Phenylketonuria (Kuvan)
(13 years ago)
Commons ChamberRead Hansard Text Read Debate Ministerial Extracts
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.
The Minister of State, Department of Health (Paul Burstow)
I congratulate my hon. Friend the Member for Spelthorne (Kwasi Kwarteng) on securing this debate and on setting out so clearly, on behalf of his constituents, the concerns that they have brought to him and the concerns that he, in turn, wanted to ensure the House properly addressed this evening. He is absolutely right: that is exactly what Adjournment debates are for. They are an opportunity for constituents’ concerns to be raised in this House, and I pay tribute to him for doing so.
My hon. Friend talked about the case of Charlie and the situation facing him and his family. He will understand that the necessity for patient confidentiality meant that until I heard his speech, I was not aware of that specific case from the briefing that I had to prepare for this debate. However, I hope that I can give him a response that will none the less address a number of the important points that he raised. As part of the preparation in advance of this debate, my private office supplied me with a copy of the postings on the Phedup website that talk about Charlie’s case. One can feel only strong sympathy for the concerns that my hon. Friend has raised on behalf of his constituents. As a 12-year-old boy, Charlie wants to be like every other young person growing up. He does not want to be different from his cousins. That point comes across clearly from the website.
I cannot deal with all the details, but I understand how important it is for children with phenylketonuria to be treated early and to receive the most effective treatment from the NHS. As my hon. Friend rightly said, the condition affects around one in 10,000 babies born in England. Those with phenylketonuria—or PKU, as it is more commonly known—are unable to break down the amino acid phenylalanine, which builds up in the blood and, critically, the brain. In most people with the deficiency, food is not broken down by the enzyme known as phenylalanine hydroxylase, or PAH—I will stick with the initials from now on, if I may. In people with PKU, the PAH enzyme function is impaired because of a genetic mutation. As a result, phenylalanine levels in the blood and other tissues rise, which can lead to brain damage and, in some cases, learning difficulties.
Without treatment early in life, the outlook for those with PKU is very poor. Most people will develop severe learning disabilities and will require constant care. With treatment, however, the outlook can be incredibly good. Since 1969, the NHS has screened newborn babies for PKU, so early detection is commonplace in this country. Following a low-protein diet and taking regular dietary supplements containing amino acids helps keep the phenylalanine levels low, avoiding the terrible damage that the condition can inflict on a person. Specially formulated low-protein foods and nutritional supplements are available on the NHS, and GPs are able to prescribe them to treat patients with PKU. As with any condition that requires constant management and attention from—
Paul Burstow
Thank you, Mr Deputy Speaker. I was not prepared for that procedural intervention.
As I was saying, any condition that requires constant management and attention from birth can be an incredibly stressful one, as my hon. Friend captured in his remarks. I appreciate how difficult it must be for children and their parents or carers to maintain the protein-free diet necessary for the majority of patients with PKU. From what I understand about the condition, children with PKU cannot eat many of the foods that we all take for granted. Some of the pleasures of life are not available to them, which makes simple day-to-day activities like going to school difficult. Again, I very much understand the desire to see a child living a life that is not different from that of their peers. That point was powerfully made, as I said, on the Phedup website when I looked at it today.
The Government recognise the importance of maintaining this diet. That is why a range of food and nutritional supplements are provided by prescription on the NHS. The committee responsible for approving these products definitely understands the importance of providing a range of products for patients and takes special care to make sure that these products are approved for NHS use.
This debate is primarily about the role of a new drug, Kuvan, and how it can change individuals’ lives if it is made available because of how it can deal with the problem of phenylalanine levels in children and adults with PKU. Kuvan is a synthetic form of a vitamin that helps PAH enzymes work better. However—here we come to what I fear is the important point—the drug is not believed to be universally effective. It works only in some cases for some types of PKU, depending on which precise PAH gene mutation is present. As I understand it, only about four in 10 patients are likely to benefit from it. As my hon. Friend said, the benefit is determined only after a month, and through a blood test. Kuvan is more likely to benefit those with the milder forms of PKU, and most people would still need to continue with their dietary treatment. The drug might enable them to eat a few grams of protein, but would not allow a complete reversion to a conventional lifestyle and diet.
Let me deal with the role of the primary care trust in all of this. PCTs are legally obliged to provide funding so that drugs positively appraised by NICE are available on the NHS. This includes drugs with European orphan designation. In the absence of NICE guidance—for instance, where NICE has not appraised a treatment or is in the process of doing so, or where NICE has not recommended a treatment for use on the NHS—the PCTs are responsible for making funding decisions based on the needs of their populations.
I understand that the Surrey NHS area prescribing committee has considered Kuvan and has taken a decision on the basis of the evidence not to make it available in Surrey. The panel, which included GPs and other clinicians, considered the available clinical evidence, together with supporting information from parents of children diagnosed with PKU. After full and careful consideration—my hon. Friend has quoted this—it concluded that there was insufficient evidence to support the use of Kuvan at this time.
Doctors can request treatments that are not usually funded on behalf of their patients through an individual funding request, if they feel that there are exceptional clinical circumstances. I understand that a further request has been made by the Great Ormond Street Hospital for Children NHS Foundation Trust. I believe it has led to further correspondence between the PCT and the trust. At the moment, the foundation trust has not supplied all the information necessary for the application to be properly considered. I hope that that information will be provided and that consideration will be given to those representations by the foundation trust. I shall also ensure that the report of today’s debate is made available to the primary care trust, so that its members can see for themselves the representations made by my hon. Friend.
Under the NHS constitution, patients have the right to expect local decisions about the funding of medicines and treatments to be made rationally, following proper consideration of the evidence. To help PCTs make those difficult decisions, the Department has issued a set of core principles. PCTs are required to have clear and transparent arrangements for local decision making on the funding of drugs, and for consideration of exceptional funding requests.
My hon. Friend asked how we could address these difficult issues in the future. We want to improve access, on the NHS, to innovative medicines that are effective, particularly those that can allow a fellow citizen to lead as normal a life as possible. We will do that by reforming the way in which companies are paid for NHS medicines, adopting to a new value-based pricing system when the current pharmaceutical price regulation scheme expires at the end of 2013. That will bring the price that the NHS pays more into line with the value that a new medicine delivers in terms of the benefits that doctors and patients gain from a drug. The aim of the new pricing system is to create a system that has the capability to include the broadest possible range of new medicines. When possible, we want to avoid creating different processes for different treatments.
I understand that some people with very rare conditions—including, I suspect, the one that we are discussing—who want to gain access to a particular treatment fear that their cases will not be considered fairly under the new system because they are among a tiny minority who would benefit in such circumstances. We will keep an open mind, and if, as we continue to develop our plans to implement value-based pricing, it becomes clear that some treatments for the very rarest conditions would be best dealt with through separate arrangements, we will consider those options.
I well understand the need for this matter to be raised in Parliament. I will ensure that the points that have been made today are taken up with the PCT as it gives further consideration to the case being advanced by the foundation trust and by the family. I assure my hon. Friend that the Government’s priority is to ensure that NHS patients, including those living with phenylketonuria, are able to gain access to the most appropriate treatments in order to manage their conditions.
My hon. Friend has raised an important point about the need to ensure that the quality of life is properly respected, and that, in the very rare cases of phenylketonuria, that respect is reflected in the provision of drugs that allow a child to enjoy a wider range of meals and, as a result, grow up as a normal child with a normal life. I hope that he feels heartened by the debate, and that, as a hard-working constituency Member of Parliament, he will be emboldened to pursue this matter further.
Question put and agreed to.