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EURORDIS MEMBERSHIP MEETING

AMSTERDAM, 13 – 14 May 2011

www.eurordis.org

RECOMMENDATIONS FOR

RARE DISEASE NATIONAL PLANS

Yann LE CAM

Chief Executive Officer, EURORDIS

Vice Chair, EU Committee Experts on Rare Diseases (EUCERD)

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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

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OUR COMMON GOAL

To build a unique EU integrated,

comprehensive and sustainable strategy to address rare disease

patients’ needs everywhere in Europe

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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 betwee n all interested parties, EU integrated approach, sharing of intrastructure tools and data so to optimize the Eu ropean community added value with an International perspec tive. Act local and short term but always with global and lon g 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

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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 Direct ive on Cross Border HealthCare, or other EU recommendatio ns 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

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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 prior ity 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

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SUSTAINABILITY

• National plans to be adopted before December 2013 a re 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 str ategies, consistent over time, with adequate resources, to a chieve measurable results.

• It’s important to build in the first national plans , the indicators on measures being implemented, the studies to colle ct 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

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Part 1

Key priorities to be covered

in national strategies and action plans for rare diseases

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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

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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 hospit al & 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 Ref erence Diagnostic Laboratories and Patient Registries, inv olving collaboration with Patient European Networks

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Information and Patient Services

• Use the OrphaCode and the future ICD 11 classificat ion• 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 Disea se 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 Europea n Networks of Rare Disease Help Lines, Information Ce ntres, Respite Care Services, Therapeutic Recreative Progr ammes, and, to integrate rare diseases into national socia l policies

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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, Euro pean

Reference Networks, Population Screening, Diagnosti c Tests, Registries, Training of Social Service Providers, M ethodology on Social Guidelines

- EU Assessment Reports on the Clinical Added Value of Orphan Drugs (CAVOD)

=> Toward more equal access to high quality care, m edicines and to social rights

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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 alli es 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-m aking 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

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Part 2

Elements of methodology for the development,

management and evaluation of national strategies and action

plans on rare diseases

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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

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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 deadli nes

• Measurable activities or results

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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

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ACCOUNTABILITY & TRANSPARENCY

• The national strategy and action plans should be ma de public

• Regular meetings of the national steering committee s

• Annual reporting (a) priority areas by priority are as (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

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EVALUATION & INDICATORS

• Common indicators should be shared across Member States

• Evaluation of activities and results should be base d on the adopted strategy and action plans

• Evaluation should include a collection of opinion f rom all stakeholders

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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

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EUCERD

• Sharing of national experiences, promotion of natio nal best practices for research, healthcare pathways and soc ial services (eg: EUCERD Website, EUCERD Workshops, European Conferences on Rare Diseases & Orphan Prod ucts, Reports “ State-of-the-Art of Rare Diseases in Europe ”)

• In the coming years, EUCERD will develop “recommend ations” & “guidelines” & “reports” on different key issues to support and guide national plans for rare diseases (e.g. ce ntres of expertise, registries, genetic testing, specialised social services…)

• 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

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National Plans in EU: an incremental process

National Plans on

Rare Diseases

EC Commission on Rare DiseasesEU Council Recommendation on Rare Diseases

EUROPLAN 2012-2014& Reports « State of

the Art of Rare Diseasesin Europe »

EUCERDRecommendations &

Guidelines

EUROPLAN 2008-2011Recommendations + Monitoring Indicators

Centresof Expertise

Diagnosis

Treatment

Registries

EU

CE

RD

Present / Future: Integration & Cooperationat Country AND Thematic Level

€ / CZK / £ …

Com

mis

sion

Com

mun

icat

ion

Council Recommendation

€

1.What is the EUCERD?

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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

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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

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EURORDIS Concrete tools & policy

• Where can I find information about national plans from all Memberstates (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 buil d mycapacities as a patient advocates for national plans?

- Eurordis Membership Meeting 2011 Amsterdam 13-14 May (&2013)

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Thank you!

www.eurordis.org

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