
diseases around the world)
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Engaging rare disease patients
in Research & Public Health actions
Partners of major EU Projects
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Promote RDs in European Programmes
Since 2000, EURORDIS has advocated for rare
diseases to be included in research and health
programmes of the European Commission
New programmes
EU Research « Horizon 2020 » (2014-2020)
Innovative Medicine Initiative – IMI 2 (2014-2020)
EU Health for Growth (2014-2020)
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Partnering in the EUCERD Joint Action
“Working for Rare Diseases”
Support for the implementation of RD Plans/ Strategies
EURORDIS & National Alliances co-organise 24 patient-driven
and multi-stakeholders National EUROPLAN Conferences to:
Foster the implementation of RD national plans / strategies
Promote EU reference documents on RD policy and assess their
transferability at national level
Address common issues based on common content guidelines
for the following areas:
Governance of the Plan
Access to Care
Codification/Inventorying
Orphan Drugs
Research
Social Services
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Partnering in the EUCERD Joint Action
“Working for Rare Diseases “
Provision of Specialised Social Services and Integration of
RD into Social Policies and Services
Mapping of Therapeutic Recreation Programmes, Respite Care
Services, Adapted Housing, Resource Centres; collection of
Guiding Principles and Case Studies
Training for Social Services Providers: collection of Guiding
Principles and Case Studies
Integration of Rare diseases into Social Policies and Services:
Report on EUCERD guiding principles for Social Care in RD and
draft of EUCERD recommendations in the social field
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RD-Connect is an integrated platform connecting databases,
registries, biobanks and clinical bioinformatics for rare disease
research
EURORDIS’ main role in the project includes engaging patients
in the governance and operations of the project, particularly
around ethical, legal and social issues important to rare disease
patients
Funded by DG Research: 2012 –2018
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RARE-Bestpractices project
Best practice and knowledge sharing in the clinical
management of rare diseases
A four year project (January 2013-Dec. 2016) funded by the
European Commission.
Objective: Creating a platform to improve the management of
rare disease patients by:
Disseminating trustworthy guidelines globally
Identifying and prioritising rare disease research needs
Facilitating timely, efficient translation of research results into
patient-oriented strategies
EURORDIS acts as a transversal partner in the different work
packages, ensuring the involvement and the capacity-building of
patient organisations across Europe.
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Work together
and learn from each other
Thank you!
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