Sesion 2 terkel 14 05 2010 (final)

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  • Because rare diseases are life-threatening or chronically debilitating diseases with a low prevalence and a high level of complexity : Need for a global approach based on special and combined efforts. Integrating all current and future initiatives at Regional and National levels Because of the limited number of patients and scarcity of relevant knowledge and expertise Rare diseases are a unique domain of very high European added-value
  • The history of RD’s in Europe is short when looking at the ”foundig documents” of action in this field
  • In fact the european action has been nourished by a number of national actions in european countries. Norway was definetely a pioneer and an inspriation to collaborative projects and initiatives in neighbouring countries. Norway: RD priority area from 1990 to 1997. Individual plans for service provision Denmark, 1993: establishment of 2 specialised centres for rare diseases. 2001: Recommendations of the Danish Board of health to define their responsibilities Sweden : Information Centre, and National Competence centre in Agrenska France created Orphanet in 1997 which now holds information on 5,200 diseases, has 20,000 daily visitors, lists 1,233 clinical laboratories offering testing for 1 504 diseasesfrom 170 countries2,003 research laboratories conducting conducting 4,198 projects on 2,040 diseases, 1,739 patients’ organisations, 10,302 professionals 521 drugs with an indication for a rare disease Spain created its first information resource sistem in 1997 Italy, 2001: rare diseases become a health care priority in the 3 years National health plan : national network for prevention, surveillance, diagnosis and treatment 21 regional governments carry the responsibility of actual implementation : over 250 regional centres designated National and regional registries Netherlands: 2001: steering committee on orphan drugs, national web-based facilitating registry
  • Main objective: ensuring access to high quality care, including diagnostics, treatments, habilitation for those living with the disease and, if possible effective orphan drugs on the basis of equity and solidarity 2005-2008 : France. 131 centres of reference designated and funded, development of Orphanet, help line, research networks and projects… Soon, publication of a second comprehensive plan 2010-2013, to consolidate progress and address unsolved issues in the 1st plan : structure networks of diagnostic labs and centres of expertise, research, information systems, improve information and proximity care… 2005 : Luxembourg. creation of a task force on rare diseases 2008 : Bulgaria. national register, improved diagnosis, training of professionals, awareness raising, clinical services, treatments… Romania. National committee with all stakeholders establish a strategy Portugal. national network of treatment centres, training, data collection 2009 : Spain. National strategy, designation of national and regional centres of reference, national registry
  • KEY FINDINGS: Patient Organisations (POs) show a high interest for research POs have a high commitment to research despite great differences in terms of number of members and budget POs show strong will to collaborate with researchers , not only by triggering encounters between clinicians, researchers and patients, but also by giving them logistical and financial support POs in Europe have devoted a total minimum of 13 million euros to research last year (not including funding from the Association Française contre les Myopathies – AFM - which gives on average 60 million euros to research every year) PATIENTS FUND RESEARCH 37% of POs funded research in the last 5 years Amongst them, half spend more than 30 000 euros yearly and one quarter spend more than 112 000 euros a year. Some devote more than 100% of their budget, which means they organise fundraisers specifically for research. POs who are 10 years or older spent about half of their budget on research Amongst those POs who funded research: •    70%  had not only funded a specific research project but had also initiated it. •    75%  had helped fund the operating budget of a research project. •    54% bought equipment and 47% had granted fellowships for young researchers WHAT KIND OF RESEARCH DO PATIENTS FUND? POs fund mostly basic research. Patients are naturally interested in all areas of research since they are concerned by many areas of research (genetics, therapeutics, social, etc) 1 out of 2 patient organisations fund basic research and 1 out of 3 fund human and social science research. Contrary to the common belief that patients only support therapeutic research, it is clear that patient groups understand and invest in long-term research projects, as well.
  • Strong patient groups, speaking with one voice in National federations or Alliances Awareness raising of the public: TV shows such as telethon in France and Italy, media campaigns, Rare Diseases Day Studies establishing the state of the art: EurordisCare study at EU level, X in Germany, ENseRio in Spain Assessment of the bottlenecks in the care of people with a rare disease, of the main patients’ needs and of possible solutions National committees with a representation of all stakeholders (Netherlands, Luxembourg, Romania..): a small group of dedicated people, including civil servants can move mountains
  • Need to stimulate national scientific societies to organize working groups Adaptation of guidelines produced by other MS.
  • Sesion 2 terkel 14 05 2010 (final)

    1. 1. The Dynamic of National initiatives for Rare Diseases Where are we? Krakow, 14 May 2010 www.eurordis.org Terkel Andersen President of EURORDIS
    2. 2. If this was intellectual capital…
    3. 3. Rare diseases, a European public health priority <ul><li>Rare diseases have been recognized as a unique domain of very high European added-value: </li></ul><ul><li>Rare diseases are life-threatening or chronically debilitating diseases with a low prevalence and a high level of complexity </li></ul><ul><li>The limited number of patients calls for transnational cooperation </li></ul><ul><li>Scarcity of relevant knowledge and expertise warrants intelligent solutions </li></ul><ul><li>The total number of RD patients constitutes a significant welfare issue </li></ul><ul><li>Research and Development in Rare diseases will contribute to European welfare and economic growth </li></ul>
    4. 4. Europe’s policy framework for rare diseases : founding texts <ul><li>Regulation of the European Parliament and of the Council of </li></ul><ul><li>on orphan medicinal products </li></ul><ul><ul><li>Adopted on 16 December 1999 </li></ul></ul><ul><ul><li>2000: Creation of the Committee for Orphan Medicinal Products at the European Medecine Agency </li></ul></ul><ul><li>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 » </li></ul><ul><ul><li>Adopted on November 11th, 2008 </li></ul></ul><ul><li>Council Recommandation on an action in the field of rare diseases </li></ul><ul><ul><li>Adopted on June 9th, 2009 </li></ul></ul><ul><ul><li>30 november 2009: Decision to create the European Union Committee of Experts on Rare Diseases </li></ul></ul><ul><ul><li>A road map for the implementation </li></ul></ul>
    5. 5. 1980- 2005 First National Initiatives in Europe <ul><li>Norway: RD initiatives from the 1980’s. Priority area from 1990 to 1993. Individual plans for service provision; Today 16 national centers </li></ul><ul><li>Nordic collaboration on Rare Diseases 1984-1989 </li></ul><ul><li>Denmark, 1990: Info service (CSH). Establishment of 2 specialised centres for rare diseases. 2001: Recommendations of the Danish National Board of Health </li></ul><ul><li>Sweden, 1990 : Information Centre, and Competence centre in Agrenska </li></ul><ul><li>France, 1997: Creation of OrphaNet </li></ul><ul><li>Spain, 1999: Sistema de Información sobre Infermedadas Raras </li></ul><ul><li>Italy, 2001: rare diseases become a health care priority in the 3 years National health plan : national network for prevention, surveillance, diagnosis and treatment: over 250 regional centres designated & National and regional registries </li></ul><ul><li>Netherlands: 2001: steering committee on orphan drugs, </li></ul><ul><li>national web-based facilitating registry </li></ul>
    6. 6. 2005 – 2009 First National plans : <ul><li>National plans: ensuring access to high quality care, including diagnostics, treatments, habilitation for those living with the disease and, if possible effective orphan drugs </li></ul><ul><li>France 2005-2008 : France. 131 centres of reference designated 2010-2013: Second comprehensive plan, to consolidate progress </li></ul><ul><li>Luxembourg, 2005: creation of a task force on rare diseases </li></ul><ul><li>Bulgaria, 2008: national register, improved diagnosis, training of professionals, awareness raising, clinical services, treatments… </li></ul><ul><li>Romania. National committee with all stakeholders establish a strategy </li></ul><ul><li>Portugal. national network of treatment centres, training, data collection </li></ul><ul><li>Spain. National strategy, designation of national and regional centres of reference, national registry </li></ul>
    7. 7. European Initiatives <ul><li>DG Public Health and Consumers: </li></ul><ul><li>1999-2003: First Community Action Programme funding projects and services </li></ul><ul><li>2003-2008 EU Public Health Programme (funding e.g. Rapsody) </li></ul><ul><li>2004: Rare Disease Task Force: OrphaNews </li></ul><ul><li>WG: Standards of Care – Centers of expertise & European Reference Networks </li></ul><ul><li>WG Coding and Classification – Revision of ICD-10 </li></ul><ul><li>WG Public Health Indicators – Databases and Registries </li></ul><ul><li>2008-13 Second Community Action Programme </li></ul><ul><li>Exchange of information and transnational cooperation </li></ul><ul><li>Funding of projects: E.g.: Evaluation of Newborn screening practices </li></ul><ul><li>Annual workshops and the ECRD conferences </li></ul>
    8. 8. European Initiatives <ul><li>DG Enterprise and Industry & EMA : </li></ul><ul><li>2000 COMP </li></ul><ul><li>2007 Regulation on Medicinal Products for Pediatric Use </li></ul><ul><li>2009 Committee for Advanced Therapies (CAT) </li></ul><ul><li>2010 EMA is transfered to DG Health </li></ul>
    9. 9. European Initiatives <ul><li>DG Research </li></ul><ul><li>European Commission’s Framework Programme for R&D (FP) – Estimated budget allocated directly to rare diseases or for projects potentially useful for rare diseases (e.g. fundamental research on genetic therapies or cell therapies): </li></ul><ul><ul><li>6th Framework Programme (FP6) – years from 2002 to 2007 (entire programme duration) = 230 million EUR </li></ul></ul><ul><ul><li>7th Framework Programme (FP7) – years from 2008 to 2009 – approx. 80 million EUR (FP7 is still ongoing, it will end in 2013) </li></ul></ul><ul><ul><li>Patient organisations support to RD research 2009: a minimum of 13 mio euros </li></ul></ul>
    10. 10. How did it become possible? <ul><li>Strong patient groups, speaking with one voice in National federations or Alliances . The growing impact of Eurordis’ initiatives. </li></ul><ul><li>Awareness raising of the public: TV shows such as telethon in France and Italy, media campaigns, The Rare Disease Day </li></ul><ul><li>Studies establishing the state of the art: EurordisCare study at EU level, X in Germany, ENseRio in Spain </li></ul><ul><li>Assessment of the bottlenecks in the care of people with a rare disease, of the main patients’ needs and of possible solutions </li></ul><ul><li>National committees with a representation of all stakeholders (Netherlands, Luxembourg, Romania..) </li></ul><ul><li>Dedicated people and pioneers can move mountains! </li></ul>
    11. 11. Europe’s overall strategy for rare diseases <ul><ul><li>Plans or strategies are to be established and implemented “preferably by the end of 2013”. </li></ul></ul><ul><ul><li>The health authorities of the 27 EU member states (MS) signed the Council Recommendation, stating their willingness to fulfil this deadline. </li></ul></ul><ul><ul><li>National strategies or plans will feed the European dynamics across member-states and future European policies </li></ul></ul>
    12. 12. 6 main priorities for National strategies or plans : <ul><li>Adequate definition, inventory and codification of rare diseases </li></ul><ul><li>Research </li></ul><ul><li>Centres of Expertise and EU Reference Networks </li></ul><ul><li>Gather expertise at EU level: protocols and guidelines </li></ul><ul><li>Empowerment of patient organisations </li></ul><ul><li>Sustainability </li></ul>
    13. 13. Europe’s active networks <ul><li>EUROPLAN project </li></ul><ul><ul><li>Draft recommendations for the development of national plans for rare diseases : The Guidance document </li></ul></ul><ul><ul><li>Selected indicators to evaluate the achievements of RD initiatives (WP5) </li></ul></ul><ul><ul><li>15 National conferences in 2010 organised by the National Alliances and EURORDIS </li></ul></ul>
    14. 14. Some ongoing initiatives <ul><li>Hungary: 2008 National Centre for Rare Disease </li></ul><ul><li>Poland: 2008 National Committee for rare diseases </li></ul><ul><li>National plans under preparation in Austria, Cyprus, the Czech Republic, Finland, France (second plan) Germany (National working group established), Greece, Ireland, Italy, Lithuania (action group), Malta, UK (5 working groups), Turkey… </li></ul><ul><li>Cross border cooperation in Central Europe </li></ul><ul><li>Council of National Alliances of EURORDIS : 2 workshops/year, regular exchange of information, advocacy fact sheets. </li></ul>
    15. 15. CoE’s & ERN to promote standards of care <ul><li>Clinical Guidelines </li></ul><ul><li>” The availability of evidence-based clinical guidelines in the field of rare diseases is extremely scarce” </li></ul><ul><li>Guidance document (5.4) </li></ul><ul><li>Care Pathways </li></ul><ul><li>” A methodology for the mutual decision making and orgnization of care for a well-defined group of patients during a well-defined period” </li></ul><ul><li>Selecting indicators to evaluate the achievements of RD initiatives (Area 4: Centers of Expertise) </li></ul>
    16. 16. Main challenges <ul><li>1. Decentralised health care systems </li></ul><ul><li>2. Insufficient resources in all countries </li></ul><ul><li>3. Need for supportive policies at EU level </li></ul><ul><li>4 Further development of cross national collaboration </li></ul><ul><li>5. Sustainability </li></ul><ul><li>6. Measuring the real impact on patients’ quality of life and health </li></ul>
    17. 17. 1. Decentralised health care systems <ul><li>How to make sure that strategies decided at national level have an impact on patients every day life? </li></ul><ul><li>Will centers of expertise catalyze or monopolize knowledge? </li></ul><ul><li>will national health strategies be translated into concrete actions in the regions of Italy, in autonomous communities in Spain, in all German Länders,, in the 24 Swiss cantons….? </li></ul><ul><li>Development of specialised social services may take even longer: help lines networking, respite care, support to carers… (one of the identified issues of the 2 nd French plan) </li></ul><ul><li>What about very rare diseases without patient groups? There is a high risk they become « super-orphans » among orphans. </li></ul>
    18. 18. 2. Need for pooling resources <ul><ul><li>Limited number of diagnosis available in each country: 1928 rare diseases can be diagnosed in France, 1650 in Germany, 1479 in Italy (source: orphanet) </li></ul></ul><ul><ul><li>Limited number of centres of expertise </li></ul></ul><ul><ul><li>Limited number of good practice guidelines </li></ul></ul>
    19. 19. Need for supportive policies at EU level <ul><li>EU directive on cross boarder health care and patient mobility : legal base and funding for EU networks of centres of expertise </li></ul><ul><li>3rd EU Public Health Programme </li></ul><ul><li>8th EU Research and Technology Framework Programme 2014-2020 </li></ul><ul><li>EU pharmaceutical legislations revisions </li></ul><ul><li>Revision of the directive on clinical trials </li></ul><ul><li>EU policies on organ donation and transplantation, gene testing and counselling, neonatal screening… </li></ul>
    20. 20. Sustainability <ul><li>5 or 10 year strategies and action plans will only partially address the needs of 30 million patients affected by rare diseases for which there currently is no cure </li></ul><ul><li>Necessity to build sustainability into National Plans and strategies as much as possible: Centres of Expertise, proper funding of complex clinical pathways, information and clinical research infrastructures: databases, registries, biobanks… </li></ul><ul><li>Support co-operation between Member States (ex: E Rare) and active participation in Reference Networks </li></ul><ul><li>Necessity to build sustainability at European level : information networks, patient associations networks, EU Reference Networks of specialised centres, databases and biobanks, other basic and clinical research infrastructures… </li></ul>European Conference on Rare Diseases, May 14, 2010, Krakow, Poland
    21. 21. A recipee for success <ul><li>will be a long-lasting process involving watchful patient groups </li></ul><ul><ul><ul><ul><li>(20) The WHO defined empowerment of patients as a ‘pre-requisite for health’ and encouraged a ‘proactive partnership and patient self-care strategy to improve health outcomes and quality of life among the chronically ill’. In this sense, the role of independent patient groups is crucial both in terms of direct support to individuals living with the disease and in terms of the collective work they carry out to improve conditions for the community of rare disease patients as a whole and for the next generations. </li></ul></ul></ul></ul><ul><ul><ul><ul><li>(21) Member States should aim to involve patients and patients′ representatives in the policy process and seek to promote the activities of patient groups. </li></ul></ul></ul></ul><ul><li>Council Recommendation of 8 June 2009 on an action in the field of rare diseases </li></ul>European Conference on Rare Diseases, May 14, 2010, Krakow, Poland
    22. 22. The need for a combined and global effort <ul><li>Rare diseases will continue to be a serious challenge to the health and welfare of EU citizens for decades to come: Continuity is a precondition for success. </li></ul><ul><li>No Member State will be able to manage this challenge alone </li></ul><ul><li>By their complex nature Rare Diseases will be a set off for increased collaboration and innovation both in science and technology as in provision of services </li></ul><ul><li>Rare Diseases will be a test case for a modern approach to disease management leading to comprehensive, intelligent and empowering solutions </li></ul><ul><li>In their own interest Member States must pool resources and support collaboration. EU must secure continuity. </li></ul><ul><li>Patient organisations will work actively to support creation of synergies, promote idea generation and maximise available resources and assess outcome! </li></ul><ul><li>Let us prepare for the future – failure is not an option! </li></ul>

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