Family's 'excruciating' wait over drug decision

Jessica is receiving a life-prolonging treatment for Batten Disease

The family of a girl with a rare neurodegenerative condition fear losing her if the NHS cannot permanently fund her treatment.

Gail Rich's daughters Nicole and Jessica, from Newcastle, were both diagnosed with Batten Disease, which is incurable.

Nicole died from complications linked to it in 2023, while Jessica, nine, still receives a life-prolonging treatment, but the decision whether to keep the drug, Brineura, on the NHS has been delayed by six months.

"It's excruciating having to wait. We've got no control. We just have to sit and wait to see whether or not our children will have a future," Ms Rich said.

In a statement, the National Institute for Health and Care Excellence (NICE) said discussions between NHS England and drug company BioMarin had so far been "constructive".

'Coming down to money'

Jessica receives a brain infusion every two weeks at Newcastle's Royal Victoria Infirmary to slow down the progression of the disease.

Ms Rich said her daughter was living proof the treatment worked because she was living an active life and loved going to school and dancing.

Since 2019, Brineura has been provided for eligible NHS patients under a managed access agreement.

But Ms Rich fears the treatment will be withdrawn and said that, without it, her daughter would quickly deteriorate.

"It's not the effectiveness of the treatment that's now being decided upon, because everyone agrees it works. It's how much they are willing to pay," she said.

"It's all coming down to money now. Our daughter's life depends upon a financial agreement being reached. That's really hard to understand…what's the price of a life?"

'Precious' time

A decision on the permanent use of the drug is now expected at the end of May 2025.

The Batten Disease Family Association's Liz Brownnutt said there were now six months of uncertainty for families.

"Their time is precious with their children and they shouldn't have to be worried about whether or not their children are going to get this treatment in the future," she said.

NICE said: "All parties will now use the extension period to try to reach an agreement that secures permanent access for current patients and allows new patients to start treatment."