NICE Appraisals: Rare Diseases Treatments

Debate between Liz Twist and James Morris

Thursday 21st March 2019 Commons Chamber

[Liz Twist (Blaydon) (Lab)]

I beg to move,

That this House believes that NICE appraisal processes do not properly address the medical treatment needs of people with rare diseases such as muscular dystrophy, phenylketonuria and cystic fibrosis; and calls on NICE to urgently review the appraisal process.

I thank my hon. Friend the Member for North Tyneside (Mary Glindon), who is chair of the all-party group on muscular dystrophy, and the hon. Member for Strangford (Jim Shannon), who is chair of the all-party group on cystic fibrosis, for their help in securing this debate.

My constituent Archie McGovern is 12 years old. He is bright and lively, and full of beans now, but it has not always been like that way because Archie has PKU— phenylketonuria. Putting it simply, PKU is a genetic condition that means Archie and others are unable to handle phenylalanine, which is found in protein—so no meat, no fish and no dairy products. There is a whole range of other things that we would not think had protein in them: the list is endless. On top of that, he has to take a protein substitute drink—if we can call it that, as it is very unpleasant—to keep the balance right.

At present PKU is not curable, and a hugely restrictive diet is the only way of controlling the condition throughout childhood and adult life. The condition is picked up by the pinprick test at birth, and for those identified as having PKU that is the start of a difficult lifetime of dietary control. For children that is especially difficult, but it is also very important because failure to control the condition can lead to serious neurological problems.

That is how it was for Archie until quite recently, but there is a treatment that can help to control PKU. It is called Kuvan, and although it was licensed 10 years ago and is widely available in many countries in Europe, and further afield, it is not available to patients in the UK. Not everyone with PKU responds to Kuvan, but it is believed that more than 20% of people will respond well and see a significant improvement in their life.

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

The hon. Lady makes an important point about Kuvan and PKU. As she will know, last year I and other Members—including the hon. Member for Dudley North (Ian Austin)—took part in the MPs’ PKU diet challenge, so I am well aware of the restricted diet that is required for those who suffer from that condition. The way that NICE evaluates Kuvan does not take into account the social costs associated with the alternative treatment for PKU. That highly restrictive diet impacts on families and wider society, and NICE should consider that point.

[Liz Twist]

Like the hon. Gentleman, I took part in the PKU diet for a day challenge. It was indeed very restrictive, even though I knew it was only for one day.

Archie is one of those who responds well to Kuvan, and last year, his parents took the difficult decision to pay to buy him the drug. That took a great deal of soul searching on their behalf, and it cost them dear—£25,000 a year, even though Archie is currently on just half a dose. They know that for many people with PKU, or for parents with more than one child who has PKU, it is simply not possible to self-fund, and they are acutely conscious of the unfairness of that. The difference that Kuvan has made to Archie is real and significant: increased concentration and energy, so that he can make the best of his education; no recurring mouth ulcers, which were a real problem; and a chance substantially to increase the number of exchanges he can have, and eat a more normal diet. For Archie, Kuvan has made a real difference.

Archie’s case, and those of many others in other constituencies, prompted us to form the all-party group on phenylketonuria, and to consider how Kuvan could be made available to those who would benefit from it. Nearly 10 years after Kuvan was approved, that treatment does not seem to have been an appraised, and in England it is still not available on the NHS. Since we set up the group, there has been a move for Kuvan to be appraised by NICE, and discussions have been held with NHS England about a managed access agreement. We were disappointed to learn just before Christmas that no agreement had been reached on that managed access agreement, and that the NICE appraisal was to be via the single technology appraisal route, and not the highly specialised technologies programme. I understand that following a legal challenge, the Department of Health and Social Care is again considering the appropriate appraisal route, and the all-party group has made representations on that point.

When talking about the NICE appraisal system it is easy to get lost in technical details—QALYs, and everything else, that means nothing to people on the street—but what really concerns people is whether or not there is a fair chance that the drugs they need will be fairly assessed and made available on the NHS.

Phenylketonuria: Treatment and Support

Debate between Liz Twist and James Morris

Tuesday 26th June 2018 Westminster Hall

[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.