Joy and anger at wonder drug on the NHS... as it is only for children up to 18
FAMILIES reacted with joy and anger after NHS chiefs finally allowed a life-changing drug to be administered – but only to children.
Kuvan has been used to treat the rare genetic condition phenylketonuria (PKU) in Europe for 12 years. But it is not used in the UK.
Sufferers cannot process the amino acid phenylalanine from protein, and it builds up to cause problems in the nervous system.
For a year the Daily Express has urged officials at the National Institute for Health and Care Excellence (Nice) to let NHS staff use Kuvan.
They have relented – but only for under-18s, for “cost-effective” reasons.
Hurley Swain was born with PKU last year and his dad Jak called the approval “amazing”, adding that he hoped Kuvan will be extended to adults. But furious campaigner Kate Learoyd said: “Nice’s decision is unrealistic and unfair, and we will be challenging it.
“People with PKU are not cured of their condition on their 18th birthday – this condition stays with you.
“Taking a teenager’s medicine away from them on their 18th birthday is simply cruel.”
Caroline Graham, 55, has two grown-up children with PKU who are desperate for Kuvan. She accused Nice of “setting children up to fail” by giving them Kuvan now, then taking it off them at 18.
“Little children with PKU grow into adults with PKU. A lifelong diagnosis should have a lifelong treatment. One of the most difficult times to manage PKU is when you turn 18, often leave home and have to survive without close family support,” she added.
Her son Callum, 26, said: “I can’t believe anyone thinks giving a child a drug and then removing it on their 18th birthday is moral. PKU for me has been a life sentence.”
While Caroline’s tearful daughter Holly, 24, added: “I work extremely hard to keep on top of my PKU but fail often. It’s not just my present affected by this decision but my whole future.That scares me.”
There are around 2,000 people with PKU under NHS care in England alone but Kuvan is esti
mated to be suitable for only around 600. Kuvan – US maker BioMarin’s brand name for the drug sapropterin – costs around £45,000 a year per adult patient but as little as £4,000 for a toddler.
BioMarin’s patents have now expired, so rival firms can offer the UK cheaper versions – but these
have yet to be approved. Officials at Nice told the Express yesterday they acknowledged sapropterin “could prevent long-term irreversible brain damage in children”.
But they claimed it was not “an effective use of NHS resources” for over-18s as dosage is based on weight, so adults need more.
However a Nice consultation document concedes that “brain development does not stop until around age 25”.
The decision is open for public consultation for a month before it is rubber-stamped in April. Children might start having Kuvan after June.
Meindert Boysen, Nice deputy
chief executive, said: “The benefit for adults is not one that we think worth paying for.
“There is a discussion to be had with the clinician and the patient about what the best timing is, but that’s our guidance.”
NHS England was approached for a comment.
ACTOR Ronald Pickup, best known for his roles in the Best Exotic Marigold Hotel films and The Crown, has died at the age of 80.
His agent Jean Diamond said: “Ronald passed away peacefully yesterday after a long illness surrounded by his wife and family. He will be deeply missed.”
Pickup worked in theatre, film and television.
His first major part came in 1964 when he starred in DoctorWho episode The Tyrant Of France as a physician.
In 2012 he told an interviewer his favourite role had been playing George Orwell in Crystal Spirit: Orwell On Jura, which told the story of the writing of the author’s classic novel 1984 on the remote Scottish island.
The actor continued to star in big productions in later life.
He appeared alongside Dames Judi Dench and Maggie Smith in the Marigold Hotel films in 2011 and 2015.
In 2016 he played the Archbishop of Canterbury in the first Netflix series of The Crown and was Neville Chamberlain in 2017’s Darkest Hour. Last year he starred in horror film End Of Term.