UK Rare Diseases Framework

Part of the debate – in Westminster Hall at 5:20 pm on 24th March 2021.

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Photo of Peter Gibson Peter Gibson Conservative, Darlington 5:20 pm, 24th March 2021

It is a pleasure to serve under your chairmanship today, Mrs Miller. It was also a pleasure to listen to Liz Twist, who opened this evening’s debate. She has been a tireless campaigner for those who suffer from phenylketonuria and I congratulate her on securing this important debate.

One in 10,000 babies across the United Kingdom is born with PKU. That rare inherited disease means that individuals cannot break down phenylalanine. If the condition is untreated, it can lead to damage of the brain and nervous system. Sadly, a number of my constituents suffer from the disorder, including, as I learned today, a three-month-old infant. I thank Samantha Parker, Kelly Thompson and Amy Duston, who suffer with PKU or have a child who does, for their engagement with me.

It was a bittersweet moment last month when NICE released its draft recommendation that, following discussion with the manufacturer of Kuvan, it could be used for children up to the age of 18. It was bittersweet because it means that as those children turn 18 they will face a cliff edge in their treatment. I fear that NICE has not fully considered the problem of an 18-year-old abruptly stopping treatment with Kuvan. The decision will lead to further difficulties down the line, and it does not address the treatment of adults with PKU.

I am hopeful on three counts for my constituents. I am optimistic that children will soon be able to access that life-changing drug, that NICE will reconsider the use of Kuvan for adults, and that as the exclusivity of Kuvan to BioMarin comes to an end it will become cheaper and more cost-effective as a treatment for all who suffer from PKU.