Considering the continental scope “the model will enable patient representatives and policy makers to monitor and compare rare disease policies and programmes in Europe. In addition, the results will be crucial to assess the cost effectiveness of new treatments and technologies to prevent, diagnose and improve health services for patients with a rare disease.” as the Project Coordinator, Dr Julio López Bastida (FUNCIS), explained.
The partners involved in this project represent a wide cross-section of the EU member states, involving 8 countries: Spain, United Kingdom, Sweden, Germany, Bulgaria, France, Italy and Hungary. Having initiated the project, the analyses in Spain are already at an advanced stage. Indeed, the Spanish on-line survey was closed on 31st December, 2011, in which a remarkable 794 responses were collected. This success and the lessons learnt have set the standard for the remaining countries.
The on-line recruitment of patients is now active in all the other participating countries.Bulgaria kicked off its survey at the middle of December 2011, Germany in January 2012, and Italy, UK, Hungary, France in February 2012 and Sweden is commencing it this month. Patients can access the questionnaires also through the project’s web page.
The prognosis of success is high in those EU countries where the active national alliances are coordinating the patients’ associations, involving many medical professionals. All of them are doing their best to contact the patients and caregivers in person, as well as via e-mail to stimulate their participation. The BURQOL-RD Board has scheduled these surveys to be open for 3 months.
EURORDIS is providing expertise on designing and implementing the survey by sharing its experience from the EurordisCare surveys and is supporting project communication to enhance its outreach.
After a thorough selection process, a set of 10 rare diseases to be targeted was established:
- Cystic Fibrosis
- Prader-Willi Syndrome
- Duchenne Muscular Dystrophy
- Epidermolysis Bullosa
- Fragile X Syndrome
- Juvenile Idiopathic Arthritis
The project is led by the Canary Foundation of Investigation and Health (FUNCIS) with the collaboration of:
11 Associated Partners:
- Federación Española de Enfermedades Raras (FEDER), Spain
- Instituto de Salud Carlos III (ISCIII) – Research Institute for Rare Diseases (IIER), Spain
- Bulgarian Association for Promotion of Education and Science (BAPES), Bulgaria
- Istituto Superiore di Sanita (ISS), Italy
- London School of Economics and Political Science (LSE-Health), UK
- Leibniz University Hannover (LUH), Germany
- The Swedish Institute for Health Economics (IHE), Sweden
- Università Commerciale “Luigi Bocconi”, Italy
- Université Paris Est, France
- Centre for Public Affairs Studies Foundation (CPASF), Hungary
- Mario Negri Institute for Pharmacological Research (IRFMN), Italy
8 National Alliances:
- National Alliance of people with rare diseases (NAPRD), Bulgaria
- Consulta Nazionale delle Malattie Rare, Italy
- UNIAMO (Federazione Italiana Malattie Rare), Italy
- Allianz Chronischer Seltener Erkrankungen (ACHSE), Germany
- Rare Diseases Sweden (Sällsynta diagnoser), Sweden
- Hungarian Federation of People with Rare and Congenital Diseases-Rare Diseases Hungary (HUFERDIS), Hungary
- Rare Diseases UK-Genetic Interest Group (GIC), UK
- Alliance Maladie Rares, France
1 Umbrella Organisation:
- European Organisation for Rare Diseases (EURORDIS)
- CRE Enfermedades Raras (CREER), Spain
- Euro-Histio-Net, an international reference network for Langerhans cell histiocytosis and associated syndromes
as well as hundreds of experts.
To follow project updates you can join the Facebook community Burqol-rd international, or you can find more information at: www.burqol-rd.com.
This article was first published in the March 2012 issue of the EURORDIS newsletter
Author: Edina Szakmari
Photo credits: © Burqol RD & EURORDIS