Conference 1 - EURORDIS’ recommendations for National Plans, Yann le Cam, EURORDIS

1. www.eurordis.org
EURORDIS MEMBERSHIP MEETING
AMSTERDAM, 13 – 14 May 2011
1

2. RECOMMENDATIONS FOR
RARE DISEASE NATIONAL PLANS
Yann LE CAM
Chief Executive Officer, EURORDIS
Vice Chair, EU Committee Experts
on Rare Diseases (EUCERD)

3. TABLE OF CONTENT
• Introduction Strategic messages
• Part 1: Key priorities to be covered
• Part 2: Key elements of methodology
• Part 3: EUCERD and National Plans
• Conclusions Key success factors
3

4. OUR COMMON GOAL
To build a unique
EU integrated,
comprehensive
and sustainable strategy
to address rare disease
patients’ needs
everywhere in Europe
4

5. OUR COMMON LONG TERM VISION
• Rare diseases is a public health priority which is complex and long
term: 30 million patients are or will be affected in the course of their
life by one of 5000 to 8000 rare diseases. It will take several
generations to address these challenges
• Hence, the only possible public policy approach = common
strategy across rare diseases, collaboration between all
interested parties, EU integrated approach, sharing of
intrastructure tools and data so to optimize the European
community added value with an International perspective. Act
local and short term but always with global and long term view.
=> 2008-2013 = 2nd EU Public Health Programme + 7th EU
Research Framework Programme + Commission Communication +
Council Recommendations + National Plans in EU 27 MSs
=> 2014-2020 = 3rd EU Public Health Programme + 8th Research
Framework Programme + EUCERD Recommendations + 2nd
generation of National Plans in EU 28 MSs and beyond in Europe
5

6. INTEGRATED
• Commission Communication + Council Recommendation
+ EUROPLAN project’s outcomes + National Strategies &
Action Plans = a unique opportunity for an integrated strategy
between the European and national levels coordinated by the
EU Committee of Experts on Rare Diseases (EUCERD)
• The Council Recommendation is not an isolated piece of policy =
it provides essential elements for national strategies on rare
diseases which are articulated with other EU Programmes such
as Research and Public Health, other EU Legislations such as
the EU Regulation on Orphan Drugs and the EU Directive on
Cross Border HealthCare, or other EU recommendations such
as the EU Pharmaceutical Forum
=> Each main objective of national strategies and action plans is to
be articulated with these EU Programmes and EU Legislations
6

7. COMPREHENSIVE
• Commission Communication + Council Recommendation +
EUROPLAN project’s outcomes + National Strategies &
Action Plans = a unique opportunity to elaborate
comprehensive research and healthcare pathways for rare
disease patients and families from diagnosis, care, information,
support, research and treatments
• The aim of national strategies and action plans is to address
the needs of patients and families. Each main priority area
supports the other: information is linked to access to diagnosis
which is directly linked to access to experts or centres of
expertise which in turn are supporting research and therapy
development which are correlated to the existence of both patient
registries and patient organisations.
=> National strategies and action plans must be comprehensive
and cover all 5 key priority areas
7

8. SUSTAINABILITY
• National plans to be adopted before December 2013 are only a
start.
• We already need to think about the next plans, the 2nd
generation of plans for 2015-2020
• We need to take a long term approach with clear strategies,
consistent over time, with adequate resources, to achieve
measurable results.
• It’s important to build in the first national plans, the indicators
on measures being implemented, the studies to collect data
about patients & families experience and needs, and the
financial mechanism for sustainable actions
=> 2010-2015 = patients need building blocks for future national
and EU strategies ; Policy and financial sustainability is a must at
EU & national levels
8

9. Part 1
Key priorities
to be covered
in national strategies
and action plans
for rare diseases
9

10. Research
Identify ongoing research projects and existing research
resources + Identify needs and priorities for basic, clinical,
translational and social research
Link these activities with centres of expertise
Encourage the participation of researchers in EU funded project
Create new additional financial resources for research
Allocate resources both to transversal infrastructure across
rare diseases and to disease specific research projects
Cover biomedical, public health and social research
=> Our objective at European level is to obtain the same
approach for EU Research Framework Programmes, to
encourage collaboration between researchers and patient
groups, to promote public-private partnership when relevant,
to put in place new funding mechanisms for the long term
sustainability of research infrastructures such as biobanks,
databases, registries, clinical research infrastructure… because
of the nature of rarity
10

11. Centres of Expertise and European Reference
Networks for Rare Diseases
• Identify Centres of Expertise or Network of Experts or
Experts at national level
• Adopt a multidisciplinary approach + comprehensive
medical & social care + coordination between hospital &
community & home care
• Put in place long-term public funding mechanism for
continued improvement of quality of care
• Support patient mobility across EU (transposition of the EU
Directive on Cross Border Health Care)
=> Our objective at European level is to integrate Centres of
Expertise, Networks of Experts and Experts with Reference
Diagnostic Laboratories and Patient Registries, involving
collaboration with Patient European Networks
11

12. Information and Patient Services
• Use the OrphaCode and the future ICD 11 classification
• Contribute to the inventory of rare diseases
• Support national and regional specific disease information
networks, registries and databases using common approach
across EU
• Raise public awareness: take part in the Rare Disease Day
• Support national rare disease information helplines with a
free number –toward a unique number in EU
• Support web-based information tools such as local Orphanet
services and patient groups web services
• Develop respite care services and therapeutic recreative
programmes for patients and families
=> Our objective is to progressively create European
Networks of Rare Disease Help Lines, Information Centres,
Respite Care Services, Therapeutic Recreative Programmes,
and, to integrate rare diseases into national social policies
12

13. Gathering of expert opinions
• It is essential that each EU 27 MS adopts measures in their
national plans to engage into European gathering of expert
opinions and to refer to common recommendations or common
assessment reports
• Main ones:
- EUCERD recommendations: Centres of Expertise, European
Reference Networks, Population Screening, Diagnostic Tests,
Registries, Training of Social Service Providers, Methodology on
Social Guidelines
- EU Assessment Reports on the Clinical Added Value of Orphan
Drugs (CAVOD)
=> Toward more equal access to high quality care, medicines and to
social rights
13

14. Empowerment of patient organisations
• Patients and families are not only « end users » of national
strategies and action plans = they are carers, health and social
actors, managing complex day to day care all along their life
span
• Patient support & advocacy groups are the best allies to take
an active role in shaping research, heathcare and social national
and regional policies for rare diseases
=> Ensure that patients and patients’ representatives are
involved at each step of the policy and decision-making
processes in the field of rare diseases
=> Ask support for patient groups and rare diseases national
alliances: awareness-raising, capacity building & training,
exchange of information, networking and outreach
14

15. Part 2
Elements of methodology
for the development,
management and evaluation of
national strategies and action
plans on rare diseases
15

16. GOVERNANCE OF THE PLAN
• Steering Committee
• All interested parties, including patient
organisations, industry, national authorithy on
health budget
• To develop the strategy and action plan
• To coordinate and manage its implementation
16

17. CONTENT OF THE PLAN
• A clear written strategy – referring to:
The Commission Communication « introduction », « issue »
and « objectives »
The Council Recommendation « whereas »
The specific national context
• Clear objectives covering each key priority area of
actions
• Well identified measures or deliverables + easy to
understand + assigned to specific competent
authorities or interested parties + possible deadlines
• Measurable activities or results
17

18. BUDGET
• Each priority or objective should have a well identified
budget
• When possible, each measure or deliverable should
have a well identified budget
• Each budget line should have well identified funding
source
18

19. ACCOUNTABILITY & TRANSPARENCY
• The national strategy and action plans should be made
public
• Regular meetings of the national steering committees
• Annual reporting (a) priority areas by priority areas (b)
on each measure or deliverable (c ) on each budget line
• Adjustments of the measures, deliverables,
assignments, deadlines, budgets
• Summary of Steering Committee meetings and Annual
reporting should be made public
19

20. EVALUATION & INDICATORS
• Common indicators should be shared across Member
States
• Evaluation of activities and results should be based on the
adopted strategy and action plans
• Evaluation should include a collection of opinion from all
stakeholders
20

21. PART 3
The EU Committee of Experts on
Rare Diseases (EUCERD)
will help shape the future of rare
disease policies at the EU level
impacting on national policies
21

22. EUCERD
• EU MS/ EEA Countries + candidate countries
• EC/EMA + ECDC + pharma indus. + reps of
EC funded RD research and public health projects
• 8 rare disease patients’ representatives
• Sharing of national experiences, promotion of national best
practices for research, healthcare pathways and social
services (eg: EUCERD Website, EUCERD Workshops,
European Conferences on Rare Diseases & Orphan Products,
Reports “ State-of-the-Art of Rare Diseases in Europe”)
• In the coming years, EUCERD will develop “recommendations”
& “guidelines” & “reports” on different key issues to support
and guide national plans for rare diseases (e.g. centres of
expertise, registries, genetic testing, specialised social
services…)
22

23. National Plans in EU: an incremental process
EC Commission on Rare Diseases
EU Council Recommendation on Rare Diseases
EUROPLAN 2008-2011 National EUROPLAN 2012-2014
Plans on & Reports « State of
Recommendations + Rare
Monitoring Indicators the Art of Rare Diseases
Diseases in Europe »
EUCERD
Recommendations &
Guidelines
23

24. Present / Future: Integration & Cooperation
at Country AND Thematic Level
Commission Communication
€ / CZK / £ …
Centres
of Expertise
EUCERD
Registries
€
Diagnosis
Treatment
Council Recommendation

25. 1.What is the
EUCERD?

26. CONCLUSION : KEY SUCCESS FACTORS
Focus on essential building blocks
Keep sight of the integrated, comprehensive
and long term strategy
Follow up (get involved) and implement the
EUCERD Recommendations
Collect your national indicators
Build-in the sustainability and think on which
base you will promote your 2nd national plan
26

27. CONCLUSION: KEY SUCCESS FACTORS
• National plans and strategies can only address
part of the needs of 30 million patients affected by
all rare diseases for which there are no cure
today!
• By joining our forces and going in the same
direction based on common strategies, we can
already improve the lives of million of people living
with rare diseases in the next five years and much
more within next ten years
27

28. EURORDIS Concrete tools & policy
• Where can I find information about national plans from all Member
states (regularly updated)?
- Section « Rare Disease Policy » / « National Policy »
- => www.eurordis.org
- Section « Rare Disease Policy » / « EU Policy »
- Information in English
• Where can I find practical tools to support your advocacy work?
- => www.eurordis.org
- Policy Fact Sheets
- Ex: Research, Centres of Expertise & European Network, Registries,
Help Lines, Orphanet, Respite care Services, Access to Orphan Drugs…
• Where to exchange information and experiences to build my
capacities as a patient advocates for national plans?
- Eurordis Membership Meeting 2011 Amsterdam 13-14 May (&2013)
28

29. www.eurordis.org
Thank you!
29

30. 30