Phenylketonuria: Treatment and Support

Paul Masterton Excerpts

Tuesday 26th June 2018 Westminster Hall

[Paul Masterton (East Renfrewshire) (Con)]

Like other hon. Members, I wanted to speak in the debate because PKU affects a family in my constituency. I will not go over what PKU or Kuvan are. The hon. Member for Blaydon (Liz Twist) did an excellent job of that, and I congratulate her on her work as chair of the APPG in bringing this issue to wider attention across the House.

Scotland has a higher prevalence of PKU than other parts of the UK, which makes the attitude of the Scottish Medicines Consortium so disappointing. It has a poor record of approving treatments for very rare diseases, and I understand from conversations with the NSPKU that the current application for PKU is not going particularly well. The decision is due next week on 3 July, and there is real concern that it will not be approved, even for high-risk groups such as pregnant women. The hon. Member for Blaydon mentioned that there is currently a pretty poor Kuvan policy for pregnant women in England. It may be poor, but at least it is a policy; we do not even have that north of the border.

Pregnancies for women with PKU are extremely high risk. We have a slightly odd fail-first approach in this country, which can lead to serious defects and lifelong disabilities for children if they survive the pregnancy. The cost to the NHS of treating those disabilities over the life of the child is significantly more than the cost of Kuvan for the mother during pregnancy. We have an odd approach to cost-effectiveness when looking at medicines in this country that I think we need to reassess. That is not only true for Kuvan; we have seen it when talking about Orkambi and for various other issues.

I completely agree with the decision of the NSPKU to apply for Kuvan to be placed in the ultra-orphan stream due to the rareness of the condition and the Scottish gene variant. I was disappointed to hear of the Scottish Government’s lack of engagement with the NSPKU, so I ask Scottish National party Members present to do something about that. While I appreciate that there is a distinction between the Scottish Government and the SMC, the Scottish Health Secretary could step up a little bit there.

As a Scot from Greater Glasgow, I was of course drinking Irn-Bru when I met the NSPKU in Portcullis House. That led to quite an interesting discussion, because Irn-Bru could previously be given to young adults with PKU as a kind of treat and something to make them feel normal, but then we introduced the sugar tax. The recipe for Irn-Bru changed overnight and they could no longer drink it. It suddenly became toxic. This is one situation in which a very well-meaning policy, such as the sugar tax, had unintended consequences.

Members may have been in touch with their diabetic constituents after the recipe for Lucozade changed and suddenly they could not drink something that had been safe for them. Unless someone has very good eyesight, it is hard to read on the can that the recipe has changed. We also have the odd situation in Scotland where, depending on the shop, someone can buy old recipe Irn-Bru or new recipe Irn-Bru. They have to check very carefully. I do not think that we do a good job of looking at the potential unintended consequences of changes in health policy for special interest groups.

I was struck in our discussions when it was put to me that, in 12 years’ time, my now four-year-old daughter will be going out and I will worry about her drinking alcohol or smoking, so imagine being the parent of a child with PKU and worrying about them going to a friend’s house and eating a bag of crisps. The difference in terms of the strain and pressure is huge. For the family in my constituency, the parents of 10-year-old Katie and 20-month-old Harry said to me:

“Having PKU is a constant shadow that hangs over the family. Everywhere you go you are surrounded by food they…can’t eat...Katie’s 10 and wishes she could eat what her friends can, or even a little bit of it, but is aware”—

even at age 10—

“of the implications of brain damage if she doesn’t stick to her diet... Kuvan might not work for her, but if it did it would be life changing…It’s really sad knowing that our one year old has all this ahead of him.”

We can and should do more to get Kuvan out there.