Rare Disease Patients Can Access Just One-Third of Medicines Available in Rest of Europe, Says IPHA

Just one-third of medicines for rare diseases licensed for patients in Europe are available in Ireland, according to new figures released on Rare Disease Day by the Irish Pharmaceutical Healthcare Association (IPHA), the representative body for the research-based pharmaceutical industry.

According to the figures, just 29% of all available orphan medicines licensed by the European Medicines Agency for use in patients in 27 European countries were available to patients here.

Ireland ranked in the bottom half of the list of European countries, and behind all other western European Union countries, including Denmark, Spain, Sweden, Italy and Austria.

The UK and Germany performed best, according to the figures, scoring 92% and 96% for availability of orphan medicines, respectively.

The figures, compiled by IQVIA for the European Federation of Pharmaceutical Industries and Associations which includes IPHA, relate to rare diseases medicines authorised by the European Medicines Agency in the years 2015, 2016 and 2017. The figures released today [Thursday] refer to the rate of availability to patients of those medicines at the end of 2018.

Rare diseases affect about 300,000 people in Ireland during their lifetime, according to Rare Diseases Ireland. Rare diseases are among the hardest medical conditions to treat. Though they affect fewer than one in 2,000 people, three-quarters of rare diseases are found in children and the mortality rate is very high. There are between 7,000 and 8,000 recognised rare diseases.

Oliver O’Connor, the Chief Executive of IPHA, said access to new innovative medicines, including to orphan medicines, remains a huge challenge that must be met through partnerships.

“Patients in Ireland are now consistently among the last in western Europe to get the benefits of new medicines. This is not due to the price of medicines in Ireland – all countries in western Europe assess, negotiate and agree prices similar to what is offered here. Sometimes, medicines that are available in other European countries are not approved here at all. This is dysfunctional.

“On Rare Disease Day, we are urging the Government to set a simple policy goal: to place Ireland in the top quartile of European countries for speed of access to new innovative medicines. This is now urgent,” said Mr O’Connor.

IPHA has established a Rare Diseases Group which though calling for a speedy the publication of the second National Rare Disease Plan, is also urging that more progress needs to be made in implementing the recommendations on medicines in the existing plan.

Rare diseases cause difficulties with daily activities, motor and sensorial functions and social interactions. The call on carers’ time is huge, and many of them are family members. Rare diseases often cause a sufferer to give up their job.

Scientific advances like genetic testing mean we have a better understanding of the causes of rare diseases which can be fatal. The biopharmaceutical industry continues to mine the science that we hope can ultimately solve the complex challenges of researching, developing and clinically testing rare disease treatments.

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