Phenylketonuria: Treatment and Support

Steve Brine Excerpts

Tuesday 26th June 2018 Westminster Hall

[The Parliamentary Under-Secretary of State for Health and Social Care (Steve Brine)]

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

Thank you, Mr Robertson, for chairing our debate. I also thank the hon. Member for Blaydon (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 the Member for East Renfrewshire (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, the hon. Member for Portsmouth South (Stephen Morgan), from the hon. Members for Dudley North (Ian Austin), for Warrington South (Faisal Rashid), for Hornsey and Wood Green (Catherine West), for Derby North (Chris Williamson), and from my hon. Friend the Member for Waveney (Peter Aldous), who always speaks so passionately, from the hon. Member for Dwyfor Meirionnydd (Liz Saville Roberts), and, as always, the hon. Member for Strangford (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 the hon. Member for Blaydon 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 the hon. Member for Washington and Sunderland (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 the Member for Chelmsford (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, the hon. Member for Linlithgow and East Falkirk (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.

[Catherine West]

Will the Minister give way?

--- Later in debate ---

[Steve Brine]

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.

[Catherine West]

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?

[Steve Brine]

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.

[Vicky Ford]

Will the Minister give way?

[Steve Brine]

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 let me say first that 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, my hon. Friend the 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.