THE ADDED VALUE OF CENTERS OF EXPERTISE

FOR RARE DISEASES

ECRD 2010 KrakowMay 14, 2010

Yann LE CAMChief Executive Officer

www.eurordis.org

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Content Outline

• Intro: Centres of Expertise in National Plans/Strategies

• Centres of Expertise are important to patients The issue: Patients’ access to diagnosis & care and their expectations The Declaration of Common Principles

• Policy elements on Centres of Expertise The Policy Fact Sheets The EU Policy Framework The definition of Centres of Expertise The criteria to identify, select and evaluate Centres of Expertise The organisation of healthcare pathways The policy indicators for Centres of Expertise

• The Added Value of Centres of Expertise Improving quality healthcare services to rare disease patients A key determinant for research on rare diseases Optimizing healthcare resources

• Conclusion: 4 Challenges ahead of us

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INTRODUCTION

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The principles of social justice and solidarity

• The most isolated patients & families,

lost in the maze of the healthcare systems,

cumulating health social and financial

vulnerabilities, desperate to be orphans

require special attention

and enhanced public health actions

• Council Conclusions on Common Values and Principles in European Union Health Care Systems:

Member States share a common commitment to ensuring universal access to high quality healthcare on the basis of equity and solidarity

These principles call for accrued action in favour of more vulnerable groups, like patients with rare diseases

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Centres of Expertise in National Plans / Strategies

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• Centres of Expertise & European Reference Networks are now key concepts of the EU Strategy on rare diseases

• Centres of Expertise are one of the six pillars for national plans / strategies – (the main one?)

• Centres of Expertise and their networking at regional & national & European levels are forming the backbone of a public health policy for rare diseases

The concepts of Centres of Expertise & European Reference Networks of Centres of Expertise have been analysed, developped, debated since 2005 … and continue to be, while being progressively adopted in several Member States and in the EU policy.

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CENTRES OF EXPERTISE ARE IMPORTANT TO PATIENTS

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Patients’ needs: access to diagnosis & care

• Centres of Expertise are not a fantasy or the latest trend: they are addressing needs expressed by patients, families and carers

• Centres of Expertise or Specialised Services or Centres of Reference for Rare Diseases have been created progressively in several Member States specificaly to address the patients needs so to improve their access to timely quality diagnosis and care

• Several studies brought to the surface

the outstanding delays, with years of

quest for diagnosis, high level of initial

misdiagnosis, leading to inappropriate

treatments, suffering and money wasting

• Other studies provided evidence of the

complexity of rare disease care, with 9.4

average different medical services needed

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Patients’ expectations

• Centres of Expertise shall: Be places where patients feel welcome and safe Be places where patients are received by knowledgeable and

understanding professionals Facilitate and improve the autonomy

of the patient Provide access to social assistance,

which respond to the special needs

of the disease Actively involve patients & their

patient representatives in the

establishment, management and

evaluation of the centre

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The Declaration of Common Principles

• The EURORDIS’ « Declaration of Common Principles on Centres of Expertise and European Reference Networks for Rare Diseases »

• Adopted in November 2008

• After 2 years of collaborative work

throughout all European Union: Membership Meeting 2006 in Berlin EurordisCare surveys – 6 000 respondents 12 national workshops & a European

Workshop in Prague 2007 - with 370

rare disease patient groups, experts and

policy makers Membership Meeting 2008 Copenhagen

• 15 key principles on two pages!

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POLICY ELEMENTS ON CENTRES OF EXPERTISE

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The EURORDIS Policy Fact Sheets

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The EU Policy Framework

• Commission Communication COMM(2008) 679 Final on Rare Diseases: Europe’s challenges

« Member States share a common commitment to ensuring universal access to high quality healthcare on the basis of equity and solidarity » «  in particular through identifying national and regional Centres of Expertise »

and « Centres of Expertise may also have an essential role in developping or facilitating specialised social services which will improve the quality of life of people living with a rare disease »

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The EU Policy Framework

• Council Recommendation on an Action in the Field of Rare Diseases (2009)

« The principle and overarching values of universality, access to good quality care, equity and solidariity are of paramount importance for patients with rare diseases »

Recommends that Member States:– Identify appropriate Centres of Expertise throughout their territory by End 2013

and consider supporting their creation– Foster the participation of Centres of Expertise in European Reference

Networks while respecting the national competences and rules– Encourage a multidisciplinary approach to care of rare diseases– Organise healthcare pathways for patients – Support the use of information & communication technologies to ensure distant

access

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The Definition of Centres of Expertise (CoE)

• Specialising in a single rare disease or a group of rare diseases

• Providing a multi-disciplinary approach with specialised competences

• Managing multi-disciplinarity in a coordinated manner

• Offering ranges of accurate services: consultation, medical examinations, specialised equipment, genetic councelling and access to social services

• Participating in research on protocols of diagnosis and care

• Participating in drug clinical trials

• Updating and increasing their expertise

• Providing guidelines on most appropriate

care for diagnosis and treatments,

integrating social and medical aspects

• Engaging into training of professionals

• Disseminating information

• Joining in European Reference Networks

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European Reference Network (ERN)

• A European Reference Network of Centres of Expertise:

Is the physical or virtual networking of knowledge and expertise of regional & national CoE in more than one country

Aim to improve overall quality and provision of care for one rare disease or a group of rare diseases

Provide added value to the existing services and expertise at national level

Promote sharing of expertise and mobility of experts rather than mobility of patients

Permit, facilitate the travelling of patients across-borders to other centres when necessary

Patients in every European country can benefit from a ERN, although CoE are not necessary in every European country

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The main criteria to identify and select Centres of Expertise

• Two essential pre-conditions: Professional qualification: both clinical and scientific experience; proven

qualification documented by publications, grants, pre-existing accreditation Commitment to cooperate and share information

• Patient access to a multi-disciplinary team of experts

• Reported volume of relevant activity

• Coordination between professionals

• A global approach integrating medical and social aspects (comprehensive or holistic)

• Participation in research activities at European and international level

• Education, information and communication outreach activities with primary healthcare professionals and possibly the public

• Training activities for healthcare professionals

• Activities to involve patients and collaboration with patient organisations

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The evaluation of Centres of Expertise

• Evaluation of CoE are not an option but a fundamental element of any regional or national policy on CoE

• Methodological approach for the evaluation of CoE are proposed in the DG SanCo Rare Disease Task Force Report of 2006

• Hard Values & Soft Values

• Common approaches on evaluation of CoE across Member States are necesssary for future progressive integration into ERN

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The organisation of Healthcare Pathways

• Clinical/care pathways are a methodology for the mutual decision making and organisation of care for a well defined group of rare disease patients during a well-defined period

• The aim is to enhance the quality of care

by improving patient outcomes,

promoting patient safety,

increasing patient satisfaction,

and optimising the use of resources

• Characteristics of care pathways: Explicit statement of goals and key elements

of care based on evidence, best practices,

and patient expectations Facilitation of communication, coordination of

roles of the multi-disciplinary team, patients,

and relatives Documentation, monitoring, evaluation of outcomes and variances Identification of appropriated resources

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The Policy Indicators for Centres of Expertise

• EuroPlan Indicators for national plans / strategies:

Existence of a policy establishing CoE at the national/regional level

Number of CoE adhering to the policy defined in the country

CoE adhering to the standards defined by the Council Recommendations –paragraphe d) preambule

Groups of rare diseases followed up in CoE

Participation of national/regional CoE into ERN

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THE ADDED VALUE OF CENTRES OF EXPERTISE

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Improving quality Healthcare Servicesfor Rare Disease Patients

• The consistent identification, establishment, designation, financial support , and evaluation of CoE throughout European countries allows patients and local health care providers to do the best use of high quality services and to benefit from the concentrated expertise by bringing it at the local level

• The European Commission and the EU High Level Group of Medical Care & Health Services have identified ERN of CoE for Rare Diseases as having a high European Added Value

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The first added value of Centers of Expertise is to provide timely access to correct diagnosis and most appropriate care practices

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A key Determinant of Research on Rare Diseases

• The co-existence of patients groups + a registry + a European network of CoE or of Research have been identified as the 3 key determinants for research on a rare disease or a group of rare diseases

• ERN is conditionned to the existence of national/regional CoE

• Registry - or patient database - is enhanced by the existence of CoE

• Clinical trials as well as research on diagnosis & treatment protocols are enhanced by the existence of CoE and ERN of CoE

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Optimizing Healthcare Resources

• CoE reduces the costs in healthcare systems by contributing to shorter delays in diagnosis, decrease of waste due to misdiagnosis, reduction of incorrect drug prescription or medical interventions, less adverse consequences and health debilitation, and more adequately adapted care

• Identification of CoE allows national health authorities to do the best allocation of financial resources to support the additional activities linked to the contractual commitments of the CoE

• Encourage travels of expertise rather than patients themselves

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CONCLUSION

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Four Challenges ahead of us

• Effective policy on CoE in all Member States + adequate financial support + built-in sustainability

• Healthcare pathways for better diagnosis and care for RDs articulating local/regional level of proximity with national level, linked to other measures in national plans such as information & training of professionals, information to patients & families & carers through national helplines, orphanet local branch and support to patient groups

• A coordinated approach at European level for (a) robust common set of criteria for identification and evaluation, (b) integration into European Reference Networks, (c ) mutualisation of expertise to develop standard of diagnosis and care

• Progressive coverage of all rare diseases, including the very and extremely rare diseases, with treatments or without treatments

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YES, WE CARE !

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… and YES, WE CAN !

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