UK Rare Diseases Framework

Part of the debate – in Westminster Hall at 5:18 pm on 24th March 2021.

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Photo of Marie Rimmer Marie Rimmer Opposition Whip (Commons) 5:18 pm, 24th March 2021

It is a pleasure to serve under your chairmanship, Mrs Miller. I pay tribute to my hon. Friend Liz Twist.

PKU is a disease that leaves people unable to break down protein. It can lead to severe brain damage. Kuvan is a life-changing drug that can help people cope with PKU. NICE’s decision to offer Kuvan only to patients up to the age of 18 is wrong. There is no miraculous cure for PKU when patients turn 18.

The transition to adulthood is a tough time already; 18-year-olds are moving away from school and often away from parental support, whether attending university, beginning an apprenticeship or starting a career. It is a difficult time. NICE’s decision strips young people of a life-changing drug when they are at their most vulnerable. Giving patients Kuvan and then taking it away turns an 18th birthday into a day of dread. Never mind the joy of a Greggs sausage roll, PKU patients cannot even grab a healthy salad or a vegetarian sandwich. The disease requires an exacting regulation of food intake.

I met Liam, a 20-year-old constituent who has PKU. The first thing that struck me was Liam’s mother bringing him bags of special ingredients. Careful planning is essential. Everything is homemade and all the ingredients have to be measured out, for Liam’s safety. Preparing the food is a full-time job. Liam has never had Kuvan and is in his second year at university studying policing. He is planned and worked hard to contribute to society, but he fears that without Kuvan this would not happen. There are hundreds like Liam who want to make a positive contribution. I asked him for his thoughts on the decision. He said:

“The overwhelming feeling right now is one of betrayal. We have spent 12 years fighting for this drug, seeing it within our sights, our hopes finally rising at the prospect of receiving such a life changing drug, only to have it snatched from us.”

I ask BioMarin and the Government to put people like Liam at the forefront of their decision making. People are being denied the quality of life that is possible and that they deserve.