For 12 years US firm BioMarin’s drug Kuvan could have eased the agony of those with genetic condition PKU – but has not been available here. NHS drugs appraiser Nice is now proposing to let children have Kuvan, but to save cash they must stop taking it on their 18th birthday. Earlier this month furious sufferer Lily Wood, 19, wrote to Health Secretary Matt Hancock pleading for adults to be allowed Kuvan too.
Now health minister Lord Bethell has replied with the olive branch of hope saying the NICE proposal could be “subject to change” after a month-long public consultation.
Currently Nice are reviewing evidence from over 240 angry sufferers who have explained why PKU sufferers of all ages must be offered the drug.
Lord Bethell added: “Please be assured NICE only publishes final guidance on the use of a drug after a very careful consideration of the evidence.”
He added that Nice “will take the comments received in response to the consultation fully into account in finalising its recommendations.”
Afterwards Lily, of West Drayton, Middlesex, told us: “Lord Bethell’s response gives me hope that Nice may change their decision.
“People who live with this disease know that what they provisionally suggested, limiting Kuvan just to kids, is not right or ethical.
“It is vital Kuvan is accessible to all PKU sufferers – as the improvement to quality of life would be immense.
“If all this evidence is finally listened to, I have hope that the 12-year fight for Kuvan will come to a very happy end.”
A further meeting of Nice will consider the 240 responses on April 7 and they expect to publish their final Kuvan guidance in June 2021.
We exclusively told how Lily penned her emotive letter to Matt Hancock after being shattered by Nice’s preliminary Kuvan decision.
She told how without Kuvan she currently lives on a highly restrictive low protein diet without meat, fish, eggs, dairy, pulses, and even some fruit and vegetables.
She wrote: “And are you aware of any drug other than Kuvan that is taken away from the patient when they reach 18? I certainly can’t.
“All of my life I have been on a highly restrictive low protein diet where I cannot eat meat, fish, eggs, dairy, pulses, and even some fruit and vegetables are restricted.
“If PKU sufferers don’t follow this diet the side effects are immense. Depression, confusion, memory loss, anxiety, motor skills and hospitalisation.
“Patients with type 2 Diabetes could be treated with a Mediterranean diet, yet Nice doesn’t take their insulin away.
“How is a parent supposed to tell their 18 year-old they now have to live a life where they will never feel full again.”
There are around 2,000 people with PKU in NHS care in England alone.
On Wednesday this week, campaigning MPs spoke out in a Westminster Hall rare diseases debate, demanding all PKU sufferers get Kuvan.
Labour MPs Liz Twist, Marie Rimmer, Catherine McKinnell, Christina Rees and Tory MPs Tom Randall, Peter Gibson and Chris Skidmore all raged at Nice’s initial decision to allow it only to children.
Chris Skidmore said: “This is a lifelong condition – we need lifelong treatment of Kuvan on the NHS.”
While Tom Randall said the idea that Kuvan will not be available for over-18s caused “understandable concerns”.
Their powerful arguments prompted Health minister Nadine Dorries to agree that withdrawing Kuvan on a child’s 18th birthday would be “problematic”.
She said: “It is important to stress that this is not the final guidance.”
NHS England (NHSE) say by law they must “implement” whatever Nice’s final decision is.
Yet NHSE have held four reviews into making Kuvan available – in 2015, 2018, 2019 and 2020 – and each time themselves decided not to make it available.