Terminally ill Limavady boy denied access to medicine

Laura Smith’s son, Callum, suffers from a condition known as Duchenne Muscular Dystrophy which causes his muscles to slowly deteriorate and drastically reduces his lifespan. Life expectancy for people diagnosed with Duchenne in Northern Ireland is 19 years.

Laura has been campaigning for a new drug, known as Translarna, to be made available to her son. The drug has been shown to lessen the impact of Duchenne for those whose condition is caused by a specific genetic mutation and could dramatically increase the lifespan of those it can treat.

She has applied for the medicine to be made available through a mechanism for specialist drugs to be made available known as an ‘Individual Funding Request’, only to be told no.

She said: “They said no. Just a flat no. They said we hadn’t proved exceptionality so he wouldn’t be getting it.”

However, the Health Minister Simon Hamilton recently announced a raft of changes to the process of applying for specialist drugs, acknowledging that the current process is flawed.

Laura said: “The Health Minister had announced he was implementing those changes but there hasn’t been any indication as to when that is coming into effect. I’ve spoken to Claire Sugden, who has written to the Health Minister but there have been no responses as yet. We are no further forward in terms of those changes. I have no idea when all that is going to start. It is still a waiting game to find out when they will be using the new rules.

“When I spoke to the Health and Social Care Board representatives they said that this one would be the final application.

However, I don’t know if there is actually a limit on the number of applications you can make. By all means, once the new rules come into place I will be applying again. Whether or not they accept it, I don’t know, but I have to try.

“NICE (the body who decide whether drugs can be made routinely available on the NHS) were originally to give their decision in January but the company who make the drug, PTC Therapeutics, they provided NICE with more information so they’ve postponed the decision.”

Laura added: “Hopefully, fingers crossed, they won’t drag it out any longer. The drug can only be used while he is still on his feet.”