Orkambi and Cystic Fibrosis

Monday 19th March 2018 Westminster Hall

16:00

[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.

[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?

[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—]

[Mr Nigel Evans (in the Chair)]

Order. Could everyone take a seat? Hon. Members can see that quite a lot of people want to participate in this very important debate, so please use some self-discipline in the length of speeches. I call Ian Austin.

16:50

[Ian Austin (Dudley North) (Lab)]

I congratulate the hon. Member for Sutton and Cheam (Paul Scully). He gave a really good explanation of this issue and why this drug should be provided. Hon. Members are supposed to start their speeches in these debates by saying how pleased they are that the debate is taking place, but I am not pleased at all. I think that this is the third debate on this issue in which I have taken part over the past few years. We have presented petitions at Downing Street. We have had campaign events in Parliament. As has been said, we had 41 Members at a roundtable just a few weeks ago. And we are still here. Three years after Orkambi was approved for use and two years after NICE said that it was “important and effective”, we are still here, waiting for it to be provided for people with cystic fibrosis. So I am not pleased that we have to have another debate about this issue, but whatever I feel about that is nothing compared with the upset and worry—indeed, the terror—that people with cystic fibrosis and their families go through as they wait while their health, life expectancy and quality of life decline.

The reason why I have taken an interest over the years in this issue is that I was contacted by Carly Jeavons, an amazing woman from Dudley who took part in the clinical trial for Orkambi. I have also been contacted by Samantha Carrier, a young mum from Dudley whose baby daughter Daisy was diagnosed shortly after birth and who now devotes her life to campaigning for access to these life-changing drugs. I want to tell everyone here what Carly told me, because it explains much better than I can why we are here and why this drug must be made available. Before being put on the clinical trial, she had had to choose between leaving work, with all the financial hardship that that would cause, and struggling in work, with her health being made worse. She had to take 90 tablets and do two hours of physiotherapy every single day. Her lung function was at about 44%, and she spent two weeks in hospital every couple of months. She told me:

“Orkambi has changed my life. I quickly became well enough to start to live a more normal life again as a working mum. My health has remained stable. I only need one or two courses of IV’s per year instead of four. Hospital visits have massively reduced and admissions are non-existent.”

But that is just part of it.

[Julia Lopez (Hornchurch and Upminster) (Con)]

The hon. Gentleman makes the important point that if people have access to this drug, they can reduce the number of times they have to go to hospital. It would be very helpful if we had an understanding of the cost of those hospital admissions and what that would be if it was offset against the cost of the drug. Does he agree with that point?

[Ian Austin]

That is a really important point and one that I hope the Minister will take into account. We should be looking not just at the cost of providing Orkambi, but at the savings that that would make in other areas. I want to develop that point in a few minutes.

What I did not understand before speaking to people with cystic fibrosis was the toll that not knowing whether they will be given these life-saving drugs takes on their mental health. I am talking about the worry that it causes them and their families and the stress and fear that it puts them through. Something else that I did not understand before meeting Carly was the impact that having a condition that reduces life expectancy has on the rest of someone’s life. Lynsey Beswick, who many hon. Members will recall was at the roundtable a few weeks ago, explained that very well. She is in her 30s and told me that, at a time when her friends are getting married, planning families, developing their careers and starting businesses—making long-term plans—people such as her are deterred from doing those things. They just cannot plan for their futures in the same way because, to put it bluntly, they do not know how long they have to live.

Since having Orkambi, Carly has been able to go on holiday abroad for the first time with her family. She has married. She has started a business. Let us think about that. She has started a business, so she is employing people and making a much bigger contribution to the economy. People talk in these debates about the cost of providing these drugs. Let us talk also about the contribution that people who are given Orkambi can make to society. Let us think not just about the cost, but about the contribution they make, the businesses they can start, the jobs they can create and the taxes that will be paid. Let us think about that as well. Let us think about the contribution that providing Orkambi can make to our economy.

What is worse, the longer people live without access to drugs such as Orkambi—I had not really appreciated this—the worse their lungs become. Every day that access to this drug is delayed is another day on which the lungs of people with cystic fibrosis are damaged. I want to repeat that point, because I really want people to think about this: every day that people who could be helped by Orkambi are denied it is another day on which their lungs suffer irreparable damage.

That damage will not be put right. It is not like some medical conditions whereby the patient is given a drug and they are cured, restored and put back to how they were originally. That is not the case here. That damage will not be put right when—or if—they eventually get this drug. The damage that has been done is permanent. Every day this treatment is delayed limits the lives of people it could help. I really want the Minister and others to understand that point, because I must confess that I had not fully understood it until I chaired that roundtable with the Cystic Fibrosis Trust and Vertex a few weeks ago.

[Alison McGovern (Wirral South) (Lab)]

My hon. Friend is making a brilliant and important speech. I am here because of my constituent Ava and her family. Ava loves horse riding and her family want her to have the opportunity to live her life as an ordinary, healthy seven-year-old. That is all they want. Orkambi could provide her with that opportunity. Is not it people such as Ava and the people my hon. Friend has mentioned whom we have to keep at the forefront of our minds?

[Ian Austin]

My hon. Friend is completely right. This is what politics is about. What are we here for? We are here to listen to people in our constituencies. It is our job to come here and speak up on their behalf, which is exactly what she has just done.

I want the Minister to look really carefully at the way in which NICE works. This is not a criticism of NICE, the Government or anyone else, but new drugs are being developed and technological changes are happening so rapidly that I want to ask whether the way in which drugs are assessed, licensed and approved still works. My central question is: how are Ministers going to ensure that these ground-breaking drugs and new developments are made available much quicker?

In 2016, NICE was not able to recommend the use of Orkambi due to uncertainty about its long-term value, impact and cost-effectiveness. Vertex submitted a fresh proposal last month. On Friday, NHS England said:

“Following advice from Nice, the NHS has asked this particular drug company to review its proposed pricing. Unless this happens, further progress at this time is frankly unlikely.”

What sort of hope does a blunt statement such as “frankly unlikely” give people with cystic fibrosis? How does it give us any confidence that new drugs such as Symdeko, which is due to have its marketing authorisation confirmed by the European Medicines Agency in the coming works, will be approved as well?

[Alex Sobel (Leeds North West) (Lab/Co-op)]

My hon. Friend is making an excellent speech. A constituent who has two children with cystic fibrosis wrote to me:

“I have lived at first hand now for 42 years in close proximity to this cruel disease…Orkambi, expensive as it is, will offer a possible lifeline to many CF sufferers. On the cost effective side I think it is worth pointing out just how expensive it is to hospitalise and treat CF patients when they are ill, as happens frequently.”

She went on to say that the local facilities are superb, but:

“The cost of any drug that reduces the number of hospital admissions would I am sure, be at least significantly off set by the savings in NHS”.

Does my hon. Friend agree that it should be proved on that basis?

[Ian Austin]

My hon. Friend is absolutely right. The Minister and the Department need to take on board that really important point, which has been and will be made repeatedly.

I understand that the Government want to pay as little as they can for these drugs—of course, it is taxpayers’ money—and the company wants to get the best price possible. However, the question for Ministers is how this impasse is going to be resolved. I repeat: delaying means that people are dying sooner. Their lungs are suffering irreparable damage. Their lives are shorter. They cannot plan for the future. These drugs are available in the USA and across Europe, in France, Germany, the Netherlands, Austria, Italy, Luxembourg, Denmark and Ireland. When does the Minister think patients here in England will get them?

The Government’s response to Sir Hugh Taylor’s important accelerated access review opens by stating:

“The Government’s ambition is that NHS patients should be among the first in the world to get life-changing treatments.”

I note the words, “first in the world”. The response continues:

“Achieving this goal is only possible by working in close partnership with our world-leading life sciences sector.”

I say to the Minister again: this system is not working. That is not a criticism of him. He is a good guy. We have worked together on lots of issues. It is not a criticism of the Government, NICE or anyone else. It is just that NICE was not designed to deal with the development of these sorts of drugs. However, I ask the Minister to commit personally to sorting this out. It is really urgent. The system has not worked. Patients are being let down. Ministers need to take charge of this personally, so that an agreement can be negotiated as soon as possible. We are asking him to take charge of it personally and to make it an urgent priority to get this sorted out. Will he meet me, other MPs and the Cystic Fibrosis Trust, to look at the issue again? Will he promise today to take charge of the negotiations and ensure that progress is made? I want the Government to look at the issue again. Vertex needs to come up with a new proposal, too; it knows that it will have to negotiate and compromise. Will the Minister lead a new set of negotiations and get these people in the room, so that these drugs can finally be provided and other people in England can benefit in the way that Carly Jeavons has?

17:06

[Sir Mike Penning (Hemel Hempstead) (Con)]

It is a pleasure to take part in this debate. Like everybody in this room, I have constituents who suffer from this terrible genetic disease. We live in a society where sometimes those who shout loudest get heard more, but interestingly, it is not possible for those who suffer from this terrible disease and their families and loved-ones to have orchestrated the petition. Members of the general public who have no contact with someone who has CF have signed it and decided that the process is fundamentally unfair. Like the hon. Member for Dudley South—

[Ian Austin]

North.

[Sir Mike Penning]

My apologies—don’t forget I am a southerner.

The hon. Member for Dudley North (Ian Austin) and I had a good meeting at the roundtable. All of us learned things. For colleagues who were not there, there are some good notes to come around.

We thought we would not have a decision by tonight from NHS England on the Vertex proposals—it usually takes much longer—so I was very disappointed when I saw not only what NHS England put out, but the press release from Vertex. As the hon. Gentleman said, it is not so much because the Department, NICE or the companies are bad—our constituents could not have any of their drugs without the R&D done by those companies. NICE is not capable, under its guidelines, of properly analysing the benefits of the drug, or the other drugs coming down the line. The Republic of Ireland must have sat in exactly the same position that we are now in. It had difficult negotiations with Vertex about a plan for not just one or two drugs, but the drugs coming down the line.

Let us not beat about the bush: this drug is not a cure. It helps some people. At the end of the day, they will either have a transplant or their lungs will give way. It is wonderful that we will have an opt-out transplant system. People are dying in this country today because the organs are being wasted. Lung transplants are vitally important. We should all campaign in our constituencies to give people the confidence to tell their loved-ones what they want done with their organs, rather than just relying on the legislation. At the end of the day, to help people today and future sufferers of this terrible disease—we know they are coming, because it is genetically in the system—we need not only drugs that slow it down and stop the lungs filling with fluid, but to get a cure. I hope we get to that position in my lifetime. Those of us who have been in the House for some time will remember taking the Human Fertilisation and Embryology Act 2008 through. It was very controversial when we started using that sort of technology, research and work, but I am pleased that we passed that Act because many people are around today who have better lives and who, without us using that technology, would have been very worried.

As my hon. Friend the Member for Sutton and Cheam (Paul Scully) said, this is not about individuals. When an individual gets CF, the whole family and all their loved ones get it. If the family is not there, what happens? The NHS and social services. Several colleagues have asked about the overall cost. If we do not give people these drugs—not just this one, but the others coming down the pipeline—the cost to the NHS is greater. If we take away the moral and ethical position that we have something that will improve and extend someone’s life and look just at what NICE looks at—the cost implications—it is plainly obvious that we need to have a better system for NICE to assess the costs.

My hon. Friend called it “physiotherapy”, but someone who suffers with CF has to have a pummelling. People have to do an amazing thing to their loved ones to get the fluid out their lungs and to stop them drowning internally. Instead of saying that drug companies are bad and NICE is good, we need to bang some heads. Frankly, the only people within Government who will do that are the Ministers. That was said to me time and again when I was a Minister. Time and again I tried, and time and again I got pushed back, but I kept going.

It is obvious—to echo what I said at the start of my comments—that those who shout the loudest should not always win. In this case, we need to shout for them. That is what we were sent here for, and that is what we should do today.

17:12

[Mr Stephen Hepburn (Jarrow) (Lab)]

It is a pleasure to see you in the Chair, Mr Evans.

I thank hon. Members on both sides of the House who are here to push this great case forward. I pay tribute to my hon. Friend the Member for Dudley North (Ian Austin), who organised a very constructive roundtable meeting where we learned a hell of a lot. I also pay tribute to all the campaigners, some of whom are represented today. They fought hard, got the petition organised and had such a loud voice that Parliament has not been able to ignore them.

We are all here because we want Orkambi to be made free on the NHS. It is a drug that works, improves lives and gives hope, so there is no reason why it should not be available. We agree that NICE’s 2016 decision to class Orkambi as not cost-effective for the NHS was wrong. Frankly, the Minister is in a position to get round the table with the drug companies, NICE and the NHS to resolve this and break the deadlock.

[Margaret Greenwood (Wirral West) (Lab)]

My hon. Friend is making a really good speech. A constituent—a proud and loving grandad—came to my surgery with two sets of photographs of his granddaughter, who is nearly six. One photograph was of a lively little girl, full of energy, and the other was of all the medication that she has to take every single day. The imagery could not have been starker. Does my hon. Friend agree that when someone is nearly six, a year is a very long time to wait for change, and the Minister should get a move on?

[Mr Hepburn]

I agree entirely and am pleased my hon. Friend has repeated my earlier plea to the Minister.

It has been said that Orkambi is recognised in a lot of countries—I do not want to go through the list, because there are many. We are getting left behind. Imagine what it is like to be a sufferer and to be told, “There is something out there that will improve your life and your family’s life, but, sorry, it is just not cost-effective.” Imagine how that makes them feel.

I have two constituents down here today: Emma and Chris Corr. They have a three-year-old daughter who has undergone more hospitalisations, more medical treatments and more operations than the vast majority of people will go through in the whole of their lives. All Emma and Chris want is to give their daughter a future and ensure that she is not limited in her life chances. I will be brief, but my plea is to think of the likes of Harriett: let her go to school and get a full-time education, without impediment; let her get qualifications; let her get a job; and let her live a long life. The alternative is unthinkable, whereby Harriett will have a deteriorating lifespan, leading to an early death, which I am sure we would all agree is unacceptable for anyone.

The final decision is with the Minister. I hope he recognises the unity of purpose in the House and acts immediately.

17:16

[Bim Afolami (Hitchin and Harpenden) (Con)]

It is a pleasure to serve under your chairmanship, Mr Evans. It is also a pleasure to be in this debate. I commend my hon. Friend the Member for Sutton and Cheam (Paul Scully). As has been said, the number of Members of Parliament here for a Westminster Hall debate is extraordinary, and shows how much Members and constituents care about this.

I will be brief because it is important that Members have a chance to make their contributions. Cystic fibrosis is the most common inherited genetic disease in the UK. We have already heard that Orkambi has been praised as being important and effective by NICE.

I am thinking about two of my constituents: Matthew Dixon-Dyer, who suffers from the disease himself, and Karen Murphy, whose son suffers from the disease. They urged me to speak in this debate. They did not just want me to urge the Minister to take charge of the negotiations with Vertex, or at least to press NHS England much harder to come to a resolution with Vertex. Nor was it to do with just cystic fibrosis or this drug. It is important that the cost-benefit analysis that NICE uses reflects chronic conditions, which drugs such as Orkambi deal with, more broadly, rather than just dealing with acute conditions, which it was typically designed for.

Like my hon. Friend the Member for Hornchurch and Upminster (Julia Lopez), I ask the Government to build the greater cost to the NHS later into the cost-benefit analysis. As we have heard from many Members, individuals may have to be in hospital for weeks on end as a result of the reduction in lung capacity. Karen Murphy’s son was in hospital for 16 weeks. I do not know what the costs of that were.

[Mr George Howarth (Knowsley) (Lab)]

The hon. Gentleman is making a very powerful point. Does he agree that, while we need processes to evaluate new drugs, sometimes the system does not work, which is why we have Ministers and such debates?

[Bim Afolami]

Yes, sometimes systems do not work and, as I said, I believe that in this case the Minister should get involved, but we also need to look at the processes. Members of Parliament cannot come to the House and demand action for every single drug. We need to build in better processes for NICE so that when there is a drug such as Orkambi, which deals with a chronic condition and can make a significant difference to people’s lives, the Government, Members of Parliament, this country and our society can act in the right way.

[Mr Paul Sweeney (Glasgow North East) (Lab/Co-op)]

It is important to note that the average life expectancy for someone suffering from cystic fibrosis is just 28 years. That is why Orkambi is so critical, because it will help people to maintain life expectancy and a good quality of life in the prime of their life.

[Bim Afolami]

My constituent, Mr Dixon-Dyer, is in his early 30s. He and his wife have a baby on the way. That gave him such an impetus that he came to me, because he wants to be around for that child’s life. I want him to be around too.

I hope the Minister takes note of the strength of feeling, which as he will be aware is not typical for a Westminster Hall debate, and looks kindly on the proposal.

17:20

[Ms Marie Rimmer (St Helens South and Whiston) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Evans. I congratulate the hon. Member for Sutton and Cheam (Paul Scully) on securing the debate. For good reason, the issue has galvanised popular opinion.

As we all know, cystic fibrosis is an extremely serious, debilitating and life-limiting—yet thankfully rare—diagnosis. As we have heard, in February, Vertex proposed a portfolio approach whereby all current and future cystic fibrosis medicines, including Orkambi, would be made available to eligible patients through a fixed budget cap to the NHS.

I was extremely disappointed to hear NHS England’s response on Friday that there will not be further funding for Orkambi. As it is a treatment that is proven to improve and prolong the lives of 50% of sufferers, that decision is a cruelty to cystic fibrosis sufferers across the country. The portfolio approach has been accepted and agreed in the Republic of Ireland, Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Greece and the US, but sadly, discussions here—with the world’s best health service—have broken down.

CF causes a build-up of mucus in the lungs that means that patients require physiotherapy, almost from birth, to keep them breathing properly. CF patients are more susceptible to lung infections and can suffer from malnutrition owing to the effect of the condition on the pancreas. When NICE made its initial evaluation of Orkambi as a treatment for the devastating condition, the evidence that it slows lung deterioration by 52% and reduces hospital admissions was not available, and therefore not costed. NICE is simply not fit for purpose any longer; it cannot evaluate a portfolio approach and precision drugs. For NHS England to turn its back on the small group of patients that suffers with this rare, debilitating illness and to ask Vertex to reapply to NICE is shocking. If it did reapply, it would get exactly the same answer—so why do it? Does NHS England understand that? Does NICE understand that? Are we going to send these seriously affected families around and around?

Vertex now has a broader pipeline of 18 drugs that will follow Orkambi in the next seven years. If the portfolio is agreed, it will advance on to those medicines. Will the Minister work towards a more flexible approach? Simply, he must get involved to benefit the small group of patients for whom a much better quality of life is in sight, but just out of reach, otherwise they will never get the treatment that they deserve and that they should be getting.

The parents of two constituents, Christian Jones and George Barton, contacted me. Christian’s mother, Hayley, wrote to me and said that Orkambi

“is not a cure but it is life altering, as it lowers admissions and reduces lung deterioration by 42%.”

She believes that Christian’s other medications will only increase unless he can access the drug. She also wrote:

“This genetic condition is awful to live with as a parent, wondering whether you will bury your child before they reach 20. I can’t begin to know how it must be for my son and other patients when they can’t breathe properly or speak without coughing or how his lungs must burn.”

She has nothing but praise for the NHS, in particular Whiston Hospital in my constituency where Christian has been treated since he was just weeks old. He is now two. In trials, Orkambi has been shown to help children aged between six and 11 to improve their oxygen uptake by up to 10%.

Orkambi would also be of immeasurable benefit to my constituent George Barton. George’s mother, Louise, wrote to me when she heard that the NHS’s provision of Orkambi was going to be debated in this place. George is eight years old and, like Christian and most other CF sufferers, was diagnosed as a young baby. CF is progressive, so George’s lungs are beginning to degenerate. Every day, he takes 30 to 40 tablets, has an hour of physio and takes two nebulised antibiotics. Louise told me that when he catches an infection, he can quickly become so seriously ill that he needs extra physiotherapy and intravenous antibiotics three times a day for a full two weeks. At times, George has to stay in Whiston Hospital, or, because he and Louise live nearby, the nurses come out and take care of him at home. In fact, when Louise first wrote to me, she was in hospital with George on a two-week stay. She wrote that although

“the damage to his lungs is becoming more serious, Orkambi can slow this down”.

George, Christian and thousands of other CF patients need that niche treatment, and there is the political will in this place to fight for them. Only about 3,000 people would benefit from the drugs. I urge the Minister to get involved, to facilitate further discussions between NHS England and Vertex, and to push for a flexible solution. Orkambi is a different type of drug that we cannot evaluate at this time. This is not only about licensing Orkambi, but the promise of those 18 drugs still to come, which Vertex will advance on to. NICE simply cannot evaluate and I urge the Minister to intervene.

17:27

[Michael Tomlinson (Mid Dorset and North Poole) (Con)]

It is a pleasure to serve under your chairmanship, Mr Evans, and to contribute to the debate. I, too, congratulate my hon. Friend the Member for Sutton and Cheam (Paul Scully) on his presentation of the debate, and I congratulate the hon. Member for Dudley North (Ian Austin) on his speech and on the way he has campaigned.

Of my constituents, 177 signed the petition. One in particular, Kerri, came to my surgery recently. She wanted the voice of her daughter, Faith, to be heard in Parliament. Faith says:

“My name is Faith. I’m 19 years old. I was diagnosed with cystic fibrosis at 8 months. I’m in my 2nd year of University. I study at Solent Uni in Southampton and enjoy it very much, however I have a low attendance due to my sickness. I’m constantly off and I miss a huge amount of lectures, even though they accommodate to help me catch up, but this is worrying to me as I have missed so much of my education due to cystic fibrosis, both in Uni and previously at school and college.

Orkambi would benefit so many children and adults just like me. It would decrease the impact of this condition, and slow down the decline in health. This would enable me to be healthier and follow my ambitions, whereas, at the moment I’m just sick, and tired of being sick and tired!

I want to do well for my future, so please consider Orkambi if you want to help the next generation! I suggest you revise your choice on allowing sufferers with cystic fibrosis access to a life changing drug. I want to live a life unlimited, after all people my age are the future and I wish to be a part of it.”

Faith’s words speak for themselves, and I look forward to the Minister’s response.

In closing, I echo the point made by my hon. Friend the Member for Sutton and Cheam in his speech and by my hon. Friend the Member for Broxbourne (Mr Walker) in his intervention. I call on Vertex to be reasonable, and for it and NHS England to get round the table and stay round the table. If it does, that could produce tremendous results. If not, CF sufferers will lose out.

17:29

[Bambos Charalambous (Enfield, Southgate) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Evans.

I was recently visited by a constituent who has twin nine-year-old daughters, one of whom—Holly—has cystic fibrosis. At present, Holly can run, play and lead an active life, but she still needs treatment and will get worse. She receives treatment at Great Ormond Street Hospital and the Brompton Hospital. Because of her genetic composition, there is still no drug available that will help her to survive beyond her thirties, but with the research and development of drugs for other cystic fibrosis sufferers by Vertex, there is hope for Holly.

For the 10,000 other cystic fibrosis sufferers, there is a chance that their lives will be improved, but that hinges on drugs being approved by NICE. We know that NICE has already approved the drug Kalydeco, which only works for the 2% of cystic fibrosis sufferers whose genetic make-up it is compatible with. However, Orkambi has not been approved, despite potentially helping a wider tranche of cystic fibrosis sufferers. NICE has ruled that that would be too costly and that there is uncertainty around Orkambi’s long-term value and impact.

As we have already heard, Orkambi is widely available in the USA and in several EU countries that have seen the clear benefits of not having people spend time in hospital. In trials for Orkambi, there has been a 61% reduction in hospitalisation among those using the drug, and over a 96-week period a 42% reduction in lung decline. It is therefore disappointing to learn that despite Vertex trying to negotiate a pipeline deal with the NHS over future drug developments to tie in with Orkambi there is still no agreement. Are we really saying that there is no scope for negotiation? What price are we placing on the lives of cystic fibrosis sufferers?

Despite Orkambi being unable to help Holly, the fact that her parents are willing to campaign for others to get help is a testament to them and to the cystic fibrosis community. I ask the Minister to put pressure on NICE to reach a solution with Vertex, so that cystic fibrosis sufferers can get the medication that they need and deserve, and get it urgently.

17:31

[Mr Iain Duncan Smith (Chingford and Woodford Green) (Con)]

I am grateful to you, Mr Evans, for your chairmanship today. As you suggested, I will be brief.

So much has been said already about this drug that I want to make only two points about it at the end of my speech. I congratulate my hon. Friend the Member for Sutton and Cheam (Paul Scully) and hon. Members from both sides of the House on having been responsible for the assistance and support that has been provided—both the roundtable that took place earlier, which I was not able to attend, and the e-petition—and on representing the sufferers and their families so well. I congratulate them, and I am pleased and grateful that they have managed to secure this debate.

I am really here because one of my constituents is standing outside hoping that there may be some resolution as a result of this for her daughter, who suffers from cystic fibrosis. I have known the family and the daughter for a little while. The contrast is interesting between two children of my constituents. On the one hand, Megan Bridge is a cystic fibrosis sufferer—her mother, Gill, as I said, is outside just now— and she has made the point to me that at the moment she is old enough to study law. Like any of our children of that age, she should have great prospects, hopes and aspirations, but hers are limited because the prognosis is that she probably does not have more than another two years if things carry on as they are.

If a decision is not taken about this drug soon and agreement is not reached, it is at Megan’s end of the spectrum that that becomes very difficult, because not only will further damage set in, as was said by the hon. Member for Sutton and Cheam earlier, but a decision might not be made within a year or two, in which case the prognosis for her and for her family is incredibly poor. She begs that we and the Minister—I will come back to this point shortly—make a clear decision with the drugs company as soon as possible.

At the other end of the spectrum is somebody called Amelia, who is only five years old. Her mother, Lucie, came to see me in my surgery to talk about her condition and about how the family copes with it. This poor girl, like so many others who have been mentioned, has to take 40 tablets a day, and has huge amounts of physiotherapy, including three nebulizer treatments, every single day. She is not yet on a feeding tube, but her mother has been told that if the current situation goes on for much longer then she will be.

My point is that these accounts are two ends of the spectrum. In a sense, they are not representative; although they are representative of sufferers, they are not representative of a vast number of people out in the country, because this is such a peculiar genetic condition that, as has been said time and again, not a huge number of people have it. That is where this place and Governments of whatever persuasion—this is not party political—sometimes have to recognise that the common good is not always about majorities and large numbers. It is more often the case that this place is set up to deal with those who are so small in number that they are unable to make the case for themselves. They are unable to drive the point home; they are unable to muster the vast number of votes that it might take a matter to the point where MPs listen very carefully.

What we have seen today is of enormous credit to the humanity of my colleagues on both sides of the House. These people are not likely to change the vote in my colleagues’ constituencies, but they care enough about the idea that these people’s lives matter that we might be able to do something for them.

[Teresa Pearce (Erith and Thamesmead) (Lab)]

I thank the right hon. Gentleman for giving way. I am listening carefully to what he is saying, and he is saying exactly what I was just about to point out, namely that this is why we are here. This is why we are sent to this place, to speak up for the people who cannot speak up for themselves. My constituent, John Bacon, who is in his twenties, sent me a very simple email that said, “Please help me. Please speak for me and advocate for me. And not just for me, but for all those with cystic fibrosis, and remove the price that has been placed on our lives. We are worth it.” They are—and if we do not speak for those who cannot speak loudly, we should not be here.

[Mr Duncan Smith]

I completely agree with the hon. Lady, and that is really the point that not just me but pretty much everyone who has spoken has been making.

I do not know what detailed conversations and discussions are going on about this drug. I sat in government, so I know how complex these things are sometimes. I say to my hon. Friend the Minister that “frustrating” was the word I often used when bodies are set up and fall back on their rules because they are not so exposed to public scrutiny.

The latest evidence—I understand that it comes from the company—shows that there is stronger data and that this drug is even more vital than it was before. It now slows lung decline by 42% and it might turn out that it is even more effective than that, which would give somebody like Megan, who I mentioned earlier, the possibility of longer. What is that delay about? The possibility of transplant, and even further down the road the genetic changes that may well be able to be introduced. So, buying time matters dramatically for this group, in the hope that things will improve for them.

When I was in Government, I know that we spent a lot of time on social impact bonds. One area we worked hard on—the Government have incredibly good data on it because we made a number of case studies—is the cost-benefit analysis, to allow us to say that local authorities and so on could set up social bonds to change the number of people going into prison, or to affect the number of people who fail at school. So it was possible to forecast how a cost-benefit analysis would work; it exists.

Right now, I am not certain that NHS England and for that matter NICE are employing that system. I urge my hon. Friend the Minister to go to the Cabinet Office and say, “We have done the work on a proper cost-benefit analysis of this drug, and once it is tied in with future developments of other drugs and balanced with health treatments in hospital, constant work with physiotherapy and all the rest of it, I am certain that this group will not only to be able to have this drug, but we will ask ourselves why that didn’t happen before when it so mattered and it benefits them. It also benefits us because instead of their being in hospital, others may be able to use that hospital treatment.” That is a good cost-benefit analysis and I urge my hon. Friend to look at it.

17:38

[Kerry McCarthy (Bristol East) (Lab)]

It is a pleasure to see you in the Chair, Mr Evans, and I hope that you received my note, saying that I would have to leave before the end of the debate—I am sorry about that.

As my hon. Friend the Member for Dudley North (Ian Austin) said, some of us very much hoped that we would not be back here making the case for Orkambi, because our being here again means that we still have not managed to persuade the Government and the National Institute for Health and Care Excellence to fund it. However, I have been absolutely overwhelmed by the fact that so many MPs have turned out today and that, as has been said, the petition gained the required 100,000 signatures within 10 days. That is phenomenal and I cannot believe that the Government will not listen to that outpouring of support.

Constituents have contacted me about their children, their grandchildren and their friends’ children—I had a lovely email from a family friend who has a little boy aged seven. I particularly want to pay tribute to Lee Partridge in Bristol for his work with the south-west parents cystic fibrosis support group. He lost his daughter Richelle in 2016 to cystic fibrosis, aged 26, and eight months later her sister Lauren died, aged 19, from the same illness. I cannot begin to imagine what that family has gone through, although I have a 13-year-old niece, Maisie, with cystic fibrosis, whom I have mentioned here before.

Maisie has been in pretty good health considering, but I have listened to Members talk about how people’s health can suddenly take a dramatic dip, with their lung function percentage going from somewhere in the 90s way down perhaps to 20, and I realise that I cannot be too optimistic. I can look at her today and say, “This is a happy, healthy child”. Apart from being hospitalised when she was first diagnosed at a few weeks old, she has had intravenous therapy only once, and that was almost a preventive measure because her lung function had dipped a little.

I have visited the children’s cystic fibrosis unit in Bristol and the adult treatment centre at Bristol Royal Infirmary, and many people spend their entire lives coming in and out of hospital, so we have been very lucky, but Maisie still has to have a cocktail of pills. When I spend time with her, her whole life is dominated by which pills she has to take. Every time she eats something she has to take her Creon. She has to have physiotherapy. She has also just been diagnosed with the very rare Wieacker-Wolff syndrome—we have suddenly discovered that one other child in the country has it. I thank the Government for supporting the deciphering developmental disorders study into 12,000 children and adults with undiagnosed conditions that has been carried out over the past couple of years, because it was only through that that we knew what was causing Maisie’s mobility problems and mild learning difficulties. The mobility problems obviously make it more difficult for the physio to be done and for her to be more independent.

[Sir Oliver Letwin (West Dorset) (Con)]

What the hon. Lady describes is a quality of life. Does she agree that, at root, what we are dealing with here is the problem that NICE measures the quality of life-adjusted years and does not measure improvements in quality of life of the kind she describes?

[Kerry McCarthy]

How do we measure the cost to the family? How do we measure Orkambi’s worth? To me, it would clearly be life changing, perhaps even life saving. When my hon. Friend the Member for Dudley North hosted his roundtable, it was reassuring to hear that Orkambi is another step forward—Kalydeco helps only 2% of people and we think that Orkambi could help 40%—and that drugs are in the pipeline, being worked on now, that may bring help to all cystic fibrosis patients in this country. It was said that it is too soon to judge whether the developments, rather than being just life-prolonging, could be life-saving, with cystic fibrosis being a condition that people live with, like diabetes, rather than one that people die from. Surely that is where we want to get to.

The overall picture is that getting one drug on the market is a stepping stone to further research, and it is about buying time, as the right hon. Member for Chingford and Woodford Green (Mr Duncan Smith) said. Maisie is now 13 and it is imperative that she get on such a drug if she turns out to be one of the children who can be helped, because, frankly, we do not have an awful lot of time before her condition might deteriorate. I cannot stress enough how important that is to us as a family. She obviously has hopes for the future. At the moment, she wants to star in musicals, which might be slightly over-ambitious. She has just got back from a horse-riding weekend in Wales.

I finish on that note of buying time. We do not have the luxury of time. We need to see action now from the Minister and we need to look at the overall cost to the health service and to the families. Maisie’s mother—my sister—is an absolute saint. She is a teaching assistant. She works with children with disabilities. She is a forest school leader. What she does in her daily life is wonderful, juggling that with a disabled child and two other children as well. If Maisie takes a turn for the worse, my sister will effectively be removed from the workforce and become a full-time carer. We ought to consider costs like that as well. Let us not look at it as a crude mathematical calculation but as what it means for people’s lives.

17:45

[Paul Masterton (East Renfrewshire) (Con)]

It is a pleasure to serve under your chairmanship, Mr Evans.

I will not go back over what Orkambi is or what it does, but it is important to remember that we are talking about only the second precision medicine for cystic fibrosis to be licensed in the United Kingdom. It has rightly been licensed for use with people over the age of 12 who have this specific form of cystic fibrosis, and both the Scottish Medicines Consortium and NICE have recognised that it is a beneficial treatment. However, neither body has recommended it for general use on the NHS, meaning that, except for on compassionate grounds in certain rare cases, many people with cystic fibrosis across the UK are simply unable to access it. Given what Orkambi can do, it is no surprise that many Scots with cystic fibrosis were disappointed by the SMC’s decision in May 2017 not to recommend it for general use on the NHS in Scotland.

Although I received a number of campaign emails ahead of the debate, it was two pieces of personal correspondence that convinced me of the need to come along today and speak. Both came from a grandparent of a young person battling cystic fibrosis: Mr Alexander from Newton Mearns whose grandson is 11, and Mrs Marshall from Eaglesham. Mrs Marshall wrote to me regarding her seven-year-old grandson:

“Through vigorous twice-daily physiotherapy and the many sports he enthusiastically takes part in, he has not lost too much of his lung capacity yet. He’s a very sociable and clever child and it is heart-breaking to know that this disease could be, at the very least, slowed down or almost eradicated by these new drugs which are not available to him. Please, too many people are suffering more than they need to and dying unnecessarily young because of the delay in approving Orkambi.”

[Kirstene Hair (Angus) (Con)]

I too am here because of the number of constituents who have been in touch but also because I lost a friend, Lucy, at the age of 19. She had so much to give. She had talent and was the most caring person you could ever meet. She dealt with it in an incredibly dignified manner, and I urge the Minister to show that same dignity in dealing with his response today.

[Paul Masterton]

My hon. Friend powerfully sums up why Vertex, the SMC, NHS Scotland, NHS England and NICE need to work together to find a way to make Orkambi available on the NHS as soon as possible, not just in Scotland but right across the United Kingdom. I understand that confidential talks are ongoing and I very much hope that there will be a positive conclusion; there is no reason to assume that an agreement cannot be reached. We have seen things happening right across Europe, and in the Netherlands a deal was struck fairly recently to allow the drug to be made available.

[Paul Girvan]

Big pharma has a strong lobby and there are those within the industry who would lose out if a drug such as this were to be licensed and used in the UK. They would fight tooth and nail to ensure that it did not get across the line. I ask the Minister: whenever a new drug is assessed, is consideration given to the fact that there are those who will not want it to see the light of day?

[Paul Masterton]

I suspect that the Minister has listened carefully to that intervention. I, too, will be interested to hear his response.

I finish by extending my hope that we are able to get the drug licensed for more children—the current license is for those aged 12 and over. In January, the European Commission extended the marketing authorisation for Orkambi to include children aged between six and 11 —just like those young people whose desperate grandparents took the time to contact me—and, just days ago, the Republic of Ireland became one of the first EU countries to make Orkambi available to children aged six and over. I hope, therefore, that Orkambi will not only become available on the NHS in the near future, but that Vertex will make a further submission to the SMC so that it can also be available to six to 11-year-olds.

Cystic fibrosis is a serious, life-limiting condition, and Orkambi can help to give people with the condition as long and healthy a life as possible. Both in Scotland and across the UK, there should be no unnecessary delay in extending access to it, either because of age or because of its availability on the NHS.

17:49

[James Frith (Bury North) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Evans. My constituent Graham Little got in touch with me. His wife Claire has cystic fibrosis. Until that moment, I was not aware of the potential life-changing drug available to sufferers of cystic fibrosis. Graham and his father-in-law Brad raised with me the status of the precision medicine Orkambi. The result of today’s debate could make the difference and give them decades more with their wife and daughter, Claire, and give her decades more with her children. I cannot imagine how that must feel.

We can either observe the steady death caused by cystic fibrosis—it has been compared to drowning—or reach for the life jacket, which is primed, ready for use and in all sizes. Our country has the largest number of cystic fibrosis patients anywhere in the world, so we ask the Government what they are going to do for our cystic fibrosis sufferers. If they will not make Orkambi available, what are they going to do? The transformative, life-prolonging impact of the drug is tantamount to life-saving. Used early enough, it can ensure that people fulfil their potential. It can prolong life and lung capacity, enabling those with the disease to contribute to the economy and wider society, as well as providing extra decades with family and loved ones.

The Government should consider the case for a commissioning body for rare diseases. It is not right that the judgment criteria used to determine funding on globally common diseases are universally applied when the uncommon traits of a rare disease bring untypical symptoms and a different economic argument. We risk overlooking the incredible efficacy of the drugs now available.

The power in numbers is not much solace to a cancer sufferer, but such is the profile of cancer treatment that we are all assured of the progress being made there. The same cannot be said for the rare diseases argument, however. We have a job to do in making the case for rare diseases. I hope the Minister will listen to the case being made for a medicine and treatment decision-making body for rare diseases, as well as consider how we bring NICE into the modern age, 20 years after it was established.

[Liz Kendall]

My hon. Friend will know that the Government review NICE once every three years. Does he agree that the next review, which will start in July this year, must look seriously at how NICE makes its decisions? Does he also agree that the results of that review should come out quickly? Last time, it took about a year and a half.

[James Frith]

I completely agree with my hon. Friend. We need to see far more nimble-footed decision making by the decision-making body, as per my call for a specific dedicated body for rare diseases. Parents in my constituency make the case about the plight of those with rare diseases. There is a constant sense of those enduring rare diseases being overlooked.

We come to the remaining agents at the table. Just this weekend, NHS England has refused to agree to the deal that Vertex proposed last month. I do not think the wording of the refusal was constructive, but I am not surprised that the NHS was unwilling to sign up in principle to the deal, which amounts to a futures prospectus from Vertex off the back of a sure thing with Orkambi. Patient groups are at the table, and I commend the work that the Cystic Fibrosis Trust is doing on behalf of CF sufferers.

We need compromise and urgency from all. Nobody should expect Vertex to surrender its drugs, but neither should we accept a failure to reach an agreed price. I say to Vertex that the pipeline might need to be for another time. When I met Vertex, I cautioned that its “portfolio of drugs” approach may be cost-prohibitive to a decision, and that appears to have been the case. We need Orkambi for patients today. It is not the time to test the innovative decision making of NHS England. Vertex should take in good faith that a fair price to the world’s largest population of cystic fibrosis sufferers will set it up well for future developments. A price somewhere between what it was offering in its recent pipeline deal and a single purchase deal is where we are now. I ask Vertex to please stay at the table. We keep in mind its proposal of having Orkambi readily available, swiftly and easily, for all. We urge focus on enabling that. The rest will surely follow.

At the table, there is a ministerial chair that needs filling by a Minister who is willing to lean in to the debate and signal their support for prescribing Orkambi on the NHS; willing to lead and lean in to the deal-making part of the job to transform lives; willing to look at the costs and to help with the price; and willing to align the political will to the possibility of life for those with cystic fibrosis. Treatment for cystic fibrosis has moved from science fiction to science fact, so the Minister is required to act and step in. The Government have a choice to make. They can ignore our arguments, or, having heard them, choose to change their mind. In changing their mind, they can change lives and lifetimes. I urge the Minister to do that and make real-life change for cystic fibrosis sufferers a reality. He would have support from Members across the House, as has been demonstrated by the magnificent strength of feeling in the contributions and arguments made today.

17:49

[Bob Stewart (Beckenham) (Con)]

Being born with cystic fibrosis is just the bad luck of the draw. Until I started researching cystic fibrosis to speak today, I had no idea that one in 25 of us carries a faulty gene that causes it. Nor did I realise that people were born with the condition and cannot catch it. The Cystic Fibrosis Trust tells me that about 10,400 people have cystic fibrosis in this country.

It is bad luck if both parents have a gene that might generate cystic fibrosis in their child. Most potential parents carrying a generating cystic fibrosis gene are totally unaware that they could have a baby cursed by the condition. I gather that the chance of having a baby with cystic fibrosis when both parents have the gene is about one in four. That was the case with Trudy and Gary Davey, my constituents, whose son suffers from the condition. Trudy is the daughter of my very close friend, Maggie Hopgood. Maggie’s grandson Zac had the standard blood test just after his birth, and he has been on cystic fibrosis medication ever since—that is for life.

Cystic fibrosis is a rotten condition to have. Most often it attacks our lungs. Sufferers get a mucus build-up in their lungs, and lungs do not work well when they are drowning, but it can be worse than that. Our pancreas is tasked with producing enzymes that break down food in our stomachs, but that organ can also often fill up with mucus, ruining what it is meant to do. Cystic fibrosis does not stop its rampage there. It can also cause diabetes and bone disease. Cystic fibrosis is literally a pain to live with. It is always life-threatening, and it is certainly long-life restricting.

I come to the main point of this debate, which is to get cystic fibrosis sufferers, such as seven-year-old Zac Davey, better access to Orkambi. Orkambi seems to make a huge difference to the lifestyles and lives of sufferers. I gather that it works on about 50% of cystic fibrosis sufferers and slows the decline in their lung function by 42%, which is quite a result.

As we have heard, Orkambi is not normally available on the NHS except in extreme compassionate cases. Of course, the reason for that is expense. Per patient, Orkambi can cost more than £100,000 a year. On the plus side, more widespread use of Orkambi is likely to reduce the requirement for hospital treatment among sufferers—there is a spin-off in reduced NHS costs. The drive must be to reduce the costs of Orkambi. That requires action from Vertex, the US-based company that produces it, and from the Government and the NHS. I gather that negotiations have been ongoing for a while. We are talking about people’s lives and their life expectancy. It must be sorted and it can be. Hopefully this debate, having raised the matter for the third time in Parliament, as we have heard, will add impetus to the resolution of the problem, and Orkambi will be made more readily available to the many who have sore need of it such as my friend’s grandson.

[Sir Roger Gale in the Chair]

18:01

[Tulip Siddiq (Hampstead and Kilburn) (Lab)]

It is a pleasure to serve under your chairmanship, Sir Roger. I pay tribute to the hon. Member for Sutton and Cheam (Paul Scully). I usually apply to speak in Westminster Hall rather than in the main Chamber because the waiting time tends to be shorter, but I have had to wait today because of the huge turnout, which is testament to his tenacity in securing this important debate. Many constituents have emailed us about how their condition has affected their lives. I will cut straight to the chase and say that all the people who emailed me were unanimous in their desire to see Orkambi made available on the NHS.

In all the representations I have received, whether from families who have been affected by the disease or from world class medical professionals in Hampstead and Kilburn, there is one clear message: cystic fibrosis patients do not have time to waste. I put this bluntly not because I want to state the obvious about a life-limiting condition, which has been made clear by all the contributors so far, nor to suggest that Ministers object to introducing the precision medicines that could extend life chances. I say it because it is the key message that must weigh upon all our contributions today.

I presume that most Members have received a briefing from the Cystic Fibrosis Trust, which does exceptional work in advocating for those suffering from the condition. I am proud to say that I have a constituent, Ffyona Dawber, who is a trustee and is passionate about this debate and patient education more broadly. Ffyona and her colleagues at the trust have told me over and over again about the possible benefits of Orkambi. They say that medicines such as Orkambi have been proven to add years to a patient’s life. They point to the fact that Orkambi has been shown to slow decline in lung function by 42% and to cut the number of infections requiring hospitalisation by 61%. As has been quoted many times, they point to the NICE appraisal of 2016 that said that Orkambi is both “important and effective”. Given that the possible benefits of Orkambi seem to be settled, it is necessary to spell out why the Government should act now and agree a sustainable solution over the cost.

It is also important to highlight the voices of those whose futures depend on the introduction of that important drug. My hon. Friend the Member for Erith and Thamesmead (Teresa Pearce), who is sitting next to me, talked about how we have to speak up for residents and constituents who cannot come here and speak for themselves. Many who have the condition cannot travel to Parliament to witness the debate.

I echo the words of a resident of mine, Caroline Brown, who wrote to me about her treatment for cystic fibrosis. She told my office that she felt “well supported” by the NHS. She paid specific tribute to the doctors and nurses at the Royal Brompton Hospital on the Fulham Road, saying that

“they are utterly amazing. I have had the best care there and I cannot fault them.”

Her tribute to the staff at the Royal Brompton reflects those paid by other local residents, especially those whose children rely on the specialist cystic fibrosis centre at Great Ormond Street. For patients such as Caroline, my constituent, the debate over Orkambi is about enabling our world-leading medical community to focus their efforts more sharply on those with advanced symptoms, and for pharmaceutical companies to get on with investing in research for aspects of the condition where the breakthrough represented by Orkambi still evades us.

It will come as no surprise to anyone to learn how Caroline felt about the situation surrounding Orkambi. As expected, she was clear in her support for its immediate roll-out. She said:

“When I look to the future it would be very comforting to know that, if I was to need it, there would be medication there that would help me. I can’t bear the thought of knowing that there is something out there that could potentially save my life, and that I am not able to take that as I couldn’t afford it. Being symptom free for someone with Cystic Fibrosis would be life changing.”

She went on to underline the consensus that exists among cystic fibrosis sufferers. A lot of them cannot meet and be in the same room for the reasons outlined by other Members, but there is one benefit of social media—I stress one benefit—which is that it has enabled a network of people with cystic fibrosis to discuss and reflect upon the key debates in the community and to share the experience of their treatments. She knows that fellow patients are unanimous:

“They all agree it should 100% be made available on the NHS. It should be available to everyone that needs it...it is sad that money is getting in the way of people's health improving.”

[Stella Creasy (Walthamstow) (Lab/Co-op)]

My hon. Friend is making an incredibly powerful speech. May I add the voice of my six-year-old constituent? She would not benefit from Orkambi, but we must recognise that, if we do not get this right, the other treatments that might help her will be subject to similar delays. She is only six years old, but she does not need any delay. Does my hon. Friend agree that this matter is not only about Orkambi, but about how we deal with life-changing drugs and whether we understand the power of them to make such a difference?

[Tulip Siddiq]

I absolutely agree with my hon. Friend. There are lots of heart-breaking stories from people who are six and even younger who share the frustrations of the people I am speaking for today.

Caroline, my constituent, was adamant that Orkambi is just one frustration that those with cystic fibrosis have over how the current range of treatments are made available. What aggravates my constituent is the fact that cystic fibrosis is the only incurable life-threatening disease where people have to pay for their prescriptions. I greatly sympathise with her view that charging patients for medication, in light of the fact that they often undertake between three to five hours of treatment a day, does not seem to square up to the rhetoric of Ministers who pledge their support for the cystic fibrosis community. As my constituent concludes, some people have to take thousands of pills over their lifetime, so paying for them makes life very difficult. Why should cystic fibrosis be an exception when people with other diseases do not have to pay?

I will close with three questions that I want the Minister to answer. Do the Government accept responsibility for the pace at which negotiations are being conducted, and will they apologise for the anxieties that patients are experiencing as a result? Secondly, what steps are Ministers taking to ensure that pharmaceutical companies will ensure that fair and responsible pricing for a deal can always be agreed when treatments are required on the NHS? Finally, will the Minister acknowledge the importance of finding a solution that guarantees we are never put in this position again for the future pipeline of treatments for cystic fibrosis and many other life-threatening conditions?

18:08

[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.

[James Heappey (Wells) (Con)]

My hon. Friend speaks eloquently about the many parents and children who have been campaigning for this cause. I add to them the voice of Helen Mann, my constituent, who has written the most wonderful, compelling book about her daughter’s life with cystic fibrosis and, indeed, her treatment with Orkambi. Her daughter, Clara, came to knock on my door yesterday lunchtime to make sure that I came to today’s debate. It was amazing to see her stood on my doorstep so full of life—a living example of just what an amazing drug it is, and how many others could benefit if only it were available to all.

[Luke Hall]

I pay tribute to all the young people such as Clara who are getting involved and making a difference in the campaign to ensure that Orkambi is available.

I was probably as disappointed as every Member in the Chamber to read the statements that came out on Friday about how a deal on the February offer by Vertex had not been reached. The petition for today’s debate was signed by 470 people from my constituency. I am here speaking on their behalf, and on behalf of Annabelle Brennan, and her parents Elizabeth and Shane. Annabelle is three years old, and is a lovely young girl. I met her at a CF campaigning event in my constituency. She does things that other three-year-olds do, of course, but she also has to endure a complex daily routine of physiotherapy, nebulisers, medication and antibiotics to help her to manage and live with the illness.

I received an email from Annabelle’s nursery leader, who is also my son’s nursery leader, explaining that Annabelle’s time at nursery, and her interaction with the other children in the group, is affected by her condition, because she is under constant supervision and is not always able to be involved in all the activities like the other children. Every winter, there is an added risk of further infection due to the common cold, and, as time passes, the potential for more complications increases. I remember hearing a devastating description from Annabelle’s parents of how every day is like waking up on a cliff edge, not knowing when irreparable damage caused by CF will come. That highlights the importance not only of these debates and the pressure, but of getting a deal, so that Orkambi can be made available as quickly as possible. That would ensure that children such as Annabelle, and people living with CF throughout the country, can realise their full potential.

A number of colleagues have laid out evidence today that shows that Orkambi works. We heard about Carly’s story from the hon. Member for Dudley North (Ian Austin)—I thank him for all he has done. We have seen international examples of the difference that Orkambi has made, but the reality is that progress on reaching a deal has been devastatingly slow. We know that Orkambi can help about 50% of people with CF. The other point we should remember is that precision medicines such as Orkambi are not just the better option—for some people, they are the only option. People have stated the countries around the world where it is already available: Austria, Germany, France, Ireland, Italy, the USA, Luxembourg, the Netherlands, Greece, Denmark, and many more. Despite the prevalence of CF in the UK, it is still not available here.

The set of statements released on Friday was disappointing, but although an agreement has not been reached, that must not be the end of those discussions; they should continue, ensuring that a deal is reached as soon as possible. It is also important that NHS England ensures that any deal is sustainable in the long term, and flexible enough to enable us to take into account any future advances in CF medication, either by Vertex or another company. The aim of NHS England and the National Institute for Health and Care Excellence should be to negotiate the best possible long-term access for people living with CF. That has to be reflected in the deal.

I ask the Minister to convey to both Vertex and NICE the importance of ensuring that a deal is reached as quickly as possible, and I encourage him to take a seat round the table himself, so that Annabelle and other people around the country have access to these drugs. I also ask him to update us on what conversations his Department has had with NHS England and Vertex since the announcement on Friday to see where progress is likely to be made in the coming weeks and months. I finish by saying to the Minister, NHS England and Vertex, “Can we please ensure that a deal is reached as quickly as possible, because this drug has the potential to change thousands and thousands of lives?”

18:09

[Patricia Gibson (North Ayrshire and Arran) (SNP)]

The development of the treatment offered by Orkambi has offered renewed hope to those living with cystic fibrosis and their families. Orkambi is suitable to treat around 50% of those with cystic fibrosis in the UK and, as we have heard today, that treatment can now be extended to those from aged six years, as opposed to the original licence, which was to treat those aged 12 and over.

We have heard today at some length about the controversy over the cost of the drug. As a Scottish MP, I was heartened by the Scottish Government Cabinet Secretary for Health and Sport, Shona Robison MSP, who strongly encouraged the manufacturer Vertex to take forward discussions with the patient access scheme assessment group secretariat. Those conversations continue. It is easy, and perhaps obvious, to point out the high cost of the drug—we have heard much about it—but who in this Chamber would not stretch every sinew for their child or loved one to access the drug if they could benefit from it? That is why parents and relatives of those living with cystic fibrosis have been campaigning so hard. What else can they do?

We have heard about the cost of the drug, but as we have also heard, the costs can be offset by reduced hospital admissions. NHS funding will always be under pressure as our technology advances. One consequence is that the cost of treatment is always likely to be driven upwards. Costs are important, and we have to be mindful of them, but I ask hon. Members, “If your child or loved one could benefit from a treatment, however expensive, what would you not do to help them to access it?” That is what drives those campaigning for access to Orkambi.

The campaign has been effective and moving, as we have heard. We must keep listening and empathising with what those people are saying, because we cannot just talk about pounds and pence when it comes to treatment. We are dealing with real lives, which must guide progress. Progress must be made—we all hope that happens sooner rather than later.

[Bob Stewart]

My point is very straightforward. It seems grossly unfair that some people can get hold of this drug and some people cannot. We ought to level the playing field.

[Patricia Gibson]

The hon. Gentleman makes an excellent point. That is why in Scotland we are setting up bodies to ensure that care and access to treatment is standardised across Scotland, and that it does not depend on the health board where someone lives or what route they can take to access the drug.

We heard from the hon. Member for Dudley North (Ian Austin) that there is a cost in not funding Orkambi. We have to keep that in mind, and find a way forward as soon as possible. We clearly need a more cost-effective proposition from Vertex, the production company. A constructive solution must be worked out with the Scottish Medicines Consortium, with an equivalent solution found for the English NHS. I am hopeful that a solution can be reached in Scotland and across the United Kingdom.

However, as we have heard, it is important that the processes about decisions for the approval of drugs are transparent and fair, and that we can be confident that a proper and extremely robust cost-benefit analysis is carried out. We hope that a way forward can be found and that progress can be made with the pharmaceutical companies about the cost of treatment—in particular, with Vertex and the cost of Orkambi, which can make a difference to the many people living with cystic fibrosis and their families. We owe it to them to do all we can to work with the pharmaceutical industry to find a way to make this happen. I sincerely hope we can do that.

18:20

[Crispin Blunt (Reigate) (Con)]

I join colleagues in congratulating my hon. Friend the Member for Sutton and Cheam (Paul Scully) and the hon. Member for Dudley North (Ian Austin) on promoting this cause and on drawing parliamentary attention to it in an extraordinary way in this debate. I have a constituent, Sharon Cranfield, whose 16-year-old daughter, Jessica, has cystic fibrosis. This is about the grassroots and all those people who are affected. She is but one of those who have done an amazing job in promoting this petition and in campaigning for access to Orkambi for the 3,000-plus people who could benefit from it in England.

Last summer, Jessica was given access to Orkambi on a compassionate use scheme for those with the lowest lung function, and her quality of life has been transformed. The medicine reduces the mucus that builds up in the lungs, causing infection and damage. She is already benefiting from fewer exacerbations, which, before the treatment with Orkambi, caused hospitalisations about four times a year for up to a month at a time, taking her out of school. Judging by stories such as Jessica’s and the growing body of evidence that shows that the medicine is working effectively to keep children and young adults what cystic fibrosis out of hospital, there is a clearly a very strong case for making it available on the NHS.

Although many other countries have moved to reimburse this medicine in their healthcare systems, in the UK Orkambi remains unfunded more than two years since it received its European licence. The UK is falling behind our OECD peers in terms of speed of adoption, overall uptake, and expenditure on the newest medicines, and it is becoming increasingly apparent that the ways in which NICE assesses the cost-effectiveness of medicines are outdated and insufficiently flexible for precision medicines for rare diseases with relatively small patient populations, such as Orkambi.

[Bob Stewart]

On my good friend’s excellent point, I wonder how much it costs to put a child in hospital for a month. I am pretty sure it is not far off £100,000.

[Crispin Blunt]

My hon. Friend leads me to my next point. How do we wish to value better and longer life for those with cystic fibrosis? How do we wish to value the savings to the NHS of reducing the 9,500 hospital admissions and the 100,000-plus hospital bed days a year? How do we wish to value the societal and economic benefits of helping young people grow up to play a fuller part in their education and employment, and the benefits to their families and care givers? How do we wish to value and reward the risk and innovation that goes into researching and developing breakthrough medicines?

NICE applies rigid health economics methodologies through its standard technology appraisal, which is designed not for rare diseases and specialised services but for primary care medicines that treat large populations with well-known diseases. Are we content for NICE to apply a threshold for valuing life that has not changed since it was established in 1999, even though healthcare inflation has almost tripled what we spend on healthcare?

My right hon. Friend the Member for Chingford and Woodford Green (Mr Duncan Smith) referred to social impact bonds and payment by results. I do not envy those who have to make those evaluations, such as the Minister and NICE. The costs fall on NICE and the health service budget, and the benefits are often felt elsewhere, not least in patients’ quality of life. Our society and the Government have to become more sophisticated about early investment. We must be able to measure the savings that come from having fewer hospital admissions and from the greater contribution to society that people who suffer cystic fibrosis will make if we improve their quality of life and reduce the degradation of their lungs.

Does the Minister accept that, although NICE has a specific evaluation process for highly specialised technologies for ultra-rare diseases, it is missing a framework for other rare diseases and precision medicines that treat sub-groups of rare diseases? He will no doubt point to initiatives such as the accelerated access review and the sector deal for the life sciences industry. I welcome those schemes in so far as they aim to address some of the access challenges, but they count for little if there is not a willingness to find innovative and flexible approaches to introducing innovation in the NHS. If we get this right, the UK has huge opportunities better to serve NHS patients and attract industry investment in clinical trials.

Vertex, the manufacturer of Orkambi, recently proposed a portfolio arrangement to NHS England, as we have heard from many colleagues, whereby all its current and future cystic fibrosis medicines could be made available to eligible patients at a fixed cost to the NHS, irrespective of the number of patients treated. Vertex wants to work with NHS England and NICE to put a long-term arrangement in place, as it has already done in Ireland. As the company introduces new medicines and line extensions, patients will get rapid access to the most suitable products for them upon regulatory approval, and the NHS will have budget certainty. Vertex expects to develop therapies that will treat 90% of cystic fibrosis patients within seven years.

In the light of the company’s proposal and the strength of feeling expressed in the petition and by colleagues in this debate, will the Minister look at mandating NHS England and NICE to prioritise discussions with Vertex to find an innovative and sustainable funding solution? It gives me hope that the offer that Vertex found unacceptable last Friday included a possible portfolio approach. It failed simply because NICE was not prepared to increase the resources it already pays for existing drugs, not including Orkambi. That plainly meant that the offer could only be unacceptable to Vertex, given that it is proposing new treatments that are going to treat 10 times as many patients as are being treated by the drugs currently available.

This is urgent. As my constituent, Sharon Cranfield, said to me:

“Each day of delay is additional delay of ‘irreversible lung damage’”

for those with treatable cystic fibrosis.

18:27

[Gerald Jones (Merthyr Tydfil and Rhymney) (Lab)]

It is a pleasure to serve under your chairmanship, Sir Roger, as we debate a petition to support patient access to Orkambi. I congratulate the hon. Member for Sutton and Cheam (Paul Scully) on making a powerful case. I also congratulate members of the Cystic Fibrosis Trust on all the work they have done to bring about this debate.

Cystic fibrosis is a hugely debilitating illness that has a massive impact on individuals’ quality of life. Research shows that Orkambi can slow down the decline in lung function, which is the main cause of death for people with cystic fibrosis. About 3,300 people in the UK could benefit from the use of that medication, including 118 people in Wales. Those research figures are from 2014, and I know they have been updated somewhat.

We have heard that many of our European neighbours, including Ireland, support the use of this medication, as do other countries across the world, such as the USA. I represent Merthyr Tydfil and Rhymney—a Welsh constituency. I am speaking for 170 of my constituents, including Mr and Mrs Bow, whose seven-year-old daughter, Sofia, lives with cystic fibrosis. At that tender age, she already takes 30-plus tablets a day. Over and above the personal impact on Sofia, that medication constitutes a cost.

CF is a debilitating condition that restricts individuals’ employment opportunities and their capacity to contribute to society. My hon. Friend the Member for Dudley North (Ian Austin), who has done so much to highlight the need for action, mentioned earlier in the debate that it is important for us to look at the bigger picture and the bigger cost, rather than only at the cost of the medication itself—at savings to the NHS in hospital admissions, treatment and medication, or reduction in tax revenue from restricting the opportunities for people with cystic fibrosis to contribute to society. The wider cost needs to be considered against the cost of not licensing this medication.

As a Welsh MP, I fully appreciate that a decision on the medication in Wales will depend on the view of the Welsh NHS and the All Wales Medicines Strategy Group. However, the outcome of today’s debate and any agreement between NHS England and Vertex will undoubtedly have an impact by setting the tone in the other regions and countries of the United Kingdom. In Wales, the strategy group has encouraged Vertex strongly to make a submission for appraisal. Apparently Vertex has agreed, but it has not yet agreed a date, which we hope will be soon.

As we have heard, the latest discussion between NHS England and Vertex, released on Friday, did not seem to go well, which has left cystic fibrosis sufferers throughout the UK and their families hugely despondent. Progress and answers are needed to offer comfort and support to families. It is time for the Secretary of State, or indeed the Minister, to take hold of the situation and commit to making progress. This has gone on for far too long and time is of the essence. I hope the Minister achieves progress in this long-standing situation—it has been a good three years. It seems to have stagnated, and we need more urgency.

I agree with the hon. Member for Cheltenham (Alex Chalk) on the need for pricing to be realistic, but we are talking about life-saving, life-changing drugs, and the cost of research needs to be covered. We also need to ensure that the companies are not looking to profit unduly from their medication, because far too much is at stake. I hope progress can be made and that the Minister will commit to ensuring action sooner rather than later.

18:32

[Mr Ivan Lewis (Bury South) (Ind)]

It is always good to see you in the Chair, Sir Roger.

I congratulate the hon. Member for Sutton and Cheam (Paul Scully) not only on securing this important debate but on his excellent opening contribution. I also pay tribute to my hon. Friend the Member for Dudley North (Ian Austin) for his work on this issue over a long period. Most of all, like other hon. Members, I thank the Cystic Fibrosis Trust—families up and down the country have come together in such a passionate and effective way to ensure that this House takes this life-and-death issue as seriously as it needs to be taken.

At the outset I want to acknowledge that it is entirely right that we have in place a system to determine objectively the effectiveness and relative value for money of new drugs. That is a policy area in which we should seek maximum consensus across the political divide in the national interest and in the long-term interests of patients and their carers. NICE does a difficult job in very challenging circumstances, and in no way should we or do we seek to undermine its work. However, any system of that nature can get it wrong or delay decisions when there is no time to waste, whether through pressure of work, changing evidence or, on occasion, a lack of nimbleness and flexibility. Sometimes pharma companies seek unreasonable commercial arrangements that have to be challenged—I am not saying that that is so in this case, but it is important for us to understand that that happens and that we always have a responsibility to ensure that we get fair value for money.

The present system truly has insufficient flexibility to take account of the true benefits of a portfolio approach. As other hon. Members have said—we can judge this from their contributions today—we may be politicians, but we are also citizens, whose human instincts are to speak up for people with life-threatening and life-limiting conditions. In any such debate, we should ask the question, what support would we expect if a member of our family or close personal friend were denied life-enhancing or saving treatment? We would not want to listen to arguments for why that was simply not possible.

I first became aware of Orkambi as an issue when a young constituent and her mother came to my constituency surgery. Alex is 10 years old and suffers from cystic fibrosis. She is a remarkable girl, whose courage and positivity are truly inspirational—that would apply to many of the young people we are here to talk about today. However, this case is perhaps a graphic example of what can happen very quickly. Alex started this year with 80% lung function, a number that at the time, understandably, scared her mother, Emma, because a few months previously it was at more than 100%. Now, Emma would give anything to see the number 80 again. Alex’s lung function continues to drop and is now at around the 54% to 56% mark.

Alex has physio and takes a large amount of medication just to manage the condition, including intravenous antibiotics every three months. Similar to what other hon. Members have rightly described, her daily life is inevitably dominated by her drug and physio regime. That is a quality-of-life issue on a day-to-day basis, as well as a health issue. Sadly, Alex now has irreparable lung damage. Recently, Alex’s consultant advised that they are running out of options with regard to the medication, explaining that had Alex been a year older—this demonstrates how random some of the decisions can be—her parents would be able to apply for Orkambi on compassionate grounds, because her lung function had deteriorated by more than 25% in less than three months. Compassionate use in the UK is used only when a patient is extremely ill, however, and NICE is not scheduled to look at Orkambi again until July 2019. But we now know that early use enables better management of the condition and reduces further complications. Emma and Alex contacted me because they cannot afford to wait another year to get the drug on compassionate grounds.

I say to the Minister and others that we talk a lot about the centrality of prevention and early intervention but in reality, when we make decisions such as this one, we do the opposite, and when public services face the kind of financial pressures that they face these days—whether that is local government services or the NHS—they resort to fulfilling core functions, statutory functions. In the real world, it is almost nonsense to talk about a shift to prevention and early intervention. This is an example of where that is crucial, in a very direct way.

Emma is understandably frustrated. Her daughter has spent her entire life fighting to stay well, and now she has to fight for a drug that could help her to feel better. For Alex, as I said, Orkambi would mean a slowing in the decline of her lung function, giving her control of her condition and a better quality of life. As Emma, her mum, movingly said:

“Alex has a real love for life and all she wants to do is live a long and happy life without having to fight to breathe.”

I would say this about disabled children and their families more generally. I coined a phrase: why do we have to constantly fight the system when we should have a right to have a system that is on our side. I have been involved in working with disabled people for about 35 or 40 years, and I am sad to say that that is the reality for most families with disabled children today. It has been the reality under successive Governments, although we tried to improve investment. We certainly made progress on stigma and in recognising that disabled children should have the right to fulfil their potential and to be treated like any other child, for example, but the constant battle every single day—when, frankly, people have enough to cope with, whether as a child or as a family—is something we should all reflect on.

Along with other hon. Members, I strongly support the call for NHS England to engage in a meaningful dialogue with the drug company and to reach a fair settlement. Having sat in the Minister’s place, I can predict that officials will say, and this is not a criticism of the officials, that we should stay out of this; that it is not a matter for Ministers; that there should not be political interference; that we have a robust system that exists and is in place; and that we should let that system run its course. Ministers may end up allowing human emotion or politics and pressure from hon. Members to influence them to make decisions that are not always consistent with what the Department wants them to make. I say to the Minister that that is nonsense. Ministers have a duty to intervene in circumstances such as this, to be a facilitator and a convener, and to bring those parties round the table and say that it is not acceptable for this situation to remain unresolved for much longer.

My hon. Friend the Member for Bury North (James Frith) said that we do not want to be back here in six months’ time, having a debate such as this one in exactly the same circumstances. Many countries around the world have reached a different conclusion and have made a different decision. Obviously, they believe that this drug makes a massive difference from a health and quality of life point of view, and that it is economically effective. I ask the Minister to please use his convening powers, his accountability to Parliament, and the desires and the will of parliamentarians as expressed today, to apply immediate pressure to the relevant parties.

18:41

[David Linden (Glasgow East) (SNP)]

It is a pleasure to serve under your chairmanship, Sir Roger. I start by thanking the 484 constituents in Glasgow East who signed the petition. The hon. Member for Sutton and Cheam (Paul Scully) made an excellent speech and was very courteous in trying to keep his speaking time down to let other Members speak. It is a pleasure to see my hon. Friend the Member for Motherwell and Wishaw (Marion Fellows) back in Parliament and on the Front Bench.

As a new Member of Parliament, the first time that a constituent came to me to raise cystic fibrosis, I am quite ashamed to say that I did not know a lot about it. I was very grateful to Karen Ashe, a constituent from Mount Vernon, who explained to me the difficulties that her daughter, who is just 14 years old, experiences, being admitted to hospital every eight weeks, and the real challenge that that brings. She impressed upon me the importance of why we need Orkambi now.

Even since the debate started, another constituent, Naomi Moore, has tweeted at me—that shows the good thing about us engaging digitally—because I said that I was taking part in the debate. She said:

“With access to amazing drugs like Orkambi/Kalydeco, I’ll be able to use my degree when I graduate. Without it, my future is uncertain and limited.”

She is a girl in Glasgow who is in her fourth year at university, studying geology. That is a very powerful point. The turnout of Members at the beginning of the debate—I must say I am quite disappointed that so many have disappeared—impresses upon us the importance of getting this right.

[Alison Thewliss (Glasgow Central) (SNP)]

My hon. Friend makes the point about young people and their future, which is a big part of the debate. Robert Kennedy and Ashley Wilson from Dalmarnock came to speak to me about their three-year-old daughter, Mirren. She goes for physio twice a day for her cystic fibrosis, but they fear that as she gets older, she may face weeks in hospital at a time. Does he agree that Orkambi ought to be there for them, so that they can access that treatment as their daughter gets older?

[David Linden]

Absolutely; I know that my hon. Friend is a passionate champion of her constituents in Glasgow Central and she is right to put that on the record.

Numerous other constituents have come to me, including Lee Bennie from Garrowhill. Again, she made the case that we need Orkambi now. Over the course of the weekend, I had the privilege of spending some time talking to a friend’s brother, Ross Moore. He is not a constituent of mine, but he has access to Orkambi through the compassionate access scheme. Ross is a remarkable young man who is incredibly articulate. I was quite struck by how frank he is; he has lived with cystic fibrosis for so long and I was very moved by the way in which he could explain why he has access to Orkambi and why he thinks that other people should have access to it.

Ross was moved on to Orkambi only in October last year because his lung function had dropped below 45%. He was quite honest in saying that the first six to 10 weeks were very tough; for some people that can be make or break, but he has got through that. My hon. Friend the Member for Glasgow Central (Alison Thewliss) has mentioned people’s ability to try to put that effort into physio, and Ross said that it is a very arduous process. He does physio for two to three hours a day. When speaking to him, I was struck that he already has access to Orkambi, and he does not want to pull up the ladder behind him. He said to me before I came into this Chamber, “Go in there and explain the benefit of Orkambi; we need to make sure that people understand that for those who do not have access to it already we have got to have that access, because it is only through that research and that ability to use it that they will have it.”

[Bob Stewart]

Does that mean that the hon. Gentleman’s constituent will have Orkambi for life, or will it be taken off him later?

[David Linden]

While I was having that conversation with Ross, that was at the back of my mind. I am very glad that he answered before I managed to ask it, because it is very difficult to ask somebody, “Will you have access to this for the rest of your life?” Thankfully, in Ross’s case, he will.

My hon. Friend the Member for North Ayrshire and Arran (Patricia Gibson) outlined very eloquently the situation in Scotland; for the purposes of time I do not want do that. I had a phone call when I left Glasgow airport this afternoon from my constituent, Helen Seery, who lives in Carmyle. Her son, Layton, was born just 22 months ago and has cystic fibrosis. The 20-week scan showed that he would have cystic fibrosis; he was born prematurely at just 28 weeks. His lungs are naturally weaker as a result of being a premature baby. My constituent Helen made the point about what a difference it makes in Ireland, where people have access to Orkambi at five and six years old. I will visit Layton in Carmyle next week. I am aware that it is a challenge for Helen to bring somebody into the house due to the risk to Layton.

It is important that we stand up and make speeches and that we will send the Hansard to the constituent afterwards, but the most important thing for me is going back to Helen and telling her and Layton that I stood up and said that I think that Orkambi should be available in Carmyle, as well as Carlisle. That is the message that we have to leave with the Minister. Constituents are absolutely clear that we need Orkambi now.

18:47

[Rachael Maskell (York Central) (Lab/Co-op)]

I want to start by thanking the 75 constituents of mine who signed the petition, not least the inspiring parents and patients who have really brought this debate to the fore. I want to mention Dr Aaron Brown, who is based in York and is living proof of what it is to live with cystic fibrosis. He was the first person with cystic fibrosis to graduate to the Royal College of Surgeons and is a GP in York. However, for him, living with cystic fibrosis asks many questions, not least as he has a young son and a child on the way. He says,

“Having CF has given me something to battle against all of my life, to prove that it won’t get me and I can achieve.”

We know that this debate is not just about the impact of cystic fibrosis but the hope that a drug such as Orkambi can bring. Although the drug may not be a panacea to all things, it certainly does slow down the disease process, including scarring of the lungs and deterioration of the lung tissue. It is also paving the way for a new generation of precision medicines. It is so important that we are at the leading edge of that research to give people real hope for the future, not just the 50% of people who will benefit from precision medicines should Orkambi be approved, as it must be, but for the next generation.

In the next five years there is potential for 13 new drugs and in seven years, 18 new drugs, impacting on 90% of the cystic fibrosis population. What an amazing opportunity that presents us. That is why we are resolute to make sure that the Minister moves on the issue.

We have heard a lot about cost, but I want to put a cost in everybody’s mind. We are talking about £285 a day. Let us think about the cumulative impact and cost of hospitalisation, perhaps of transplants, drugs and economic loss. We are talking about just £285 a day, a figure that Ministers of State will be familiar with because it matches how much they earn. We can recognise that this is a cost that the NHS can and must afford. Even if there are final negotiations to be had about a portfolio of medication, which is important, to ensure that we usher in this new generation of drugs, what is the cost of a life? I leave that question in the air. I am sure we would all argue that it is a lot more than £285 a day. If we are determined to save lives, that is affordable.

I worked as a physio in respiratory medicine for 20 years, so I have first-hand experience of making this journey with so many patients. The physio may not be pummelling, but it is certainly intense. I celebrate the way that physio has developed over that time. It has become much more dynamic, rather than passive, and a number of techniques have been developed. We heard about active exercise—that is so important for building lung capacity—positive pressure techniques and postural drainage to ensure that we minimise stress on the lungs as we clear secretions. Just think what a life-changing opportunity those advances in physiotherapy, as well as a good diet and precision medicine, present for people with cystic fibrosis compared with their opportunities just a few years ago.

We have a real opportunity to change people’s life chances, but systems have got in the way, and that is what we plead with the Minister to change. The European Medicines Agency approved these drugs two years ago. It is therefore incumbent on us to stress to the Minister that we need effective movement, quickly. NICE itself recognises the benefits of the drugs: in July 2016, it called them important and effective treatment. So much time has been lost through so-called negotiations.

The Minister cannot hide behind NHS England or other bodies. It is time for leadership. It is time for him to step up, step into the debate and ensure that access to the drugs is delivered. I plead with him to call Vertex, to ensure that discussions are held with NICE and NHS England before Easter and to get the table set for finalising the negotiations. If a portfolio of drugs can be achieved in Northern Ireland and the Netherlands, it can be achieved here. Even if there has to be an interim agreement to ensure that people with cystic fibrosis have immediate hope, it is his responsibility to deliver that. With each day that passes, people’s future lung capacity, and therefore their lives, are seriously impacted.

Of course, some people already have access to these precision drugs, and the outcomes for people who benefit from access to Orkambi are really positive. It really is unethical and both economically and clinically illiterate to make Orkambi available to people who are seriously deteriorating but not to people who could stop their lungs being damaged in the first place if they had access to it in advance.

I am grateful to the many constituents who contacted me, but I want to relay the words of Abigail, who wrote very passionately. She has children aged four and seven, both of whom have been diagnosed with cystic fibrosis, and she expressed the real hope that these drugs would bring:

“Those drugs…are here now, which makes it even more heartbreaking that Orkambi, which could benefit 50% of people with CF, is not available on the NHS. To know that there is a drug out there that could change my children’s lives and allow them to live longer and in better health, but which they have been denied access to is absolutely devastating…simply because of cost reasons…It is absolutely imperative that some kind of agreement is reached that makes these drugs available to everyone with CF who could benefit from them. Living with CF is already difficult enough, please don’t make it any harder.”

18:54

[Vicky Foxcroft (Lewisham, Deptford) (Lab)]

It is a pleasure to serve under your chairmanship, Sir Roger.

Like many other right hon. and hon. Members, I was inspired to take part in the debate by two constituents. The first told me about his younger sister, who has cystic fibrosis. She has always been very healthy, but she is now in her 30s and reaching the point at which she will need to use additional medications. As he was emailing me, his sister was on her way to hospital to undergo an operation to put a port in her chest so that she could receive intravenous antibiotics. He ended his email by saying:

“I understand that the NHS is under pressure, but this illness is one of the beatable ones”.

The second message I received was from the parents of a 10-year-old girl named Ruby, whom I met soon after I was first elected as an MP when she came here to see me as part of the environmental change lobby. Ruby is a bright and optimistic girl who loves science and maths, swimming in the sea and making emojis to send to her friends. However, unlike her contemporaries, she has to take more than 30 tablets a day to help manage her condition. She also has to nebulise further drugs and have physiotherapy every morning and evening. Her condition brings with it regular visits to hospital for scans and tests.

Although Ruby copes brilliantly with her condition, her parents know that with each passing year, the likelihood of her health declining increases significantly. As well as the obvious effects cystic fibrosis will have on her lungs, it carries a vastly increased risk of diabetes, liver disease and chronic digestive problems. As children with cystic fibrosis grow older and begin to realise the condition’s potential impact on their future, it takes a major toll on their mental health. Ruby’s parents worry about that continually. Knowing that they might live only to their mid-30s is an incredibly heavy burden for a young person to carry. It is not difficult to see that being able to access Orkambi would have a profound effect on children such as Ruby by slowing the progression of that cruel disease, if not stopping it completely.

I will leave hon. Members with a few words Ruby’s father wrote to me that perfectly sum up the impact that access to Orkambi would have on families such as his:

“Whilst it is Ruby who carries by far the most weight of her cystic fibrosis, a drug like Orkambi has the potential to free all of us in her family – and the families of so many other children with cystic fibrosis – from the sometimes overwhelming worries about the future – knowing that next week, next month, next year, a new infection or new problem could arise and the downward spiral that has taken the lives of so many with cystic fibrosis could begin.

We know society has to balance the cost of treatments for all with the needs of individuals. We know society has difficult choices to make. But we believe there is a resolution that can do just that, by bringing together the brilliance of science and a health system that is, and always should be, free for all. Enabling access to treatments like Orkambi would literally change our daughter’s life for good.”

18:58

[Marion Fellows (Motherwell and Wishaw) (SNP)]

It is a pleasure to serve under your chairmanship, Sir Roger.

Where do I start to sum up this debate? It has been a truly awesome experience, and I congratulate and thank all the Members who took the time to attend. I have rewritten my speech all the way through the debate, which I will try to summarise as best I can.

The petition was signed by 371 of my constituents. As has been said, the general public care about this. I thank the Butterfly Trust, which is the CF charity in Scotland, the Cystic Fibrosis Trust and the CF support group for parents in Scotland for all the assistance they gave me to prepare for the debate. We heard many passionate and informed speeches. There is true cross-party agreement. We heard how CF sufferers could benefit from Orkambi and how it could transform their lives.

I must declare a personal interest. My 16-month-old granddaughter Saoirse was diagnosed with cystic fibrosis shortly after she was born. On hearing about the debate, my daughter-in-law enlisted all of her friends and family to sign the petition, even though she knows that Orkambi will not help her daughter. Vertex is developing new treatments all the time, and the use of Orkambi can only help that research.

Saoirse is lucky—she is pancreatic sufficient, which means she can digest food—but she is on daily antibiotics and additional treatments. As a grandmother, watching her going through physiotherapy is heartbreaking. Her parents were told that they must not comfort her as she needs to get used to it, because she will need physio for all of her life. She cried, and I cried. Her grandfather, who is here today, cried, too. However, she is getting used to it. CF sufferers and their families are amazing people—I discount myself from that. They care about each other. The proof of that is here today, in more than 100,000 signatures and so many Members taking part in the debate.

I pay special regard to the hon. Member for Dudley North (Ian Austin). I apologise that I could not attend his roundtable—I have serious family illness at the moment. The fact that so many Members attended shows how important this is to both Members and their constituents. We do things not just because we want to, but to help our constituents. He talked a lot about the cost of Orkambi and how we should look better at cost-benefit analysis. We should also look at the contribution that CF suffers could make, were they able to. We must all deeply reflect on that. As he said, when an everyday drug is denied to CF suffers, that is another day when their lung power is damaged. We cannot allow that to continue.

As one of my colleagues stated, the Scottish Government are working hard to improve access to drugs such as Orkambi through changes to the individual patient treatment request to ensure that patients in Scotland get access to the right treatment at the right time. Orkambi could be accessed through that, but the Scottish Government’s role is to try to ensure that everyone in Scotland gets equal treatment, which is important all over the UK. There should be no postcode lottery for treatment.

[Mr Sweeney]

Does the hon. Lady agree that the Scottish Government and the UK Government should consider an interim acceptance period for drugs under active price negotiation when organisations such as the Scottish Medicine Consortium have agreed that there is a clinical benefit? That would be a good way to ensure that people get immediate access. Two years is two years too late for many people.

[Marion Fellows]

I agree with the hon. Gentleman. We are talking about people whose lives are shortened and people whose lives are deeply affected. These people could be of such benefit to society as a whole. It is difficult for me to think beyond my granddaughter, and it is also difficult to listen to stories of what might happen. I know she is lucky, but I also know and am passionately concerned about other children who are even more affected by CF than she is. We owe it to them and to people who will inherit CF in future.

I have to be a carrier of the CF gene, and my husband must also be a carrier. We were so lucky that none of our children were affected. It is a terrible, silent disease. The fact that CF sufferers cannot even get together for comfort and mutual support—it all has to be done through parents’ groups—is even more heartbreaking.

[Bob Stewart]

I am deeply affected by listening to the story of the hon. Lady’s granddaughter. When did the hon. Lady and her husband know that they were carriers? That is pretty important.

[Marion Fellows]

I probably am a carrier—unfortunately, for another reason, we have not been tested yet. That is another issue that we will deal with. It would help future research. Actually, after my daughter was tested, she said to me, “Mum, you and dad must go out and buy a lottery ticket—you must be the luckiest people around,” because I have three children and none of us has CF.

The Scottish Government are encouraging patient representatives to participate in Scottish Medicine Consortium meetings. That must be a good driver to let clinicians and other medical people see with their own eyes the difficulties undergone by people who cannot access these drugs.

Sir Roger, you have been most considerate, and I will now sum up. There is an overarching need for a change in the way in which new drugs are approved for use. Cost-benefit analysis needs to be used to calculate the true costs. For example, we should look at reductions in hospital care costs and include the benefits to the economy from the use of these drugs—especially Orkambi, which is our exemplar for now. Of course, CF suffers must be able to play a full part in society and, as we have heard, they could do so if they could access drugs.

Orkambi could be just the start of a whole line of drugs that will benefit CF suffers. No one can cure the disease at the moment, but such drugs will lead to so many more better and productive lives. Yes, there are issues around pricing new drugs. Pharmaceutical companies are not there for charitable purposes, and we all know and understand how expensive it is to create new drugs, but there must be a better way of pricing the cost of new drugs at their introduction across years of their use rather than huge up-front costs.

I was asked by the Scotland parents support group to demand that the Minister takes responsibility for the negotiations with Vertex, and that he brings NHS England and Vertex Pharmaceuticals to the table. Deals and negotiations should never be conducted by email. I am happy to do so, and I wait with interest for his response.

19:07

[Mrs Sharon Hodgson (Washington and Sunderland West) (Lab)]

It is a pleasure to serve under your chairmanship, Sir Roger. I thank the hon. Member for Sutton and Cheam (Paul Scully) for his excellent opening speech, and I thank the 114,000 people who signed the online petition to enable us to debate it. As we have heard, the petition received more than 107,000 signatures in just 11 days—probably a record for such a petition—which shows how important Orkambi is to people with cystic fibrosis and their families.

I also thank my hon. Friend the Member for Dudley North (Ian Austin) for his sterling campaigning on this issue over a number of years, for his passionate speech and for organising the excellent roundtable in Parliament. I was unable to attend the roundtable, but a member of my staff took extensive notes and briefed me fully. I also thank all hon. Members who spoke in the debate. I was going to list them all, but so far there have been 23, and with me and the Minister that will make 25, so I will save everyone from the roll-call. Indeed, at the start of the debate it was standing room only, which shows the strength of feeling on this issue across the House. We have heard very moving accounts about family members and constituents, and that alone should be more than enough to make the case for Orkambi to be made available on the NHS as soon as possible. Finally, I thank the Cystic Fibrosis Trust for its continued “Stopping the Clock” campaign and for all the work that it does to support people with cystic fibrosis.

As we have heard, cystic fibrosis affects about 10,400 children and adults in the UK today. It affects one in 2,500 people, and one in 25 of us carries the gene that causes it. It is a life-shortening genetic condition, with the median survival for an individual with cystic fibrosis currently at just 47 years. Patients with cystic fibrosis therefore have to spend three to five hours every day on aggressive and indiscrete physiotherapy, and need nebulised treatments and strong antibiotics just to keep well. The medicines tackle the symptoms of cystic fibrosis rather than the root cause. That is why new precision medicines such as Orkambi will change cystic fibrosis care for the better.

Kalydeco is the first precision medicine used in this country for people with cystic fibrosis. It has transformed the lives of those receiving it. Patients report no longer needing a tank of oxygen to support their breathing, and greater health stability, so that they can plan their lives more securely. Some are taken off the transplant waiting list, as their clinical status improves dramatically. However, it works for only one in 20 people with cystic fibrosis. In comparison, Orkambi works for eight in 20 people. About 50% of individuals with cystic fibrosis in the UK have the genetic mutation that Orkambi tackles, so the approval of Orkambi for use on the NHS could benefit about 5,200 people living with cystic fibrosis.

Orkambi has been shown to slow decline in lung function by 42% and cut the number of infections requiring hospitalisation by 61%. It gives patients not only more control over their lives but a greater quality of life. Orkambi is available for patients in nine other countries: Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Greece and the United States. Yet here in the UK, NICE has deemed it to be cost-ineffective, and at least two and a half years after being approved for use it is still not available for cystic fibrosis patients on the NHS. I welcome reports that, after over a year of waiting, dialogue between the drug company Vertex, NHS England and NICE has reopened, and that last month Vertex announced it had proposed a new portfolio approach to the Government on the funding of Orkambi.

I would be grateful if the Minister would elaborate on that in his response and tell us whether the Government are considering the offer seriously. I understand that some hon. Members may have had an update email from Vertex this morning, although I did not; there may be news on that front. I would also appreciate it if the Minister would acknowledge that, given the example of Orkambi, more needs to be done to change how drugs for rare long-term conditions are assessed by NICE. The longer patients go without those precision drugs, the longer they go on suffering irreversible lung damage. That is why we need urgent change.

[Mr Sweeney]

Many hon. Members have referred to the Scottish Medicines Consortium. Of course, healthcare in Scotland is a devolved issue but the issues are the same: there is still a two-year wait to get the matter through. Does my hon. Friend agree that there is a need for a closer working relationship between NICE and the SMC? Perhaps sometimes, with collective bargaining, a deal on price could be reached sooner.

[Mrs Hodgson]

Absolutely, I agree. I do a lot of work, as the Minister does and has done in the past, on access to cancer drugs. The same can be said in that context—that it is a postcode lottery not just within nations but between the nations, and that it need not be. The issue could be explored further.

NICE considers all the different benefits that a treatment could give, including living longer, but also having a better quality of life. That is hard to establish for some rare long-term conditions such as cystic fibrosis, making it hard for drugs to meet NICE’S requirements. There is a need for high-quality data on treatments, so that an accurate quality-adjusted life year model can be created. However, that is very difficult to achieve in short trials, particularly for rare long-term diseases such as cystic fibrosis, where “powering” a trial with enough patients is very difficult. People with long-term conditions often score their quality of life more highly than people who have developed acute conditions after being well, often because of differences of perception. If, during trials, people score their quality of life as high prior to treatment, subsequent quality-adjusted life year gains are lower.

The cost of developing a treatment for a rare disease is also high. Understanding the condition, developing an effective treatment and running the required trials in a small population is expensive. Treatments often have no competitors, so there is dampening of market forces, with negative consequences for the consumer or payer. NICE found that Orkambi was important and effective, but that the cost was too high. Vertex must therefore work as a matter of urgency to bring down the cost of Orkambi, so that thousands of cystic fibrosis patients can benefit from the drug on the NHS. The Opposition are committed to ensuring that all NHS patients get fast access to the most effective new drugs and treatments.

In response to the online petition, the Government responded:

“We want patients to benefit from clinically and cost effective treatments.”

What steps are they taking to bring that about? NICE’S guidance on Orkambi is scheduled to be reviewed in July 2019. Will the Government work with Vertex, NICE and NHS England to ensure that the review is brought forward so that thousands of patients can benefit much sooner from the drug on the NHS? It is clear from today’s debate that we need more and better treatments for cystic fibrosis in the UK. Last year, half of all people who died with cystic fibrosis were under the age of 31. It is unacceptable that while pharmaceutical companies, NHS England and NICE barter, people’s lives are at stake. The Government must therefore take responsibility for negotiating an agreement as soon as possible. I hope that the Minister will take on what he has heard today. I hope he will acknowledge how important it is to find a solution that guarantees that the current situation affecting the pipeline of treatments for cystic fibrosis will never happen again, and that thousands of patients will get access to the most effective drugs and treatments as soon as possible.

19:17

[The Parliamentary Under-Secretary of State for Health (Steve Brine)]

It is a pleasure to serve under your chairmanship, and that of Mr Evans, Sir Roger. May I be the final speaker to thank my hon. Friend the Member for Sutton and Cheam (Paul Scully), who has done his job as a member of the Petitions Committee in introducing the debate on the petition today. As the hon. Member for Washington and Sunderland West (Mrs Hodgson) said, the number of Back-Bench MPs who have spoken is 22. My Parliamentary Private Secretary reckons that we have had 60 Members through the Chamber in the past three hours. Of course that is only just over 9% of MPs, but, as we all know, having been to many Westminster Hall debates between us, that is a lot of MPs in one debate. That did not even include the hon. Member for Strangford (Jim Shannon)—I am worried for him. As we know, so many MPs attending the debate suggests a great deal of interest in the subject, and the way everyone spoke was excellent. Perhaps I may mention that the speech of the hon. Member for Motherwell and Wishaw (Marion Fellows) cannot have been an easy one to make, and she delivered it with grace.

With more than 115,000 signatures—my goodness—the petition shows what huge public support there is for tackling this terrible disease. The passionate way in which hon. Members across the board have spoken on behalf of their constituents is testament to that support. I want to make a special mention of the “Bury brothers”, who represent the 202 people in the Bury constituencies who signed the petition: the hon. Members for Bury North (James Frith) and for Bury South (Mr Lewis) both spoke well and in a sensible and balanced way. I can only assume that the hon. Member for Bury South, from what he said, must be a former Health Minister. Am I right?

[Mr Lewis]

indicated assent.

[Steve Brine]

I thank 163 of my own constituents who signed the petition and who have been in touch with me, and one who has been to my constituency surgery. I may be the Health Minister responding to the debate, but I am also a constituency MP and a parent of two young children.

I pay tribute to the Cystic Fibrosis Trust, which does a tremendous job on behalf of all our constituents. I know we are not meant to address the Gallery. I will not do so, but if I did I would thank those who have made the trip in the snow and ice, probably not all from SW1, to come to Westminster today. It is a credit to them that they have made time to do that.

I recognise the progress that Vertex has made to date in its mission—I believe it is a mission—to discover ultimately a cure for cystic fibrosis, and recognise the treatments that it brings to the market. I know Vertex understands the importance of the UK market, and NHS England as a customer, to its future and to that mission.

This petition is calling on the British Government—that would be me—

“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.”

I am here today to do exactly that. It is not a difficult call to agree to. The negotiations must remain constructive and be undertaken with the utmost urgency, for all the reasons we have heard—I will not repeat them all—or, as my hon. Friend the Member for Mid Dorset and North Poole (Michael Tomlinson) said, people living with cystic fibrosis will suffer.

It seems to me that the main ask I have heard from pretty much every hon. Member speaking today is, “Get involved.” I am not the Minister directly responsible. That falls to Lord O’Shaughnessy, the Parliamentary Under-Secretary who sits in the other place, but I can say on his behalf and on mine that we are involved, and you can bet your life that we will continue to be involved. I do not think I can be clearer.

[James Frith]

The Minister says, “You can bet your life” that he will continue to be involved. Any further delay is literally betting lives and the ever-diminishing lung capacity of those who go without Orkambi. What will he commit to do differently from today, and when will Orkambi be prescribed by the NHS?

[Steve Brine]

The hon. Gentleman knows that I cannot give the answer to his when question. I will come to the other bits of his question. His point about the reducing lung function is well made, and has been made by almost everybody. The Government wholeheartedly support efforts to ensure that the precision medicines we have heard about are made widely available to CF patients and other patients. It is true that high-cost precision medicines represent a challenge to the NHS, but they are also a tremendous opportunity to deliver high-quality outcomes through highly specialised treatment. I hope I can go some way to ensuring that people with comparatively rarer conditions such as CF get the same quality, safety and efficacy in medicines as those who have more common conditions, and to doing so in a way that is sustainable for the NHS.

There has been much talk about NICE, which has a difficult job, as the hon. Member for Bury South rightly said, but as we know, England has the second-highest number of cystic fibrosis patients in the world and there is sadly no cure. Current treatments generally target the complications rather than the cause of the disease. Of course, I can appreciate the huge daily burden of treatment for patients and their families, and the uncertainty that they live with. Uncertainty is a huge burden—the hon. Member for Dudley North (Ian Austin) mentioned the mental health burden. It is so important that patient voices are heard during any appraisal process to ensure that that burden is fully understood.

NICE’s technology appraisal programme makes recommendations for the NHS on whether drugs represent an effective use of NHS resources in what we must remember is a publicly funded health system, as many hon. Members have said. There are other parts of the world where, if someone could not afford it or their insurer could not afford it, this would not even be a discussion. That system means that patients can have the confidence that the price paid by the NHS is consistent with the improvement in health outcomes a medicine brings, ensuring fairness as well as the best possible use of funding for patients and the NHS.

Those are very difficult decisions to make, but it is essential that patients are getting the maximum benefit from every pound of our constituents’ money that is spent by the NHS. NICE takes its decision independently of Ministers. The Government rightly have no say in whether a new medicine is recommended for the NHS at the price proposed by the manufacturer. NICE published its final guidance on the use of Orkambi in July 2016 and, as we have heard, did not recommend it for use in treating cystic fibrosis. That, of course, is not the end of the story, and I do not want it to be. I will return to that in a moment—although, ironically in a three-hour debate, time is short for me to give our position.

Since 2013, NHS England has been responsible for securing high-quality outcomes for patients with cystic fibrosis. Six years ago, it agreed to fund Ivacaftor and Kalydeco for cystic fibrosis patients with the relevant genetic mutation. An additional possibility that may further the pool of treatment options for CF in the near future is the double combination therapy branded Symdeko, which has shown positive results and is currently undergoing European Medicines Agency licensing.

More broadly, the number of medicines for cystic fibrosis patients expected within the next three years is promising, with products being developed by a range of manufacturers as well as Vertex. NICE is aware of 31 other technologies by 19 different companies—not Vertex—that are in the pipeline for cystic fibrosis. Clearly, the prices for any new treatment will also be considered by NICE and we must ensure that the arrangements NHS England enters into now do not restrict options for patients to have the best available medicine in the future. The hon. Member for Bury North made that point very competently.

The NHS is in discussions with Vertex about Orkambi. Vertex has approached NHS England with a proposed deal to reduce its prices. While I cannot share the details of this proposal due to their commercially confidential nature—believe you me, I wish I could—I can assure hon. Members that, at the level that Vertex has proposed, the products are still far from cost-effective. Therefore, there is more talking to do.

Last week NHS England made a counter-proposal, which would ensure that the drugs could be used at a price that is cost-effective. I understand that NHS England has agreed to meet with Vertex—I would jolly well hope so, and of course it is not over email—to discuss that counter-proposal further. I have seen the statements made this weekend—I must say I am no fan of Twitter diplomacy in this or any other form of diplomacy. The statements this weekend were not exactly encouraging, but they must not be the end of the story.

While it is not for Ministers to approve, I truly believe this can be a mutually satisfactory arrangement for both parties. I know I speak for my colleague, Lord O’Shaughnessy, when I say that we are both impatient for a breakthrough and are watching the matter like hawks. I get the message loud and clear: the House has said, “Get involved.” The House has that assurance from me.

I thank all hon. Members who have spoken so passionately. They have helped the cause on behalf of their constituents. On Orkambi in particular, I hope above all that hon. Members can see that this is a live issue and that it is work in progress.

19:28

[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.

19:29

Sitting adjourned.