Phenylketonuria: Treatment and Support

Vicky Ford Excerpts

Tuesday 26th June 2018 Westminster Hall

[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.

--- Later in debate ---

[Vicky Ford (Chelmsford) (Con)]

Thank you, Mr Robertson, for the opportunity to speak in this important debate. I add my congratulations to the hon. Member for Blaydon (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 the Member for Thurrock (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.

--- Later in debate ---

[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.