Swindon family of girl with rare disease 'horrified' at latest trial update

The Swindon family of a young girl with a rare brain disease welcomed the news that her trial of lifesaving treatment will continue indefinitely.
Addy Clarke is just one of 100 children in the world to be diagnosed with Batten Disease (CLN2), which is gradually taking away her ability to talk, eat and walk independently, and see.
Her parents Hayley and Dave have previously spoken to the Adver about their heartbreak at seeing their daughter fade before their eyes while she is supported by vital cerliponase alfa treatments.
Earlier this month, they celebrated NICE and the NHS coming to a commercial agreement to continue that treatment for Addy and all other children with CLN2 after the trial ends in 2025, describing the news as 'amazing'.
But they were saddened to learn that the same access to the treatment would not be given to any child diagnosed after January 1, 2026.
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NICE explained that although its committee took into account the condition's rarity, severity and the effect of the treatment on quality and length of life, the most likely cost-effective estimate based on the proposed price of the medicine is not within what it considers to be an acceptable use of NHS resources.
In a statement on Addy's Batten Adventure Facebook page, the Clarke family said: 'Whilst we are happy for our own little Addy, we sadly know firsthand the devastation that this news can cause on a family - which would be made all the worse if you knew other children in the country were on the same treatment that your child couldn't access because of the timing they were diagnosed, and cost.
'For us, this treatment has never been simply just about our Addy, but all children diagnosed with this earth-shattering disease - therefore we will fight on.
'The decisions by NICE have been horrific and it feels like they are trying to manipulate the pharmaceutical companies by using families as pawns in game.
'Thank you for all your support you have shown us through the ongoing questions, the donations, prayers, meals and many other things over the past five years of this journey.
'We take a big deep breath, gather our thoughts and go again.'
The family has met with South Swindon MP Heidi Alexander as they try to raise awareness of this disparity in the treatment of children with the rare disease.
As for Addy herself, she recently celebrated her ninth birthday by having a party with her friends and classmates at Swindon's hydrotherapy pool.

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