Family of Chesterfield girl, 4, who has fatal Batten Disease, plead for ‘crucial’ drug to become available on NHS to help prolong her life
“It really is a matter of life or death.”
Four-year-old Grace Thompson, of Chesterfield, is a happy, bubbly and active little girl. She loves her friends, animals and playing outside on her scooter.
In February this year she was not her usual, playful self. Her speech became slurred and she started to fall over a lot.
Worried about her health, her loving parents Izabela, 37, and her partner David, 52, took her for some tests.
And sadly in July their worst fears came true. Grace was diagnosed with Batten disease, a very rare inherited degenerative condition that affects between one and six babies each year in the UK.
Batten disease takes away vision, ability to walk, talk and hear, and eventually life.
Those who are diagnosed with the condition tend to only live until about the age of 10 or 12.
But there is some hope for Grace.
Although there is no cure, there is a drug called Brineura which costs around £500,000 a year. It has been proved to work and could extend Grace’s life by as many as 20 years.
However, the drug is not available on the NHS.
The National Institute for Health and Care Excellence (NICE), the independent body which provides the NHS with evidence-based guidance on the clinical and cost effectiveness of drugs, was expected to make a decision on Brineura this week but has now delayed it. NICE said ‘further work’ is being carried out which will be completed ‘in the near future’.
But time is not something the Thompson family have.
Grace’s mum, Izabela, of Cordwell Avenue, Newbold, told the Derbyshire Times: “We do not know how much time we have got. For us, it is absolutely crucial to get this treatment now. We just do not have time to waste.
“At the moment our lives have been turned upside down. It has been absolutely devastating news for us. My heart has broken into thousands of pieces and I am just losing every piece every day. She is my world.”
She added: “When people come to see Grace they do not realise what is going on.
“She is getting worse day-by-day. Every day is crucial.
“The more people that are aware the better.”
In a statement in February, Nice said that an independent committee had agreed that, although Brineura is not a cure for Batten disease, ‘it had shown substantial short-term benefits in slowing the rate at which it progresses’.
The statement read: “However, in the absence of long-term evidence about its effectiveness in stabilising the disease and preventing death, and having taken all the health and non-health-related benefits of cerliponase alfa (Brineura) into account, the independent committee considered that the drug was not value for money within the context of a highly specialised service (the NHS).”
When the Thompson family found out about Grace’s condition, they understandably hit rock bottom.
“I felt angry, devastated and scared for her,” Izabela said. “Our lives turned upside down. We just have hope. We have to be strong to fight for Grace.”
So what would it mean to the Thompson family if Brineura became available now?
“It is the difference between life or death for Grace,” Izabela said.
Backed by Chesterfield MP Toby Perkins, the Thompson family is keen to spread the word and raise awareness of the need for the treatment to be made available immediately.
Mr Perkins said: “We have written to the Secretary of State for Health, NICE and the CCG (Clinical Commissioning Group).
“This is a case of the Government making a judgement on whether Grace’s life is worth saving or not. It is a matter of life or death.
“We know that the drug works and, yes, it is expensive but what price do you put on a child’s life?”
A NICE spokesperson said: “After the NICE Highly Specialised Technologies committee meeting held on 25 July, NHS England and the company have agreed with NICE to undertake further work on the value proposition of cerliponase alfa (Brineura) for consideration by the committee. We envisage this work will be completed in the near future. NICE will not issue draft final guidance on this topic while these discussions are ongoing.”
Seema Kennedy, Minister for Public Health and Primary Care, recently told the House of Commons: “We have to rely on the NICE process to be independent. And yes these are exceptionally difficult cases and this is why as custodians of NHS funds we have to be very, very careful because every pound we spend on one drug is a pound we can’t spend on another.”
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