A Wigan family are fighting against a decision by the NHS not to fund a new epilepsy drug which has drastically improved the life of their son.
Linda and Phil Gavin have been left devastated after Everolimus, a drug used in the treatment of people with Tuberous Sclerosis Complex (TSC), was rejected by NHS England over concerns about its cost.
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It has greatly helped their son Stephen, who is being treated with the drug on a trial basis. But his health now hangs in the balance, and his parents fear his severe convulsions will “come back with a vengeance” if he is denied access to the treatment.
TSC is a genetic condition that can lead to growths in various organs of the body, and it is estimated to affect 10,000 people across the UK. Seizures are just one of the symptoms, as are learning difficulties and irregular heart rhythms.
Everolimus has been part of medical trials across the UK, and was recently approved for use by the NHS in Scotland. But to the family’s dismay last weekend it was knocked back by NHS England, despite recent reports showing positive signs that it would get their approval.
An assessment report estimated that the annual cost of providing Everolimus to eligible adults and children living with TSC in England would be just under £60 million.
The 39-year-old, who lives with parents Linda and Phil in Appley Bridge, has been taking the drug for the past three years as part of a trial at York Hospital.
Since it began, the number of Stephen’s daily seizures has halved, and their severity has also been reduced.
His parents said the drug has had an amazing effect on their son, and the NHS’s decision not to fund it was “devastating”.
Dad Phil said: “It has made the most significant difference to his seizures, they have reduced by half.
“It also reduced the severity of them too, so it made a massive difference to his life. There have even been days when he hasn’t had seizures at all. That is unheard of for Stephen.”
Mum Linda said: “This is the only drug that we feel has had an impact on Stephen’s quality of life. The decision is so disappointing, it’s devastating.
“We want to stress how much good this drug has done, not just for Stephen, but for us as a family. He is striving for the best life he can have. It’s so unfair that Scotland have approved this and England have not. They have seen the same evidence, and yet one approved it and one did not. We are completely shocked by it.”
Linda added: “It’s not just our son. Right across the globe, everyone has seen a significant impact. I just can’t understand. We are very shocked.”
Maxine Smeaton, Chief Executive of the Tuberous Sclerosis Association (TSA), said: “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.”
They may be devastated, but the family have far from given up hopes that the drug will become available in England.
Phil said: “We will continue the fight with the TSA. We won’t give up the fight until this is overturned.
“We are hoping that by the time the trial is over, the NHS will have approved it. But if it stops, Stephen’s seizures will come back with a vengeance.”