Members of MDI call on the HSE to fund treatment for Duchenne Muscular Dystrophy
Members of MDI are calling on the HSE to fund the first ever treatment for Duchenne Muscular Dystrophy. Translarna (also known as ataluren) has been developed to treat children whose Duchenne muscular dystrophy is caused by a ‘nonsense’ mutation and is the first drug to address an underlying genetic cause of muscular dystrophy to receive approval from the European Medicines Agency.
Translarna received conditional marketing approval from the European Medicines Agency in August 2014. Since then boys have had full unrestricted access to this medication in over 20 EU countries. In April 2016 The NHS in England agreed to allow access and in August 2016 The Minister for Health in Northern Ireland confirmed access for boys North of the Border.
Meanwhile in the Republic of Ireland children still have no access to Translarna.
According to our information there are no more than 5 young boys currently living in Ireland with DMD resulting from a nonsense mutation for whom this drug may be applicable. Furthermore, of these 5 boys only 2 boys are currently aged over 5 years and still walking and therefore deemed to be suitable for the drug. The remaining 3 boys are still under age 5 years but may become eligible for the drug if they are still walking at the age of 5 years.
In January 2017 MDI received the news that the HSE have decided not to reimburse the treatment.
The HSE decision is also at odds with other European Countries. Translarna has been approved by the EMA and is currently available in over 20 other European countries including Spain, France, Italy, Germany and Greece. Individual funding requests can also be made in countries such as Poland, Romania, Slovakia, Estonia, Hungary and Czech Republic and Sweden and a access via a managed risk programme is available in the UK.