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EC sets up expert group on rare diseases

Will revamp EUCERD to better handle development of orphan medicines

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The European Commission (EC) is setting up a panel of experts to help direct its activities and policy-making in the area of rare diseases.

The new group is a revamp of the European Union Committee of Experts on Rare Diseases (EUCERD) - which was set up in 2009 but whose term of office came to an end on July 26 - and will come into line with updated EC rules designed to boost transparency and reduce red tape.

The EC's decision to appoint a new panel came on July 30 and kicks off a search for new committee members, who will be drawn from member state representatives, patients organisations, healthcare professionals and industry, according to the official notice. 

They will be appointed by Director-General for Health and Consumers Tonio Borg for initial three-year terms, and the intention is to have the new group in place in time for an inaugural meeting in the late autumn, according to EUCERD.

In the EU, a rare disease is defined as one which affects fewer than 5 people per 10,000, and there are around 7,000 diseases that come under that category, collectively affecting between 6 and 8 per cent of the EU population in the course of their lives. Most are caused by genetic defects, but environmental exposure during pregnancy or later in life can be another cause.

At its last meeting in June, the 51-member EUCERD adopted a series of recommendations on issues relating to rare disease, including the need for improved patient registries and data collection to support research and for post-marketing surveillance of orphan medicinal products and medicines used off-label.

Rare diseases tend to be characterised by patchy knowledge, scattered expertise and patients, and research duplication, it said, and registries would enable data to be pooled to “reach sufficient statistically significant numbers for clinical research and public health purposes”.

It also called for EU member states to develop and adopt - ideally by the end of this year - national strategies to improve the handling of rare diseases. A report published earlier this year found that rare disease patients typically face a five- to seven-year wait for proper diagnosis.

The announcement of the new expert group came as pharma company Boehringer Ingelheim warned that inflexibilities in the EU regulatory environment for clinical trials of medicines to treat rare is holding back new treatments.

The EC is currently in the process of seeking public comment on plans to revamp its system for reviewing marketing applications for orphan drugs for rare diseases, having been ordered to approve a previously-rejected drug - CTRS' Orphacol - earlier this month.

The Commission is also trying to stimulate and support research in this area. Earlier this year it announced €144m in funding for research to "help improve the lives of some of the 30 million Europeans suffering from a rare disease".

Among the projects receiving funding are a new 'bioartificial' liver support system to treat acute liver failure; data processing operations for novel diagnostic tools, biomarkers and screening strategies for drugs to treat rare kidney diseases; and the clinical development of a drug to treat alkaptonuria, a genetic disorder which leads to a severe and early-onset form of arthritis.

2nd August 2013

From: Research, Regulatory

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