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 netayme@orpha.net

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

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

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

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

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

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

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

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

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

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

Orphanet in European countriesOrphanet in European countries

O h t di t f 2 500 diOrphanet directory of 2,500 rare diseases published at national level

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

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