The father of a nine-year-old girl diagnosed with a rare neurodegenerative disease has called on health authorities to extend access to a life-saving drug, which he describes as “miraculous.”
Addy Clarke, from Swindon, was diagnosed with Batten Disease, a rare form of childhood dementia, when she was four years old. The condition progressively impairs motor skills, including the ability to walk, talk, and eat.
Addy’s parents say the drug Brineura has been instrumental in preserving their daughter’s abilities and extending her life expectancy. However, with the treatment’s managed access agreement set to expire on May 27, 2025, there are concerns over whether continued access will be guaranteed.
The National Institute for Health and Care Excellence (NICE) has confirmed that Brineura will not be included in standard NHS care beyond this date, although discussions with the drug’s manufacturer are ongoing.
Dave Clarke, Addy’s father, expressed his hope that the government will take action to ensure the treatment remains available for children like Addy, describing it as a “miracle” for those suffering from the disease.
“Your child grows up absolutely normal, and the first sign something is wrong is usually a seizure,” Clarke told BBC Radio Wiltshire. “Then, they begin losing skills—walking, talking, even basic abilities like feeding themselves—until they’re diagnosed. For children who have been on this drug for several years, the results are nothing short of miraculous.”
Since Brineura was introduced in clinical trials, the number of children diagnosed with the specific form of Batten Disease Addy has been diagnosed with has grown from 15 to 40 in the UK. Clarke emphasizes the drug’s proven effectiveness in prolonging both the quality and length of life for affected children.
Despite its effectiveness, concerns are mounting about the future of the treatment due to the looming expiration of the managed access agreement, which began in 2019. Under this agreement, the drug was provided with the understanding that additional data would be needed to assess its long-term viability. A six-month extension was granted in October 2024, but the future beyond May 27 remains uncertain.
The cost of Brineura is high, with each patient requiring a fortnightly dose priced at £522,722 annually. However, Clarke argues that the drug’s efficacy in improving the lives of children like Addy justifies the cost.
“I don’t know the exact price of the treatment per child, but what we do know is that it works. These children are thriving on it,” Clarke said.
The uncertainty surrounding continued access to Brineura has led to increasing anxiety among families. On Wednesday, dozens of affected families gathered outside Westminster, urging NICE and the government to negotiate a deal that would ensure the drug remains accessible.
“The waiting is prolonging the agony for families like ours. It’s creating an immense amount of mental stress,” Clarke added. “We just want our children to have the best chance at life.”
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