Parents ‘in shock’ after drug victory
THE parents of two terminally-ill children say they are ‘in utter shock’ after a drug they have been fighting for has been approved.
We reported just last month how Lucy and Mike Carroll were taking their battle to the High Court after a health watchdog decided lifesaving treatment should not be funded on the NHS.
Their children, Ollie and Amelia Carroll, eight and six, from Poynton, have been diagnosed with rare genetic condition; Late Infantile Batten’s Disease, which could cut their lives devastatingly short.
Over time, both children are at risk of losing their speech, mobility, eyesight and ability to swallow.
Thanks to a specialist treatment, known as cerliponase alfa - which has been provided to the kids on ‘compassionate grounds’ - the effects of the disease have been halted.
But in February last year, they were left heartbroken after health watchdog NICE concluded it could not recommend the drug for use by the NHS because it was too expensive.
But now the family has been given fresh hope with a U-turn from NICE, and committee members agreeing its use on the NHS ‘ subject to a managed access agreement’.
Sharing the news on their Ollie’s Army Battling Against Battens Facebook page, Lucy and Mike said: “We are in utter shock right now so we will write a full update later, but right now we sit in tears with the news that The National Institute For Health And Care Excellence have now RECOMMENDED the ONLY treatment for Batten Disease to children in England.
“We are in talks with NICE as to what happens next, thank you to everyone from the bottom our hearts.”
Hundreds of supporters have reacted to the post, with many commenting on how they thrilled they are for the family.
Barbara Brooks said: “I am so thrilled for you all and all children suffering with this awful condition, well done for all your fighting.”
And Jen Elizabeth added: “I have goosebumps reading this. I am so relieved for your family and all the others you have helped. I cannot imagine how happy you are. WELL DONE!!!! Now you can relax and focus on your children and your lives again.”
It is estimated that the drug could boost the children’s life expectancy from 13 years to 60.
The family had been granted permission to take their fight to the High Court where a full hearing had been expected to take place in October.
It is estimated that between 30 and 50 children in the UK have Batten disease.
The specialist drug is already funded by health services in 23 other countries, including Wales.