Cheshire siblings will continue life-saving treatment after long legal battle
Ollie and Amelia Carroll from Poynton have Batten Disease, which causes seizures, sight loss and takes away their ability to walk and talk
Last updated 15th May 2025
Medical experts are continuing a treatment programme which is currently keeping two children from Cheshire alive, after their parents took their fight to Parliament.
Ollie and Amelia Carroll from Poynton, are aged 14 and 12. The siblings have Batten Disease which causes seizures, sight loss and takes away their ability to walk and talk.
The medical body which advises the NHS, NICE, has recommended Ollie and Amelia keep having brain infusions which are slowing down the disease.
Their Dad, Mike, is relieved the fight for his children's treatment is now over: "They can receive that treatment for the rest of their lives now which is absolutely amazing, incredible news because this has been going on for such a long time, it's been such a long fight...
"To get that news now, it's just quite emotional, to be honest.
"It's taken so much out of us as a family, battling daily, legal process, it's just been a long old journey and to get that news that we did, is absolutely fantastic."
However, Mike said the fight must continue as future patients will not recommend the treatment’s use because of its high price, and limited evidence of long-term effectiveness: "At the moment, what the draft is saying, if you get diagnosed in January and onwards, you will not receive the treatment.
"So this is the next fight now, that cannot happen, and that will not happen.
"When our children got diagnosed, we got told there was no treatment, there's no cure and just enjoy the time with your children...
"And the thought that other parents will have to sit in a room and listen to them words again, that is absolutely disgraceful and it's not going to happen.
"The newly diagnosed children from next year, we're fighting for them now, and we're not going to stop fighting for them."
What NICE has said
In a statement, NICE said: "The draft guidance does not, at present, recommend the treatment’s use for future patients with this rare, life limiting disease due to its high price and the limited evidence of long-term effectiveness.
"However, with the support of NICE, NHS England (NHSE) and BioMarin have agreed a deal to provide permanent access to cerliponase alfa (also called Brineura) within its licensed indication for people with CLN2 who have already been initiated onto treatment or will be started on treatment before the end of the newly-extended managed access period.
"This is due to close when NICE’s evaluation concludes or by the end of 2025, whichever is sooner.
"This means that people will be able to stay on the treatment even if NICE is unable to recommend it for new patients beyond that point.
"NICE, NHSE and the company continue to work towards a solution to secure access to all future patients but at the moment the treatment is not considered cost effective."
Helen Knight, director of medicines evaluation at NICE, said: “We’re pleased that NICE has been able to support NHSE and the company in reaching an agreement to make access to cerliponase alfa permanent for everyone who has already started treatment and those who will start treatment before the managed access agreement ends in December. NICE, together with NHS England, remains committed to working with the company to try to reach a long-term deal that will give access to cerliponase alfa to all eligible people after that time.”
The treatment has been available to NHS patients while further evidence has been collected on its use and benefits. That ‘managed access period’ has been extended several times, although without the requirement for additional data collection, and will now end in December 2025 (or when NICE publishes its final recommendations if earlier). The additional evidence collected prior to the extension of the MAA is being evaluated by NICE to determine whether it can be used routinely on the NHS.
After looking at the new evidence from children having treatment in the NHS in England, the independent NICE committee concluded that, although cerliponase alfa has substantial short-term benefits in slowing the rate at which CLN2 gets worse, there is not enough evidence to show what benefits it provides in the long term. In particular this relates to how a person’s health state when they start treatment dictates how much they will benefit from treatment.
The committee also took into account the rarity of CLN2, its severity and the effect of cerliponase alfa on length and quality of life. It recognised the significant benefits it provides by applying a cost-effectiveness threshold 1.5 times higher than that normally applied to treatments for ultra-rare conditions and more than 5 to 8 times higher than that normally applied to treatments being evaluated outside of the HST programme.
But, because of the lack of long-term evidence of effectiveness, the price of the medicine proposed by the company is still far higher than NICE can consider an effective use of NHS resources.
Helen Knight continued: “We know this is not entirely the news people in the Batten disease community were hoping for. However, this is not the end of the story. We will continue to work with all parties towards a solution.”
With a list price of more than £500,000 per patient for each year’s treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.
It is estimated that in the UK there are around 30 to 50 children living with CLN2 with around 3 to 6 children newly diagnosed each year.