Western Daily Press (Saturday)
MAX FORCED TO MOVE FOR TREATMENT
NHS REFUSES TO PAY FOR DRUG:
ABOY with a rare genetic disorder that is slowly killing him has had to move to The Netherlands this week so he can get access to a drug that could prolong his life.
Max Sewart and his dad Simon travelled on Tuesday to set up home in Rotterdam, where Max can be treated for a rare type of Batten Disease, leaving mum Ivana and younger brother Felix back in Britain.
The eight-year-old from Somerset has become the victim of a stand-off between NHS drugs chiefs and the manufacturer of medication that can slow down the onset of Batten Disease, an extremely rare group of fatal, inherited disorders of the nervous system.
NHS bosses in England are refusing to pay the asking price for the drug called cerliponase alfa, marketed as Brinuera, even though it is available in 20 other countries including Wales.
It can reportedly slow down the progression of Batten disease and even stabilise it in cases.
Max, from East Harptree in the Chew Valley, was only diagnosed with the condition
a few months ago, and has rapidly deteriorated even though the drug that could stop that exists.
His family set up a crowdfunding page to raise money to try to fund it themselves, as well as a treatment called critical enzyme replacement therapy.
But they have only managed so far to raise around £15,000 of a half a million target, which they say they need to fund what they hope will be a cure for this devastating and fatal disease.
Mr Sewart has joined forces with the families of just over a dozen other children in the same position, fighting for the NHS to end the limbo that sufferers are going through after talks between the NHS and the drug’s manufacturers broke down.
Max and Simon have set up home in Rotterdam to be eligible for treatment under the Dutch health service – something that would not have been possible had they simply moved to Wales.
“It’s an absurd situation. It’s bad enough with the diagnosis, but to have to do this nonsense too,” said Mr Sewart.
“I feel so badly let down by the NHS – it’s not what the service is about. It’s about looking after your society. Their negotiators are not humane people.”
Mr Sewart said the drug available is “highly effective” – but concedes it’s also pricey.
“It’s a rare disease drug, so it’s very expensive,” he said.
“We want to preserve Max in the state he’s in. We’ve seen his decline in the last four months. We’ve given up on the NHS.”
The Sewarts’ MP, Jacob Rees-Mogg, has backed their campaign, and raised their case – and those of other children – in the House of Commons several times.
Last week, he did so again, demanding answers from the NHS and health ministers.
“The terrible thing is that in the month that has passed since I first raised this matter in the House, Max’s condition will have slightly deteriorated.
“In every month that goes on while we are debating this, not just Max but all the children with this condition will go downhill. That is what happens with this disease,” he told MPs last week.
“Although I accept, of course, that there is a need to look at costs, when we are talking about eight-year-old children, we are not talking about a cost for people who only have weeks or months to live, but about a child who could have years of a high quality of life ahead of him.”
At the weekend, Mr Rees Mogg was one of hundreds who attended the village fete in East Harptree, which was this year held as a fundraiser for Max.
Organised jointly between the school and the church, a record £5,000 was raised towards the public appeal, to help the family.
Max and Felix’s headteacher at the village primary school, Jane Bailey, said she was proud of the response from the village.
“We are striving to support Max, his younger brother and parents at this desperately challenging time within school,” she said.
An NHS England spokeswoman said: “The National Institute for Health and Care Excellence (NICE) have not recommended this drug, since British taxpayers simply cannot offer a blank cheque to this particular American pharmaceutical company BioMarin, who have sadly so far shown a complete lack of willingness to price their products fairly, as independently assessed by NICE.”
A spokeswoman for BioMarin said: “BioMarin is deeply disappointed by the NICE and NHS England decision to not approve cerliponase alfa for the treatment of CLN2 disease, this is a tragic outcome for children living in England with this debilitating disease.
“Patients with ultra-rare diseases in England face greater barriers to access effective new and innovative medicines through restrictions imposed by the changes in the NICE HST appraisal process, compared to other countries worldwide.
“It should be noted that the 11 patients already receiving treatment in England are all currently being given treatment free by BioMarin.
“By doing so BioMarin is doing everything it can to help patients and their families, but the company cannot be expected to simply treat all patients for free – it is not a sustainable solution.”
It is estimated that there are approximately 100 to 150 children, young people and adults currently living with an NCL diagnosis in the UK.
Symptoms include visual impairment, epilepsy, seizures, difficulty sleeping and the loss of mobility.
Death is inevitable and, depending on the age of onset and specific genetic diagnosis, may occur anywhere between early childhood and young adulthood.
I feel so badly let down by the NHS – it’s not what the service is about. It’s about looking after your society. Their negotiators are not humane people
SIMON SEWART