Phenylketonuria (Kuvan)

Kwasi Kwarteng Excerpts

Thursday 1st December 2011 Commons Chamber

[Kwasi Kwarteng (Spelthorne) (Con)]

I am grateful for the opportunity to raise the issue of access to Kuvan for sufferers of phenylketonuria. I believe that the most important part of our job in the House is to represent our constituents as conscientiously as possible. It is also our job to bring forward issues of national interest that, even though they might come to us initially as matters of individual complaint, relate more widely to national concerns. In raising this issue of access to Kuvan, I hope to fulfil both these important functions.

I want to raise this issue tonight for one simple reason: a constituent of mine, Mandy Macedo Box, who lives in Shepperton, has a six-year-old son who suffers from PKU. Mrs Macedo-Box’s son, Charlie, happens to have a particularly severe form of the illness. PKU is a relatively rare and unknown liver disease. Its sufferers have to restrict all protein-containing food in their diet in order to survive and to avoid damage to both the brain and the nervous system. About one in every 10,000 people has PKU. Sufferers can never eat food that has a high protein content, such as meat, dairy, bread and cakes, while foods containing lower amounts of protein, such as vegetables, can be eaten, but only in small amounts. Only fruit can be eaten safely.

To maintain this low-protein diet, sufferers have to work out a complex diet of “exchanges”, which often includes prescription products, such as imitation rice or protein-free bread. As well as this very restrictive diet, sufferers often have to take a number of supplements to help their bodies function normally and to reduce their appetite. I am sure that Members can imagine how restrictive and detrimental to the quality of life this can be, especially for children and their families.

There is now a drug on the market that has been developed to help with the treatment of mild and moderate cases of PKU. This drug, Kuvan, enables sufferers to double the amount of protein that they can tolerate, which obviously means an increase in the amount of real food that can be ingested. I am sure that you can imagine, Mr Deputy Speaker, the huge improvement in the quality of life that such a drug would bring to many sufferers of PKU. Unfortunately, however, as is often the nature of these things, the drug is not guaranteed to work in Charlie’s case. It has therefore been proposed that Charlie take the drug for a trial period of one month so that doctors can determine whether it can help his condition.

The drug company that manufactures Kuvan has offered to make the drug available to Charlie’s local health authority, NHS Surrey, on this trial basis on the understanding that should the trial be successful, NHS Surrey would agree to fund the drug for patients with PKU in Surrey in the future. I understand that similar arrangements have been offered to other primary care trusts. However, the crux of the problem is that in Surrey the local area prescribing committee is simply unwilling to make this undertaking because—it claims—there is

“limited evidence of ongoing clinical effectiveness, and lack of cost-effectiveness”

of the drug.

To put it simply, we are now in the absurd position where the drug company will not commit to a trial period unless the PCT can guarantee future funding should the trial be successful, while, on the other hand, the PCT will not commit to a trial period because it cannot guarantee future funding because it is not sure that the drug will work. While this stand-off continues, the quality of life for a young boy in my constituency and many other sufferers of PKU continues to be seriously impaired simply because they have no access to a drug that might help to alleviate the symptoms of the disease.

To make matters even more frustrating for Charlie and others like him who are desperate for some non-dietary treatment, we know that Kuvan, although not available to many PKU sufferers in the UK, is routinely made available to patients in European countries such as France, Germany, Italy and Spain. It simply does not seem right that, despite the many miracles that we all know the NHS performs every day, British patients should be disadvantaged in that way, compared with their peers on the continent. It is in that context that I feel that a solution to the impasse must be found. Why can British sufferers of this disease not access the drug on the national health service? An answer might lie in the fact that the National Institute for Health and Clinical Excellence has not yet positively approved the drug. If that were to happen, some progress may well be made.

Finally, I find it quite striking that in an area where we are supposed to be supporting a great British business—pharmaceuticals—we should not be able to provide funding for the drug, thereby restricting the opportunities of many thousands of people in this country.

I am grateful to the House and to you, Mr Deputy Speaker, for the patience that you have shown in this debate—and, in fact, for your consideration in granting it. This may seem a small issue, but it has a massive bearing on the quality of life of those affected—both the sufferers and their families. It is quite right that it should be aired in the highest possible arena, which is what this House represents. I urge the Government to do all they can to find a solution to this grave dilemma.