The parents of three terminally ill children are to meet with health bosses in a bid to challenge a preliminary ruling which decided treating them is not cost effective for the NHS.
Ollie and Amelia Carroll from Cheshire, and James Toohey from Liverpool all have Batten disease, a rare, hereditary neurological condition which, without treatment, will deprive them of their ability to live without round the clock care.
Siblings seven-year-old Ollie and five-year-old Amelia show a stark difference in the progression of their condition. Amelia began her treatment before her symptoms developed, while Ollie's treatment was delayed.
According to the National Institute of Neurological Disorders and Stroke, Batten disease develops in childhood, typically between the ages of five and ten. Early symptoms include personality and behavior changes, slow learning, clumsiness, or stumbling. Over time, affected children suffer cognitive impairment, worsening seizures, and progressive loss of sight and motor skills. Those with the condition have a life expectancy into the late teens and early twenties.
The three children travel to Great Ormond Street Hospital every fortnight to have an enzyme drug injected into their brain, but at a cost of £550,000 a year, in February the National Institute of Clinical Excellence (NICE) made an initial decision deeming deemed the drug too expensive for use on the NHS.
While a committee researching the drug admitted "it is an innovative treatment that is effective in the short-term...and given the significant burden this disease...anything that can help to improve the treatment of these children is to be welcomed. In the absence of long term evidence about its effectiveness in stabilising the disease and preventing death... the committee considered that the drug was not a good use of NHS resources."
Ollie and Amelia's parents dispute the ruling.
They plan to meet with NICE in Manchester on Wednesday, where they will argue that they can see the difference the treatments make, especially in light of the way in which preventative measures have slowed the progression of the disease in Amelia, when contrasted with Ollie.
They argue that the treatment is allowing their children to live a quality life: