THE mum of a six-year-old boy with a rare genetic disorder has hit out at an NHS decision not to supply the drug that has transformed his life in this country.
Philippa Ward’s son, Thomas, has a disorder called tuberous sclerosis complex (TSC).
The illness causes non-cancerous tumours to develop on his brain and these growths cause epilepsy and learning difficulties.
Thomas was suffering up to eight severe seizures a day and the family had tried a range of treatments before he was given the drug everolimus about four years ago as part of a clinical trial.
The treatment is available on the health service in Scotland, but patients in England suffering from the condition will be denied the same drug.
Philippa, from Wigginton, said: “It meant Thomas couldn’t really have a life.
“You always had to be extra cautious about taking him out or leaving him with people. His development was affected.
“It had a disastrous impact on the whole family, it’s heartbreaking to have a child with this condition.
“But since going on the drug trial Thomas’ development has come on loads and he seems happy.
“He’s funny and cheeky and naughty - he’s developed a love of Youtube and he dances around the kitchen.
“In his own way he’s just loving life and without the drug he wouldn’t be like this. It has changed our lives.”
But on Saturday NHS England announced it would not be funding the treatment for people suffering from epilepsy like Thomas, even though NHS guidance said there is enough evidence to consider making the drug available.
Philippa said the news was “devastating” and added that it is “crazy” that the drug has been approved for patients living in Scotland, but not England.
The 40-year-old, who also has an eight-year-old son named Ben, said: “This is known as the drug you want your children to be on if they have this condition.
“Within a few months of taking it his seizures pretty much stopped. The effects have been amazing for him and the tumours have not grown during that period.
“Other parents I know are devastated. They were hoping that their children will be able to get this drug and it’s heartbreaking.
“I wouldn’t be surprised if people did move to Scotland. People go to extraordinary lengths for their children.”
She added that the family hopes Thomas will be able to stay on the drug as part of the clinical trial but is calling on NHS England to reconsider its decision not to fund the treatment.
“It’s devastating that other children will not be able to have this quality of life,” she said.
More than 10,000 people suffer from the condition and more than half of them have epilepsy which will not respond to normal treatments.
Maxine Smeaton, chief executive of the Tuberous Sclerosis Association, said NHS England had made the “wrong decision”.
She added: “NHS England’s decision to ignore the evidence base and feedback from experts treating and living with TSC is disappointing beyond words.
“The difference that everolimus makes to the lives of people with TSC who have refractory epilepsy is unprecedented, and the TS community in England will be devastated by this news.”
The NHS estimated that the annual cost of providing the drug to eligible adults and children would be just under £60million.
An NHS spokesperson said: “The panel of doctors, clinicians, and the public assessed everolimus for refractory seizures associated with tuberous sclerosis complex and concluded that it delivered limited benefits compared to other treatments being considered.
"This treatment can be considered again for funding in November but drug companies have an important part to play by pricing their treatments responsibly.”
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