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www.eurordis.org




               12o Convegno
    Patologia Immune e Malattie Orfane
              “Malattie Rare: Le R...
www.eurordis.org




« L’Esperienza di Eurordisquot;

      Michele Lipucci di Paola
EURORDIS IN BRIEF

    Founded in 1997
    Patient-driven
    Non governmental - Not for profit

    > 345 member organisa...
EURORDIS’ PRESENCE IN EUROPE
                             345 members
                            in 38 countries
        ...
EURORDIS’ MISSION

    To build a strong pan-European community of
    patient organisations and people living with rare
 ...
WHAT IS A RARE DISEASE?


    A rare disease in Europe is a disease affecting
              less than 1 in 2,000 citizens
...
CHARACTERISTICS OF RARE DISEASES



    80% genetic diseases
    Patients are few and geographically
    spread
    Releva...
PRIORITY 1: NETWORKING &
               INFORMATION SHARING
    More than 345 members in 38 countries

    Council of 13 N...
PRIORITY 2: ADVOCACY &
                  POLICY DEVELOPMENT
    Contribution to the adoption of the EU Regulation on Orpha...
EURORDIS IS PRESENT IN MANY EUROPEAN
         INSTITUTIONS AND PLATFORMS

 European Institutions:

     Committee for Orph...
PRIORITY 3: ACCESS TO INFORMATION,
        DIAGNOSIS, TREATMENT & CARE


     Guidelines (9 languages) to support creation...
PRIORITY 4:
     THERAPEUTIC DEVELOPMENT & RESEARCH
     Contribution to transparent and quality information on
     medic...
EUROBIOBANK


        European network of biobanks of DNA,
       cells and tissue for rare disease research



     Budge...
EUROBIOBANK

     30 standard operating procedures
     A web-based catalogue displaying 971
     sample collections
     ...
TRAINING SESSIONS FOR PATIENTS


     ‘Providing information on rare
     diseases’
     ‘Searching for validated biomedic...
THE ‘EURORDISCARE’ PROGRAMME


     A series of surveys to compare access to care for
            rare disease patients ac...
THE ‘EURORDISCARE’ PROGRAMME


     2004: Compare needs and access to care
        6 rare diseases, 17 countries
        5...
THE ‘EURORDISCARE’ PROGRAMME

     2006:
     Compare experience and expectations of patient and families
     on health c...
ONLINE PATIENT COMMUNITIES

     ‘Mailing Lists for Patients with Rare Diseases’: a pilot

       4 lists as of July 2006
...
THE RARE DISEASE PATIENT
                SOLIDARITY PROJECT
     Creation / development of services & networks for people ...
Other Projects


     Europlan 2008-2011
      • Project leader: D. Taruscio, CNMR/ISS
      • 25 countries
      • Object...
EURORDIS PATIENT REPRESENTIVES IN ODs

     • Patient groups play a key role in policy development:
            EU Regulat...
STATUS OF ORPHAN APPLICATIONS


       893 Applications for orphan medicinal products

       577 Designations of orphan m...
Availability of OMPs by country in 2007

                  Availability for OMPs approved before
                    2004 ...
EURORDIS’ FUNDING SOURCES

                                               Total Revenue =
                                ...
CONCLUSION



     Empowerment of Patients: “ the process of increasing capacity of individuals or
     groups to make cho...
Rare Disease Day 2009



     • Date:    28 February 2009


     • Logo:




     • Coverage Europe, Canada, USA, Argentin...
Theme + Focus 2009



     •Theme: (2008-2011)

            • Rare Diseases: a Public Health Priority


     •Focus 2009 :...
European Event
                                 March 2009
     European Patron: Commissioner Androulla Vassiliou
     • P...
www.rarediseaseday.org


     •




30
www.rarediseaseday.org




31
www.eurordis.org




                 Acting Together
               For the Challenge
           To Build a EU Coordinati...
FOR MORE INFORMATION


     Michele Lipucci di Paola
      Former Vice President of Eurordis
      Board Member Pan-Europe...
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2009 Convegno Malattie Rare Lipucci Di Paola [22 01]

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Transcript of "2009 Convegno Malattie Rare Lipucci Di Paola [22 01]"

  1. 1. www.eurordis.org 12o Convegno Patologia Immune e Malattie Orfane “Malattie Rare: Le Reti” Torino 22 Gennaio 2009 PHOTO
  2. 2. www.eurordis.org « L’Esperienza di Eurordisquot; Michele Lipucci di Paola
  3. 3. EURORDIS IN BRIEF Founded in 1997 Patient-driven Non governmental - Not for profit > 345 member organisations in 38 countries ( 25 EU ) > 1.000 rare diseases covered 18 staff in Paris and Brussels 120 volunteers involved in task forces, steering committees, working groups and the Board of Directors 700 patient groups participate in Eurordis’ activities in 2008 Partner in the Rare Disease Platform 3
  4. 4. EURORDIS’ PRESENCE IN EUROPE 345 members in 38 countries (25 EU) Country with a National Alliance 4
  5. 5. EURORDIS’ 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 and – directly or indirectly – to fight against the impact of rare diseases on their lives. 5
  6. 6. WHAT IS A RARE DISEASE? A rare disease in Europe is a disease affecting less than 1 in 2,000 citizens < 230,000 EU citizens per rare disease 6,000 to 8,000 rare diseases 30 million people affected in the EU. 6 to 8 % of the EU population 6
  7. 7. CHARACTERISTICS OF RARE DISEASES 80% genetic diseases Patients are few and geographically spread Relevant information is scarce Experts are few Specialised care centres for each disease cannot exist in every country Resources are limited Research is fragmented 7
  8. 8. PRIORITY 1: NETWORKING & INFORMATION SHARING More than 345 members in 38 countries Council of 13 National Alliances & Network of > 300 Allied members Organisation of annual membership meetings (2002 Barcelona, 2003 Namur, 2004 Cork, 2005 Venice, 2006 Berlin, 2007 Paris, Copenaghen 2008, Next AMM in Athens 2009) Organisation of the European Conference on Rare Diseases (2001 Copenhagen, 2003 Paris, 2005 Luxemburg, 2007 Lisbon, Next ECRD in 2010, Poland) Website in 6 languages: English – French – Spanish – Italian – German – Portuguese (www.eurordis.org) Monthly electronic newsletter in 6 languages 8
  9. 9. PRIORITY 2: ADVOCACY & POLICY DEVELOPMENT Contribution to the adoption of the EU Regulation on Orphan Medicinal Products in 1999 Contribution to the adoption for the EU Regulation on Medicinal Products for Paediatric Use in 2006 Contribution to the promotion and maintenance of rare diseases as a priority in the EU Research Framework Programmes Contribution to the adoption of EU Regulation on Advanced Therapies April 2007 Contribution to the promotion and maintenance of rare diseases as a EU public health policy priority: November 2008 Adoption EU Commission Communication on European Actions in Rare Diseases; Council Recommendation First European Rare Diseases Day, 29 February 2008: 18 countries Second European Rare Diseases Day, 28 February 2009 9
  10. 10. EURORDIS IS PRESENT IN MANY EUROPEAN INSTITUTIONS AND PLATFORMS European Institutions: Committee for Orphan Medicinal Products at the EMEA (COMP) Committee for Pediatric Use Medicinal Product at the EMEA EMEA/CPMP Working Group with Patients’ Organisations Rare Disease Task Force at DG Health and Consumer Protection EU Health Policy Forum at DG Health and Consumer Protection European Platforms: European Patients’ Forum (EPF) European Platform for Patients’ Organisations, Science, and Industry (EPPOSI) European Forum for Good Clinical Practice (EFGCP) International Alliance of Patients’ Organizations (IAPO) Pan-European Blood Safety ( PBSA ) 10
  11. 11. PRIORITY 3: ACCESS TO INFORMATION, DIAGNOSIS, TREATMENT & CARE Guidelines (9 languages) to support creation and management of rare disease patient groups and information services Mapping of rare disease patient organisations’ profiles and activities in Europe Scientific surveys to increase our advocacy power Training sessions for patient representatives 11
  12. 12. PRIORITY 4: THERAPEUTIC DEVELOPMENT & RESEARCH Contribution to transparent and quality information on medicines for patients, improvement of pharmacovigilance: participation in the European Medicines Agency Working Group with Patients’ Organisations Creation of a European network of rare disease Biological Resource Centres (EuroBioBank) for DNA, cells and tissue Partnering in many European research projects: EuroClinGene, ECRIN, Orphan Platform, TREAT-NMD etc Contribution to obtaining EU funding for rare disease research projects 34 pharmaceutical companies are members of the Eurordis Round Table of Companies 12
  13. 13. EUROBIOBANK European network of biobanks of DNA, cells and tissue for rare disease research Budget: 1,219,321 € (funded by the European Commission within the 6th Framework Programme) and by major patient groups 16 partners in 8 countries (Belgium, France, Germany, Hungary, Italy, Malta, Slovenia and Spain) 13
  14. 14. EUROBIOBANK 30 standard operating procedures A web-based catalogue displaying 971 sample collections 7 languages (English, French, German, Hungarian, Italian, Slovene, Spanish) 170,000 samples 64,000 samples collected during the project 6,047 samples exchanged in the network 41 scientific publications have mentioned EuroBioBank 14
  15. 15. TRAINING SESSIONS FOR PATIENTS ‘Providing information on rare diseases’ ‘Searching for validated biomedical information on the Internet’ ‘Understanding clinical trial protocols’ ‘Getting involved into the EU drug regulatory decision making process” ‘Patient databases, registries and cohorts’ ‘Gaining access to rare disease research resources’ 15
  16. 16. THE ‘EURORDISCARE’ PROGRAMME A series of surveys to compare access to care for rare disease patients across Europe Scientific methodology to collect new and solid data from patient groups, to increase their advocacy power Collaboration with scientific research teams (Inserm, London School of Economics) 16
  17. 17. THE ‘EURORDISCARE’ PROGRAMME 2004: Compare needs and access to care 6 rare diseases, 17 countries 50 patient groups, English language 2005: Compare delays of access to diagnosis and the causes 8 rare diseases and 17 countries 70 patient groups, 12 languages, 6 000 questionnaires filled The objective of the study was to show the delays to diagnosis and to identify the main causes. 17
  18. 18. THE ‘EURORDISCARE’ PROGRAMME 2006: Compare experience and expectations of patient and families on health care and specialised services in Europe 12 rare diseases, 20 countries, 8,500 patients Access to and organisation of health and social care and medical services. This survey will provide patient and family experience on centres of reference for rare diseases and their expectations. 18
  19. 19. ONLINE PATIENT COMMUNITIES ‘Mailing Lists for Patients with Rare Diseases’: a pilot 4 lists as of July 2006 • Ichthyosis • Fragile X • Prader-Willi • Sanfilipo 6 more lists at the end of 2007 • Guenther’s porfiria • Behcet • Anorectal malformations • Sarcoidosis • Dancing eyes November 2008 total lists 22• 11q Good practice guidelines http://eurordis.medicalistes.org/ 19
  20. 20. THE RARE DISEASE PATIENT SOLIDARITY PROJECT Creation / development of services & networks for people living with rare diseases • Help lines • Integration at school • Summer camps • Respite care centres Reflection process on the organisation of care for rare diseases, including centres of reference in Europe • Patient survey (EurordisCare) • National workshops on centres of reference will be held in 10 European countries 2006-2007 • European workshop on centres of reference to be held in Prague on 12-13 July 2007 20
  21. 21. Other Projects Europlan 2008-2011 • Project leader: D. Taruscio, CNMR/ISS • 25 countries • Objective: to develop recommendations how to define a national plan on rare diseases Polka 2008-2011 • Project leader: Eurordis • 27 countries • 21 languages • Objective: innovative patient consultation process on RD policies by DECIDE method 21
  22. 22. EURORDIS PATIENT REPRESENTIVES IN ODs • Patient groups play a key role in policy development: EU Regulation on Orphan Medicinal Products in 1999 EU Pharmaceutical legislation Review in 2003 (including conditional approval, EU centralised procedure for all orphan drugs) EU Regulation on Paediatric Use of Medicines in 2006 (including extension of orphan drug market exclusivity to 12 years). EU Regulation on Advanced Therapies in 2007 • Patient representatives are in the decision making process 3 members in the COMP + 1 Observer: policy, guidelines, designation opinion, significant benefit opinion) Patient experts in Protocol Assistance procedure Patient experts in Risk Management Programme 2 members in the PDCO 2 members in the CAT • Patient groups validate information to public Public Summaries of COMP Opinions European Public Assessment Reports 22
  23. 23. STATUS OF ORPHAN APPLICATIONS 893 Applications for orphan medicinal products 577 Designations of orphan medicinal products 47 Orphan medicinal product MA As of December 2008 23
  24. 24. Availability of OMPs by country in 2007 Availability for OMPs approved before 2004 (12 OMPs) & 2006 (22 OMPs) 20-21 15-19 10-14 5-9 0-5 OMP authorised before 01/01/04 OMP authorised before 01/01/06 24
  25. 25. EURORDIS’ FUNDING SOURCES Total Revenue = 2,626 M euros Revenue % by source of funding in 2007 25
  26. 26. CONCLUSION Empowerment of Patients: “ the process of increasing capacity of individuals or groups to make choices and to transform those choices into desired actions and outcomes” Networking and Information Sharing Training Policy Development and Coordination: Member States, EU High and Firm Commitment 26
  27. 27. Rare Disease Day 2009 • Date: 28 February 2009 • Logo: • Coverage Europe, Canada, USA, Argentina… 27
  28. 28. Theme + Focus 2009 •Theme: (2008-2011) • Rare Diseases: a Public Health Priority •Focus 2009 : Patients Centered Care Slogan: “Patient Care, a Public Affair!” 28
  29. 29. European Event March 2009 European Patron: Commissioner Androulla Vassiliou • Press Conference “Rare Disease Day” at the European Commission : • Presentation of the Declaration of Common Principles on Centres of Expertise and European Reference Networks • Launch of Eurordiscare Survey book “The Voice of 12,000 Patients – Experiences and Expectations of Rare Disease Patients on Diagnosis and Care in Europe” • Dinner Debate in the European Parliament •Rapporteur: Trakatellis, PPE Greece •DG SANCO, DG Research, DG Enterprise •RDTF, COMP •Stakeholders 29
  30. 30. www.rarediseaseday.org • 30
  31. 31. www.rarediseaseday.org 31
  32. 32. www.eurordis.org Acting Together For the Challenge To Build a EU Coordination of Health Systems and Policies For Equity Access across Europe Thank You PHOTO
  33. 33. FOR MORE INFORMATION Michele Lipucci di Paola Former Vice President of Eurordis Board Member Pan-European Blood Safety ( PBSA ) Yann Le Cam - Chief Executive Officer Francois Houyez – Health Policy Officer Fabrizia Bignami - Therapeutic Development Officer www.eurordis.org 33
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