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Northern Ireland ‘way behind’ in vital PKU drug legislation, says Armagh campaigner

"The wonderful staff in the Royal Hospital in the Metabolic team up in Belfast are being put into a ring with one hand tied behind their backs because they're having to deal with the archaic legislation and rules around this drug."

Christopher and his son Daragh, who suffers from PKU

The father of an Armagh schoolboy who suffers from a rare medical condition which restricts his diet to 13g of protein a day has said Northern Ireland is lagging behind the rest of the UK in provision of a “life-changing” drug to help treat the illness.

Eleven-year-old Daragh Cassidy suffers from a condition known as Phenylketonuria, or PKU, a metabolic condition which affects on average 1-in-10,000 people in the UK.

It means that the person lacks the ability to break down the protein in food properly.

In Daragh’s case, he can only have the equivalent of two or three slices of white bread a day.

His dad Christopher is an advocate for a change in legislation to better help those with the condition.

Christopher explains that the legislation around the drug Kuvan in Northern Ireland is “archaic”, with the “life-changing” medication only available for those under the age of 22.

In England, Scotland and Wales, however, the treatment is open for persons of any age.

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.

Related: Armagh schoolboy’s rare medical condition that restricts diet to 13g of protein a day

This difference in legislation has a number of implications for those with PKU in Northern Ireland.

The most profound is that when those taking the medication in Northern Ireland reach the age of 22, they will be pushed back onto their low-protein diet.

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 raise the previous limit of 22, which is still the limit in Northern Ireland.

Christopher says that even the trial process takes much longer here than in the rest of the UK.

However, for those who do respond to the drug, the results are revolutionary.

Children at the NSPKU day conference held in Armagh on June 10.

Christopher explained: “I know of one person that’s been on the drug now for a short period of time and she herself has gone from having the maximum amount of protein each day allowed to be 12 grammes to now having 60 grammes of protein a day. 

“The young girl in particular is only 20. In another two years, if things don’t change, she will then have to come back off that and will have to go back down to her original limit of 12 grammes of protein a day.”

He added: “The wonderful staff in the Royal Hospital in the Metabolic team up in Belfast are being put into a ring with one hand tied behind their backs because they’re having to deal with the archaic legislation and rules around this drug.”

Christopher says that the legislation is lagging “vastly behind” in Northern Ireland in comparison with the rest of the United Kingdom, citing the situation on the hill as the reason why the legislation has not changed.

“Our big stumbling block is that we don’t have that working Executive to help and support us over here,” he said.

He explains there is a push to circumvent the Northern Ireland Executive and seek direct advice with the Commissioner for Northern Ireland to see if they can expedite the process. However, Christopher concedes it’s going to be a “slow process”.

“The more direct and more effective way would be to just have the Northern Ireland Executive back up and running,” he added.

Christopher describes the current system in Northern Ireland as “very dire”.

He said: “How can we as one of the G7 countries be sitting here, with part of that country not being able to use life-changing drugs, whenever you have members of less advanced countries in Europe having this drug available for many a year?

“December 2021 was the legislation change for the rest of the UK, so it’s now coming up to two years later and it’s still not changed in Northern Ireland. We’re lagging behind big time. You just can’t take the rug from underneath somebody at the age of 22.”

Kate Learoyd, the Campaign Manager for NSPKU, commented: “It is really frustrating that patients in Northern Ireland do not have equal access to Sapropterin (Kuvan).  PKU is more prevalent in people of Irish ancestry and it is a really challenging condition to manage. Sapropterin is a life-changing drug for patients.”

She added: “The drug is now available in a cheaper non-branded form which meant the NHS in England, Scotland and Wales has made it available for patients of all ages.

“As it’s now cost effective, there is no logical reason why the same sensible decision can’t be made in Northern Ireland. It seems really unfair that people with PKU are being left waiting.”

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