RAPSODY Rare Disease Patient Solidarity...
Transcript of RAPSODY Rare Disease Patient Solidarity...
RAPSODYRare Disease Patient Solidarity project
EC2005120
Yann Le CamEURORDIS, Chief Executive Officer
RAPSODY, Project Leader
Luxembourg June 20 2007
www.eurordis.org
Progress Report June 2007
PHOTO PHOTO
Eurordis survey on patients’ experience and expectations
concerning access to health services in Europe(EurordisCare3 - Rapsody Work Package 5)
Presentation to the DG SanCo Rare Disease Task Force June 20th 20073
Represent a wide diversity of prevalence, age of onset, clinical manifestations,handicap generated and availability of treatments.
• Marfan syndrome (3,00 / 10 000)• Fragile X syndrome (1,42 / 10 000)• Williams syndrome (1,33 / 10 000)• Ehlers-Danlos syndrome (1,25 / 10 000)• Cystic fibrosis (1,20 / 10 000)• Prader-Willi syndrome (1,07 / 10 000)• Epidermolysis bullosa (1,00 / 10 000)• Tuberous sclerosis (0,88 / 10 000)• Myasthenia (0,85 / 10 000)• Osteogenesis imperfecta (0,65 / 10 000)• Huntington disease (0,62 / 10,000)• Ataxias (0,22 / 10 000)• Aniridia (0,17 / 10 000)• Pulmonary arterial hypertension (0,15 / 10 000)• Chromosome 11qdisorders (150 published cases)• Alternating hemiplegia (200 published cases, about 30 in France)
16 rare diseases
Presentation to the DG SanCo Rare Disease Task Force June 20th 20074
A true European dimension
• Commitment of 116 patient organisations as of 07/06/2007
• Representing 23 countriesOverall: 21 EEE states/28, plus Switzerland and CroatiaField work completed: France, Hungary, Spain Field work in progress: Austria, Belgium, Cyprus, Czech Republic, Denmark, Germany, Greece, Ireland, Italy, Malta, Netherlands, Slovakia, Sweden, Switzerland, United KingdomField work to be started: Croatia, Finland, Norway, Poland, Romania,
• Target: 16 languagesTranslation completed: Danish, Dutch, English, French, German, Greek, Hungarian, Italian, Slovak, Spanish, SwedishTranslation in progress: Croatian, Finnish, Polish, Romanian, Still to be translated: Norwegian
Presentation to the DG SanCo Rare Disease Task Force June 20th 20075
EurordisCare 3 Questionnaire progress
• 116 organisations agreed in principle as of 07/06/200718 additional patient organisations have joined since May 2007
• State of progress69 : pack of questionnaires already sent out24 : being printed9 : in progress9 : translation and/or validation of less frequent languages in
progress (Croatian, Finnish, Norwegian, Polish, Romanian)
• For each organisation: between 20 and 450 questionnaires sent out (between February and May)
• 3 135 questionnaires returned as of 07/06/20072 853 questionnaires analysed out of 12 480 sent (resp. rate: 25%)From Fra, Spa, Dnk, Ita, UK, Ire, Swe, Bel, Ger, Swi, Hun, Slk, Aus
Presentation to the DG SanCo Rare Disease Task Force June 20th 20076
Analysis of results
• Preliminary analysis :Deadline for responses 11/06/2007Data entry : May and JuneAnalysis : June and JulyFirst presentation and workshop : 12-13th July 2007 during the “European Workshop on Centres of Expertise/Reference networks”
• Overall analysis will last until 2008 Answers received after June 11th will be included, as well as participation of patient organisations that joined the study later (Deadline for participation agreement : 30/08/2007 // for responses : 30/09/2007)Second presentation : 27-28th November 2007 “4th European Conference on Rare Diseases (ECRD 2007)”
• In addition to overall results, each organisation will receive :• Statistical results of the analysis based on responses of their members,• Analyses obtained with data collected on their disease in other countries,• And data on the other 15 diseases in their own country.
Centres of Expertise / European Reference Networks(Rapsody Work Package 8)
Presentation to the DG SanCo Rare Disease Task Force June 20th 20078
11 National workshops held
Ass. Haemophil PortugalAna Rita DagninoMarch 30thPortugal11
EGANAlastair Kent GIGMelissa WinterMarch 28thUnited Kingdom10
VSOPPauline EversApril 11thThe Netherlands9
Swedish AllianceElisabeth WalleniusMarch 16thSweden8
FEDERRosa Sanchez de VegaMarch 23rdSpain7
ALLAN asblBettina VogelMarch 22ndLuxembourg6
UNIAMOSimona BellagambiMarch 24thItaly5
ACHSEMirjam MannMarch 19thGermany4
Alliance Maladies RaresFrançoise AntoniniMarch 29thFrance3
RDDTorben GrönnebaekMarch 26thDenmark2
SUKLMaryna KrenkovaMarch 2ndCzech Rep.1
OrganisationParticipantsDatesMember state
Presentation to the DG SanCo Rare Disease Task Force June 20th 20079
263 Participants & 10 Synthesis report
Participants total: 263
received14 PO, 10 HCP, 1 PM = 25March 30thPortugal11
received16 PO, 12 HCP, 1 PM = 29March 28thUnited Kingdom10
received9 PO, 11 HCP, 2 PM = 22April 11thThe Netherlands9
received30March 16thSweden8
received17 PO, 16 HCP, 4PM = 37March 23rdSpain7
pendingMarch 22ndLuxembourg6
received10 PO, 7 HCP, 1 PM = 18March 24thItaly5
received11PO, 6 HCP, 5 PM = 20March 19thGermany4
received12 PO, 6 HCP, 7 PM, 1 Pharm = 26March 29thFrance3
received10 PO, 7 HCP, 2 PM = 19April 20thDenmark2
received21 PO, 12 HCP, 4 PM = 37March 2ndCzech Rep.1
ReportParticipantsDatesMember state
Presentation to the DG SanCo Rare Disease Task Force June 20th 200710
Next steps
• Advisory group meeting (synthesis of 10 reports): June 11Question N° 1: Needs and expectations for national rare diseases centres ofreference/expertiseQuestion N° 2: Proposals for the evaluation of national centres of reference in your countryQuestion N° 3. Cooperation with other countries and recommendation for European reference networks
• Information to EU HLG WG ERN (Brussels): June 14• Information to DG SanCo RDTF (Luxembourg): June 20• First results EurordisCare survey (Paris): End June• European Workshop (Prague): July 12-13• Report to European Conference on Rare Diseases Nov 27-28
Presentation to the DG SanCo Rare Disease Task Force June 20th 200711
European Workshop in Prague – Day 1
EurordisCare Survey results, 2.30 – 4.30 pmCo-chairs: Terkel Andersen +/- Panos Kanavos LSE
Survey on patient experience and expectations in access to health services (EurordisCare3)Pierre Chauvin, INSERM, Unit Public HealthPanel with representatives of the selected diseases
Lunch 1.00 pm to 2.30 pm90 min
Introduction 11.00 to 12.00 amCo-chairs: Birthe Holm (Rare Disorders Denmark), Martin Benes (SUKL director)Welcome speechCzech patient representativeWorkshop objectivesFrançois Houyez EURORDIS, EUPresentation of the proposals of the High Level Group on Health Services and Medical CareAude Marlier Sutter, Ministry of Health, FrancePresentation of the DG Sanco Rare Disease Task Force reportDr Ségolène Aymé, DG SanCo Rare Disease Task Force, EUDiscussion
Presentation of selected European networks 12.00 – 1.00 pmCo-chairs: Birthe Holm (Rare Disorders Denmark) Martin Benes (SUKL director)- Establishing the six first European Reference Networks: practical experienceProl Lara Fragonese, Rare Bleeding Disorders Network, Netherlands- Cystic fibrosis, a European Reference Network supported by DG SanCoProf Thomas Wagner, ECORN-CF, Germany- EuroAtaxia, a European Network of Centre of Expertise supported by DG. ResearchProf Olaf Riess, EuroAtaxia, GermanyDiscussion
5 min
5 min
20 min
20 min
10 min
10 min
10 min
10 min
30 min
First day: starts at 11.00 am
Presentation to the DG SanCo Rare Disease Task Force June 20th 200712
European Workshop in Prague - Day 2
Ends at 4.00 pm
Closing remarks 3.45 to 4.00 pmThe way forward form the Commission^’s perspectiveToni Montserrat, DG SanCo, EU (tbc)
15 min
Methodology to assess the outcomes of European Reference Networks, 2.30 to 4.00 pmChair person: Prof Birgitta Strandvik, Sweden and Prof Olaf Riess, GermanyDiscussion based on DG SanCo RDTF meeting June 2007Dr Edmund Jessop, Ministry of Health, UKDescription of the evaluation plan as proposed by the French policy on centres of expertiseHAS representative (France)Discussion
20 min
20 min
30 min
Lunch 1.00 pm to 2.30 pm
Synthesis of national workshops 9.30 am – 1.00 pmChair person: Terkel Andersen (EURORDIS), Maryna Krenkova (SUKL, Czeck Republic)Overall presentation of the national workshops
Synthesis of responses to question 1 by Christel Nourissier, EURORDIS, EUDiscussionResponses to question 2 by Simona Bellagambi, UNIAMO, ItalyDiscussionCoffee break 11.00 am to 11.20 amResponses to question 3 by Birthe Holm, Rare Disorders DenmarkDiscussionProposed priority of criteria and their content for European Reference NetworksYann Le Cam, EURORDIS, EUDiscussion
10 min
10 min30 min10 min30 min11.00 am10 min30 min12.00 am
30 min
Second day
Presentation to the DG SanCo Rare Disease Task Force June 20th 200713
Participantsas of June 8th: 54 confirmed, 47 pending
Anders Fasth
Manuel Posada
Dr Domenica Taruscio
Prof. Flora PayvandiDr François Faurrisson
Prof Jan StolkDr Pierre Chauvin
President HLG HSMCDr Frédéric Sicard
Claire Joan Scharf-KrönerProf Dian DonnaïDr Edmund Jessop
Martin TerbergerFrançoise GrossetêteProf Thomas Wagner
Martin DorazilDr Alexandra FourcadeProf Jean Charles Deybach
John RyanDr Kerstin WestermarkProf Olaf Riess
Dr Catherine BerensDr Susan WebbDr Edmund Jessop
Manuel HallenDr Jill Clayton-SmithDr Ségolène Aymé
Dr Andrzej RysOctavie Quintana-TriasToni Montserrat
PendingNot attendingConfirmed
Presentation to the DG SanCo Rare Disease Task Force June 20th 200714
Participants as of June 11th 2007 (2)
• Confirmed (54)22 patient representatives from national workshops 7 members Advisory Committee WP8 & WP5 10 health care professionals3 MS representatives4 policy makers7 Eurordis Board members/staff
• Pending (47)7 MS representatives / EU HLG HSMC WG ERN12 participants from disease networks EurordisCare survey9 EC representatives8 patient representatives from national workshops4 MEPs5 members Advisory groups WP5 & WP82 health care professionals
• Expected total: 101
European Conference on Rare Diseases2007 in Lisbon
(Rapsody Work Package 2)
Presentation to the DG SanCo Rare Disease Task Force June 20th 200716
Organisation
• On-line registration opened: www.rare-diseases.eu
• See Electronic announcement for more information
This paper was produced for a meeting organized by Health & Consumer Protection DG and represents the views of its author on thesubject. These views have not been adopted or in any way approved by the Commission and should not be relied upon as a statement of the Commission's or Health & Consumer Protection DG's views. The European Commission does not guarantee the accuracy of the dataincluded in this paper, nor does it accept responsibility for any use made thereof.