The health watchdog is being sued for claims that he has violated human rights by not approving medication for the treatment of a cruel and deadly syndrome.
NICE controversially rejected the only medication available to treat one type of Batten disease for use on the NHS in England in draft guidelines in February.
Angered families – including the parents of a boy who was once rocked into the hospital by Prince Harry – launched legal action to make cerliponase alfa available.
The High Court has now announced that it will conduct a judicial review of the legality of the decision. Lawyers fighting to annul the decision claim it was unlawful.
Campaigners now hope that the lawsuit will encourage officials to make a deal with the drug manufacturer, which is believed to cost around £ 500,000 per patient per year.
According to the Batten Disease Family Association, the drug, marketed as Brineura, will help children fight the disease & lead the life they should live & # 39 ;.
Angered families – including the parents of a boy who was once rocked into the hospital by Prince Harry – launched legal action to make cerliponase alfa available (Oliver Carroll & # 39; s situation hit the nation in 2016 when a striking image of the six-year-old emerged who was embraced by the Prince – he called his & # 39; Diana moment & # 39;)
The drug – a form of enzyme therapy – is already available in 20 other countries, including Wales, where healthcare is distributed, and the Netherlands.
There is currently no cure for the CLN2 form of the disorder, which affects an estimated 30 to 50 children in the UK – about one-fifth of all Batten's disease patients.
Brineura gives neuronal ceroid lipofuscinosis type II patients a synthetic version of a crucial enzyme that they lack for a healthy brain function.
Without treatment, life expectancy varies between six and 13. It slowly robs children of their ability to walk, talk, see, and breathe.
Studies have shown that Brineura can slow the progression of the disease, often labeled by affected families as a form of dementia in children.
Samantha Barber, CEO of the BDFA, said she was dragged to the last minute tomorrow in a meeting with NICE, NHS England and the manufacturer of the drug BioMarin.
She told MailOnline: & # 39; We have had an incredibly short message from this meeting … I have no idea whether it is a good or a bad thing. & # 39;
However, officials refuted her claims and said it & # 39; weeks ago & # 39; was arranged whereby all major stakeholders in the discussion were informed at that time.
Caroline Day (left), mother of a three-year-old Connie who is affected by Batten's disease (right) told MailOnline how she is afraid of a meeting tomorrow between officials and the drug manufacturer is just a & # 39; checkbox & # 39;
Amelia Carroll, Oliver's brother, who started treatment before developing symptoms of Batten's disease, is a happy, healthy little girl, pictured with Prince Harry in 2016
Parents are skeptical – a three-year-old mother who was afflicted with Batten's disease told MailOnline how she feared the meeting would just be a & # 39; tick box exercise & # 39; is becoming.
Mrs Barber added: & # 39; It is really sad that it came to that. I'm really happy that the court listens to the cases – but it shouldn't be.
& # 39; I call on all parties to remind the children in all their actions. In the end we are talking about children who would be dead very, very quickly without this treatment.
& # 39; I am sad that it took so long. We should not have been fighting so hard, and the families have experienced so much more stress than necessary. & # 39;
She spoke about Brineura and said: & # 39; (It) will help them live the life they could and should live. They don't have to die, they just have to get the treatment. & # 39;
And Mrs. Barber added that they are all families affected by Batten's disease & # 39; the largest party ever & # 39; had promised if NICE overturned its position.
The body's original decision ruled that Brineura & # 39; could not be recommended as a cost-effective use of NHS agents & # 39 ;. It is unclear when the final guidance will be issued.
After the decision, the deeply grieved parents of two children – Oliver and Amelia Carroll – with Batten's disease took legal action through Irwin Mitchell.
Lawyers concluded the process that NICE used to arrive at the first draft decision, which was open for stakeholder consultation, & # 39; may be defective & # 39 ;.
In a statement on its website, the law firm said that its lawyers believe that the decision is unlawful on various grounds and is also a violation of human rights law.
Oliver, eight and six-year-old Amelia, are currently gaining access to Brineura as part of a trial at Great Ormond Street Hospital, funded by BioMarin – the manufacturer.
Before Oliver was given the medicine, he was deprived of his ability to walk and talk – but his family, from Poynton in Cheshire, says his health is now stable.
Brineura gives neuronal ceroid lipofuscinosis type II patients a synthetic version of a crucial enzyme that they lack for a healthy brain function
WHAT IS BATTEN DISEASE?
Cat disease is a rare genetic disease, caused by waste accumulation in the cells that causes neurodegnerative effects, including:
- Epileptic attacks
- 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 diagnose and not tested at birth.
There are 14,000 children worldwide who are diagnosed with the condition.
The Batten Disease Family Association estimates that around 17 people are diagnosed with a form of the disease every year.
There are between 100-150 affected individuals currently living with Batten's disease in the UK.
There is currently no known cure or treatment for Batten's disease.
Amelia started treatment before one of the paralyzing symptoms showed up. She goes to a regular school and loves dancing, gymnastics and swimming.
The couple's 34-year-old mother, Lucy, said: “Our children mean everything to us. We remain determined to fight so that Ollie and Amelia get the treatment they need.
& # 39; It is heartbreaking to think that individuals have decided that our children should not have access to the only treatment that could save their lives.
& # 39; We continue to hope that the NHS, NICE and BioMarin will work together to ensure that all children with Batten's disease in this country have access to this life-saving treatment. & # 39;
Gail and Matthew Rich, a couple from a village near Newcastle, who also have two daughters with CLN2, took legal action with the same company shortly thereafter.
Nicole, seven, and her three-year-old sister Jessica both use Brineura, one via a drug trial and the other via a scheme paid for by BioMarin.
Since taking the medicine, Nicole's attacks and speech problems have been kept at bay. Jessica has yet to develop signs of the condition. She starts nursey in September.
Mrs. Rich, 41, said: “Our children mean the world to us and we only want to help them achieve everything they can in life.
& # 39; This treatment has been enormously effective and has given us real hope for the future, so it is hard to believe that it is not recommended here.
& # 39; How can it be available in 23 other countries, but not here? Something needs to change and we think NICE should show some compassion and urgently need to reconsider how they came to this conclusion. & # 39;
A full hearing in the Supreme Court is now scheduled for October 16 and 17. The families have received legal aid.
MOTHER CLAIMS THAT DO NOT GIVE THE DRUG TO HER DAUGHTER WILL BE A & # 39; DEATH SENTENCE & # 39;
Mrs. Day said her three-year-old Connie might not reach her sixth birthday without the life-prolonging medicine
A mother of two claims she did not give Brineura to her daughter with Batten's disease, as if she were giving her a "death sentence."
Caroline Day, from Leeds, said her three-year-old Connie might not reach her sixth birthday without the life-prolonging medicine.
Connie, whose full name is Constance-Annakin, was diagnosed with the CLN2 form of Batten's disease in June after two months of testing.
Mrs. Day, 41, MailOnline said: "If Connie were to receive this medicine, it would give us back our normality that I think we took away from."
Without Brineura she becomes blind, bedridden, gets "seizure after seizure," she loses her ability to walk, talk, and eat, Mrs. Day added.
"I just want her to live a normal, happy life and not keep a life short. She may not even reach the six without treatment. & # 39;
She has been on sick leave since April, when her daughter was first hit by epileptic seizures – a symptom of the paralyzing condition.
Mrs. Day, who provides care to Connie 24 hours a day, told MailOnline: "Realistically, I can't go back. That may be different if she is treated. & # 39;
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