Child with rare disease will still get vital drug

A family has been told their child will continue to get access to a drug which she began taking as part of a trial, and is helping to keep her alive.

Nine-year-old Addy Clarke, from Swindon, has Batten Disease - a rare degenerative disease that has no cure.

An agreement has been reached that existing patients will continue to have the drug Brineura, which costs £500,000 per patient per year, via the NHS.

However, it has not been recommended for future patients "due to its high price and the limited evidence of long-term effectiveness", said the National Institute for Health and Care Excellence (NICE).

Addy's mother Hayley Clarke said she is "absolutely thrilled and relieved and so thankful" at the news but wants to get access to the drug for other children.

"There's still another half a fight to go to get it secured for all children, future children most importantly," she added.

She said the family could have lost Addy by now, that she should be blind, but her eyesight has been preserved and she can still make herself understood.

One of the main symptoms of Batten Disease is childhood dementia.

"We're just so thankful for the time that this drug is giving us with her," said Hayley, who also told the BBC about her daughter's passions for lions and swimming.

"I think I'm still processing that it's a yes for our daughter," she added.

"[I'm] almost trying to be really guarded with hope because we've had too much shattered hope along the way."

Hayley said the family has had a lot of support from people around them as well as local hospice charities and the council.

The family went with other with members with Batten Disease to Westminster to ask for continued access to Brineura.

Draft guidance from NICE did not recommend using it for future patients due to cost and long-term effectiveness, but said it had reached a deal with the NHS and manufacturer BioMarin for those already using it and anyone signed up by the end of 2025.

Helen Knight, director of medicines evaluation at NICE said: "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."

The charity Batten Disease Family Association said it was pleased with the agreement, but said as the NICE guidance is a draft and not final, it will continue to push to make Brineura accessible to all children who need it.

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