BETRAYED BY THE SYSTEM
Sam’s denied new miracle drug
A DAD has said he is “horrified, devastated, sad and angry” after a decision by health officials to reject a drug that could change his young son’s life.
Gary McKie had hoped that his six-year-old child Sam, who suffers from Spinal Muscular Atrophy (SMA), would benefit from a new treatment approved in the US.
A DAD has said he is “horrified, devastated, sad and angry” at a decision by health officials to reject a drug that could change his young son’s life.
Gary McKie had resigned himself to the fact that his six-year-old child Sam would be spending the rest of his life in a wheelchair after being diagnosed Spinal Muscular Atrophy (SMA).
He was given hope last year when Spinraza - the first ever treatment for the rare genetic disorder - was approved for patients in America.
But now Gary has been left “heartbroken” after the National Institute for Health and Care Excellence (Nice) has not recommended the treatment in the UK in new draft guidance.
Muscular Dystrophy UK has said that, without access to the treatment, children with SMA - which affects up to 2,500 people across the UK - will lose the ability to crawl, walk and swallow.
But Nice said it would welcome further discussions about access to the drug, also known as Nusinersen, manufactured by Biogen.
Gary, from Forest Hall, said the treatment could give Sam more strength in all his muscles.
“It would give him better control. At the moment we can be up five or six times a night because Sam can’t turn himself over.
“Even for him to do something like that for himself, which most of us take for granted.”
The 45-year-old said he was “horrified, devastated, sad and angry, all at the same time”.
“I’m devastated because I now have to explain to my child why he can’t get treatment.
“He knows there is a treatment that could change his life and now I have to break his heart and tell him that the UK will not fund it to help him and others.
“I’m sad because every day I have to look at photos and videos of people all over the world who are receiving treatment and posting on media about how beneficial Nusinersen is to them.
“I have to watch as others progress and know that without treatment my son will decline more and more as each day, month and year passes.”
Sam was diagnosed with SMA type 2 shortly after his first birthday.
Nice said the drug shows substantial benefits but added there are uncertainties over its long-term benefits, and the cost is extremely high.
Infants with the most severe forms of SMA usually die before the age of two.
The condition affects the nerves in the spinal cord which control movement. This causes muscle weakness, progressive loss of movement, and difficulty breathing and swallowing.
Spinraza is the first treatment to address the cause of motor neurone degeneration in
SMA - it is injected directly into the spine and is a lifelong treatment.
Gary added: “I am determined to push forward and do what we can to get this decision overturned. This is not the end, we have to pull together as a community and fight this decision.”
Muscular Dystrophy UK, along with SMA Support UK and the SMA Trust, called for a temporary scheme so patients can access the drug while evidence is collected on its long-term benefit.
“Spinal muscular atrophy can be devastating and the news will be heartbreaking for the families of those living with the condition,” said Robert Meadowcroft, chief executive of Muscular Dystrophy UK.
“Once again we are seeing families suffer due to the appraisal process being too limited to assess costly but life-changing rare disease drugs. The one glimmer of hope is for a temporary scheme that ensures access.
“We call on Nice, NHS England and the drug company, Biogen, to urgently come together and find a solution to provision and pricing so families can get Spinraza before more lives are lost.” Meindert Boysen, director of the centre for health technology evaluation at Nice, said: “Nusinersen is a promising treatment that has been shown to improve a range of outcomes important to patients.
“We are actively engaging with Biogen to discuss how they might address the uncertainties identified by the committee, while demonstrating the potential for Nusinersen to be considered cost-effective and managing the risk to the NHS of allowing access to this treatment.”