North East mum delighted daughter's Batten disease treatment continues but says fight isn't over yet
9-year-old Jessica Rich will still be able to access treatment
A North East mum tells us they are delighted that their daughter's treatment for Batten disease is continuing, but says the fight is not over.
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.
However, the treatment at the moment is said to be not considered cost effective for new patients.
9-year-old Jessica Rich has the condition. Her sister Nicole also had Batten disease, but died in 2023.
Gail Rich, their mum, says that they will not give up until Brineura is available for every child in the future who needs it.
She said: "When we found out that Jessica's treatment had been approved and she was safe, it would not be taken away from her, it was as such a huge weight lifted off our shoulders.
"We've been fighting for years. We've been living with the agonising weight, lengthy approval processes and feeling like we've had to campaign and advocate and justify why our daughter, and all of the other children in the UK who are currently receiving it, justify the worth of our daughter’s life.
"We're determined that we just would not give up, so to have confirmation that NHS England have agreed to fund this treatment for as long as Jessica and the children need it, is just the best news.
"It's just giving families hope. It's giving families time and it's alleviating the rapid decline that is typical and historical in this disease. We should be celebrating this treatment."
Gail continued to say: "The fight is not over because the committee have said they cannot recommend Brineura for children in the future because of uncertainties over the cost effectiveness, so they're not happy with the price basically. It's now back to negotiations.
"It's unthinkable to think that children in this country would be denied access to this life changing drug. We should be celebrating it. We shouldn't be justifying why our children should be able to get access to it.
"As a community, we are coming back together. We've celebrated the huge victory, the huge milestone in achieving Brineura for existing children. But the fight is truly not over until we secure it for children of the future."
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.”
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.