Phenylketonuria: Treatment and Support

Faisal Rashid Excerpts

Tuesday 26th June 2018 Westminster Hall

[Faisal Rashid (Warrington South) (Lab)]

I commend the fantastic work of my hon. Friend the Member for Blaydon (Liz Twist) and the National Society for Phenylketonuria, who have worked incredibly hard to raise awareness of PKU in Parliament in the past few months.

Until I met with representatives from the NSPKU a few months ago, I knew very little about PKU, like many other hon. Members. Since, I have learned a huge amount about it, and it has become clear that this devastating disease requires action. It affects one in 10,000 people across the UK. Although it is rare, an estimated 3,000 individuals in England alone live with it and could benefit from increased awareness and improved treatment of it.

As we have heard, the only treatment of PKU available on the NHS is a severely restrictive diet. Patient voices from the NSPKU illustrate how much of a daily struggle that diet is. It often leads to eating disorders and unhealthy relationships with food. It forces patients into social exclusion and is a great burden on their carers, who often also report psychological distress and strained family relationships. Studies show that half of parents will stop work or reduce their hours to accommodate the extra work needed to take care of a child with PKU.

I am grateful to my constituents, who shared with me the story of life with their five-year-old daughter who has PKU. Their openness helped me to understand what that life is like, and the difficulties their daughter faces daily, especially when she is unable to eat the same meals as other children at school.

The fragmented nature of NHS services provided to PKU patients often leads to confusion and mistakes in the chain. The inconsistent quality of those services has made the system needlessly complex and has added to the patients’ burden. There have been multiple reports of clinical commissioning groups restricting funding for PKU dietary products. The variable quality of even the most basic treatment is simply not good enough.

Beyond dietary treatments, a drug called Kuvan has been developed, as has been mentioned. It can treat up to 25% of PKU patients and allow them to eat substantially more natural proteins, which fundamentally improves their quality of life. Despite having been licensed in the USA since 2007 and the EU since 2008, and being used in almost every EU country as a routine treatment for PKU, it is unavailable for the vast majority of patients in the UK.

Of course, Kuvan comes with a price tag, but as we approach the 70th birthday of our treasured NHS, we must allow it to uphold its fundamental principle of providing healthcare on the basis of need.