EUCERD releases state-of-the-art information capturing rare disease and orphan drug activity throughout Europe
An elaborate document detailing the rare disease-related activities throughout Europe has been released. Produced by the Scientific Secretariat of the European Union Committee of Experts on Rare Diseases (EUCERD) through the three-year Joint Action to support the Scientific Secretariat of the former-RDTF/EUCERD (N° 2008 22 91), the 2011 Report on the State of the Art of Rare Disease Activities in Europe of the European Union Committee of Experts on Rare Diseases provides a comprehensive overview of rare disease and orphan drug activities at both the European Union (EU) and Member State (MS) levels up to the end of 2010.
The lengthy report is a hybrid of updated data from the previous report and brand-new information that was carefully selected to enhance understanding of the rare disease activities across Europe. As such, it presents an informed overview of rare disease and orphan drug activities, elaborated in concertation with a wide range of stakeholders from each of the MS and at the EU level. The information has been divided into three principal sections:
- Part I, available for the first time, presents the Overview of Rare Disease Activities in Europe and Key Developments in 2010
- Part II delineates the European Commission and Other European Activities
- Part III outlines Activities in EU Member States and Other European Countries
The report outlines the political context that has spawned the growth of rare disease activities across Europe and also summarises the political frameworks of other regions across the world. Amongst the topics examined at both the MS and EU levels are the national strategies for rare diseases either implemented or under elaboration; centres of expertise; registries; newborn screening policies; genetic testing resources and activities; patient organisation activities; information resources; guidelines for best practice; educational initiatives; research and funding mechanisms and participation in EU-level projects; rare disease conferences and events; orphan drug and device incentives, availability, reimbursement and pricing policies; and specialised social services. New to this year’s edition are the topics of genetic testing, Europlan national conferences, orphan devices, other initiatives to improve access to orphan drugs, and orphan drug pricing policy. Each section of the report finishes with a bibliography of sources used, including a list of any European Commission documents referred to and a list of web addresses organised by country listing national sources of information on rare diseases and links to documents concerning national plans or strategies for rare diseases when appropriate. The report also furnishes a list of contributors, organised by country and with mention of the validating authority for each country.
The report has met with praise at both the EU and MS levels for providing valuable insight into understanding the current resources and activities in the field of rare diseases across Europe that will help determine future strategies to meet the needs of rare disease patients and their families in Europe and further afield. Its publication comes just in time for the upcoming meeting of the International Rare Disease Research Consortium (IRDiRC) next month in Canada (learn more about the IRDiRC).
Consult the 2011 Report on the State of the Art of Rare Disease Activities in Europe of the European Union Committee of Experts on Rare Diseases