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.