It will be disgrace if boys suffering from degenerative disease are denied access to the drug they so badly need, says Iain Gray
The decision last week by the Scottish Medicines Consortium (SMC) refusing NHS funding of the drug Translarna for boys with Duchenne was a devastating blow to families living with the condition.
Rubbing salt in the wound, it was quickly followed by a positive recommendation by the equivalent body in England.
Duchenne muscular dystrophy affects around one in 3600 boys and results in muscle degeneration and early death. Translarna is the first therapy available to treat the underlying cause.
I have supported the campaign for Translarna ever since my constituent Gary Fegan of Longniddry contacted me. Gary’s five-year-old son, Cormac, was diagnosed with Duchenne in 2014. Since then Gary has become a relentless campaigner and fundraiser and he has also lodged a successful “individual funding request” with NHS Lothian and so Cormac is one of only two or three boys in Scotland receiving Translarna.
Anyone who hears Gary explain the difference this drug has made to Cormac will be left angry by the decision of the SMC. Translarna will not cure Cormac, but it has improved his balance and walking, giving the hope of much longer before he becomes dependent on a wheelchair. Even more dramatically it transformed Cormac’s behaviour overnight, stopping his tendency to bite other children.
Cormac now depends on Lothian renewing his discretionary funding. The Fegan family do not know what will happen. This is not a party political issue. But prior to the decision Michael Young, another Duchenne boy, met Nicola Sturgeon to ask her to support Translarna. She explained that the SMC is independent, and that she could not intervene.
I know this is “officially” correct. But Michael is not going to accept it, nor is Gary Fegan. At the very least, if re-elected First Minister, Nicola Sturgeon must find a way to get the SMC and the pharmaceutical company to negotiate a lower price for Translarna.
Michael Young has launched a petition. It says: “I am asking for help from the First Minister to change the SMC’s mind to make sure that me, Cormac, Ross and the other boys with Duchenne can get the medicine in Scotland like lots of other boys do in Europe.”
The petition is at www.change.org/p/nicola-sturgeon-ensure-access-to-duchenne-drug-translarna-in-scotland. I have signed it. Please do so too.
• Iain Gray is the Labour Party candidate for East Lothian