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Duration: 16 April 2011 – 16 April 2014
EC Contribution: € 661 402,00
Website: EPIRARE
General objectives:
- to build consensus and synergies to address regulatory, ethical and technical issues associated with the registration of RD patients and to elaborate possible policy scenarios
- creation of a EU platform for the collection of data on RD patients and their communication among qualified users, based on a feasibility study
- define the options for the preparation of a legal basis, the possible scopes to achieve most effective synergies, the corresponding governance framework and possible options for sustainability. The feasibility of registration of a minimum data set common to all rare diseases, designed to inform policy-making, the conditions to admit research-driven disease or treatment-specific modules and the ways to ensure a sustainable data flow will be assessed
Coordinator: Istituto Superiore di Sanità
EURO-WABB is a newly launched project that will create a rare diseases registry for Wolfram, Alstrom and Bardet Biedl syndromes. Funded by DG Sanco for 36 months, the project is based in the UK. Its vision is to make the EU health service a more supportive environment for rare diabetes syndrome patients, to reduce morbidity and premature mortality and to improve quality of life. The general objective of this project is to support efficient diagnosis, treatment and research for the overlapping rare genetic diseases Wolfram, Alstrom and Bardet Biedl syndromes and other rarer syndromes in Europe. It will be achieved by implementing an EU registry for Rare Diabetes, containing clinical, genetic diagnostic and outcome data. For more information, please visit EURO-WABB website.
The new issue of BAPES official newsletter is now published. The highlights include summary of Rare Disease Day 2011 events in Bulgaria, as well as interview with patients and medical professionals on the recent changes in the provision of rare diseases medication. In “Rare diseases library” you can find an updated review on amyotrophic lateral sclerosis.
The European register of clinical trials has been launched recently. It is an online register where users can find information on the design, the sponsor, the investigational medicinal products and therapeutic areas involved and the status of the clinical trial. It covers clinical trials in EU Member States and the European Economic Area and clinical trials which are conducted outside the EU/EEA if they form part of a pediatric investigation plan. The website is hosted by the European Medicines Agency (EMA). For further information, please visit www.clinicaltrialsregister.eu.
ICRDOD announces the publication of the updated and expanded version of its review on the access to orphan drugs in Bulgaria. The report contains 4 sections: orphan drug designation and marketing authorization; pricing, inclusion in the Positive drug list and reimbursement; mechanisms for accelerated access to innovative medicines; conclusions. There are 2 annexes, attached to the review: list of orphan drugs in EU and Bulgaria, which contains information about the trade name, ATC code, active substance, indication(s), marketing authorization holder and date of marketing authorization for each item (additionally, it is indicated whether the drug is present in the Positive drug list of Bulgaria and if it is reimbursed by public funds); list of references. You can read the review at the Registries & Statistics section.
Issue 2 / March 2011 (click here to read the PDF file, 705 KB)