Introduction
Centres of expertise : the key stone of national plans and strategies for rare diseases and a step towards European networks
• | 1 European country with designated CE RD in the scope of a national plan for rare diseases |
• | 5 European countries with officially designated CE RD |
• | 15 European countries with non-designated CE RD acknowledged by health authorities to varying degrees |
• | 9 European countries with CE RD recognised by reputation only |
• | 16 European countries with plans to designate CE RD in their national plans/strategies for RD |
• | EUCERD Recommendations on quality criteria for centres of expertise for rare diseases in Member States adopted on 24 October 2011 |
• | Consensus on 45 recommendations covering the mission and scope, criteria for designation, process of designation and evaluation, and European dimension of CE RD |
European Reference Networks for rare diseases: the European dimension of health care pathways
• | Rare diseases cited in Directive on the application of patients’ rights in cross border healthcare (2011/24/EU) (9 March 2011) as priority area for ERNs |
• | EUCERD Recommendations on European Reference Networks for Rare Diseases adopted on 31 January 2013 |
• | Consensus on 21 recommendations covering mission, vision and scope, governance, composition, funding and evaluation, and designation of ERNs for Rare Diseases |
Patient registration and data collection: Gathering information at European level on rare diseases
• | Around 640 rare disease registries in Europe1 |
• | Majority of registries are academic |
• | Some RD have more than one registry, many RD have no registry |
• | EUCERD Core Recommendations on Rare Disease Patient Registration and Data Collection adopted on 5 June 2013 |
• | Consensus on 6 main areas : international operability, sources of data, collection of data, good practices, use of data for regulatory purposes, and sustainability |
Steps towards better and timely access to orphan medicinal products for rare diseases : Improving the Clinical Added Value of Orphan Medicinal Products information flow
• | 1234 positive opinions for orphan product designation from 1798 applications submitted since 2000 at EU level and a total of 1219 European Commission designations |
• | 85 orphan designated products have received marketing authorisation by end of 2013 at EU level |
• | EUCERD Recommendation on Improving Informed Decisions Based on the Clinical Added Value of Orphan Medicinal Products Information Flow adopted in September 2012 |
• | Recommendation proposes four key time points for information sharing to improve the pricing and reimbursement decision process |