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Other websites/documents
Directorate General for Health and Consumers
DG Sanco's website has a section dedicated to rare disease policy, publications and events at the European level link.
This section also includes information on the European Union Committee of Experts on Rare Diseases link.
Orphanet
Orphanet is the European portal for information on rare diseases and orphan drugs.
Orphanet provides information for all stakeholders including:
- an inventory of rare diseases
- an inventory of orphan drugs
- an encyclopaedia
- a directory of services: specialised clinics, laboratories, ongoing research, registries, clinical trials and patient organisations
- a newsletter (in English and in French)
- thematic studies and reports
EU documents
1. Directives, Regulations, Communications, Council Decisions, Council Recommendations and other related documents
Communication from the Commission on Rare Diseases: Europe’s Challenge link
Council Recommendation on an action in the field of rare diseases (June 8th 2009) link
Summary of the Impact Assessment Accompanying the Communication on Rare Diseases: Europe's challenges link
Commission Decision of 30 November 2009 establishing a European Union Committee of Experts on Rare Diseases (2009/872/EC) link
Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare link
Regulation (EC) N°141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products link
Directive 2001/20/EC of the European Parliament and of the Council of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use (Clinical Trials Directive) link
Commission Directive 2005/28/EC of 8 April 2005 laying down principles and detailed guidelines for good clinical practice as regards investigational medicinal products for human use, as well as the requirements for authorisation of the manufacturing or importation of such products (Good Clinical Practice Directive) link
Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use link
Regulation on advanced therapies (Regulation (EC) 1394/2007) link
Regulation on medicinal products for paediatric use (Regulation (EC) N° 1901/2006) link
EMEA Public Statement on Fee Reductions for Designated Orphan Medicinal Products link
Council Decision No 1982/2006/EC of 18 December 2006 concerning the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007-2013) link
European Commission Communication on Action Against Cancer: European Partnership link
European Commission Communication on a European initiative on Alzheimer’s disease and other dementias link
2. Reports
Directorate General Health and Consumers
Eurobarometer 74.3: European awareness of rare diseases link
All reports of the EC Rare Disease Task Force and European Union Committee of Experts on Rare Diseases can be found here
Directorate General Research and Innovation
International Rare Disease Research Consortium (IRDiRC) link
International Rare Disease Research Consortium (IRDiRC) First Workshop Summary Report link
International Rare Disease Research Consortium (IRDiRC) Second Workshop Summary Report link
DG Enterprise and Industry
Inventory of Community and Member States' incentive measures to aid the research, marketing, development and availability of orphan medicinal products (2005 revision) link
Final Conclusions and Recommendations of the High Level Pharmaceutical Forum (2nd October 2008) link
Initial investigation to assess the feasibility of coordinated system to access orphan medicines (Gesundheit Österreich GmbH, May 2011) link
EMA
Recommendation on elements required to support the medical plausibility and the assumption of significant benefit for an orphan designation (EMEA/COMP/15893/2009) link 1 link 2
European Medicines Agency Annual Report for 2008 link
European Medicines Agency Annual Report for 2009 link
European Medicines Agency Annual Report for 2009/2010 link
3. Programmes of Community Action, Work Programmes and Calls
EU Programme of Community Action in the Field of Health 2003-2008
link
EU Second Programme of Community Action in the Field of Health 2008-2013 link
DG Sanco Work Plan 2009 link
DG Sanco Work Plan 2010 link
DG Research and Innovation FP7 Calls link
EMEA Work Programme 2009 link
EMA Work Programme 2010 link
EMA Work Programme 2011 link
4. Other European documents
Orphanet Report Series
Orphanet Report Series: Prevalence of reported number of published cases listed by alphabetical order by disease
link
Orphanet Report Series: Prevalence by decreasing prevalence or number of published cases link
Orphanet Report Series: Patient registries in Europe
link
Orphanet Report Series: List of orphan drugs in Europe link
Orphanet Report Series: European collaborative research projects funded by DG Research and by E-Rare in the field of rare diseases and European clinical networks funded by DG Sanco and contributing to clinical research in the field of rare diseaseslink
Report on Rare Disease research, its determinants and the way forward (May 2011)link
Eurordis
The Voice of 12,000 Patients: Experiences & Expectations of Rare Disease Patients on Diagnosis & Care in Europe link
Rare Diseases: Understanding this Public Health Priority” (November 2005) link
Results of the 4th Eurordis Survey on Orphan Drug Availability in Europe (2007) link
Other reports
Final Report: Impact on clinical research of European legislation link
EU websites
DG Enterprise and Industry
Eudra-CT (European Clinical Trials Database)
website
DG Sanco
Rare Diseases information on the DG Sanco website
High Level Group on Health Services and Medical Care on the DG Sanco website
Public health initiatives and other institutions responsible for rare diseases on the DG Sanco website
Former Rare Diseases Task Force website
OrphaNews Europe archives
DG Research and Innovation
CORDIS: the gateway to European Research and development website
European Commission research and innovation website website
International Rare Disease Research Consortium (IRDiRC) website
European Medicines Agency
EMA Committee for Orphan Medicinal Products website
Other European websites
Orphanet website
EURORDIS website
EUROPLAN: Guidance for National Plans and Conferences/ Final National Conference Reports website
Country documents
Belgium
Recommendations and Proposed Measures for a Belgian Plan for Rare Diseases - Final Report (2011) - English link
Recommendations and proposals for the Belgian National Plan for Rare Diseases (2010) - English link
Recommandations et propositions de mesures en vue du Plan belge pour les Maladies Rares (2010) - French link
KCE 112B «Politiques relatives aux maladies orphelines et aux médicaments orphelins, 2009» (Report of the Belgian Federal Centre of Healthcare Expertise) link
Bulgaria
National Plan for Rare Diseases 2009-2013 (Genetic, congenital malformation and nonhereditary disease - Unofficial English translation link
ICRDOD Report on Access to Orphan Drugs in Bulgaria link
Czech Republic
Press release concerning material being considered by the Czech Government: National Strategy for rare diseases 2010-2020 link
National Strategy for rare diseases 2010-2020 link
Denmark
Rare Diseases Research in Denmark: Barriers and Prospects (2004) link
France
French National Plan for Rare Diseases 2005-2008 (Ensuring equity in the access to diagnosis, treatment and provision of care) - English link
Plan national maladies rares 2005-2008 - French link
Evaluation du Plan national maladies rares 2005-2008 - French link
Propositions pour le Plan national maladies rares 2010-2014 - French link
Plan national maladies rares 2010-2014 - Qualité de la prise
en charge, Recherche, Europe :une ambition renouvelée - French link
Plan national maladies rares 2010-2014 - Dispositif de suivi - French link
Plan national maladies rares 2010-2014 - Financement - French link
Second French National Plan for Cancers (2009-2013) - French
link
Second French National Plan for Rare Handicaps - French
link
Germany
Maßnahmen zur Verbesserung der gesundheitlichen Situation von Menschen mit Seltenen Erkrankungen in Deutschland (Measures to improve the health situation of persons with rare diseases in Germany - German
link
Joint Press Release: National Action League for People with Rare Diseases Launched (08/03/2010)
link
Joint Declaration and Agreement on the Establishment of the National Action League for People with Rare Diseases
link
Greece
Greek National Plan on Rare Diseases
link
Ireland
IPPOSI Information Document on Rare Diseases (19/02/09)
link
Luxembourg
Les maladies rares: Enquête sur la situation des personnes atteintes de maladies rares au Grand-Duché de Luxembourg - French link
Norway
A Rare Guide: Information on the Norwegian programme for persons with rare conditions (produced by the National Directorate for Health)link
Rare Disorders in Norway: How users experience the health services (report produced by SINTEF December 2008) link
Portugal
Programa nacional para doenças raras (Portuguese National Plan for Rare Diseases) - Portuguese link
Romania
Romanian National Plan for Rare Diseases 2010-2014 (Draft) - English link
Romanian National Plan for Rare Diseases 2010-2014 (Draft) - Romanian link
Spain
National Strategy for Rare Diseases - English link
Estrategia en Enfermedades Raras del Sistema Nacional de Salud - Spanish link
Rare Diseases in Extramadura (2004 Report) link
Country websites
Austria
Gesundheit Österreich GmbH / Austrian Health Institute link
Information provided by the Main Association of Austrian Social Security Institutions link
Belgium
RaDiOrg.be website
Institut national d’assurance maladie-invalidité website
Bulgaria
Information Centre for Rare Diseases and Orphan Drugs website
Croatia
Croatian Society for Rare Diseases website
Croatian Society of Patients with Rare Diseases website
Croatian Agency for Drugs and Medicinal Products website
Cyprus
Gene Net Cyprus website
Cyprus Alliance for Rare Disorders website
Czech Republic
Ministry of Health website
Czech National Strategy website
Neonatal screening website website
Denmark
Danish Centre for Rare Diseases and Disabilities website
Rare Disorders Denmark website
Estonia
Estonian Agrenska Foundation website
Ravimiamet – State Agency for Medicines website
Finland
Vaestoliitto website
Harvinaiset website
Terveysportti website
France
Dossier on Rare Diseases on the French Health Ministry website
Plateforme Maladies Rares website
Alliance Maladies Rares website
AFM Téléthon website
Germany
BMG German Federal Minstry of Health Website - Rare Diseases website
Rare Diseases – The Networks: BMBF website
NAMSE - Nationales Aktionsbündnis für Menschen mit seltenen Erkrankungen website
German Institute of Medical Documentation website
German Clinical Trials Register website
BMG - German Federal Minstry of Health website
BMBF website
ACHSE website
Greece
Greek Alliance for Rare Diseases – PESPA website
EOF – National Organisation for Medicines
website
HUFERDIS website
Ireland
IPPOSI website
Genetic and Rare Diseases Organisation – GRDO website
Medical Research Charities Group website
National Centre for Medical Genetics website
Italy
National Centre for Rare Diseases - Istituto Superiore di Sanità website
National Registry for Rare Diseases website
National Registry of Orphan Drugs website
Italian Ministry of Health - Rare Diseases Information website
Italian Ministry of Health – Rare Disease Research Programme 2008 link
Rete Nazionale Malattie Rare website
UNIAMO website
Consulta Nazional Malattie Rare website
Telethon website
Latvia
The State Agency of Medicines website
National Rare Disease Association Caladrius website
Palidzesim.lv website
Luxembourg
Groupe de travail maladies rares website
Alan absl. website
Lithuania
Lithuanian State Medication Control Agency website
The Netherlands
Dutch Organisation for Health Research and Development (ZonMw) website
National Genetic Resource and Information Centre website
Steering Committee on Orphan Drugs Netherlands website
Forum Biotechnologie en Genetica website
Farmanco – List of Orphan Drugs registered in the European Union (in Dutch) website
VSOP website
Rare Disease Day website
List of genetic diagnostic tests in Netherlands website
Norway
National Directorate for Health and Social Affairs website
Frambu Centre for Rare Diseases website
Resource Centre for Rare Diseases – SSD website
Frambu Centre for Rare Diseases website
Rarelink website
Poland
National Forum on the Treatment of Orphan Diseases website
Portugal
FEDRA - Portuguese Rare Disease Alliance website
Alianca Portuguesa de Associaçoes das Doenças Raras website
Rarìssimas website
Romania
RONARD - Romanian National Alliance for Rare Diseases website
Slovakia
SUKL - State Institute for Drug Control
website
Spain
CISATER – Information Centre for Rare Diseaseswebsite
IIER – Research Institute for Rare Diseases website
Information System on Rare Diseases in Spain website
CIBERER – Biomedical Research Network on Rare Diseases website
Advisory Committee on Rare Diseases Catalonia website
National-Provincial Atlas of Rare Diseases website
FEDER – Spanish Rare Disease Alliance website
Sweden
Swedish Information Centre for Rare Diseases website
Agrenska website
Rare Diseases Sweden website
National Quality Register website
Switzerland
Swiss Telethon website
Gebert Rüf Stiftung Foundation website
Proraris website
Association Enfance et Maladies Orphelines website
United Kingdom
National Commissioning Group website
Advisory Group for National Specialised Services (AGNSS)website
UK Genetic Testing Network website
Genetic Interest Group website
Rare Disease UK website
Contact A Family website
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