Eight-year-old Callum McCorriston’s quality of life has improved greatly since he started taking a ground-breaking drug to treat his muscular dystrophy, according to his mother, Laura Smith.
“Callum is lucky and has been doing very well on Translarna. He is strong and able to walk and he has learned to ride a bike. He is also more mentally alert and able to remember words,” she says.
Translarna slows down the progress of Duchenne muscular dystrophy, a rare muscle-wasting disease that sees sufferers consigned to a wheelchair in mid-childhood as they lose the use of their legs.
By delaying the onset of the disease, the drug aims to give children more years of mobility with a longer-term objective of increasing life expectancy from the current level of just 25-30 years.
In the Republic, the HSE was unconvinced of the effectiveness of the treatment and unhappy at the cost, likely to be about €200,000 a year after price negotiations with the drug company.
The drug treats only a minority of forms of Duchenne, so only two young boys in the Republic would benefit from treatment. The overall cost was low, therefore, in the context of the HSE’s €14 billion budget, but reimbursement under the State drug schemes was rejected this summer.
In the North, however, Translarna got the go-ahead last year and Callum from Limavady, Co Derry, was one of the first patients to benefit. Ms Smith, who campaigned for two years for access to the treatment for her son, says waiting for so long was “an awful feeling”.
“I don’t know what is worse: being told that Callum has Duchenne and there is no cure; or being told there is a drug that could prolong his life but you cannot get access to it.”
“You are told of the condition and that there is no cure. Then you have this new treatment dangled in front of you and you fight to get it for your son.”
“The challenge is harder, when you see other boys throughout Europe getting access to the drug as you and your son wait, hope and pray you can get it in time.”
With new, highly-expensive treatments coming on stream, the HSE is under enormous pressure to meet patient expectations and yet control costs. The politically-influenced decision to fund a raft of new cystic fibrosis drugs earlier this year at a 10-year cost of more than €600 million has left little in the kitty for other medicines. And while the exposure arising from using Translarna to treat muscular dystrophy is limited, the drug can be used too for treating CF – at far greater potential cost.
The fact that families in a similar position south of the Border cannot access the treatment angers Ms Smith: “It really makes me mad, these political decision-makers playing God with children’s lives”.
She acknowledges Translarna is expensive