13° CONVEGNO
   Patologia immune e malattie orfane 2010
         Torino 21 - 23 gennaio, 2010




Network europei per le m...
I RIUNIONE: LUSSEMBURGO / BRUSSEL - 26 Febbraio 2010
EU NETWORKS FOR RARE DISEASES


              Care & treatment



                                                      Ad...
Programme of Community action on rare
    diseases within the framework for action in
      the field of public health (19...
Projects supported in the framework of the
Programme of Community Action on Rare
 Disease (1999 – 2003) and the EU Public
...
Second programme
      of Community action in the field of health
                    (2008-
                    (2008-13)...
Raccomandazione approvata: giugno 2009

                                  www.iss.it/cnmr
www.iss.it/cnmr
www.iss.it/cnmr
www.iss.it/cnmr
EUROPLAN
EUROPLAN progetto europeo per lo sviluppo di piani
nazionali sulle malattie rare
è un progetto triennale, finanzi...
Obiettivi specifici di EUROPLAN
-    Raccogliere informazioni sulle iniziative già
     esistenti nei vari Paesi
-    Anal...
Partners di EUROPLAN

EUROPLAN
include
30 Paesi
      e




EUROPLAN collabora con
l’Office for rare diseases (NIH-USA)

 ...
Paesi che
collaborano con
  EUROPLAN




                  www.iss.it/cnmr
EUROPLAN: un progetto inclusivo
• Associated partners




• Collaborating partners


    Rappresentanti delle Autorità san...
A che punto siamo
       nel progetto EUROPLAN?

(1) Raccolta delle informazioni già
realizzate nei vari Paesi
• Piani naz...
(2) Elaborazione delle
Raccomandazioni di EUROPLAN


            Principali capitoli:

                1. Introduzione

  ...
3) Sviluppo di indicatori per il monitoraggio,
       implementazione e valutazione
  dell’impatto di piani / strategie na...
(4) Discutere le Raccomandazioni
   nei vari Paesi con tutti gli stakeholders
Conferenze nazionali saranno organizzate da
...
Meeting futuri

• Cracovia, EU Conference; 13-15 Maggio,
  2010

• Conferenze nazionali nei 16 Paesi scelti da
  EURORDIS ...
http://www.europlanproject.eu/




                         www.iss.it/cnmr
E-mail: europlan@iss.it
National Centre for Rare Diseases
   Istituto Superiore di Sanità
     Via Giano della Bella, 34
 ...
www.iss.it/cnmr
E-Rare: Networking research programmes
             on rare diseases in Europe




• EU funded project (DG Research)
• Sci...
E-Rare roadmap

                   Exchange of information
                       and best practices
                   At...
Facts about the first transnational call
 Budget: over 9 million euros
 Partners: Mean of 5 partners/proposal
 Proposals:
...
What next
                                       next?

•    Success of E-Rare call reflects expectations and needs of the...
Projects supported in the framework of the
Programme of Community Action on Rare
 Disease (1999 – 2003) and the EU Public
...
Projects supported in the framework of the
          Programme of Community Action on Rare
        Disease (1999 – 2003) a...
Projects supported in the framework of the
      Programme of Community Action on Rare
    Disease (1999 – 2003) and the E...
Projects supported in the framework of the
  Programme of Community Action on Rare
Disease (1999 – 2003) and the EU Public...
Reference networks pilot projects
•   The 2006 Annual Work Programme for grants from the European
    Commission ….the dev...
The RDTF recommendations

•   the wording "centre of reference" is not used in the
    future when referring to the nodes ...
Taruscio Domenica Torino 13° Convegno Patologia Immune E Malattie Orfane 21 23 Gennaio 2010 [Modalità Co
Taruscio Domenica Torino 13° Convegno Patologia Immune E Malattie Orfane 21 23 Gennaio 2010 [Modalità Co
Taruscio Domenica Torino 13° Convegno Patologia Immune E Malattie Orfane 21 23 Gennaio 2010 [Modalità Co
Taruscio Domenica Torino 13° Convegno Patologia Immune E Malattie Orfane 21 23 Gennaio 2010 [Modalità Co
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Taruscio Domenica Torino 13° Convegno Patologia Immune E Malattie Orfane 21 23 Gennaio 2010 [Modalità Co

  1. 1. 13° CONVEGNO Patologia immune e malattie orfane 2010 Torino 21 - 23 gennaio, 2010 Network europei per le malattie rare Domenica Taruscio Direttore Centro Nazionale Malattie Rare Istituto Superiore di Sanità Roma
  2. 2. I RIUNIONE: LUSSEMBURGO / BRUSSEL - 26 Febbraio 2010
  3. 3. EU NETWORKS FOR RARE DISEASES Care & treatment Advocacy Research Surveillance Policy development Collaboration Development and sharing of information Patients & patients associations Health care Public health specialists professionals Researches Experts
  4. 4. Programme of Community action on rare diseases within the framework for action in the field of public health (1999 to 2003) • Promote the development of, and access to, a coherent and complementary European information network on rare diseases … The availability of this information must be made as widely known as possible, including via the Internet. • Contribute to training and refresher courses for professionals in order to improve early detection, recognition, intervention and prevention in the field of rare diseases. • Promote transnational collaboration and networking between groups of persons directly or indirectly affected by the same rare conditions or volunteers and professionals involved and coordination at Community level in order to encourage continuity of work and trans-national cooperation. • Support at Community level the monitoring of rare diseases in the Member States and early warning systems for clusters, and promote the networking and training of experts concerned with the handling of rare diseases and with rapid response to the phenomenon of clusters
  5. 5. Projects supported in the framework of the Programme of Community Action on Rare Disease (1999 – 2003) and the EU Public Health programme 2003-2008 2003- 1. To strengthen collaboration at European level among patient organisations 2. To increase the visibility and operational capacity of organisations and networks active in the field of rare diseases.
  6. 6. Second programme of Community action in the field of health (2008- (2008-13) • DG SANCO Work Plans for the implementation of the Public Health Programme lines of action: – exchange of information via existing European information networks on rare diseases, and – development of strategies and mechanisms for information exchange and co-ordination at EU level to encourage continuity of work and trans-national co-operation. – Furthermore, regarding rare diseases projects, DG SANCO prioritises generalist networks, which centralise information on as many rare diseases as possible - not just a specific group or a single disease - to improve information, monitoring and surveillance.
  7. 7. Raccomandazione approvata: giugno 2009 www.iss.it/cnmr
  8. 8. www.iss.it/cnmr
  9. 9. www.iss.it/cnmr
  10. 10. www.iss.it/cnmr
  11. 11. EUROPLAN EUROPLAN progetto europeo per lo sviluppo di piani nazionali sulle malattie rare è un progetto triennale, finanziato dalla Commissione europea nell’ambito del Programma “Sanità Pubblica” durata: Aprile 2008 – 2011 Responsabile scientifico: Dr. Domenica Taruscio Obiettivo generale: elaborare raccomandazioni per la realizzazione di piani e/o strategie nazionali e/o regionali sulle malattie rare www.iss.it/cnmr
  12. 12. Obiettivi specifici di EUROPLAN - Raccogliere informazioni sulle iniziative già esistenti nei vari Paesi - Analizzare le informazioni raccolte e identificare le azioni di maggior successo e le “lessons learned” - Elaborare le raccomandazioni di EUROPLAN - Elaborare indicatori per il monitoraggio, la implementazione e la valutazione di piani/strategie nazionali - Discutere le raccomandazioni nei vari Paesi - Diffondere nei vari Paesi le raccomandazioni www.iss.it/cnmr
  13. 13. Partners di EUROPLAN EUROPLAN include 30 Paesi e EUROPLAN collabora con l’Office for rare diseases (NIH-USA) www.iss.it/cnmr
  14. 14. Paesi che collaborano con EUROPLAN www.iss.it/cnmr
  15. 15. EUROPLAN: un progetto inclusivo • Associated partners • Collaborating partners Rappresentanti delle Autorità sanitarie & health care planners Medici, operatori socio-sanitari, ecc. Ricercatori Pazienti www.iss.it/cnmr
  16. 16. A che punto siamo nel progetto EUROPLAN? (1) Raccolta delle informazioni già realizzate nei vari Paesi • Piani nazionali e/o strategie nazionali • Ricerca scientifica (es. presenza di programmi o azioni) • Empowerment delle Associazioni dei pazienti • Presenza di specifici programmi sui farmaci orfani (uso compassionevole, ecc.) • Finanziamenti specifici dedicati alle malattie rare • Servizi sociali specializzati www.iss.it/cnmr
  17. 17. (2) Elaborazione delle Raccomandazioni di EUROPLAN Principali capitoli: 1. Introduzione 2. Definizione di Piano o Strategia nazionale 3. etc. Amsterdam, 17-18 settembre 2009 Roma (ISS), 18-19 gennaio 2010 www.iss.it/cnmr
  18. 18. 3) Sviluppo di indicatori per il monitoraggio, implementazione e valutazione dell’impatto di piani / strategie nazionali Workshops: 1) Madrid :18-19/06/2009 2) Amsterdam:17-18/09/09 3) Roma: 18-19/01/2010 www.iss.it/cnmr
  19. 19. (4) Discutere le Raccomandazioni nei vari Paesi con tutti gli stakeholders Conferenze nazionali saranno organizzate da EURORDIS, mediante le Alleanze Nazionali a) presentare le raccomandazioni di EUROPLAN b) discuterne la trasferibilità nei diversi Paesi, discutendole con tutti le parti interessate c) presentare la Communicazione della Commissione e la Raccomandazione del Consiglio I Paesi che EURORDIS ha selezionato sono 16: Bulgaria, Croazia, Danimarca, Francia, Germania, Grecia, Irlanda, Italia, Lussemburgo, Olanda, Polonia, Romania, Spagna, Svezia, Ungheria, UK www.iss.it/cnmr
  20. 20. Meeting futuri • Cracovia, EU Conference; 13-15 Maggio, 2010 • Conferenze nazionali nei 16 Paesi scelti da EURORDIS (2010) • Conferenza conclusiva: Roma, ISS (2011) www.iss.it/cnmr
  21. 21. http://www.europlanproject.eu/ www.iss.it/cnmr
  22. 22. E-mail: europlan@iss.it National Centre for Rare Diseases Istituto Superiore di Sanità Via Giano della Bella, 34 000162 – Rome Tel. +39 06 4990 4371 Fax. +39 06 4990 4370
  23. 23. www.iss.it/cnmr
  24. 24. E-Rare: Networking research programmes on rare diseases in Europe • EU funded project (DG Research) • Scientific responsable: France, Italy – ISS (WP Leader) • Objective: coordinate existing national research programmes on RD to: – Reduce fragmentation in research – Launch multinational joint calls and other joint initiatives – Eventually develop joint programmes on RD
  25. 25. E-Rare roadmap Exchange of information and best practices At program level & at project level Survey & Electronic tool for project analysis Strategic activities & Research policy development Workshops & consultations of experts (identification of needs, new technologies, new ethical challenges) Transnational activities Joint activities Development of common administrative and Mobility & training legal procedures for research funding into RD research Mutual access to technology platforms Opening of national calls to international participation Implementation of transnational calls
  26. 26. Facts about the first transnational call Budget: over 9 million euros Partners: Mean of 5 partners/proposal Proposals: 123 received; 18 thematic areas 13 funded; 7 thematic areas % of projec ts per thematic area 4 7 8 9 5 15 8 5 6 10 15 18 4 2 8 38 2 6 2 2 8 9 2 2 5 Autoinmune and rare systemic diseases Bone diseases Cardiovascular diseases Complex syndromes Dermatological diseases Embryonic developement abnormalities Endocrinological diseases Hepato Gastro Entero diseases Immunodeficiency Kidney diseases Lung diseases Lysosomal storage disorders Metabolic diseases Mitochondrial diseases Neurological diseases Neuromuscular diseases Non malignant hematological diseases Sense organs diseases
  27. 27. What next next? • Success of E-Rare call reflects expectations and needs of the RD research community • Legal and administrative barriers among countries are still significant E-Rare will continue its efforts towards: Facilitating access of RD researchers to the best technology platforms regardless their localization Fostering multidisciplinarity thorough training and exchange programs Establishing joint programs for rare diseases research E-Rare 2 has been submitted to the EU Commission 19 / 01 /2010
  28. 28. Projects supported in the framework of the Programme of Community Action on Rare Disease (1999 – 2003) and the EU Public Health programme 2003-2008 2003- The EU has supported EURORDIS projects to gather the information required to help define a public policy on rare diseases, to improve access to quality information on rare diseases and orphan drugs, to organise workshops at European and national level, as well as to produce guidelines and pedagogical documents.
  29. 29. Projects supported in the framework of the Programme of Community Action on Rare Disease (1999 – 2003) and the EU Public Health programme 2003-2008 2003- • Network of Public Health Institutions on Rare Diseases (NEPHIRD) The project aimed at discussing and analysing the epidemiological data collection for RD in participating countries in order to identify and suggest possible approaches for estimating epidemiological indices. It aimed also at analysing the state of the art with regard to RDs focusing on health care services accessibility and quality for RDs patients and undertaking a specific assessment of the quality of life of RDs patients. • The EU Surveillance of congenital anomalies in Europe project, (EUROCAT) a European network of 51 registries in 28 countries for the epidemiological surveillance of congenital anomalies. • European educational programme on Rare Diseases The project involved several public conferences with the aim of improving communication and co-operation between people professionally interested in rare diseases in Europe and offered education and training opportunities to health professionals and others involved in the field of rare diseases (scientists, doctors, nurses, patient support groups).
  30. 30. Projects supported in the framework of the Programme of Community Action on Rare Disease (1999 – 2003) and the EU Public Health programme 2003-2008 2003- • ENERCA (European Network for Rare Congenital Anaemias) • European network on the epidemiology, pathophysiology and treatment of severe chronic neutropenia • The EU Rare Forms of Dementia projects • The EU EUROMUSCLERNET project - Muscle diseases - prototype of rare and disabling disorders: Creation of a European information network • The EU CAUSE Project - Charge Association and Usher Syndrome in Europe • The European Information Network on Paediatric Rheumatic Diseases Project • The EU project establishing European Neurofibromatosis Lay Group Network • Health promotion, improving health information and knowledge for neurofibromatosis (NF) in Europe • The EU Information network for immunodeficiencies • The EU TEAM project - Transfer of expertise on rare metabolic diseases in adults
  31. 31. Projects supported in the framework of the Programme of Community Action on Rare Disease (1999 – 2003) and the EU Public Health programme 2003-2008 2003- 2005 • EU Primary Immunodeficiency Consensus Conference • European Myasthenia Gravis Network • European Autism Information System 2006 • Rare Diseases portal (Orphanet) • Surveillance of rare cancers in Europe (RARECARE) • European Register on Cushing’s Syndrome (ERCUSYN) 2007 • European Haemophilia Safety Surveillance System • European Project for Rare Diseases National Plans Development (EUROPLAN) • Patients' Consensus on Preferred Policy Scenarios for Rare Diseases
  32. 32. Reference networks pilot projects • The 2006 Annual Work Programme for grants from the European Commission ….the development of European Network of Centre of Expertise for rare diseases as a prority, priority 2.1.4(e). • Six networks of centres of reference for a specific rare disease or a group of rare diseases have been selected, spanning six EU countries. They will serve as pilot projects for reference networks of Centres of expertise: – European Centres of Expertise Network for Cushing Syndrome (Dr Susan Webb, University of San Pau, Barcelona) – European Centres of Expertise Network for porphyria (Prof. Jean-Charles Deybach, Paris) – European Centres of Expertise Network alpha-1 antitrypsin deficit (Prof. Jan Stolk, Leiden) – European Centres of Expertise Network for dysmorphic syndromes (Prof. Jill Clayton-Smith, Manchester) – European Centres of Expertise Network for cystic fibrosis (Prof. Thomas Wagner, Frankfurt) – European Centres of Expertise Network for hemorragic syndromes (Prof. Flora Payvandi, Milan).
  33. 33. The RDTF recommendations • the wording "centre of reference" is not used in the future when referring to the nodes of a network to be established. The preferred wording is “centre of expertise centre expertise”. “Centres of expertise” exist everywhere, whereas “centres of reference” are confined to a few countries. • ... Countries with established centres of reference should be encouraged to share their experience and the results of their outcome measures. • the European Commission plays an important role in supporting the identification of centres of expertise and in the diffusion of the information about them.

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