Phenylketonuria: Treatment and Support — [Mr Laurence Robertson in the Chair]

– in Westminster Hall at 9:30 am on 26 June 2018.

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Photo of Laurence Robertson Laurence Robertson Conservative, Tewkesbury 9:30, 26 June 2018

Given the number of Members who will seek to catch my eye, I am imposing a four-minute time limit on Back-Bench speeches.

Photo of Liz Twist Liz Twist Labour, Blaydon

I beg to move,

That this House
has considered access to treatment, support and innovative new medicines for phenylketonuria patients.

It is a pleasure to serve under your chairmanship in this important debate, Mr Robertson. It is just over 12 months since I was elected Member of Parliament for Blaydon, and only a few days less than that since I first heard of phenylketonuria—commonly known as PKU, which is easier to pronounce. One of my constituents, Barbara McGovern, had called at my office and spoken to a member of my staff about whether I would attend an event in Westminster on 28 June. Barbara’s son Archie has PKU, and she described his condition and some of the restrictions that he has to cope with every day. Her explanation of what PKU means, and her determination to get the best for her son, impressed my staff, who made sure that I attended the reception to learn more about the condition. Barbara and her husband David, and Archie, have travelled from the north-east to listen to the debate, and I welcome them.

Since that first encounter, I have been lucky enough to be introduced to the officers of the National Society for Phenylketonuria, and have had the chance to work with them and its other members to raise the profile of PKU, get people to understand it and its effects, and press for access to treatment and support. I will give a few of the many names that I could mention. Kate Learoyd and Caroline Graham both have children with PKU, and they have dedicated much of their time to talking to Members about the condition and the need for action for people who live with PKU. Professor Anita MacDonald OBE, of Birmingham Children’s Hospital, also does much to raise awareness of the condition and goes above and beyond in advising families affected by PKU on diet.

Photo of Ian Austin Ian Austin Labour, Dudley North

I commend my hon. Friend on securing the debate and on her leadership of the all-party parliamentary group on phenylketonuria. There are lots of health-related campaigns for more resources, new treatments and drugs. Many have plenty of resources and are funded by major pharmaceutical companies, which hire expensive lobbyists and all the rest of it. The remarkable thing about Kate Learoyd and Caroline Graham is that they are parents of children with the condition, and their campaign is entirely voluntary. They have to fit it around their family and other commitments, which makes their work all the more remarkable.

Photo of Liz Twist Liz Twist Labour, Blaydon

My hon. Friend is absolutely right, and of course there are many others working hard in the NSPKU.

PKU is a rare inherited disorder, affecting about one in 10,000 babies. Most people are familiar with the pinprick test that newborn babies are given; that is how the condition is picked up. It is one of a series of tests given. People with PKU are unable to break down the amino acid phenylalanine, which is found in proteins. They must have a diet very restricted in protein to prevent problems. If the condition is not properly controlled, it can lead to severe neurological and brain damage, as well as to behavioural problems. Untreated PKU causes profound, irreversible intellectual disability, seizures and behavioural problems. As the damage is not reversible, early diagnosis and early consistent treatment are vital.

I say that the condition can be treated by diet; that sounds easy, but it is not. Imagine, as a child or young person, trying to cope without all the foods that most children and adults take for granted. When we think of food that is high in protein, we probably think of meat, but that is the straightforward bit. All meats are on the red list, and so is fish. Everyday bread is too high in protein, so people with PKU must have special bread without protein, much of which must be baked at home using a specially prescribed flour.

Photo of James Morris James Morris Vice-Chair, Conservative Party

Having learned about the disease and issues connected with it, I think that the hon. Lady is right to argue for better treatment, but I am struck by the peculiar and intense pressures that PKU puts on parents and carers. Does she agree that we should explore ways to support them in coping with those pressures?

Photo of Liz Twist Liz Twist Labour, Blaydon

I agree. There is incredible strain on parents—and of course on people with the disease, but we must not forget about the parents.

Photo of Catherine McKinnell Catherine McKinnell Labour, Newcastle upon Tyne North

I commend both my hon. Friend for securing the debate, and the many parents and families who work to raise the issue. She is right to highlight how challenging the condition can be for children, particularly in adolescence. A constituent told me that her daughter asked whether, for her 18th birthday, she could eat a pizza in a restaurant—seemingly a normal activity for a teenager, but a clear challenge for such children, who want to fit in with their peers.

Photo of Liz Twist Liz Twist Labour, Blaydon

My hon. Friend is right, and I want to touch on that issue.

As well as having to have bread specially baked with prescribed flour, people with PKU can have no cheese, eggs or dairy products. Even some vegetables, such as cauliflower, are problematic, and so are potatoes, so there can be no chips or crisps. The daily intake of food needs to be monitored constantly. Imagine how that must be for a child at school who just wants to join in with classmates, or for young people who want to go out and socialise with friends—perhaps to go for a pizza, as my hon. Friend Catherine McKinnell mentioned—and get on with their life. Everything must be measured and calculated to make sure that the appropriate level of protein for the child or adult with PKU is not exceeded.

Let us not forget that the condition is lifelong, and adults, too, must restrict their intake of protein. On top of all that I have described, both children and adults must take a protein supplement. I and other MPs have had the chance to try it, courtesy of our friends at the NSPKU, and it is not a pleasant experience. That puts additional pressure on parents, who often struggle to get their children to take the supplements, which they really need, three times a day. Making sure a child has the right diet, including when parents are not around to control it, and trying to make food interesting, often by starting from scratch with basic low-protein foods, is a minefield.

Photo of Neil Gray Neil Gray Shadow SNP Spokesperson (Social Justice)

I congratulate the hon. Lady on securing the debate and on her work for the all-party group. A constituent shared with me some experiences similar to the ones that she has outlined. They are heartbreaking, and other Members who have constituents with PKU will share that feeling. Does the hon. Lady agree that alongside the medicinal treatments that might be available, it is important to raise awareness, through debates such as this one, and through Thursday’s “diet for a day”, in order to help provide and incentivise more support for parents who are supporting their children with PKU?

Photo of Liz Twist Liz Twist Labour, Blaydon

I agree; it is important to look at the question in the round.

Many parents find that they need to give up work or reduce their hours to maintain their child’s diet and keep them healthy. At the end of last year, the NSPKU produced a booklet and video, “Patient Voices: Listening to the experience of people living with PKU”, which clearly and movingly sets out the practical and psychological impact of the condition on individuals.

Photo of Jim Cunningham Jim Cunningham Labour, Coventry South

I congratulate my hon. Friend on getting the time for a debate on the issue; not enough is known about it. It strikes me, as I listen to her, that a child with PKU is a prisoner of their body, in a way, and so are their parents, because of the regimented way they must deal with the child’s needs. Does she agree?

Photo of Liz Twist Liz Twist Labour, Blaydon

I most certainly agree that it places an incredible strain on parents, who must live with that all the time. I recommend the “Patient Voices” booklet and video to anyone who has not already seen them.

In this debate, I will highlight very specific concerns about treatment and support for PKU. The first is the issue of access to a drug treatment, sapropterin, which is thankfully more commonly known as Kuvan. Although it is available in 25 countries across Europe, and was licensed for marketing over 10 years ago in the European Union, Kuvan is not available to people with PKU in the UK.

Photo of Tim Farron Tim Farron Liberal Democrat Spokesperson (Environment, Food and Rural Affairs)

I congratulate the hon. Lady on this important debate. She makes a hugely important point about the licensing of Kuvan. The European Medicines Agency licensed the drug in 2008, and 10 years on we have buck-passing between the National Institute for Health and Care Excellence, the Department of Health and Social Care and NHS England. It is deeply concerning. I am representing two children with PKU—I am sure there are many more—in my constituency. I got a letter back from NICE just a few weeks ago that said that the condition and the treatments for PKU are

“the subject of a NHS England commissioning policy…not covered by any existing NICE guidance.”

It went on to pass the buck back to NHS England. Does the hon. Lady agree that it is time for the buck-passing to stop, and for the treatment to be licensed?

Photo of Liz Twist Liz Twist Labour, Blaydon

I absolutely agree. I hope that one of the good things that will come out of this debate will be that the buck stops being passed, and the assessment gets done as quickly as possible. It is so important for people to have access to this drug; we need that to be sorted.

There is evidence that for a significant proportion of people with PKU—about 25% —this drug can significantly improve their condition. It does not cure it, but it does make it much easier to deal with the dietary issues, which have such an impact on the way people live their lives. Despite the drug having been around for so long, NHS England has only recently considered it for the management of PKU. The drug has now been referred to NICE for assessment and technology appraisal. The APPG on PKU recently heard from NICE about the process, but there is concern about the timescales and how the benefits of the treatment will be assessed. Understandably, there is huge frustration on the part of the PKU community that there are children and adults who could be benefiting from Kuvan now, and there is substantial evidence to support its benefits.

There is a particular issue about prescribing Kuvan for pregnant women with PKU, who can understandably find it hugely difficult to control their diet, and who fear the effect of any problems on their unborn child. While there is a 2013 commissioning policy in place that allows Kuvan to be prescribed to some pregnant women, it can be difficult for women to be prescribed it in a timely way.

Some people, some of whom are in this room, have had access to Kuvan through individual funding requests, or on a trial basis. Those people have found real benefits from the drug. My constituent Archie, who is here, started on the treatment earlier this year. Archie tells me he has benefited from having Kuvan, not just because his diet is now much less restricted and he is able to do what many of his school mates do, but because it has improved his energy and his life. As his mum Barbara said to me, “If we had been coming here before the treatment, we would have been bringing our own special breakfast for Archie to eat in the hotel, and would have had to watch everything he ate very carefully. It has made a real difference.” I hope that the Minister will be able to assure us that the assessment of Kuvan will be done very quickly, and that it will be available to the people it can help.

Photo of Vicky Ford Vicky Ford Conservative, Chelmsford

I congratulate the hon. Lady on securing this important debate. Kuvan, the brand name for sapropterin, is clearly deeply beneficial for about 20% to 30% of sufferers. I met the manufacturers last week, and they told me they had written to NHS England twice to ask for meetings to discuss price, but they are still waiting for a response. Will she join me in urging the Minister to use all his best offices to ensure that that meeting and the price negotiation can go ahead?

Photo of Liz Twist Liz Twist Labour, Blaydon

I certainly do join the hon. Lady in that wish. I too have met BioMarin, which says it is prepared to negotiate significantly on the price, but we need to get in there and ensure that it happens quickly.

Photo of George Howarth George Howarth Labour, Knowsley

I congratulate my hon. Friend not only on securing this debate, but on the knowledgeable and passionate way in which she is putting the case. Does she agree that this is an example of the way that we in this country are very bad at dealing with rare conditions and potential therapies or treatments for them? Does she believe that, while this needs to be hurried up, the whole process needs to be streamlined and sped up?

Photo of Liz Twist Liz Twist Labour, Blaydon

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.

Photo of Nigel Mills Nigel Mills Conservative, Amber Valley

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?

Photo of Liz Twist Liz Twist Labour, Blaydon

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.

Photo of Paul Masterton Paul Masterton Conservative, East Renfrewshire 9:49, 26 June 2018

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. 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 expensive 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.

Photo of Stephen Morgan Stephen Morgan Labour, Portsmouth South 9:53, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I must admit that I had not heard of PKU before my election to the House, let alone of some of the medical terms, which I still cannot pronounce. I think that the same is true for many across our country. In fact, after hearing that it meant a fault with enzymes, leading to an inability to break down the amino acid phenylalanine—PHE—I was not much the wiser.

However, after hearing my constituent Holly Mae’s story and what the condition meant for her everyday life and tasting the various concoctions that replace typical meals for PKU patients, I was left in no doubt as to the potential debilitating impact of the disease. That is why I will begin with the impact on everyday life and what that means in practice for people with PKU.

As has been mentioned, people with PKU have to eat a diet with virtually no protein, meaning that they must take chemical supplements to avoid malnutrition. The briefing I was sent by the excellent NSPKU describes the protein replacements as “unpalatable”. I have tasted those replacements and can assure hon. Members that the NSPKU is being polite; they are absolutely rank. However, they do not just taste disgusting. They form part of an incredibly prescriptive and restrictive diet that not only consumes a huge amount of time—approximately 19 hours a week—but makes living a normal life difficult and social activities intolerably difficult.

Hollie Mae’s mum, Tara, says they hardly ever eat out. When they do, they have to bring separate food. It is the same at friends’ houses. It is inevitably a difficult diet to manage, and because no young person likes to stand out as different, PKU makes sensitivity and insecurity around food and eating particularly pernicious among its teenage victims. These young people just want to live normal, happy lives, but PKU often exacerbates teenagers’ vulnerability to eating disorders and so also becomes a mental health issue. I therefore urge the Minister to do all he can to improve access to psychological support for people with PKU. Clearly, the pressure of PKU and the diet it necessitates puts patients in immense difficulty and can be overwhelming.

We have just celebrated national Carers Week, and it is important to consider the burden that falls on families too, as we have heard. Half of parents stop working or reduce their hours to accommodate the extra work of caring for a child with PKU. What is more, the burden inevitably falls on women, with 81% of respondents to an NSPKU survey saying that it was the mothers who did most of the PKU-related work. I pay tribute to Tara and all the other parents and families who care for those with PKU. However, they do not want warm words, they need action.

I hope the Minister agrees that the opportunity to offer those with PKU and their families hope of a better life cannot be missed. There are practical things that can be done. I suggest that that means meeting with the manufacturers of a tablet can make a massive difference. It means making life a little bit easier for families and PKU patients by fixing the fragmented service on offer and smoothing and simplifying the chain from specialist metabolic clinic to GP to pharmacist to courier, complications in which constantly cause grief for patients. It definitely means ending the exclusion of PKU treatments from the prescription charge exemption.

The fact that PKU is a rare disease should not mean that it deserves any less of our attention. On behalf of my constituents and Hollie Mae, I urge the Minister to implement these changes.

Photo of Peter Aldous Peter Aldous Conservative, Waveney 9:57, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I congratulate Liz Twist on securing the debate and on championing so well the cause of PKU patients and their families and carers.

It is also appropriate to highlight the pioneering work of my hon. Friend Kwasi Kwarteng. In an Adjournment debate on 1 December 2011, he highlighted his constituent’s problems in obtaining a Kuvan prescription. He successfully secured a prescription for his constituent, but it is most unfortunate, as we have heard, that six and a half years later we are no further forward in making this drug, which has the proven potential to change many people’s lives, more widely available.

Finley Walsh lives in Lowestoft with his parents, Michelle and David. He is two and half years old and he was born with PKU—a genetic condition that will be with him for his whole life. All Finley’s foods have to be weighed, using a calculation that takes account of the amount of protein in the food. His parents have to take weekly heel-prick blood tests, which are sent to Addenbrooke’s hospital in Cambridge for analysis. The results are then sent to the Norfolk and Norwich Hospital, where dieticians phone through the results that enable Michelle and David to prepare Finley’s food intake for the coming week. That is a critical, delicate and often worrying process for them. It is vital to get Finley’s protein levels right; if they go too high, there is a real risk of brain damage.

The challenges that the family face daily are immense and place a real strain on them. Quite often, the blood test results do not come through on time and must be chased up. At present, they have had no feedback for two weeks. Food must be ordered on prescription to enhance Finley’s diet. Products such as those from Violife and Hooba are not only expensive but, quite often, not immediately and readily available. At present, there is also the worry that Finley was due to have a review with his specialist in May and that appointment has yet to take place.

Children with PKU suffer patchy care and support, which depends on where they live. That could be addressed by setting up specialised metabolic centres with an experienced metabolic physician and dietician. Psychological support should also be available in the centres to assist children if they experience learning difficulties and to ensure that they receive an education that enables them to realise their full potential. The centres could also administer prescriptions for PKU foods and dietary supplements so as to provide a more efficient service and to overcome the problems that the Walshes are experiencing.

On Thursday I, too, will take part in the PKU diet for a day challenge. In no way does that replicate the real experiences of people such as Finley, but I hope that together, we in this Chamber and around the House can highlight the need for modern treatments and better care for those who face such an enormous challenge daily.

Photo of Ian Austin Ian Austin Labour, Dudley North 10:01, 26 June 2018

Like other hon. Members, I had never heard of this condition until I was contacted by a constituent, Kirsty Thornton from Dudley. She is the reason why I am taking part in the debate. To hear how the condition had affected her was very moving, and as a result of meeting her, I met Kate Learoyd and Caroline Graham, to whom I paid tribute earlier. I want to mention one other person at the outset. Professor Anita MacDonald OBE, who is with us today, is the brilliant head of a dedicated team of dieticians caring for children with rare inherited metabolic disorders at the Birmingham Children’s Hospital. I want to tell the Minister that Kate Learoyd, Caroline Graham and Anita MacDonald are here today. I hope that, at the conclusion of the debate, he might find a moment to say hello to them and arrange to meet them properly and at length subsequently.

It is deeply moving to hear how families manage this condition, particularly for toddlers, who cannot understand why they are not allowed to eat the same food as their siblings or have the same food as their friends at a birthday party. One parent has said of their child:

“She resents the fact that her family can eat normally and she can’t. At mealtimes, she will go into a depression. Often she will ask to eat the crumbs of normal bread off our plates or we catch her licking our plates. It is awful to see.”

It is very distressing to hear how a simple mistake can have huge ramifications, affecting a young person’s concentration and even their mental health and then their ability to study or to work. One young person said:

“When I have high phe levels I slur my words, struggle with balance, lose my train of thought and stop speaking...I am hit with such fatigue that I lose sight of what it is like to feel awake.”

I would therefore like to encourage the Minister and the Opposition spokesperson, my hon. Friend Mrs Hodgson, to join us on the PKU diet challenge this Thursday—indeed, I hope that as many hon. Members as possible will take part—to highlight the difficulty of adhering to the strict rules that people with this condition have to follow. We have to do this properly and stick to the rules. We have to check all the ingredients, as we heard earlier, weigh foods properly and keep a tally throughout the day of the amount of protein that we have eaten. Most of all, as we have just heard, we have to remember that we are doing it only for a day and not a lifetime, like the constituents on whose behalf we are speaking today.

I want to ask the Minister three other questions before I conclude. First, as he has heard, we are calling for an examination of the failure to use Kuvan in the UK. That treatment can transform people’s lives. It has been licensed for almost 10 years and is used in lots of other countries in the EU. We would like the Minister to take personal charge of this matter and work out what can be done to sort out provision of it in the UK, too.

Secondly—we have raised this issue in respect of other conditions and other drugs—will he look at the appraisal rules for new treatments for PKU? This is a very rare disease, but it has a wide impact outside direct health costs. It is not a criticism of NICE or the Minister or Government, but the fact is that these are conditions and treatments that NICE was not really designed to deal with. How can the appraisal system be altered to work for conditions such as this?

Finally, will the Minister work with the NSPKU to review the provision of treatment to patients, including basic dietary treatments? How can that be made consistent across the country? It is not the case at the moment. That is a very urgent thing that it should be possible to sort out without too much difficulty.

Photo of Vicky Ford Vicky Ford Conservative, Chelmsford 10:05, 26 June 2018

Thank you, Mr Robertson, for the opportunity to speak in this important debate. I add my congratulations to Liz Twist on securing it. I speak as a chair of the all-party parliamentary group on rare, genetic and undiagnosed conditions, but also on behalf of my constituent Cait, and other constituents who suffer from PKU. I was very honoured to lead an Adjournment debate on this condition in March. I will not repeat everything that I said then, but I want to add some points.

PKU is a very rare condition, affecting about one in 10,000 people, but it is not ultra-rare, and that is part of the problem. Living with PKU is extraordinarily challenging, but for the 20% to 30% of sufferers who react positively to the drug sapropterin, there is a glimmer of hope. That is only about 150 children in the UK, and about 350 people in total, but for them, sapropterin is life-changing. Sapropterin is available in Austria, Belgium, Bulgaria, the Czech Republic, Denmark, Estonia, France, Germany, Hungary, Italy, Latvia, Lithuania, Luxembourg, the Netherlands, Norway, Portugal, Romania, Russia, Slovakia, Slovenia, Spain, Sweden, Switzerland, Turkey and Ukraine, as well as in the United States. However, except for a small number of people—truly exceptional cases—and women during pregnancy, it is not available in England on the NHS, and that must change. It should be available for all those who would benefit, not just those who cannot stick to the diet. Those who do stick to the diet should not be excluded for good behaviour.

I understand that NICE sometimes has a very challenging time in considering whether to approve drugs that can be very expensive, but sapropterin does not fall into that category. In my Adjournment debate, I pointed out that when the broader economic benefits of prescribing PKU are compared with the costs of not doing so, the pure financial calculation alone suggests that it may even be financially beneficial to the public purse to prescribe the drug. Furthermore, as I stated back in March, BioMarin, the manufacturer, told me that it was willing to make a substantial reduction in the price. During that debate, the Under-Secretary of State for Health and Social Care, my hon. Friend Jackie Doyle-Price, suggested that a patient should go down the individual funding request route, but I understand that only one child has ever managed to receive the drug by that route. It simply does not work for the patients who need it.

I have written to the Under-Secretary of State for Health in the House of Lords a couple of times and I am grateful for his responses, but in his latest response, he suggests, in relation to the approval process, that the drug has now been prioritised for potential guidance development through NICE’s technology appraisal programme. Both patient stakeholders and the manufacturer are really concerned that that could lead to even more delays. BioMarin tells me that it has written twice to NHS England, offering to meet to discuss price. It wants to negotiate on price; it wants to make a generous offer, but its letters have apparently not been responded to. The decision needs to be made. It should not need 21 Members of Parliament standing here in Westminster Hall to get this drug for 150 children.

Looking at the broader picture, there is a fundamental problem with NICE’s commissioning programme. It works well for common conditions, and the highly specialised technologies process seems to work well for ultra-rare conditions, but conditions such as PKU fall through the gap. We need to find a way to get modern, personalised, specialist medicines to those who need them, where their condition is rare, but not unique.

Photo of Liz Saville-Roberts Liz Saville-Roberts Shadow PC Spokesperson (Home Affairs), Shadow PC Spokesperson (Women and Equalities) , Plaid Cymru Westminster Leader, Shadow PC Spokesperson (Justice), Shadow PC Spokesperson (Business, Energy and Industrial Strategy) 10:09, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I thank Liz Twist for securing this important debate and for her cogent argument. I am here on behalf of Mark Edwards of Llanegryn, Tywyn in Gwynedd. We have heard much about the impact on families and children, but the most effective thing I can do is talk about how Mark, as an adult, deals with this condition; how he manages from day to day; and what would make a difference to the quality of his life and the lives of others living with PKU.

Mark first contacted me about the sugar tax, which I had blithely thought to be a good thing. The sugar-sweetened drinks issue sums up how people with PKU struggle to strike the excruciatingly difficult balance between a medically essential specialist diet and fitting in with society’s rigid norms about food and drink.

Our social lives revolve around food and drink, from children’s tea parties to teenagers’ fast-food binges to adults’ restaurant dates. I have been told time and again how difficult it is for people with PKU to cope with the stigma of being “difficult” and “different”, especially over something as integral to our lives as food. If they cannot enjoy cake, jelly, burgers and chips, at least they can enjoy a fizzy drink—Irn-Bru or whatever the brand—like everyone else. Now, however, it costs more, because aspartame is on the red list. People with PKU are being taxed on one of the few social drink and food experiences that they are able to share with everybody else, simply because of their condition.

Mark is keen to stress how much he appreciates Wales’ policy on universal free prescriptions, which allows him to receive the special food and medical dietary supplements that he needs through Tywyn health centre’s dispensary, when he needs them and for free. That means that people with PKU in Wales have a much better arrangement, he said, than those in England. None the less, Mark still faces public prejudice towards his invisible condition. He has had to explain to people that he is not “freeloading on food”, as though it were a matter of lifestyle choice, and not medical necessity.

A number of us in this Chamber have committed to the PKU “diet for a day” on Thursday. A great number of people in Wales have also done so, and they deserve a shout out. They are: Alex Jones of Cambrian News; Janet Davis, the supervisor of Brighter Foods, where Mark works; school friends Carys Hughes and Nicci Hughes; and Tywyn solicitor Andre Bright, who has committed to keeping to the diet for a week. He deserves respect, even if a week is nothing compared to what the families face. I am only brave and organised enough to do it for a day; I apologise. We are doing this in solidarity with PKU families, but we also know that this in no way fully reflects or replicates the reality of their lives. Most of us will do this by being fussy and awkward—I anticipate living off aubergine for a day—but we are only doing it for one day. We will not face this fraught, potentially toxic relationship with food, and the stigma associated with it, every day of our lives.

PKU affects every aspect of one’s life. Controlling the condition by diet alone causes immense strain, and any possible medical intervention will make an immense difference to the quality of people’s lives. Wales is alert to England’s NICE guidelines, and I urge the Minister to do all he can to press NICE to move ahead, so that his Department can recommend Kuvan. I also urge that other drugs, such as Pegvaliase, be considered. I ask the Government to consider the health implications of the wider use of aspartame, which is associated with the sugar tax, and the way that it affects a number of other health conditions as well. Diolch yn fawr.

Photo of Jim Shannon Jim Shannon Shadow DUP Spokesperson (Human Rights), Shadow DUP Spokesperson (Health) 10:13, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I congratulate Liz Twist on bringing this debate. I also congratulate Vicky Ford on her work in the past and on bringing her Adjournment debate to the House, which I supported.

I am the Democratic Unionist party health spokesperson, so it is important to be heard on this issue, which grossly affects people in Northern Ireland, as well as in the Republic of Ireland. Not many people know this, as Michael Caine always says, but there is a higher per capita prevalence of PKU in Northern Ireland than in the rest of the UK. In fact, one in 4,000 people in Northern Ireland has this condition, compared to one in 12,000 in England.

Photo of Gregory Campbell Gregory Campbell Shadow DUP Spokesperson (International Development), Shadow DUP Spokesperson (Cabinet Office)

Given the higher incidence in Northern Ireland, but also instances across the United Kingdom, does my hon. Friend agree that that it is imperative for the Minister, and all health Departments across the UK under the devolution settlement, to ensure that the best possible treatment and support is given to PKU sufferers and their families?

Photo of Jim Shannon Jim Shannon Shadow DUP Spokesperson (Human Rights), Shadow DUP Spokesperson (Health)

My hon. Friend is absolutely right. We always look to the Minister for support on these issues, and he is always very forthcoming, so we look to him again with that in mind. It is clear that this condition affects my constituents and those of everyone else who is speaking today.

PKU is usually diagnosed shortly after birth by the heel test. Many women can tell us that when the heel prick is done and they hear their child crying in the hands of the midwife, their automatic reaction is to reach out and grab the child. That test is so important at a very early stage. For those families who receive a diagnosis of PKU, however, the pain begins when they realise just what that means.

People with PKU have a faulty version of the enzyme that breaks down the amino acid phenylalanine, a component of protein. Untreated, it can cause brain damage when it builds up in the blood and brain. Untreated PKU causes profound and irreversible intellectual disability, seizures and behavioural problems. The damage is not reversible, so early diagnosis and early consistent treatment is vital. That is why, in 1969, we added this test to the routine blood test at birth. Some people here were not born then; I was just a young child.

The only treatment for PKU that is currently funded by the NHS is a very restrictive diet. I am a type 2 diabetic, so I understand a wee bit what it means to be careful with what I eat. I know that if I had a wee bit of honey with my toast this morning, I probably should not have done, but by and large I know what I have to do, and what I can and cannot eat. For those with PKU it is much more difficult, and the restriction is great. Most sources of protein are removed from the diet to prevent brain damage.

I want to give a few quotes from the parents of PKU sufferers, so that we can understand a bit better the life lived by those with this disorder. One parent said:

“The low protein prescription breads and pastas give her stomach ache—another reason she refuses to eat them.”

A parent whose daughter has PKU said:

“My daughter struggles with drink supplements as they all upset her tummy so she has to take 50 tablets per day.”

Another parent said:

“PKU causes arguments between us. My husband and I have suffered with stress, we argue about the management of her diet. I had hoped after 12 years things would get easier but this diet is met with anger, frustration, resistance and annoyance all aimed at me.”

Ian Austin already referred to the following case, but it is worth repeating simply because of its importance. One parent said that her daughter cannot normally eat, adding,

“she will ask to eat crumbs of normal bread off our plates or we catch her licking our plates.”

That is the impact PKU has on some children and their families. That is why parents throughout the UK are demanding that more be done. We look to the Minister to see if more can be done through his office. If there is something to help these people, we must make it available. We all know what must be made available: Kuvan. We all know what it can do. One young girl took a one-month trial of Kuvan and could eat a normal vegetarian diet. She had more energy, her mood lifted, her nightmares stopped and she could do ordinary activities at home and at school. What a difference it made to the child’s quality of life, and that of the entire family!

I have read that the cost of Kuvan is on average £14,535 for a child and £43,597 for an adult, based on list pricing. The pharmaceutical company BioMarin has publicly stated its willingness to offer substantial discounts in a deal with the NHS. I am asking the Minister, as other Members have done, to broker that deal, and enter into meaningful discussions on providing the medication, as the High Court ruling has said that we should. I urge the Minister to instruct his Department to find a way of making this available, rather than simply checking a box.

In conclusion, I ask that no parent be forced into this situation when there is something available to prevent it. I stand with the PKU sufferers of Strangford, Northern Ireland and the whole of the United Kingdom of Great Britain and Northern Ireland.

Photo of Faisal Rashid Faisal Rashid Labour, Warrington South 10:19, 26 June 2018

I commend the fantastic work of my hon. Friend Liz Twist and the National Society for Phenylketonuria, who have worked incredibly hard to raise awareness of PKU in Parliament in the past few months.

Until I met with representatives from the NSPKU a few months ago, I knew very little about PKU, like many other hon. Members. Since, I have learned a huge amount about it, and it has become clear that this devastating disease requires action. It affects one in 10,000 people across the UK. Although it is rare, an estimated 3,000 individuals in England alone live with it and could benefit from increased awareness and improved treatment of it.

As we have heard, the only treatment of PKU available on the NHS is a severely restrictive diet. Patient voices from the NSPKU illustrate how much of a daily struggle that diet is. It often leads to eating disorders and unhealthy relationships with food. It forces patients into social exclusion and is a great burden on their carers, who often also report psychological distress and strained family relationships. Studies show that half of parents will stop work or reduce their hours to accommodate the extra work needed to take care of a child with PKU.

I am grateful to my constituents, who shared with me the story of life with their five-year-old daughter who has PKU. Their openness helped me to understand what that life is like, and the difficulties their daughter faces daily, especially when she is unable to eat the same meals as other children at school.

The fragmented nature of NHS services provided to PKU patients often leads to confusion and mistakes in the chain. The inconsistent quality of those services has made the system needlessly complex and has added to the patients’ burden. There have been multiple reports of clinical commissioning groups restricting funding for PKU dietary products. The variable quality of even the most basic treatment is simply not good enough.

Beyond dietary treatments, a drug called Kuvan has been developed, as has been mentioned. It can treat up to 25% of PKU patients and allow them to eat substantially more natural proteins, which fundamentally improves their quality of life. Despite having been licensed in the USA since 2007 and the EU since 2008, and being used in almost every EU country as a routine treatment for PKU, it is unavailable for the vast majority of patients in the UK.

Of course, Kuvan comes with a price tag, but as we approach the 70th birthday of our treasured NHS, we must allow it to uphold its fundamental principle of providing healthcare on the basis of need.

Photo of Catherine West Catherine West Labour, Hornsey and Wood Green 10:23, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I congratulate my hon. Friend Liz Twist on securing this important debate. I will speak on behalf of my 11-year-old constituent, Olivia, and describe a bit of her struggle. I congratulate her parents on fighting so hard for her, because the stress on families is incredible. We have heard from many hon. Members about the impact that PKU can have on the whole family and just how isolating it is. The National Society for Phenylketonuria is helping us as parliamentarians to get to grips with the issues.

We have heard how restrictive the diet is. My hon. Friend Catherine McKinnell, who is no longer in her place, talked about going to a pizza restaurant at the age of 18. Olivia is 11, but she is allowed only 8 grams of protein a day. She was fortunate enough to participate in the one-month trial of Kuvan, and she was found to be a responder. Her protein tolerance increased from 8g to 27g. On Kuvan, she had safe, low blood results and ate healthy, natural, normal vegetarian food. She could have baked beans at school on a Friday with a normal-sized portion of chips, just like her friends. It filled her plate—and her, instead of leaving her hungry. Her teachers commented on how bright and focused she was in lessons. Ironically, they asked, “What did she have for breakfast today?”. Olivia loves life, and, during that month on Kuvan, it showed, just as it does for everyone else.

As many hon. Members have pointed out, it seems anomalous that a drug that is licensed in the US and the EU is still so difficult to obtain for our constituents. NHS England has handed the draft policy on commissioning Kuvan to NICE for review, but no date has been set. I press the Minister for a date, because it is absurd that Kuvan has been prescribed for years as a routine treatment in the rest of the developed world.

For Olivia, the waiting is taking its toll. She has frequent and severe migraines that cause vision disturbance, light, sound and taste sensitivity, vomiting and gastroparesis. During such times, she loses her PKU supplement and exchanges food, which takes her off diet for the duration of the migraine. They are difficult times for the whole family. Her illness and temporarily altered sense of taste mean she cannot drink the supplement and wants the normal food she prefers. How could anyone refuse a sick child because the food contains too much protein? She can be off diet for 60 hours.

Last year, Olivia was also diagnosed with moderate to severe scoliosis—a curved spine—for which she has to wear a back brace for 23 hours a day until she is 16 years old. It is uncomfortable, painful, and often prevents sleep. Her consultant said that spinal fusion surgery would likely be advised, but PKU is likely to result in low bone density, especially among girls. Olivia had a scan that showed that her bone density was abnormally low and getting worse.

Nothing more can be done dietetically; the amino acid supplement contains the correct levels of calcium, vitamin D, vitamin K and potassium, but it does not contain whole protein, which plays a major role in bone density. By definition, the diet goes to great lengths to avoid whole proteins, and that is one of the consequences. Olivia may therefore never be able to have the spinal fusion surgery that she needs, because her bones are not strong enough to take the screws and rods that would need to be drilled and fixed to her spine. Kuvan would at least allow her to eat more whole natural protein. Would that not be better for her, and everyone else who has been mentioned? The only access route to Kuvan remains an individual funding request. I hope that the Minister will consider that issue in his response.

Photo of Chris Williamson Chris Williamson Labour, Derby North 10:27, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I congratulate my hon. Friend Liz Twist on securing this important debate. We have heard some heartrending accounts from hon. Members about the impact of PKU on their constituents.

My constituent, Denise Clayton, whose family I have known for 30 years, has lived with PKU for nearly 70 years, so she was born long before any real screening programme was in place. Consequently, she did not receive the required treatment, and the treatment she did receive was too little, too late to avoid neurotoxicity. Her family have done everything they could to support Denise. A few weeks ago, I spoke to her father, Norman, who recounted some of the traumas and difficulties that they experienced down the years. His view was that nothing could be done for Denise, but it seems that a treatment is now available.

I join hon. Members in their pleas with the Minister to use his good offices to ensure that the new life-changing drug Kuvan is made available so that the sufferers of PKU in the UK—like sufferers in other countries—can obtain the benefits of it and lead something approaching a normal life. Let us remember that we live in a wealthy nation; we are the sixth-biggest economy in the world.

PKU is a very rare condition that affects only a very few people in total across the country. The cost of the drug would be minimal in comparison to the life-changing impact it could bring about for young people—our fellow citizens. I plead with the Minister to listen to the impassioned pleas we have heard from Members today. He should use his good offices to ensure that the drug is made available so that we can change the lives of so many young people in our country who are suffering from this terrible condition.

Photo of Laurence Robertson Laurence Robertson Conservative, Tewkesbury

We now come to the wind-ups. I would like to leave two minutes for the mover of the motion to wind up at the end.

Photo of Martyn Day Martyn Day Scottish National Party, Linlithgow and East Falkirk 10:30, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I congratulate Liz Twist on securing time for this important debate and on her comprehensive and detailed summary of the issue. I am also grateful for the NSPKU briefing she kindly sent round to colleagues in advance of the debate. I must admit I had been in touch with Library specialists before I received the briefing to find out the pronunciation. The hon. Lady sensibly told us how to pronounce it. I will refer to it as PKU for the rest of the debate, despite my hatred of acronyms. It is fair to say that without that guidance, I would have been mispronouncing it.

I welcome the NSPKU members who are here to watch the debate. In preparation for the debate, I found out just how awful the condition is. I am grateful to all Members who have taken part and shared their constituent cases, which have helped highlight how truly horrendous the situation is. For my part, I am aware of no cases in my constituency, but as health is a devolved matter, they would more likely go to Scottish Parliament counterparts.

We have heard that PKU is a rare inherited disorder sufferers of which are unable to break down the amino acid phenylalanine. It is a truly horrific condition, and it is worth putting on record that there is currently no cure. Left untreated, it can cause serious damage to the brain and nervous system, which can lead to learning disabilities and other symptoms. As has been pointed out, the condition affects about one in 10,000 babies in the UK. As Paul Masterton mentioned, the situation is worse in Scotland. The stats I have seen suggest that the condition affects one in every 8,000 babies in Scotland, which represents about six or seven cases a year.

Early intervention is vital. Without it, irreversible damage can occur. The Scottish Government take the condition seriously, which is why at around five days old, babies are offered newborn blood spot screening to check whether they have PKU or a number of other conditions. If PKU is confirmed, treatment will be given straight away to reduce the risk of serious complications. If the right treatment is followed, babies with PKU are well in early life and do not develop symptoms. It can be managed with a low protein diet, but as has been pointed out by many speakers today, that is far from an easy option.

We have heard much about access to new treatments such as sapropterin, also known as Kuvan, which is available in 25 countries and has been licensed in the EU since 2008. In May this year, Scotland’s Health Secretary Shona Robison wrote to the Health and Sport Committee to provide a further update on the Scottish Government’s progress in delivering the recommendations from the review of access to new medicines. She confirmed that the pharmaceutical company BioMarin has made a submission to the Scottish Medicines Consortium for sapropterin or Kuvan to be used for the treatment of PKU. The SMC will publish its advice within the next few weeks. Let us hope that we can see progress in the matter as a consequence of the Montgomery review and the definitions of new processes for ultra-orphan drugs.

Decisions made by the Scottish Medicines Consortium are independent of Ministers and the Scottish Parliament, and it is worth remembering that our involvement in that process can be limited, but I would be happy to offer my support to the hon. Member for East Renfrewshire to do any joint working we can to bring pressure to bear, as the drug would be very beneficial for sufferers. The Scottish Government do not intervene in SMC processes, but they have sought to consider with all parties how issues identified in previous submissions could be resolved in new applications to achieve a best-value deal for NHS Scotland.

The Scottish Government have significantly increased access to new medicines in recent years. Between 2011 and 2013, the combined SMC acceptance rate for orphan cancer medicines was 48%. Between 2014 and 2016, the rate was 75% for ultra-orphan, orphan and end-of-life medicines. There are some positives that we can look at in that process. A responsible funding model is key, however. The Scottish Government are actively examining an improved negotiating function that seeks to ensure that the NHS in Scotland pays the same effective price for medicines as in the rest of the UK.

I thank everyone who has taken part. My sympathies go to anyone who is living with the condition. I would be interested in supporting the diet for a day challenge. My diet needs serious improvement at a range of levels, but I would be up for putting in the effort.

Photo of Sharon Hodgson Sharon Hodgson Shadow Minister (Public Health) 10:35, 26 June 2018

It is a pleasure to serve under your chairmanship, Mr Robertson. I thank my hon. Friend Liz Twist for securing this important debate, for her excellent speech and for all her campaigning on the issue. I also thank all the Members who have spoken this morning. There has been a good number. I thank Paul Masterton, my hon. Friend Stephen Morgan, Peter Aldous, my hon. Friend Ian Austin, Vicky Ford, Liz Saville Roberts, Jim Shannon, my hon. Friends the Members for Warrington South (Faisal Rashid), for Hornsey and Wood Green (Catherine West) and for Derby North (Chris Williamson) and Martyn Day for their excellent contributions to this important debate.

I also thank the all-party parliamentary group on phenylketonuria, which is more commonly known as PKU. I understand the group was only recently set up by my hon. Friend the Member for Blaydon and others. It is already providing an invaluable forum for PKU to be discussed. Finally, I thank the National Society for PKU for the help and support it provides to sufferers of PKU, for its sponsorship of medical research into PKU and for the work it does with medical professionals in the UK. Just last week, it held a particularly informative event in Parliament sponsored by my hon. Friend. I attended it, and I know it will have been helpful in spreading awareness of this extremely serious disease. I found it very useful, and I know other Members did, too.

I had not heard of PKU. It is a rare metabolic disease that causes an inability to break down the amino acid phenylalanine, which can then build up in the blood and brain. Left untreated, PKU causes severe intellectual disability, seizures and behavioural problems. Damage caused by the disease is, tragically, irreversible. That makes early diagnosis and treatment essential. The only treatment available on the NHS for PKU is an extremely restrictive diet. A PKU diet involves avoiding most forms of protein, and taking a special protein replacement—as we have heard, it does not taste as good as what it replaces—to avoid malnutrition. I would like to briefly highlight a number of the problems with the treatment.

First, it is extremely restrictive. Only a small number of foods can be eaten without severe limitation. It is easy to think that almost all food allergies and requirements are catered for in the modern supermarket these days, but with PKU that is not the case. Some of the necessary food replacements are only available by prescription. For some sufferers, the nature of the diet can have a detrimental impact on their social lives, particularly for younger people, as we have heard from a number of Members. Sadly, a high number of PKU patients also suffer from eating disorders and other mental health problems because of it. The NSPKU recommends that all people with PKU should automatically have follow-up appointments with an integrated specialist metabolic physical dietician, along with support from a psychologist and support worker. Is that something the Minister agrees with? Are the Government looking into providing that kind of support?

The second problem with the current available treatment is that it places a huge amount of pressure on those who care for children with PKU. In order to administer the necessary diet, a significant amount of measuring and preparation is required. As we heard at the event last week, dietary care takes on average 19 hours a week according to a recent study.

Photo of Catherine West Catherine West Labour, Hornsey and Wood Green

Is the shadow Minister aware that nearly 60% of mothers who care for their children with PKU have some kind of other psychological stress associated with this type of lifestyle?

Photo of Sharon Hodgson Sharon Hodgson Shadow Minister (Public Health)

I was not aware of that fact, but it is hardly surprising when we realise how complicated the diet is. As the child grows, the calculations have to be changed. As we know, children’s sizes change every week, so it is a constant battle to try to get it right, and it is not surprising that that figure is so high.

As the consequences of a child with PKU consuming the wrong type of food are so severe, it is easy to see how much stress a carer can go through in ensuring not only that they are preparing the right food but that a child follows the diet, particularly when away from home, as we have heard, or in school. With that in mind, I want to know whether the Minister believes that patient-centred care should be extended to school support, psychological support and counselling in order to relieve some of the pressure on carers.

When we consider the fact that the consequences of failing to adhere to the necessary diet are so extreme, one would imagine that all treatments that could improve outcomes would be available. Sadly, as we have heard, that is not the case. As has already been discussed this morning, a non-dietary treatment for PKU does exist, and yet it is not available to patients here in England. Kuvan is a licensed medicine that comes in the form of a simple tablet. In some 20% to 30% of people with PKU, taking Kuvan considerably increases the amount of protein that they can eat each day while maintaining a safe phenylalanine level. Indeed, some patients are able to stop or decrease the use of specially manufactured prescription foods while taking the drug. For those people, having access to Kuvan would literally change their lives and in some cases it would allow them to come off their restricted diet.

Unfortunately, the treatment is not currently commissioned by the NHS, except in a very small number of cases and for women during pregnancy. That is despite its having been licensed in the USA since 2007 and in the EU since 2008. It is used by thousands of patients across Europe and around the world. We heard the full list from the hon. Member for Chelmsford, but I will simply mention such countries as Ukraine, Estonia and Turkey by way of example. Many patients who suffer from PKU will rightly ask why, if the treatment is available there, it is not here?

Although Kuvan is available to women during pregnancy, it can be difficult to get hold of. Tragically, some women with PKU avoid having children altogether owing to fear of the risks to the foetus associated with high levels of phenylalanine. I understand that NHS England has recently referred Kuvan to NICE after it went through its internal clinical panel. Can the Minister explain why there has been such a delay in commissioning Kuvan, and when we can expect it to be available to all patients? Indeed, I understand that it was under the appraisal process in NHS England for seven years.

Access to Kuvan is not the only PKU treatment that has been impeded by the structure of the NHS. For sufferers of PKU, there is a significant risk of variable outcomes and health inequalities, something that is exacerbated by access to special protein replacements and manufactured low protein foods. Many PKU patients have reported difficulties in accessing the prescriptions they rely on, and some clinical commissioning groups have been found to actively restrict funding for PKU products. Has the Minister had any conversations with Public Health England and the CCGs to ensure that people with PKU have easy access to prescription-only foods and amino acid supplements?

As a parent who watched her children have the heel prick test as babies, I had no idea how important that test was. I had not heard of PKU back then and I thought the test was just a little test in which they check the hips and prick the heel. I can only imagine how it must feel to be the one out of 10,000 parents who receive a life-changing diagnosis for their child, only to find out that their life and the health of their child will be harder than it needs to be because of what can only be described as rationing by their CCG and NHS England.

Photo of Catherine West Catherine West Labour, Hornsey and Wood Green

Does my hon. Friend the shadow Minister agree with me that it is a disgrace that it takes a court case to get the NHS, NICE and all the other bodies to respond, even when they have heard about the difficult times that families have? Does she agree that a family should not have to take the Government to court to get the treatment that their child needs?

Photo of Sharon Hodgson Sharon Hodgson Shadow Minister (Public Health)

I absolutely agree. It is shocking. I want to end by saying to all the campaigners here and across the country that I hope we have shown in this debate today that we are listening. All their campaigning has not been in vain. It has led to us having an amazing champion in my hon. Friend the Member for Blaydon, and it has led to this well-attended debate today. The Minister has heard all the powerful speeches. He is a compassionate man, so they cannot fail to have had an impact on him. I look forward to his response. This is his opportunity to give hope to thousands of people. Let us hope that he does so.

Photo of Steve Brine Steve Brine The Parliamentary Under-Secretary for Health and Social Care 10:45, 26 June 2018

No pressure, then. I will try to give some hope.

Thank you, Mr Robertson, for chairing our debate. I also thank Liz Twist for introducing the debate with such humanity. She speaks so well and passionately on this subject. We also heard from my hon. Friend Paul Masterton, who mentioned the Irn-Bru issue. The Treasury has a policy on the sugar tax, which is part of our child obesity plan. We published the update on that yesterday. The policy long predates me. This subject has not been raised with me before, but we cannot let the bad be the enemy of the good. Taking sugar out of fizzy drinks is a good thing for society, but the unintended consequences of that need to be addressed, and he is right to raise it.

We also heard from the hon. Gentleman from my own county, Stephen Morgan, from the hon. Members for Dudley North (Ian Austin), for Warrington South (Faisal Rashid), for Hornsey and Wood Green (Catherine West), forDerby North (Chris Williamson), and from my hon. Friend Peter Aldous, who always speaks so passionately, from Liz Saville Roberts, and, as always, Jim Shannon, all of whom—I think everybody—touched on the subject of Kuvan. Many touched on the dietary aspect and everybody gave personal examples of constituents. I hope to address all of those subjects.

I congratulate Liz Twist and the all-party group on the work that they do. When I was a Back Bencher I was involved in many all-party groups, including the APPG on breast cancer with Mrs Hodgson, who speaks for the Opposition. So much of the good work of this place goes on in APPGs. I hope that the public watching inside and outside today can see that.

The House debated PKU and Kuvan in March this year, led by my hon. Friend Vicky Ford, who spoke well again today. I was not able to attend that debate in person back in March, so I am grateful to have the opportunity today to hear the issues around PKU and access to treatments. I have learnt a lot today, as I did in my reading ahead of today. The importance of rare diseases, of which PKU is one, is of course recognised by us and by policy makers and healthcare service providers in the UK and internationally, and rightly so. One in 17 of us will suffer from a rare disease at some point in our lives.

With the number of known rare diseases steadily growing as our diagnostic tools improve, the Government remain focused on and dedicated to improving the lives of those living with a rare condition. That was reinforced in the Prime Minister’s words last Monday at the Royal Free. I was fortunate to be there when she set out a vision for the long-term plan for the NHS, underpinned by increased funding for the service. She said the UK had an opportunity,

“to lead the world in the use of data and technology to prevent illness, not just treat it;
to diagnose conditions before symptoms occur, and to deliver personalised treatment”, informed by our own data, including our genetic make-up. I will say more about that in a moment.

Early and accurate diagnosis of rare conditions is essential for the best outcome for patients with rare diseases such as PKU. We know that without early treatment the outlook for those born with the condition is very poor, as the Scottish National party spokesperson, Martyn Day, and the Opposition spokesperson, the hon. Member for Washington and Sunderland West, said.

With early treatment, however, the outlook can be good, which is why, as a number of speakers have said, screening has such a vital role to play. I have two children, and equally watched the pin heel prick with trepidation—little did I know what it could have found. I had no idea what they were doing—I was in that daze—let alone what it could have found, so I have great compassion for people in that moment.

The current newborn screening programme in the UK is based on the blood spot test—the heel prick test that we have referred to—and screens for nine rare but serious conditions, including PKU. With that early diagnosis, treatment can start straight away. For patients with the condition, that treatment includes a special diet and regular blood tests. We have heard so many incredible examples today.

We have heard how severely limiting a protein-restricted diet is and how difficult it must be for any patient to stick to, but particularly for young children. Those of us with young children can really feel that. Children with PKU, as has been said, cannot eat most of the foods that we all take for granted, such as meats, fish, milk and treats such as chocolate—everything in moderation—and that is just to name a few.

I stand here as a Minister, but also as a constituency MP. I, too, had not heard of PKU until constituents brought the condition to me. I recently met with one of my constituents, Sarah, who was a doctor and, like many people, as we have heard, had to give up her job to look after her children. Her three-year-old daughter, who is a beautiful little girl, lives with the condition. I heard first-hand of much of the daily strain that it puts on her daughter and the family. My constituent, like many carers, cares for the child full time—preparing the meals, calculating ingredients and going to doctor appointments—and has had to give up her career. As the hon. Member for Blaydon said in her introduction, when we say that the condition can be treated by diet it sounds quite easy. However, in an email last night my constituent said to me,

“If she goes off ‘the diet’, she will suffer permanent and irreversible brain damage.”

If my seven-year-old boy goes off diet and drinks a fizzy drink we certainly suffer the consequences, but it usually lasts for only an hour. I have a great understanding from today’s debate about that.

I understand that even in adulthood, as the hon. Member for Dwyfor Meirionnydd said, PKU can cause harrowing symptoms that make any attempt at a normal life and contributing to society very difficult and sometimes impossible. The availability of specially formulated low-protein foods and nutritional supplements through the NHS is therefore vital. Since its development in the ’50s, it has saved the lives of and improved outcomes for many patients.

I cannot deny that PKU is not on the list of medical conditions in England that are exempt from prescription charges. As such, only the usual age-related pre-paid certificate exemptions apply to such patients. That is the current situation, but everything can be challenged and can change. As I said at the start, the power of all-party groups is incredible, and perhaps that is something that the all-party group may wish to look at and campaign on.

An awful lot of information is available. My constituent Sarah is also the editor of the National Society for Phenylketonuria’s magazine. She sent me the summer 2018 edition last night, which I read overnight. It was a really interesting read, and I might touch on a couple of things in it before I close. That magazine and its website contain all sorts of information on foodstuffs, advertisements for foodstuffs, products and recipes—and yes, avocado does keep coming up.

Photo of Steve Brine Steve Brine The Parliamentary Under-Secretary for Health and Social Care

I will briefly, but then that will be it, because I know hon. Members want to hear from me, as the Minister. We have heard from Back Benchers.

Photo of Catherine West Catherine West Labour, Hornsey and Wood Green

As the Minister knows, there have been five applications for an individual funding request. Two of those were allowed and one, which I mentioned earlier in the debate, had to go to the High Court. The judge declared that the decision that had been made was irrational and unlawful. Will the Minister not just speak about the dietary supplements, which we can all find out from Google, but about what he is doing to push these requests? Specifically, what is he doing on behalf of Olivia, aged 11, whose mother is here today, who would like to know whether he will personally support her application for Kuvan?

Photo of Steve Brine Steve Brine The Parliamentary Under-Secretary for Health and Social Care

I was going to come on to talk about Kuvan; obviously, I stopped to listen to the hon. Lady’s intervention. No, I will not personally support an individual request. That would not be appropriate for a Minister at the Dispatch Box. That is not how our system works, but if she wishes to write to me with the specific example then of course I will see that she gets a reply. That should be handled through the right processes. I know that the processes for individual funding request applications are sometimes torturous, and I am sure that we could do them better.

Let us touch on Kuvan, which everybody has raised. It is one treatment option that has been found to lower blood phenylalanine levels in some patients with mild or moderate PKU. We know that the drug is effective in a small number of patients, depending on their genetic make-up, and is more likely to benefit those with milder forms of the condition. If patients respond to treatment, it is likely that they will still need to continue with some form of dietary restrictions—everyone understands that.

As we have heard, Kuvan is not currently routinely commissioned for use in children and adults. That is due to the lack of evidence of its effectiveness on nutritional status and cognitive development at the time the policy was developed in 2015. NHS England does, however, have a commissioning policy for PKU patients with the most urgent clinical need—namely, pregnant women, as we have heard.

Photo of Steve Brine Steve Brine The Parliamentary Under-Secretary for Health and Social Care

No, I will not. Although the decision taken by NHS England was not to commission Kuvan routinely, the system has the flexibility to review that decision if new evidence emerges. As the House heard during the debate in March, NHS England received a preliminary policy proposal for the use of Kuvan in the management of PKU for adults and children, because new evidence has now been published to support its use. Kuvan was subsequently referred to the National Institute for Health and Care Excellence for assessment through its topic selection process—the process through which NICE prioritises topics for appraisal in its technology appraisal or highly specialised technology programme.

The NICE process is important. It is independent of Ministers and provides a standardised, governed procedure to ensure that NICE addresses topics that are important to the patient population, carers, professionals and commissioners and, similarly, helps to make the best use of NHS resources. To update the House on progress, Kuvan has progressed through the first stage of the topic selection, and NICE is currently considering whether the drug should proceed to the draft scope creation stage. We are expecting that decision to be taken in the autumn. I will press NICE, along with the relevant Minister in the Department—the Under-Secretary who sits in the other place—to bring that to a conclusion as swiftly as possible.

People have asked today for me to personally get involved in access to Kuvan. NICE’s process is important and sits independently of Ministers. It would be a very strange situation if Ministers were able to sit in the Department of Health and, like a Roman emperor, give a thumbs up or thumbs down. I do not think that any Minister in this Government or previous Governments would want to be in that inappropriate position. As I said, we expect the decision to be taken in the autumn and we will press for that to be brought to a conclusion as soon as possible.

I will give the hon. Member for Blaydon time to wind up the debate, but there are other promising treatments on the horizon. NICE is currently considering pegvaliase, an enzyme substitution therapy indicated for adults, through its topic selection process, and recently consulted stakeholders on its suitability for the technology appraisal. I can update the House that a scoping workshop on this topic is scheduled to take place tomorrow, 27 June.

Finally, the hon. Member for Chelmsford said that there had not been a response on BioMarin. She mentioned that point to me last night, and I am worried to hear it. As I said, Kuvan is currently going through the independent NICE assessment. If the topic goes ahead, there will be many opportunities for BioMarin to engage in commercial discussions, as per NICE’s usual process. BioMarin and NHS England are already in discussions about a number of other drugs, so it has the opportunity to raise the issue. However, it seems to me that NHS England could at least communicate better, because no answer sounds like a bad answer. I will take that away from the debate and ensure that it happens ASAP.

I know you want me to stop, Mr Robertson, and let the hon. Member for Blaydon close the debate, so I will do that.

Photo of Liz Twist Liz Twist Labour, Blaydon 10:58, 26 June 2018

What can I say in less than one and a half minutes? I thank all hon. Members who have taken part today. I thank the hon. Members for East Renfrewshire (Paul Masterton), my hon. Friend Stephen Morgan, Peter Aldous, my hon. Friend Ian Austin, the hon. Members for Chelmsford (Vicky Ford), for Dwyfor Meirionnydd (Liz Saville Roberts) and for Strangford (Jim Shannon), my hon. Friends the Members for Warrington South (Faisal Rashid), for Hornsey and Wood Green (Catherine West) and for Derby North (Chris Williamson), Martyn Day, my hon. Friend Mrs Hodgson and the Minister for his reply.

I am rather disappointed that we did not have a bit more promising news on the future of Kuvan, but we will be back; we will ensure that the Minister hears from us again, and we will continue our campaign. Finally, I thank all the families who are here today to show how strongly people feel about the issue. I thank everyone for attending. The issue will not go away. We will be back and will keep pushing this agenda.

Question put and agreed to.


That this House
has considered access to treatment, support and innovative new medicines for phenylketonuria patients.

Photo of Laurence Robertson Laurence Robertson Conservative, Tewkesbury

Would hon. Members leaving please do so quietly? Thank you. We have another debate.