Phenylketonuria: Treatment and Support

Liz Twist Excerpts

Tuesday 26th June 2018 Westminster Hall

[Mr Laurence Robertson (in the Chair)]

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

[Liz Twist (Blaydon) (Lab)]

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.

[Ian Austin (Dudley North) (Lab)]

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.

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[Liz Twist]

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.

[James Morris (Halesowen and Rowley Regis) (Con)]

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?

[Liz Twist]

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

[Catherine McKinnell (Newcastle upon Tyne North) (Lab)]

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.

[Liz Twist]

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 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.

[Neil Gray (Airdrie and Shotts) (SNP)]

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?

[Liz Twist]

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.

[Mr Jim Cunningham (Coventry South) (Lab)]

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?

[Liz Twist]

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.

[Tim Farron (Westmorland and Lonsdale) (LD)]

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?

[Liz Twist]

I absolutely agree. I hope that one of the good things that will come out of this debate is 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.

[Vicky Ford (Chelmsford) (Con)]

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?

[Liz Twist]

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.

[Mr George Howarth (Knowsley) (Lab)]

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?

[Liz Twist]

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

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

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

[Nigel Mills (Amber Valley) (Con)]

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

[Liz Twist]

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

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

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

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

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[Liz Twist]

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 the Member for Portsmouth South (Stephen Morgan), the hon. Member for Waveney (Peter Aldous), my hon. Friend the Member for Dudley North (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), the hon. Member for Linlithgow and East Falkirk (Martyn Day), my hon. Friend the Member for Washington and Sunderland West (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.

Resolved,

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

[Mr Laurence Robertson (in the Chair)]

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