The health watchdog is being taken to court over claims it has breached human rights by not approving a drug to treat a cruel and deadly syndrome.
NICE controversially rejected the only available medication to treat one type of Batten disease for use on the NHS in England in draft guidance in February.
Outraged families – including the parents of a boy once cradled by Prince Harry in hospital – launched legal action to get cerliponase alfa available.
The High Court has now announced it will hold a judicial review into legality of the decision. Lawyers fighting to overturn the decision claim it was unlawful.
Campaigners now hope the court battle will prompt officials to strike a deal with the manufacturer of the drug, believed to cost around £500,000 per patient per year.
The Batten Disease Family Association say the drug, marketed as Brineura, will help children battling the illness ‘live the lives they should be living’.
Outraged families – including the parents of a boy once cradled by Prince Harry in hospital – launched legal action to get cerliponase alfa available (Oliver Carroll’s plight touched the nation in 2016 when a striking image emerged of the six-year-old being hugged by the Prince – dubbed his ‘Diana moment’)
The drug – a form of enzyme therapy – is already available in 20 other countries, including Wales, where health care is devolved, and the Netherlands.
There is currently no cure for the CLN2 form of the condition, estimated to strike only 30 to 50 children in the UK – around a fifth of all Batten disease patients.
Brineura gives neuronal ceroid lipofuscinosis type II patients a synthetic version of a crucial enzyme they are missing that is required for healthy brain function.
Without treatment, life expectancy ranges from between six and 13. It slowly robs children of their ability to walk, talk, see and breathe.
Studies have shown Brineura can slow down the progression of the disease, often labelled by affected families as being a form of childhood dementia.
Samantha Barber, chief executive of the BDFA, said she has been hauled into a last-minute meeting with NICE, NHS England and manufacturer of the drug BioMarin tomorrow.
She told MailOnline: ‘We have had incredibly short notice of this meeting… I have no sense whether it’s a good thing or a bad thing.’
However, officials refuted her claims, saying it was arranged ‘weeks ago’ with all the key stakeholders in the discussion being notified at the time.
Caroline Day (left), mother of a three-year-old Connie who is struck down with Batten disease (right) told MailOnline how she fears a meeting tomorrow between officials and the drug manufacturer is just going to be a ‘tick box exercise’
Amelia Carroll, Oliver’s brother, who started the treatment before she developed any symptoms of Batten disease, is a happy, healthy little girl, pictured with Prince Harry in 2016
Parents are sceptical – a mother of a three-year-old struck down with Batten disease told MailOnline how she fears the meeting is just going to be a ‘tick box exercise’.
Ms Barber added: ‘It’s really sad that it’s come to this. I’m really glad that the court is listening to the cases – but it shouldn’t be this way.
‘I call on all parties to remember the children in all of their dealings. At the bottom line, we are talking about children who, without this treatment, would be dead very, very quickly.
‘I’m saddened it’s taken this long. We shouldn’t have had to fight this hard, and the families have been through so much more additional stress than they needed to be.’
Discussing Brineura, she said: ‘[It] will help them live the lives they could, and should, be living. They don’t need to die, they just need to get the treatment.’
And Ms Barber added that she promised all of the families affected by Batten disease ‘the biggest party ever’ if NICE overturns its stance.
The body’s initial decision ruled Brineura could ‘not be recommended as a cost-effective use of NHS resources’. It’s unclear when the final guidance will be issued.
Following the decision, the heartbroken parents of two children – Oliver and Amelia Carroll – with Batten disease launched legal action through the firm Irwin Mitchell.
Lawyers concluded the process NICE used to reach the initial draft decision, which was open for consultation for stakeholders, ‘may have been flawed’.
In a statement issued on its website, the legal firm said its solicitors ‘believe the decision is unlawful on several grounds and is also a breach of human rights legislation’.
Oliver, eight, and six-year-old Amelia currently receive access to Brineura as part of a trial at Great Ormond Street Hospital funded by BioMarin – the manufacturer.
Before being given the drug, Oliver had been robbed of his ability to walk and talk – but his family, of Poynton in Cheshire, say his health is now stable.
Brineura gives neuronal ceroid lipofuscinosis type II patients a synthetic version of a crucial enzyme they are missing that is required for healthy brain function
WHAT IS BATTEN DISEASE?
Batten disease is a rare genetically-inherited disease, caused by waste build-up in the cells which creates neurodegnerative effects including:
- Personality changes
- Loss of motor skills and the ability to walk, talk and communicate
Because it is a rare disease, it is often difficult to diagnosed and untested at birth.
There are 14,000 children worldwide that are diagnosed with the disorder.
The Batten Disease Family Association estimate that around up to 17 people are diagnosed with a form of the disease each year.
There are between 100 – 150 affected individuals currently living with Batten disease in the UK.
There is currently no known cure or treatment available for Batten disease.
Amelia started the treatment before any of the crippling symptoms emerged. She attends a mainstream school and loves to dance, gymnastics and swimming.
The pair’s 34-year-old mother, Lucy, said: ‘Our children mean everything to us. We remain determined to fight so that Ollie and Amelia receive the treatment that they need.
‘It is heartbreaking to think that individuals have made the decision that our children should not have access to the only treatment that could save their lives.
‘We continue to hope the NHS, NICE and BioMarin will work together to ensure all children with Batten disease in this country will have access to this life-saving treatment.’
Gail and Matthew Rich, a couple from a village near Newcastle who also have two daughters with CLN2, launched legal action through the same firm shortly after.
Nicole, seven, and her three-year-old sister Jessica both use Brineura, one through a drug trial and the other through a scheme paid for by BioMarin.
Since taking the drug, Nicole’s seizures and speech problems have been kept at bay. Jessica has yet to develop any signs of the condition. She starts nursey this September.
Mrs Rich, 41, said: ‘Our children mean the world to us and all we want to do is help them achieve everything they can in life.
‘This treatment has been hugely effective and has given us real hope for the future, so it is difficult to take that it has not been recommended for use here.
‘How can it be available in 23 other countries but not here? Something has to change and we think NICE need to show some compassion and urgently rethink how they came to this conclusion.’
A full hearing in the High Court is now scheduled to be held on 16 and 17 October. The families have been granted legal aid.
MOTHER CLAIMS NOT GIVING THE DRUG TO HER DAUGHTER WILL BE LIKE HANDING HER A ‘DEATH SENTENCE’
Ms Day said her three-year-old Connie may not even reach her sixth birthday without the life-extending drug
A mother-of-two claims not giving Brineura to her Batten disease-suffering daughter will be like handing her a ‘death sentence’.
Caroline Day, from Leeds, said her three-year-old Connie may not even reach her sixth birthday without the life-extending drug.
Connie, whose full name is Constance-Annakin, was diagnosed with the CLN2 form of Batten disease in June, after two months of tests.
Ms Day, 41, told MailOnline: ‘If Connie got this drug it would give us our normality back that I feel we’ve had taken away from us.’
Without Brineura, she will go blind, be bed-ridden, have ‘seizure after seizure’, lose her ability to walk, talk and eat, Ms Day added.
‘I just want her to live a normal, happy life and not have a life cut short. She might not even make it to six without treatment.’
She has been on sick leave since April, when her daughter was first struck down with seizures – a symptom of the crippling condition.
Ms Day, who gives 24-hour care to Connie, told MailOnline: ‘Realistically, I’m not going to be able to go back. That might be different if she gets treatment.’
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