Max’s wonder drug to be funded on NHS
‘Amazing news’ for those with genetic condition
A Perth mum has said the decision to fund her son’s life-saving drug on the NHS is “amazing news”.
Little Max Farmer, who is only 18-months-old, was diagnosed with the life-limiting condition spinal muscular atrophy (SMA) type one when he was only nineweeks-old.
The condition means his muscles do not work as they should, leaving him with extremely limited mobility, respiration and swallowing ability.
However, he has been receiving the wonder drug Spinraza since he was 14 weeks old, and on Monday this week the decision was taken to fund this on the NHS.
The drug is injected into Max’s spine every four months, and each injection costs £75,000. Up until now, this has been funded by the pharmaceutical company Biogen.
Mum Elaine Donoghue, who said her son would not still be alive if it wasn’t for this drug, welcomed this week’s decision.
She said: “This is absolutely amazing news for Max and all future children with SMA type one. It has been approved for type one with a strict criteria in line with the clinical trial, so children will need to be under six months and be symptomatic to get access to it on the NHS.
“It is a significant announcement, and significant news for us as a family. Type one is life-limiting and Max would have died without it. This is the first time there is a drug available to change the course of this disease.
“Max has been doing brilliantly, he is going from strength to strength. We have a standing frame for him now so he can go out into the garden, which has opened up a whole new world for him. He is doing really well, so fingers crossed that continues.”
Despite Max’s family’s joy at the news he will be given this drug on the NHS, mum Elaine is concerned how this decision will affect those with SMA type two.
SMA type two is when a child is diagnosed with the disease after seven-months-old. Max’s treatment will now be funded on the NHS
She continued: “It is bittersweet because there are children who are type two who would have benefited greatly from getting access to this. I secretly hoped the decision would cover both type one and type two so most young people in Scotland could have the chance to make huge improvements.
“They will not have this same opportunity, and their parents will have to watch them deteriorate without it. But I can understand there is a huge cost, and those with type one responded best in the clinical trials.
“Countries like Australia have just announced they will fund the drug for types one, two and three, so that will take children into young adulthood.
“And in America, if someone is pregnant and the genetic test shows the baby will be born with SMA, the child can have the drug in the first week of life. That means they will walk and crawl normally.
“We are very aware of other families this has affected, so we will do all we can to raise awareness of SMA and show Max’s progress to fight for other children to get access to this.”
Max’s care has been fully transferred from Biogen to NHS Max Farmer and mum Elaine Donoghue Scotland as of this week, and he is due to get his first injection funded by the NHS at the end of this month.
A spokesperson for SMA Support UK said: “The Scottish Medicines Consortium has just recommended that this treatment should be funded only for children with SMA type one. This is a positive step forward and a lifeline for these children and families.
“However, it is hugely disappointing that children, young people and adults with SMA type two or type three who wish to access, and could potentially benefit from, this treatment still don’t have this opportunity.
“This is despite the hard work of the patient groups and the powerful testimonies from the SMA community.
“We will not though, give up advocating for broader access and will, with the other charities, be working on the next steps.”
Max is going from strength to strength. We have a standing frame for him now