International Rare Diseases Research Consortium

Posted - February 2013 in HSPRF News

Focused on finding therapies

 

The goal: to deliver 200 new therapies for rare diseases and means to diagnose MOST rare diseases – by the year 2020!!!

 

The International Rare Diseases Research Consortium (IRDiRC) is a group whose members are composed of funding body/organisations investing a minimum of $10 million (US) over 5 years in research projects/programs contributing towards IRDiRC objectives, and also invited patient advocacy groups. Currently the 26 members include organizations from several European countries, the USA, Canada and include the Western Australian Department of Health. Members of the Executive Committee, Members of the Scientific Communities and Members of the Scientific Secretariat include a similarly rich and distinguished blend of individuals from Australia, the USA, Canada and many European nations. See who is involved.

 

The Global Genes Project estimates that there are 350 million people worldwide affected with a rare disease while EURODIS estimates that there may be as many as 5,000 to 7,000 rare diseases in existence. Individual researchers and consortia have been studying rare diseases for several decades. However, the rare disease research community was very fragmented mainly due to the large heterogeneity of rare diseases. Indeed, for many diseases, there are few researchers and very limited resources available. There’s been a complaint for some time in groups trying desperately to fund cures or effective treatment for rare diseases, that research takes too long, researchers and pharmaceutical companies are competing rather than working together and getting treatment from lab to patient can take decades, if at all, and cost millions.  Concerted efforts to organize the rare disease research community and funding were emerging in several countries but so far, international coordination was rather limited.

 

Now these challenges are being addressed through actions such as providing access to harmonized data and samples, boosting clinical research and streamlining regulatory procedures on a world scale.

 

In 2009, the idea to establish IRDiRC came during a meeting between Dr. Ruxandra Dragha-Akli (European commission) and Dr. Francis Collins (NIH). Their main objective was to accelerate medical breakthroughs for people affected by rare diseases by establishing a network of research funders.

 

In October 2010, the European Commission and the US National Institute of Health announced, in the first preparatory workshop in Reykjavik (Iceland), their intention to join forces on rare diseases research. The two institutions planned to coordinate their research funding on rare diseases and to make major investments in this research field in the years to come.

 

In April 2011, IRDiRC was officially established and launched during the second preparatory workshop in Bethesda (USA). The group of funding agencies develop common scientific and policy frameworks to guide the activities of the participating IRDiRC members.

 

In October 2011, in Montreal (Canada), the third preparatory workshop gathered around 100 participants representing public and private funding organizations, scientists, regulators, industry and patient groups. It focused on continuous efforts to develop common scientific and policy frameworks to guide the activities of the participating IRDiRC members.

 

IRDiRC’s website already shows 56 new therapies toward their goal of 200 therapies by the year 2020, and 1883 new diagnostics.  A conference is scheduled for Dublin Ireland in April 2013.

 

The NEUROMICS consortium, comprising leading academic institutions as well as businesses, will be utilising the latest technologies to revolutionise diagnostics and to develop new treatments based on the mechanisms of each disease. The focus will be on 10 rare diseases, including ataxia, spastic paraplegia, Huntington’s disease, muscular dystrophy and spinal muscular atrophy. The results will then help hospitals to apply directly, giving patients immediate benefits. Read more

 

You can learn more with their 13 minute introductory video on the IRDiRC’s website homepage.

 

 

 

 

 

 

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