Rare disease policies to improve care for patients in Europe

Biochimica et Biophysica Acta - Molecular Basis of Disease

Volumn 1852, Issue 10, 2015, Pages 2329-2335

  Rodwell, Charlotte ^a   Aymé, Ségolène ^a  

^a INSERM   (France)

Author keywords

Codification; Healthcare planning; Orphan drugs; Policy; Rare diseases; Registries

Indexed keywords

ARTICLE; DISEASE REGISTRY; EUROPE; EUROPEAN UNION; HEALTH CARE POLICY; HEALTH CARE QUALITY; HUMAN; INFORMATION SERVICE; LAW; NEURONAL CEROID LIPOFUSCINOSIS; PATIENT CARE; POLITICAL SYSTEM; PREVALENCE; PRIORITY JOURNAL; RARE DISEASE;

EID: 84940791702     PISSN: 09254439     EISSN: 1879260X     Source Type: Journal    
DOI: 10.1016/j.bbadis.2015.02.008     Document Type: Article

References (32)

1. Regulation (EC) N°141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products December 16 1999, (accessed 5 December 2014). http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDF.
2. List of rare diseases and synonyms listed in alphabetical order, Orphanet Report Series, Rare Diseases collection July 2014, (accessed 5 December 2014). http://www.orpha.net/orphacom/cahiers/docs/GB/List_of_rare_diseases_in_alphabetical_order.pdf.
3. Communication from the Commission to the European Parliament, the Council, the European Economic and Social Committee and the Committee of the Regions on Rare diseases: Europe's challenges COM (2008) 679 final November 2008, (accessed 5 December 2014). http://ec.europa.eu/health/ph_threats/non_com/docs/rare_com_en.pdf.
4. 2014 Report on the state of the art of rare disease activities in Europe July 2014, (accessed 5 December 2014) [http://www.eucerd.eu/?page_id=163#StateArt]). C. Rodwell, S. Aymé (Eds.).
5. OrphaNews (accessed 5 December 2014). http://www.orphanews.org.
6. Council recommendation on an action in the field of rare diseases (2009/C 151/02) June 2009, (accessed 5 December 2014). http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0007:0010:EN:PDF.
7. Aymé S., Rodwell C. The European Union Committee of Experts on Rare Diseases: three productive years at the service of the rare disease community. Orphanet Journal of Rare Diseases 2014, 9:30. (28 February 2014).
8. Commission Expert Group on Rare Diseases (accessed 5 December 2014). http://ec.europa.eu/health/rare_diseases/expert_group/index_en.htm.
9. List of projects concerning rare diseases in the field of health financed by the Second Community Programme (accessed 5 December 2014). http://ec.europa.eu/health/rare_diseases/projects/index_en.htm.
10. European Commission: implementation report on the Commission Communication on Rare Diseases: Europe's challenges [COM(2008) 679 final] and Council Recommendation of 8 June 2009 on an action in the field of rare diseases (2009/C 151/02) September 2014, (accessed 5 December 2014). http://ec.europa.eu/health/rare_diseases/docs/2014_rarediseases_implementationreport_en.pdf.
11. Directive 2011/24/EU of the European Parliament and of the Council on the application of patients' rights in cross-border healthcare (accessed 5 December 2014). http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2011:088:0045:0065:EN:PDF.
12. Orphanet (accessed 5 December 2014). http://www.orpha.net.
13. European Union Committee of Experts on Rare Diseases: recommendations on quality criteria for centres of expertise for rare diseases in Member States (accessed 5 December 2014). http://www.eucerd.eu/?post_type=document&p=1224.
14. European Commission: Directorate General for Health and Consumers, European Reference Networks website (accessed 5 December 2014). http://ec.europa.eu/health/ern/policy/index_en.htm.
15. European Union Committee of Experts on Rare Diseases: recommendations on European Reference Networks for rare diseases (accessed 5 December 2014). http://www.eucerd.eu/?post_type=document&p=2207.
16. European Union Committee of Experts on Rare Diseases: preliminary analysis of pilot European Reference Networks for Rare Diseases May 2011, (accessed 5 December 2014). http://www.eucerd.eu/?post_type=document&p=1357.
17. World Health Organization ICD11 Beta Draft (accessed 5 December 2014). http://apps.who.int/classifications/icd11/browse/l-m/en.
18. European Commission Expert Group on Rare Diseases, Recommendation on ways to improve codification for rare diseases in health information systems November 2014, (accessed 5 December 2014). http://ec.europa.eu/health/rare_diseases/docs/recommendation_coding_cegrd_en.pdf.
19. European Commission: Directorate General Research & Innovation, Horizon 2020 (accessed 5 December 2014). http://ec.europa.eu/programmes/horizon2020/.
20. E-Rare (accessed 5 December 2014). http://www.erare.eu/.
21. International Rare Diseases Research Consortium (accessed 5 December 2014). http://www.irdirc.org.
22. Rare Disease Registries in Europe, Orphanet Report Series, Rare Diseases collection January 2014, (accessed 5 December 2014). http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf.
23. European Union Committee of Experts on Rare Diseases: recommendations on rare disease patient registration and data collection (accessed 5 December 2014). http://www.eucerd.eu/wp-content/uploads/2013/06/EUCERD_Recommendations_RDRegistryDataCollection_adopted.pdf.
24. Report of the December 2013 Meeting of the European Medicines Agency's Committee on Orphan Medicinal Products (accessed 5 December 2014). http://www.ema.europa.eu/docs/en_GB/document_library/Committee_meeting_report/2014/01/WC500159429.pdf.
25. European Union Committee of Experts on Rare Diseases, Recommendation on improving informed decisions based on the clinical added value of orphan medicinal products information flow (accessed 5 December 2014). http://www.eucerd.eu/?post_type=document&p=1446.
26. European Commission, DG Enterprise & Industry, Platform on access to medicines in Europe (accessed 5 December 2014). http://ec.europa.eu/enterprise/sectors/healthcare/competitiveness/process_on_corporate_responsibility/platform_access/index_en.htm#h2-2.
27. EURORDIS (accessed 5 December 2014). http://www.eurordis.org.
28. Lists of medicinal products for rare diseases in Europe, Orphanet Report Series, Orphan Drugs collection October 2014, (accessed 5 December 2014). http://www.orpha.net/orphacom/cahiers/docs/GB/list_of_orphan_drugs_in_europe.pdf.
29. Report on the state of the art of rare disease activities in Europe. Edited by S. Aymé, C. Rodwell, July 2010, July 2011, July 2012, July 2013, July 2014 (accessed 5 December 2014) []. http://www.eucerd.eu/?page_id=163#StateArt.
30. Prevalence of rare diseases: Bibliographic data, Orphanet Report Series, Rare Diseases collection, May 2014, Number 1: Listed in alphabetical order of disease or group of diseases (accessed 5 December 2014). http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence:of_rare_diseases_by_alphabetical_list.pdf.
31. Prevalence of rare diseases: Bibliographic data, Orphanet Report Series, Rare Diseases collection, May 2014, Number 2: Listed in order of decreasing prevalence or number of published cases (accessed 5 December 2014). http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence:of_rare_diseases_by_decreasing_prevalence:or_cases.pdf.
32. Orphadata (accessed 5 December 2014). http://www.orphadata.org.