Overview of Member States Public Health Policies for Rare Diseases: 2007 … · 2018. 1. 22. ·...
Transcript of Overview of Member States Public Health Policies for Rare Diseases: 2007 … · 2018. 1. 22. ·...
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O f SOveview of Member StatesPublic Health PoliciesPublic Health Policies
for Rare Diseases: 2007 Update
Ségolène AyméRare Diseases Task ForceRare Diseases Task ForceOrphanet / Paris / France
ayme@orpha [email protected]
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Source: Inventory by DG EnterprizeSource: Inventory by DG Enterprize• Article 9.3 of Regulation (EC) N° 141/2000 c e 9 3 o egu a o ( C) / 000
of 16 December 1999EC m st p blish a reg lar pdate of the• EC must publish a regular update of the inventory of all incentives– To support research into orphan drugs– To support their development and availabilityTo support their development and availability
• Last inventory: 2006• Accessible on the EC portal
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Source: OrphaNews EuropeSource: OrphaNews Europe
• Online newsletter of theRare Diseases Task Force
• Over 8,000 registeredreadersreaders
• Political News• Scientific News• Events• Publications• Register on Orphanet• Register on Orphanet
website
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Empowerment of patients organisations
N ti l i ti• National organisations:Feder, Uniamo, Achse, Alliance Maladie RaresAlliance Maladie Rares, Gard, Vsop….
• Registered with Eurordis
• Diversity of initiativesPolitical lobbying– Political lobbying
– Help lines / services– Fund raising for researchFund raising for research– Communication
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Research funding: targeted callsResearch funding: targeted calls• Belgium
– New: Foundation Roi Baudoin fund• France:
B i d li i l h +– Basic and clinical research + networks / databases
– 22 Million Euros for 4 yearsy• Germany:
– 10 Networks with 30 Million for 5 years
• ItalyBasic and clinical research + with 10– Basic and clinical research + with 10 Million for 3 years
– academic clinical trials with 5 Million per year
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Research funding: targeted callsResearch funding: targeted calls• Netherlands
– Budget of 1.4 Million Euro per year• Spain
12 N t k ith 20 Milli E– 12 Networks with 20 Million Euros over 3 years
– National network: CIBERER• Sweden
– Swedish Research Council: 1.1 Milli i 2005Million euros in 2005
• TurkeyBudget of 0 5 Million per year– Budget of 0.5 Million per year
• Transnational funding (E-Rare)– France Germany Italy Israel Spain– France, Germany, Italy, Israel, Spain,
Turkey
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Types of Research Projects in the field
Genetic research Development of a new diagnostic protocolGene mutation research Development of a new medical deviceGene expression Development of a new vaccineGenotype/phenotype correlation Clinical trial of a new drugPhysiopathology and model in vitro Clinical trial of a new diagnostic instrumentPhysiopathology and model in vivo Clinical trial of a new protocolNatural history Clinical trial of a vaccineGene therapy Epidemiological studiesCell therapy Registries/ Observatories/ CohortsBiomarkers Epidemiological studiesBiological collections / resources Research in health sociologyPreclinical development of a new drug Research in health economicsDevelopment of a new diagnostic Public Healthinstrument
Key figuresIn Europe : 4 625 projects of which 13% are clinical trialsIn Europe : 4 625 projects of which 13% are clinical trials
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Measures for Orphan DrugsMeasures for Orphan Drugs• Belgium:
N C itt j t t bli h d– New Committe just established• France:
– ATU system (temporary use autorisation +ATU system (temporary use autorisation national funding + Committe with stakeholders
• Hungary:C itt th t t t f diti– Committee on the treatment of rare conditions
• Ireland– Irish Platform of Patients academics andIrish Platform of Patients,academics and
Industry• Netherlands:
– Dutch Steering Committee on Orphan Drugs (2001)
• Poland:Poland:– National Forum on the treatment of Orphan
Diseases
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Availability at national level in 2007 of the 22 first OMPs authorised at EU levelof the 22 first OMPs authorised at EU level before 1 January 2006 (source: Eurordis Survey)
20-2120-21
15-19
10-1410-14
5-9
0 50-5
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Centres of ReferenceCentres of ReferenceOffi i l CR f R Di• Official CR for Rare Diseases:– Denmark, France, Italy,
S edenSweden– In preparation: Spain….
• Official networks of CR:– France, Germany, Spain
• Official CR but not for Rare Diseases specifically:– Austria, Belgium, Finland, Greece,
Ireland, Netherlands, Spain, UK• Non official CR / established by• Non-official CR / established by
reputation: all countries
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Public Information MeasuresPublic Information Measures• National Information CentresNational Information Centres• National website/helpline by
governmental agenciesgovernmental agencies– Belgium, Bulgaria, Denmark,
France Netherlands NorwayFrance, Netherlands, Norway, SwedenIn preparation: Greece Hungary– In preparation: Greece, Hungary, Italy
National website/helpline by• National website/helpline by patient/professional organisation
S– Italy, Netherland, Spain, UK
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Orphanet in European countriesOrphanet in European countries
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O h t di t f 2 500 diOrphanet directory of 2,500 rare diseases published at national level
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National plans in preparationNational plans in preparation• Effective National PlanEffective National Plan
– France (2005-2008)
• In preparation (first semester 2008):2008):– Greece, Italy,Portugal, Romania,
SpainSpain
• In discussion:• In discussion:– Bulgaria, Ireland, Netherlands,
Sweden SloveniaSweden, Slovenia
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CONCLUSIONCONCLUSION
• Many national / regional initiatives and incentives in the last few months
• To be made widely known• To be monitored to learn about their efficacy andTo be monitored to learn about their efficacy and
cost/effectiveness• Indicators to be defined• Indicators to be defined • Encourage round table of stakeholders• National plans all over Europe• Coordination at EU level