Phenylketonuria: Treatment and Support

Debate between Liz Twist and Vicky Ford

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.