‘NHS DRUG BAN IS KILLING OUR KIDS’ Fight to save Amelia and Ollie
PARENTS of two children with a terminal rare condition have been told the NHS will not fund lifesaving treatment.
Mum and dad Lucy and Mike Carroll should be spending today celebrating their daughter’s sixth birthday.
Instead, they will be continuing to fight for their little girl’s life.
Amelia and her brother Ollie, eight, have the genetic condition late infantile Batten disease and are being kept alive by a wonder drug.
Its manufacturer BioMarin had been covering the cost of the treatment – an estimated £500,000 per child, per year – as part of a clinical trial.
But it has emerged that the breakthrough treatment will not be funded by the NHS because it is too expensive.
Last night, dad Mike, 33, said: “This is a life and death situation. You can’t put a price on a child’s life.
“If the drug is taken away from our children, they will deteriorate rapidly and die.”
Liverpool-mad Ollie was a healthy and active child who loved playing footie with his older brothers at the family home in Poynton, Cheshire.
But by the age of four he had started having seizures and his speech and mobility became more limited until he could no longer walk or talk. Within a year he had lost his sight and had to be fed through a tube.
Lucy said: “When the neurologist told us the news it was devastating.
“We were in shock and cried a lot. We asked if there was any treatment, any cure, anything, in any country, that would help.
“To hear that there was nothing was heartbreaking.”
Lucy, 32, and Mike were soon hit with another life-changing setback when blood tests revealed that Amelia also had the disease.
Lucy said: “Getting the news a second time was even worse than the first.
“We had no questions to ask – we already knew the answers.”
The family launched a crusade to find some form of treatment and, after a two-year battle, were handed a lifeline.
Ollie was started on the drug, an enzyme therapy, on grounds of compassionate use at London’s Great Ormond Street Hospital, where he was visited by Prince Harry. The benefits of the treatment were instant.
Lucy said: “Ollie has been seizure-free for 18 months, he is pain-free and he enjoys a life full of fun and laughter.
“He laughs, smiles and rolls his eyes at us. We never see him sad any more.”
Amelia also had the treatment, called cerliponase alfa, and has thrived. Lucy said: “She can jump, run, hop and climb, she can speak in sentences, sing her favourite songs and is learning her animals and colours.
“She is showing the world what can be achieved by treating young children with CNL2 Batten disease.”
The family’s lives had been turned around, but two weeks ago that lifeline looked to have been snatched away.
The treatment will not be recommended for NHS use over “concerns about its long-term effectiveness”. The National Institute for Health and Care Excellence (NICE) said the decision not to recommend the drug came “at the end of year-long negotiations” between the company which produced it and NHS England.
It said the company was unable to “price the treatment” at a level that would have addressed the problems highlighted during NICE’s assessment.
Lucy said: “We have been handed hope only to have it ripped away.
“If the treatment is stopped then Ollie will not be with us in 12 months. Amelia will be in a wheelchair, she won’t be able to talk and she’ll be fed through a tube. She’ll also die.”
Mike added: “We won’t take the decision lying down. We’ll keep campaigning.”
The draft guidance from NICE is now out for consultation.
The drug company, healthcare professionals and patient/carer organisations all have the opportunity to appeal against the recommendations.
DAILY STAR SUNDAY SAYS
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