NICE Appraisals: Rare Diseases Treatments

Kerry McCarthy Excerpts

Thursday 21st March 2019 Commons Chamber

[Kerry McCarthy (Bristol East) (Lab)]

I will be brief because I have spoken on this topic many times, specifically about cystic fibrosis, but before I get on to that I congratulate my hon. Friend the Member for Blaydon (Liz Twist) on her speech about PKU. I popped into the PKU drop-in event a few months ago purely by accident when I was doing one of those classic MP things where we are asked to go here, there and everywhere to be photographed holding up a sign or wearing a silly hat. I knew nothing about PKU, but I talked to an 11-year-old boy there about his diet. I am somebody who chooses to have a fairly restrictive diet in that I do not eat meat, dairy or fish, but I look for protein alternatives, and I was struck by the sheer difficulty that the boy has in functioning and by the fact that he is unable to enjoy the normal life of an 11-year-old. My heart went out to him, and I do not need to add any more, because my hon. Friend made such an excellent speech.

We have also had excellent speeches about the difficulties of obtaining Spinraza. I do not claim to be an expert, as some other Members are, but constituents have been in touch with me with similar problems. I would therefore support any efforts to get the drug approved, because that would make a huge difference to their lives.

As I said, I am here to talk about cystic fibrosis, and I have a personal interest in that my 14-year-old niece Maisie has CF. She must be the most mentioned niece in Parliament, because she gets name-checked quite a lot. Before I talk about Maisie, I want to mention the recent evidence to the Health and Social Care Committee from Oli Rayner, who lives with cystic fibrosis. He has just turned 40 and had a lung transport around 18 months ago, so he is at the healthier end of the spectrum, and he talked about being quite lucky until he reached his thirties, when his health started to deteriorate.

However, many cystic fibrosis patients are not as lucky. My constituent Lee Partridge lost both his daughters within eight months when they were both in their late teens, and it is common for people to start to deteriorate when they reach their teens. Luckily, my niece is very much at the healthier end of the spectrum, but that does not influence my judgment; I want to make the case for Orkambi to be available not just for her when it is needed, but for everybody else with the condition.

I have been trying to get my head around quality-adjusted life years, how the calculations are made, whether NHS England is doing the right thing and whether NICE is using the right sorts of calculations compared with other countries. NICE says the calculations factor in societal benefits, but it is not clear how they do that.

The hon. Member for Reigate (Crispin Blunt), who made an excellent speech, spoke about the cost of hospital treatment to society as a whole. If a person has to go into hospital several times a year, there is the cost of care, but there is also the cost of lost work time, the cost of care by family members and even the cost of transportation to hospital if they live in a rural area. There are so many factors. Although NICE says it takes those things into account, I cannot quite see them. Was it Hugh Dalton or Clement Attlee who was called a “desiccated calculating machine”? Anyway, the system seems to be based on desiccated calculations.

It may be incredibly naive of me, but why cannot the system just think of the impact on people’s lives and of the hell they are going through? It is incredibly difficult for teenagers to come to terms with discovering that they have a life-limiting condition, and there is also the issue of the availability of mental health support and counselling.

It is striking that everyone who gave evidence to the Health and Social Care Committee said that the drugs work. There is no question but that this would be of benefit to people. Kalydeco, the first drug, helped only 5% of patients, and it was funded in part because not many people would benefit and, therefore, the costs were lower. We now have Orkambi, and there will soon be a successor that will benefit about 40% of patients. For the future we are looking at triple therapies that will cover about 90% of the cystic fibrosis community. If 90% of people can benefit, of course the overall cost will go up, but the decision should not be made on the basis that we fund one thing because not many people will benefit.

I am concerned that Vertex and other companies would be deterred from exploring further therapies and treatments if they cannot get a commercial deal. The line that jumps out of the Health and Social Care Committee’s letter to Vertex, which it wrote shortly after the meeting, is:

“Vertex appears to have decided on the pricing of its therapies on the basis of the return it wants to make, rather than the value which they bring. NHS England is right to continue to take the wider patient population for whom it is responsible into account.”

The Minister has previously responded to such debates, so can we have an update on where the conversations have got with Vertex, including the Secretary of State’s meeting? There are so many cystic fibrosis patients waiting to hear the outcome. Last time I looked, the petition was up to about 80,000 signatures. Can we try to get some answers soon?

--- Later in debate ---

[Ian Austin (Dudley North) (Ind)]

I thank the hon. Members for Blaydon (Liz Twist) and for North Tyneside (Mary Glindon), and the Backbench Business Committee, for arranging this debate. I am here to speak up for people in Dudley with cystic fibrosis, phenylketonuria—or PKU—and spinal muscular atrophy.

I have been campaigning with others to make Orkambi and other new treatments available for people with cystic fibrosis because I was contacted by Carly Jeavons from Dudley, who took part in the clinical trial for Orkambi, and by Samantha Carrier, whose baby daughter Daisy was diagnosed shortly after birth. Samantha has given up her career and now devotes her life to campaigning for access for these life-changing drugs. While listening to this debate, I have been receiving emails, texts and other messages from people around the country—heroic parents of children with cystic fibrosis who work so hard and campaign tirelessly for the treatments that their children need. I know that the Minister has met some of these parents, and I hope he will have some new answers for them today, because they have worked so hard to raise these issues, which really do need to be sorted out.

Before the clinical trial, Carly Jeavons had to take 90 tablets and do two hours of physiotherapy a day. She had a lung function of around 44% and spent two weeks in hospital every three months. She had to choose between the financial hardship of leaving work or her health being made worse as she struggled on at work. She told me:

“Orkambi has changed my life. My health has remained stable. I only need one or two courses of IV’s per year instead of the four previously, hospital visits have been massively reduced and admissions are non-existent.”

Since having Orkambi, she has been able to go on holiday abroad for the first time with her family, and she has got married. She has also started a business and is employing people, so she is making a much bigger contribution to the economy. The Government need to look at the contribution that people who get these drugs can make to the economy, not just at the costs of providing the drugs. I believe that NHS England and NICE are with Vertex this afternoon for yet another meeting about whether these cystic fibrosis drugs can be provided. But this comes three years after NICE appraised Orkambi. I really hope that the Minister will explain how this situation can be resolved and how other situations like it can be avoided, so that patients can get can get access to these drugs.

Let me turn to the issue of treatments for people with PKU. Some of the people who have been leading this campaign are sitting in the Public Gallery, watching this debate. Again, I hope that the Minister will have some good news for them today. As we have heard, PKU is a rare metabolic disease that leaves people unable to metabolise protein properly, leading to a toxic build-up of material that can cause irreversible brain damage. The only existing treatment is a strict diet of extremely low protein, meaning that almost all normal foods are off limits. The diet is lifelong, and sufferers find it stressful and difficult to cope with. I had never heard about this condition until a woman in Dudley called Kirsty Thornton got in touch with me. Since then, I have met the campaigners and taken part in a PKU diet challenge. I have also joined the cross-party parliamentary campaign led by the hon. Member for Blaydon to ensure that people with PKU get access to the treatments and supplements they need.

It is heartbreaking for parents of young kids with PKU who do not understand why they cannot go to their friends’ birthday parties in case they eat the wrong foods that will make them tired, sick and ill for the rest of the day, or for longer. Students say how difficult it is when their friends are going on nights out, or they move to university and the people they share a flat with are ordering in pizzas and so on, or they cannot go out on a date because they do not know what they are going to be able to eat or not eat. This must be really tough for young people.

[Kerry McCarthy]

My hon. Friend will probably be aware that the Government are looking at a new food strategy, and one of the things under consideration is better food labelling. A lot of people think that those who make certain dietary choices do it almost to be trendy, or just because it is the fashionable thing to do, but there are people whose lives are at stake if they cannot get the information on food that they need. Food manufacturers and anyone else involved in the provision of food need to be alert to the fact that it is important that people know what they are going to be eating.

[Ian Austin]

That is a really important point. The PKU campaigners have explained to me that on some occasions when manufacturers have changed the ingredients in food or drinks, that has had a terrible impact because people with PKU have not necessarily known about it, so they have continued to drink or eat things that they have consumed without any problems in the past, but because the ingredients have changed, it has caused them a big problem.

For many people with PKU, taking Kuvan considerably increases the amount of protein that they can safely eat. We are therefore urging the Government, the NHS and BioMarin, which manufactures these supplements, to agree a deal so that people with PKU can enjoy a normal healthy life. I spoke to the National Society for Phenylketonuria this morning, and it told me that the whole PKU community are demoralised. They say that they are working hard but feel that not much progress is being made. What is the Minister going to do today to give these people, some of whom are in the Public Gallery, and others who are sitting at home with their kids watching this debate, to give them hope of this situation being resolved?

My next point is about Spinraza treatment for people with spinal muscular atrophy, or SMA, which affects an estimated 1,300 people across the UK. It can cause irreversible loss of a child’s ability to crawl, walk, breathe and swallow. In the most severe cases, it can cause death. Spinraza is the first possible treatment for those who have SMA types 1, 2 and 3. It can slow its progression and prolong life. From April, this treatment is going to be routinely available in Scotland, and it is already available in 24 other countries in Europe, yet it is still stuck in the NICE process for England, Wales and Northern Ireland.

That is why, in the end, the purpose of this debate is to ask the Government to look carefully at the way that NICE works. New drugs are being developed, and technological changes are happening, so rapidly that the Minister needs to be able to tell us how the way drugs are assessed and licensed, and then approved, will work in future. How is he going to ensure that these ground-breaking drugs are made available to the people who need them, when they need them? In 2016, NICE was not able to recommend the use of Orkambi because of uncertainty around its long-term value, impact and cost-effectiveness. But this drug is available in the USA, across Europe, and, more recently, in Scotland, so when do the Government think patients in England are going to get it? This is really urgent. The system has not worked and patients are being let down.

On PKU, NICE decided to start an appraisal of Kuvan in 2018, but this has since been suspended. NICE is currently reconsidering which appraisal process to use to access Kuvan, and the NHS is considering whether to fund an interim policy for the use of the drug. But, again, this is not enough, and not quick enough. The NICE process sees PKU as rare, but not rare enough. As we have heard, the majority of PKU treatments are assessed by the STA process, which is designed for non-rare treatments. NICE’s approach evaluates the lifelong costs of treatment, meaning that the cost thresholds and the approach to evidence are all designed for more common diseases than PKU. SMA sufferers are waiting for NICE, NHS England and Biogen, which manufactures Spinraza, to come to a deal. I hope we will hear better news on all those things from the Government than we have in the past.

It is my job to listen to people in Dudley who are living with cystic fibrosis, PKU, SMA and other rare diseases, to come down to London and speak up for them in Parliament and to demand, as we are doing this afternoon, that the Government ensure that they get access to the treatment they need and deserve.