The NHS has finally made a deal for a life-prolonging drug that is given to children who fight against a cruel disease that slowly robs them of their ability to walk, talk and breathe.
Health watchdogs earlier this year rejected cerliponase alfa – the only drug available to treat one type of Batten disease because it was too expensive.
But officials have now agreed a "fair price" for the drug, marketed as Brineura, with the manufacturer after nearly two years of fierce negotiations and just weeks before affected families would appeal the decision.
The medicines are estimated to cost around £ 500,000 per patient per year, but the discount price offered to the NHS was not revealed after the U-turn.
Simon Stevens, CEO of NHS England, today warned pharmaceutical companies that health care "will not simply write blank checks at the expense of taxpayers."
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;)
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
He said: "This is another concrete step to ensure that NHS patients with rare conditions have access to important new treatments.
"After lengthy negotiations, the new deal reached today is a reminder that companies must succeed in being flexible and realistic."
Brineura is offered as part of a managed access agreement (MAA) for the next four and a half years. In the meantime, evidence of its effectiveness will be collected.
Angered families – including the parents of a boy who was once rocked into the hospital by Prince Harry – started legal action after the first decision by NICE.
The Supreme Court announced last month that it would hold a judicial review of the legality of the decision. Lawyers who fought to annul the decision claimed it was unlawful.
A full hearing in the Supreme Court is scheduled on October 16 and 17. However, it is currently unclear whether this will continue.
Samantha Barber, director of the Batten Disease Family Association, said the charity was "absolutely delighted" with the decision to approve Brineura.
She said: "Today's announcement will be a huge relief for all the affected families who fought day and night for this outcome.
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;
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.
"Brineura offers real hope for these children and the priority must now be to ensure that everyone who needs it has access as quickly as possible.
"Although we recognize the efforts of both parties to reach this agreement, the reality is that it took almost two years to reach this point.
"These human costs of this delay and the anxiety caused cannot be underestimated and we hope that efforts will be made to help others prevent this heartache in the future."
Professor Stephen Powis, National Medical Director of NHS, said, "This is important news for children and their families suffering from this devastating disease."
Children who fight the condition get access to Brineura on the NHS at Christmas at the latest. It is administered directly into the brain via a surgical implant.
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.
Brineura gives neuronal ceroid lipofuscinosis type II patients a synthetic version of a crucial enzyme that they lack for a healthy brain function
Studies have shown that Brineura can slow the progression of the disease, often labeled by affected families as a form of dementia in children.
The original draft decision of NICE in February ruled Brineura – owned by pharmaceutical company BioMarin – could not be recommended as a cost-effective use of NHS agents & # 39 ;.
It led the deeply saddened parents of two children – Oliver and Amelia Carroll – with Batten's disease to take legal action through Irwin Mitchell.
Lawyers concluded the process that the body used to reach its decision, which was open to stakeholder consultation, & # 39; may be incorrect & # 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.
Amelia started treatment before one of the paralyzing symptoms showed up. She goes to a regular school and loves dancing, gymnastics and swimming.
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, the seizures and speech problems of Nicole have been kept at a distance. Jessica has yet to develop signs of the condition. She starts nursey in September.
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;
Donate to their fundraising here
. (TagsToTranslate) Dailymail (t) health (t) nhs