Today is rare disease day.  These are diseases we do not often talk about and they do not get the attention that other diseases do. These are often genetic, chronic and serious diseases and they affect a serious amount of people. Throughout the EU and the US over 60million people are affected by over 6000 rare diseases. Yes, those are large numbers and they are numbers that should be getting more attention from all of us. We in the pharmaceutical sector are looking for more ways to be able to treat these diseases, this through continued research and innovation by our members. The European institutions have also pushed to ensure that treatments are made available. The Orphan Medicinal Product Regulation was introduced in the European Union to incentivise the development of medicines for rare diseases and has pushed companies to act- and they have.

The number of designations for orphan diseases has been steadily increasing over the last five years.  107 designations were granted in 2011, 148 in 2012, and more than 150 are expected in 2013. In line with this trend, in 2012, 19 applications for marketing authorisation concerned designated orphan medicines, compared with 14 in 2011.

To add to this the EMA last month announced that its Committee for Orphan Medicinal Products (the COMP) was going to seek to strengthen its relations with international partners and patient groups to have the maximum amount of impact while not replicating initiatives and gaining a full understanding of the demands by patient groups.

We are all in this together but this is just a start and I know we can do better. The access to orphan medicines is still very slow and unequal. Many patients, especially in newer Member States simply have no access at all to these medicines, and the current context of austerity this is not getting better.

Today all those affected by these diseases, those who work with them and their families deserve all our attention. Take a few minutes to look at the Rare Disease Day website done by the Eurordis ( European Organisation for Rare Diseases), look at some of the great work being done and understand the difficulties being overcome by extraordinary people all around us.

 About EFPIA:

EFPIA represents the pharmaceutical industry operating in Europe. Through its direct membership of 33 national associations and 39 leading pharmaceutical companies, EFPIA provides the voice of 1,900 companies committed to researching, developing and bringing new medicines to improve health and quality of life around the world. The pharmaceutical industry invests 27.5 billion on research and development per year in Europe and directly employs 660,000 people including 116,000 in R&D units in Europe. 

EFPIA members are committed to delivering innovative medicines to address unmet needs of patients and reducing the burden of chronic diseases for Europe’s ageing population. EFPIA believes in close cooperation with its stakeholders to help create sustainable healthcare   systems and to develop prompt responses to health threats in Europe.

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