Phenylketonuria: Treatment and Support

Debate between Nigel Mills and Liz Twist

Tuesday 26th June 2018 Westminster Hall

[Liz Twist]

I certainly agree with my right hon. Friend. It has become clear, as we have looked into the issue, that there is a very complex way of assessing drugs. Clearly, we want to get the process right, but there needs to be a rigorous look at that process, not just for Kuvan, but for other treatments for rare diseases. PKU perhaps falls between the cracks, because it is not quite rare enough to be in that group, but it is still very rare, and will be pushed to the back of the queue if it goes into a more general group.

For those whom Kuvan will not help, and who still need to manage their diet carefully, there is another issue that must be addressed: access to low-protein foods, which help to maintain the diet. Individuals may be advised through their dietician and their specialist centre that they need a particular level of foodstuff supplies, such as the low-protein flour I mentioned, which I am told can be used in many ways to try to make the diet more palatable. General practitioners, however, may not have a complete understanding of the condition or the dietary needs, and may feel that patients are just trying to get food on the cheap, and they may limit or deny prescriptions for those foodstuffs. They may feel that they are like gluten-free foods, which can be bought at supermarkets.

The fact is that those foods and supplements cannot be bought; they are available only on prescription, and the absence of them creates a real injury to those affected. It is not just that they are not there; it is actually damaging if they are not available. It would be good if the Minister could address how we can ensure that GPs prescribe the specialist foodstuffs that form part of the treatment that those with PKU need, and how we can close the gap between the specialist services, clinical commissioning groups and GPs.

[Nigel Mills (Amber Valley) (Con)]

I add my congratulations to the hon. Lady. Does she agree that there should be clear guidance, so that GPs or CCGs that are thinking about stopping prescribing that stuff can be told quickly and clearly that that is the wrong thing to do, and that there is no other way of getting this bread, and so that if one of them is foolish enough to go down that line, there can be a quick resolution?

[Liz Twist]

I thank the hon. Gentleman for that comment, and I agree. Last week at the APPG meeting, we heard some terrible stories; over the years, people have felt as if they were asking for a favour in asking for those goods. They are not; the goods are absolutely essential, and they cannot be bought over the counter. We must do something about that. We need to square that circle.

Finally, for the 75% who will not benefit from Kuvan, it is important that new, innovative treatments are developed and assessed quickly, so that more people can benefit from treatments that enable them to live well and safely with the condition.

To conclude, it is important that we listen to individuals and families who are living with PKU day in and day out. It is time that this condition was acknowledged, and that we addressed the need for effective treatment. I hope that the Minister can give us positive news that will move us forward in helping those with PKU.

This Thursday, on National PKU Awareness Day, I and many other hon. Members in this room and across the House will undertake the PKU “diet for a day” challenge. We will restrict our protein intake to 10 grams a day, avoid all those things we normally eat without thinking, such as that piece of toast in the morning or Rice Krispies with real milk rather than coconut milk, and drink tea or coffee without milk. We know that we will not really face those restrictions day in, day out, or the relentless grind of getting the diet right to stay well, but we hope that it will help raise public awareness of PKU, and help to bring about change.