Orkambi and Cystic Fibrosis

Paul Scully Excerpts

Monday 19th March 2018 Westminster Hall

[Paul Scully (Sutton and Cheam) (Con)]

I beg to move,

That this House has considered e-petition 209455 relating to access to the drug Orkambi for people with Cystic Fibrosis.

I will first read the petition into the record, if I may. It is entitled “Make Orkambi available on the NHS for people with Cystic Fibrosis”, and states:

“Conventional CF treatments target the symptoms of CF, precision medicines like Orkambi tackle the cause of the condition. Though Orkambi is not a cure, it has been found to slow decline in lung function, the most common cause of death for people with CF, by 42%.

In July 2016, the National Institute of Clinical Excellence (NICE) recognised Orkambi as an ‘important treatment.’ They were, however, unable to recommend the drug for use within the NHS on grounds of cost effectiveness and a lack of long-term data.

We are calling on the British Government to call for a resolution to ongoing negotiations between Vertex Pharmaceuticals, NHS England and NICE as a matter of the utmost urgency. It is essential that a fair and sustainable agreement is found.”

To date, there are 115,419 signatories to the petition.

It is a pleasure to serve under your chairmanship, Mr Evans. This is my first debate here—hon. Members have not come to see me, but to support this important cause. It is incredible to see so many people, and to see these Benches and the Public Gallery full. I thank my hon. Friends and colleagues for supporting this noble cause.

[Ian Paisley (North Antrim) (DUP)]

Does the hon. Gentleman agree that the reason why there is such public and political interest in this matter is that the Government have acted in a prehistoric way when it comes to treating patients? This drug should be purchased and made available as widely as possible and as soon as possible, so that patients who suffer from this disease can have a better quality of life.

[Paul Scully]

I understand the hon. Gentleman’s position and concern, especially so when many people in Ireland in particular are cystic fibrosis sufferers, but I remind him that NHS England is the procurer, not the Government—it is done through that independent process.

[Mr Charles Walker (Broxbourne) (Con)]

I discussed this with my hon. Friend the Member for Meon Valley (George Hollingbery), who is in the Chamber. We decided that it is essential for Vertex and NHS England to be reasonable. There is common ground and a deal to be done. Both sides need to work towards that with the encouragement of the Minister and the Department of Health and Social Care.

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[Paul Scully]

I totally agree. When I say that the Government are not the purchaser of the drug, it does not mean that they are without responsibility. The Government have a position, which I will flesh out in a short time, but it is important that they play the role of middleman to encourage negotiations between Vertex and NHS England to ensure that sufferers can get the cutting-edge treatment they deserve.

[Mr Jim Cunningham (Coventry South) (Lab)]

We are told the drug is not economical, but has anybody done the figures on that, given the seriousness of the disability? I wonder whether the hon. Gentleman or more probably the Minister have any figures. People cannot take things out of thin air and say, “It is not economical,” without letting us know the figures.

[Paul Scully]

I clearly do not have the figures that may have been done on the economics, but I know there is a test, which I will talk about later, in which the treatment falls between two stools. In effect, the system does not suit this kind of condition. It suits acute conditions far better—I will come to that shortly.

[Alex Burghart (Brentwood and Ongar) (Con)]

My hon. Friend is being extremely generous with his time. Is it the case that this treatment might prevent certain conditions from escalating, and so prevent people from having to move on subsequently to more expensive treatments?

[Paul Scully]

It is exactly the case—there is no doubt about that. There are many people in the Public Gallery whose children are sufferers from cystic fibrosis. When I was at the Cystic Fibrosis Trust last week, I could not meet many sufferers, because they cannot be in the same room due to the risk of infection. As my hon. Friend says, cystic fibrosis can escalate quickly, and the more we can treat the underlying causes, which Orkambi and subsequent drugs will allow us to do, the better.

[John Spellar (Warley) (Lab)]

Can we not see, during the week in which Stephen Hawking died in his 70s from a condition that was anticipated to kill him in his 20s or early 30s, that in fact conditions can allow for a much longer life? Is it not therefore incumbent on the Government to put pressure on both NHS England and the manufacturer to get an acceptable solution that will enable younger patients in particular to have the same opportunity that Stephen Hawking had?

[Paul Scully]

That is very much the point. Half the people who die of cystic fibrosis are under the age of 31. The average life expectancy for someone born now with cystic fibrosis is 47, so we see how it is starting to extend. When I was at the Cystic Fibrosis Trust just last week, I thought that the two people I was able to meet were just staff of the trust and did not realise they were sufferers. Why was I able to meet them, and why did I not notice? Because they had had lung transplants. In effect, they had pressed the reset button on their suffering. They had fresh pairs of lungs and Orkambi would not necessarily be right or necessary for them. The right hon. Gentleman is absolutely right in identifying that it is for the next generation coming through.

[Sir Greg Knight (East Yorkshire) (Con)]

In view of the national importance of this matter, would one way forward be for Vertex, the drug manufacturer, to be willing to be more flexible on costings and price?

--- Later in debate ---

[Paul Scully]

We are effectively in a position of negotiation with Vertex. When the petition was originally started, the issue was, “Here is Orkambi; let’s set a price and buy it.” It was a very high price, which is why the National Institute for Health and Care Excellence made its decision. We have now moved on to a portfolio system that Vertex is proposing. Vertex has a drug that has been available here for a little while, Kalydeco. Orkambi is available in Ireland, the Netherlands and other countries, but not here—and even then, it would not be right for every mutation of cystic fibrosis. Symdeko is coming out in the future, and there is a pathway of drugs that will have ever-increasing effectiveness on a wider group of people.

Vertex has proposed a system, which has been knocked back by NHS England, but I hope both sides can come to a reasonable conclusion as a result of this debate and as a result of pressure. The important thing is to get access for those people who are suffering and whose lung capacity can reduce so quickly at any moment.

[Sir Edward Davey (Kingston and Surbiton) (LD)]

The hon. Gentleman is making a powerful case. Is the problem that the current system of NICE analysis, using its quality-adjusted life year—QALY—measures, is unable to take account of negotiated discounts through pipeline agreements such as the one he has outlined? Is it time that this House and the Government made it clear that that needs to be taken account of, and that NHS England and Vertex need to get together and show that the cost is good value for money when we take account of pipeline deals and negotiated discounts?

[Paul Scully]

The right hon. Gentleman’s contribution comes to the nub of the issue. I will scrap most of my speech, which I had not written anyway, and dash around. There are two issues. We can talk about the Government getting involved in pushing NHS England and Vertex together to make a sensible deal in this case, but I come back to the point that this is an analogue system in a digital age. I will try to do it justice, but it is a case of inequality. About 400 patients have access to Kalydeco, which I mentioned earlier. They have a particular mutation to which Kalydeco responds. Around 3,000 patients would be eligible to access Orkambi. However, the point about cystic fibrosis is that, because it is a genetic disease, it cannot be caught, so we know pretty well the number of people we will need to treat over the next few years. There are around 10,400 sufferers in this country, which is extraordinary, because there are only 70,000 sufferers around the world.

Cystic fibrosis is a pernicious disease, and I have not even started talking about its effect on the children of the people I have met. However, it is not big enough to require an international epidemic-style solution, and it is not small enough to be a rare disease. It fits somewhere in the middle.

[Bob Stewart (Beckenham) (Con)]

Nobody can catch cystic fibrosis, but as my hon. Friend mentioned, two sufferers in the same room together can really affect one another. The bugs from their lungs can transfer, presumably through the air.

[Paul Scully]

Absolutely. A lot of the people I have met have aspergillus, which is a mould-based infection. It is around in the air in many homes, which for us does not matter, but for sufferers can cause a severe reaction and a severe loss of lung capacity.

[Alan Mak (Havant) (Con)]

My constituency, and that of my right hon. Friend the Member for Portsmouth North (Penny Mordaunt), were among the top three constituencies for people signing the petition. Will my hon. Friend join me in calling on the Minister to get Vertex, the NHS and NICE to continue their negotiations, because time is of the essence?

[Paul Scully]

My hon. Friend is absolutely right. It is important, because every day counts for people who have the condition. I said that cystic fibrosis cannot be caught, but neither can it be cured. The people who have cystic fibrosis now will be the same people who have it for the rest of their lives, which basically means that the supply of Orkambi would have a fixed price. We know pretty well, within a margin, how much we would spend on this lifelong treatment.

The current evaluation process turns on an incremental cost-efficiency ratio, which is the total additional lifetime cost of a treatment divided by the additional quality-adjusted life years resulting from that treatment. For acute conditions, the additional quality-adjusted life years resulting from the shorter-term treatments moderate the efficiency ratio, even if the drug is expensive. However, because drugs for chronic and lifelong conditions have to be taken every day for life, the cost of the treatment prevents that downward moderation. Basically, it is easier, under the NICE appraisal system, for medicines for acute conditions to attain a more favourable cost-effectiveness outcome than for innovative medications for chronic conditions, like Orkambi. It is basically a one-size system.

We then have to take into account section 13G of the National Health Service Act 2006, as amended by the Health and Social Care Act 2012, which requires NHS England to have regard to the need to reduce inequalities in health outcomes. Those two imbalances in the system need to be looked at if we are to have a system that is far fairer for people with illnesses such as cystic fibrosis.

[Douglas Ross (Moray) (Con)]

My hon. Friend’s excellent speech rightly focuses on NHS England, but does he agree that there has been a great campaign to get Members from across the country to come to the debate? Some 74 of my constituents signed the petition, because cystic fibrosis sufferers across the UK want action.

[Paul Scully]

I agree. It was a fantastic effort from—it was a remarkable achievement—the petitioners to get 114,000 signatures within 10 days. I have been on the Petitions Committee since its start and, short of having a go at Donald Trump and a few of the Brexit debates, this is one of the most potent petitions, and one of the more productive.

[Alex Chalk (Cheltenham) (Con)]

Does my hon. Friend agree that drugs companies need to behave ethically in pricing their vital medicines? While profit itself is not a dirty word, when it comes to cystic fibrosis treatment, profiteering is.

[Paul Scully]

One thing I will say in favour of Vertex in this case is that, although the amount of people who suffer from this condition is fortunately restricted, the research and development still has to go in. It is not like developing the next ibuprofen or cancer drug, which will go out to millions of people; this would go out to 70,000 people in the world. In order to build that research and development budget in, Vertex needs to charge reasonable costs for the drug. None the less, those costs do need to be reasonable. That is why the negotiation needs to be absolutely above board, transparent and sensible for the sake of the sufferers, who, frankly, do not have time to wait for a prolonged negotiation.

[Paul Girvan (South Antrim) (DUP)]

So much emphasis is put on the cost of treatment, yet if we add together the costs of the cocktail of drugs that many sufferers are currently on, there probably would not be a big difference from the cost of the new drug.

[Paul Scully]

I will come back to that important point in just one second. We were talking about the campaign being brought together, and I want to pay tribute to the hon. Member for Dudley North (Ian Austin), who has done a fantastic job in raising the profile of cystic fibrosis. I am sorry that I was not able to be at the roundtable that he hosted—I was out of the country—but 41 Members were there who were keen to learn more about this. That is because of his efforts and the efforts of the petitioners, and that is absolutely to be welcomed.

[Ian Austin (Dudley North) (Lab)]

That is very generous of the hon. Gentleman, and I am grateful, but the credit is due not to me but to the fantastic campaigning of the Cystic Fibrosis Trust, the army of people around the country who have this condition and their families. Let us be honest: that is why there are so many Members in the Chamber today, just as there were at the roundtable. However, I am grateful for what he says.

[Paul Scully]

Take it while you can—I am joking, of course.

We have talked a little about cystic fibrosis, and we are all here because we know what it is, so I will not talk about that. I know that lots of Members want to speak, so I will finish with the little hint that I have seen of what it must be like to live with cystic fibrosis. It goes to the point about medicines from the hon. Member for South Antrim (Paul Girvan). I saw a fantastic video—it is private at the moment but I hope the Millers will make it public—of Grace Miller, who is 15 and likes to run. What many people do not understand about cystic fibrosis is that many people have to do physio. They actually do more training than a lot of professional athletes. These kids, who may be aged three or four, have to get up and use a nebuliser for an hour or so. I met a mother who talked about her three-year-old daughter who would use a nebuliser with salty water. It would make her cough and make her sick, and she would then have to do her physio. She would have to run on the spot and run around the house for a while, and then she would have to go and eat.

However, cystic fibrosis also suppresses nutrient intake, so sufferers have to eat far more than normal people—50% more in many cases—with some people taking up 3,000 calories or maybe more each day. It is therefore no surprise that sufferers often have a really unhealthy relationship with food. They just do not want to eat. We talked about one girl who filled her pockets with coins because she did not want to go to school and be underweight. That relationship with food is relatively minor in some ways, compared with the shortage of breath and the actual illness. We have not even started to talk about the underlying illness that cystic fibrosis causes.

[Liz Kendall (Leicester West) (Lab)]

The hon. Gentleman is being very generous with his time. One hundred and eighteen of my constituents signed the petition, so I am very grateful for the debate. He speaks with great passion about the huge challenges facing people living with cystic fibrosis. I agree that NHS England and the drug company need to get together to resolve this issue as quickly as possible. However, does he agree that the bigger issue is about ensuring that NICE has the proper processes in place to make sure that new drugs are agreed as quickly as possible? Does he also agree that the Government should review that, to make sure, not only with this issue but with others, that people have confidence in the process and that that follows the right principles and guidelines?

[Paul Scully]

I thank the hon. Lady for that intervention, and I totally agree with her.

I will finish on the issue of living with cystic fibrosis by saying that in addition to having physio and nebulisers, sufferers often have to take a huge cocktail of drugs, as the hon. Member for South Antrim said. The cupboard that I saw for Grace was bigger than one of my kitchen cupboards and it was just full of medicines. There have to be savings in that respect. To return to the hon. Lady’s point, it is really important that we have a system that is fit for purpose. We can sit there and put sticking plasters, as it were, on all these things, but that is what we need. When we are talking about the health service as a whole, it is not always about the big headlines that we argue about across the Chamber. It is about efficiency and ensuring that things work in the modern age. For modern diseases, there need to be modern treatments and so on.

[Hugh Gaffney (Coatbridge, Chryston and Bellshill) (Lab)]

The hon. Gentleman is making a great speech. Before he finishes, I would like to say that I have had more than 250 emails on this issue alone from my small constituency. The point I want to make to the Minister is that we have a full house in this Chamber and a full Public Gallery. I want this noted. The Government have to act on this issue—not just talk about it, but act on it. Let us take some action.

[Paul Scully]

I thank the hon. Gentleman for that intervention. I will give way one more time and then conclude.

[Matt Western (Warwick and Leamington) (Lab)]

The hon. Gentleman has been very generous with his time. What struck me most—I am sure that this was the case for many people in the Chamber—was just how many countries, particularly in Europe, have agreed and licensed this product. Therefore the cost-benefit analysis has been done, and therefore it is now incumbent on the Government—the Secretary of State—and the NHS to sit around the table with Vertex and discuss how we can deliver it as soon as possible.

[Paul Scully]

I thank the hon. Gentleman for that intervention. Let me be the first to mention Brexit. This is not quite as random as it might seem, because I met someone last week who lives close to the Irish border. Of course, that means that his daughter cannot access Orkambi, but if they were only a few miles across the border, in the Republic of Ireland, she would have access to it. We have to address that inequality, and that will be done, as I keep repeating, by two things. If we can ensure that we use any of our good offices to get NHS England and Vertex to come to a sensible agreement quickly, we can get the portfolio process started now. But I also ask the Minister to see what more he can do to adapt the system long term, so that it is fit for purpose for those modern-style, portfolio-type deals that are done elsewhere.

I keep talking about the urgency of this issue. We were taken to the Cystic Fibrosis Trust by someone who works here, one of the parliamentary outreach team. Alasdair lost his partner. Her lung capacity had dropped to 20% before she died. Some of the people we met had seen their lung capacity drop from 90% to 15% within two or three months. That is why things such as infections are so dangerous. It does not take a lot. Something that we would just shake off can be, for these people, literally a matter of life or death, or it can be a matter of being able to get a lung transplant somewhere down the line or not. That is why there is this sense of urgency. They have been described as a limited number of people, but none the less they are crucial, important people who, if we give them the treatment that they deserve, can go beyond the 31 or 47 years and live a full life, as we would expect to do. With that, I will conclude and let others speak.

[Several hon. Members rose—]

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[Luke Hall (Thornbury and Yate) (Con)]

I am delighted to have a chance to speak in this debate, Sir Roger. I thank my hon. Friend the Member for Sutton and Cheam (Paul Scully) for securing it. When it was initially scheduled by the Petitions Committee, they probably had no idea about how timely it would be three days after the announcement that a deal has not been reached. I thank my hon. Friend for his work. I know he has worked with the CF Trust to learn more about cystic fibrosis. I also thank the CF Trust for all the work it has done not only in setting up the roundtable and for spending time with me speaking about CF, but for what it has done to educate all Members.

[Paul Scully]

Because of the nature of the disease and the fact that there are only 70,000 sufferers around the world, the CF Trust has 99% of the entire clinical diagnostic information of sufferers around the world, which is a good resource to be able to feed back into future treatments. Will my hon. Friend pay tribute to that?

[Luke Hall]

Absolutely—that example highlights the positive work that the Cystic Fibrosis Trust is doing. I want to put on record my thanks and appreciation to all the parents and campaigners, whom I find completely inspirational. Not only are they parents, which is challenging enough in itself sometimes, but they are parents of children with CF, and on top of that they manage to find time to lobby their MPs, to get groups of people together, and to get support for petitions and debates such as today’s.

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[Paul Scully]

I thank the Minister for listening to us today. I congratulate not only all hon. Members who have spoken today, but Gemma and Michelle, who started this petition, and the people I met at the Cystic Fibrosis Trust.

We have talked a lot about suffering over the last three hours, because of the obvious necessity, but there are a lot of young people with cystic fibrosis who are watching this, and I want to end on a positive note. I started by talking about the video I watched. When we talked about running, I remember Grace Miller’s parents looked at a bag of trophies that she had won running. She was sad, because she missed the ability to run because of her lung capacity.

I would say: “Don’t be sad. Don’t look back at what you have done. Look into the future at what you are doing. Enjoy every day, because it is your stories that you are telling us today, and the constituents we have heard from today, that have got us to the point where we can hopefully get the Minister, the Government, NHS England and Vertex around the table and have a bright future for our constituents who are suffering from cystic fibrosis, with that progressive medicine.”

Question put and agreed to.

Resolved,

That this House has considered e-petition 209455 relating to access to the drug Orkambi for people with Cystic Fibrosis.