Orkambi and Cystic Fibrosis

Lord Austin of Dudley Excerpts

Monday 19th March 2018 Westminster Hall

[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.

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[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?

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[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?

[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.