The father of an Armagh schoolboy who suffers from a rare medical condition which restricts his diet to 13g of protein a day has welcomed news that provision in Northern Ireland for a drug to help treat the illness is to be brought into line with the rest of the UK.
Twelve-year-old Daragh Cassidy suffers from a condition known as Phenylketonuria, or PKU, which means he lacks the ability to properly break down the protein in food.
It affects on average 1-in-10,000 people in the UK; in Daragh’s case it means he can only have the equivalent of two or three slices of white bread a day.
His dad Christopher has been battling for a change in legislation around the drug ‘Kuvan’, which has been hailed as potentially ‘life-changing’ for those with PKU.
It is estimated about 20-30% of patients in the UK will be responsive to Kuvan treatment, with the effect being a significant increase in the amount of protein that person can consume in their day-to-day life.
In Great Britain, the National Society for Phenylketonuria (NSPKU), were able to lobby with the National Institute for Health and Care Excellence (NICE) to help bring a more cost-effective version of the drug Kuvan into play. It was then introduced to all patients that were successful on the trial.
In December 2021, it was announced that the NHS would make the drug available to patients of all ages for the first time. A deal had been struck by the NHS to secure the non-branded ‘generic’ version of the drug. This more cost-effective version helped eradicate the previous limit of 22.
While the treatment is open for persons of any age in England, Scotland and Wales, the medication is currently only available in Northern Ireland for those under the age of 22, meaning those on the drug here would be pushed back onto low-protein diets when they reach that age.
However, much to the relief of Christopher, and indeed many families battling PKU, that legislation is set to change in the near future, in spite of previous cost concerns.
The change in policy position was outlined in a letter sent to a number of political parties on January 16, in which Peter May, the Permanent Secretary at the Department of Health, said the Department endorsed the provision of treatment for patients of all age.
He said this was in line with, “the overall policy aim of ensuring that the people of Northern Ireland can benefit from timely access to effective and cost-effective medicines on an equitable basis to elsewhere in the UK”.
He added that there would be, “specific and immediate provision in conjunction with the service to ensure that no patient established on treatment and approaching their 22nd birthday ceases treatment in any interim, intervening period while longer term arrangements are developed and implemented”.
Speaking to Armagh I after the announcement, Christopher Cassidy said he was “overwhelmed” by the news, which means that the campaigning by himself and others in the PKU community in Northern Ireland has finally paid off.
“It’s just been a continuous bombardment from people in the community contacting their local MLAs,” he said. “I’m hoping that he had no option to do nothing but change it.”
He continued: “I’m very, very pleased with the outcome. I speak for the rest of the people I know in the PKU community that they’re overjoyed, even those that won’t benefit from it because they’ve been told it just will not work for them.
“If it works, it’ll offer them that reassurance that once they turn 22 they’ll not lose this opportunity and it means they can continue their better standard of life that they’ve been given through the use of this drug.
“It also now offers the opportunity for those that are already over the age of 22 to the drug as well, which offers them a bit of light at the end of the tunnel.”
While Daragh is yet to start trials on the drug – it’s hoped he will start in the coming weeks – and although there’s no guarantee it will work, the drug still offers a fighting chance to help him work towards keeping an ordinary diet.
Said Christopher: “It may not work for him, it may work for him, but either way it’s the bigger picture that there’s people in the community that now have the same opportunity as people in the rest of the UK and aren’t being discriminated against just because of their age.”
He added: “I know of one person in the community who has went from 10g of protein a day before starting the trial and is now on 55g of protein a day, which pretty much could be classified nearly as a normal diet.
“Without this legislation being changed, at the age of 22 the person in question would have then had to either revert back to a low-protein diet of 10g a day or stay on a high-protein diet and know the consequences of potential health risks to them.”
While the change is undoubtedly welcome, Christopher says he can’t help but feel it could have came a lot sooner.
“I believe that had we had a functioning government, this wouldn’t have been the case and it wouldn’t have took so long to have been the case. In fact, it could have been done at the same time as the rest of the UK.
“But the voice of the people has won in this situation and as they always say – power to the people.”
