A Limavady mum says she is “heartbroken” after being told her application for funding to secure a life-enhancing drug for her terminally ill little boy has been turned down.
Limavady boy Callum McCorriston has a form of muscular dystrophy known as Duchenne. His mum, Laura Smith says the condition has no cure and, such is the nature of her son’s condition, the prognosis is grim. It means that for Callum, who has been taking heart medication since March, his muscles will deteriorate until he is wheelchair-bound.
Laura said she has been told Callum is unlikely to live beyond his early twenties.
Callum, a pupil at Termoncanice PS in Limavady, is amongst a small group of Duchenne sufferers in the UK who could benefit from a drug called Translarna. She said five boys are currently on a trial for the treatment, but Callum doesn’t meet the criteria because of his age.
Laura has been campaigning for Callum to get access to the drug under ‘Team Callum’, and on Monday, with other parents and charities, met with Health and Social Care Board officials at Stormont hoping to find out more about the status of the application.
On Tuesday afternoon, however, Laura said she was left “angry and disgusted” when she was told the application for individual funding for Callum to access Translarna had been turned down.
My heart is broke and my hope is fading, but I’ll fight for Callum.Laura Smith
“Health officials have turned down a drug to help my baby stay ambulant, stay active and protect his muscles. They have denied him his basic human right, the right to live,” said Laura.
“I am totally disgusted with their decision. I will not accept this answer, and will do anything I can to get him what he needs. My heart is broke and my hope is fading, but I’ll fight for Callum.”
Laura, who said the treatment would cost £226,000 per year, has urged the decision makers to reverse their decision.
“I can’t believe it. My child is on heart medication at five years old. If that’s not exceptional, tell me what is? I feel sick to my stomach, but I will not take ‘no’ for an answer.”
Callum’s dad, Paul, wrote on social media via Facebook: “People see fit to value my son’s life based on his condition and, as the time ticks past, they are robbing him of his right to treatment that could extend his quality of life.”
A spokesperson for the Health and Social Care Board said: “It is not appropriate for the Health and Social Care (HSC) Board to comment on the circumstances of an individual patient case.
“In considering a request for specialist drug treatment on behalf of an individual patient, the HSC Board must take account of the evidence of the drug’s effectiveness and, in this regard, applies NICE guidance where it is available. The Board also takes account of guidance from other national bodies, for example the Scottish Medicines Consortium (SMC), particularly if NICE guidance has not been issued.
“For Translarna, advice in regard to its cost effectiveness has not yet been issued by NICE or the SMC. In this circumstance Translarna is not currently commissioned in Northern Ireland.
“The HSC Board does, however, have a clear process by which individual patient requests can be considered. If the patient’s clinical circumstances are identified as exceptional, funding for the requested treatment can be approved by the Board.
“As part of that process the patient’s hospital consultant puts in writing the exceptional clinical circumstances which apply to a particular patient. This information is expected to demonstrate why that patient would be more likely than others with the same condition to benefit from the proposed treatment. Such requests require to be supported by nominated senior clinicians and managers within the relevant HSC Trust.
“It should be emphasised that cost is not the determining factor in reaching a decision regarding the appropriateness of an individual request for a specialist drug. Rather, the priority is to consider the evidence to support the drug’s use and the extent to which an individual may benefit from it.”