Cystic Fibrosis Drugs: Orkambi

Paul Scully Excerpts

Monday 10th June 2019 Westminster Hall

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

I beg to move,

That this House has considered e-petition 231602 relating to access to Orkambi and other drugs for people with cystic fibrosis.

It is a pleasure to serve under your chairmanship, Mr Bailey. I will read the petition, if I may:

“Children & young adults with CF endure lifelong suffering & early death. They need Orkambi and other precision medicines as they are developed. Sufferers in the EU, US & Australia can access the drugs, but not the UK. Hundreds have died in the 3 years since these drugs were licensed. All MPs who debated the petition in March were passionately in favour of supplying the drugs. Despite an ever-increasing abundance of evidence as to the drugs’ effectiveness, CF sufferers still do not have access. Case studies report miraculous improvements in health. Consultants nationwide have asked the NHS to make the drugs available. Doctors have expressed distress at seeing children die whilst the drug they need is on the shelf. After 3 years, conclude negotiations and fund these drugs.”

I pay tribute to Catherine Meredith, who started the petition, and the 108,144 people who had signed it as of this morning. Many hon. Members present are here because of their constituents and their long-lasting championing of cystic fibrosis treatment and the need to get these drugs to market as soon as possible.

Cystic fibrosis is a severe, devastating and progressive disease. It is a genetic disease, so we know the exact number of people living with it—70,000 around the world. It is considered by many to be a Celtic disease; the UK has 12% of all sufferers, which makes it an important place for research and an important market for pharmaceutical companies, such as Vertex, that have been working on treatment.

[Alberto Costa (South Leicestershire) (Con)]

I congratulate my hon. Friend on securing a clearly important debate. If Orkambi were available on the NHS, my constituent Amelie-Rose Sullivan, who is only two years old, would be able to take that life-saving medicine. Having spoken with her family, I understand that she has needed antibiotics on five occasions, which involves a two-week course each time—a cost to the NHS. Antibiotics might not have been needed if she were taking Orkambi, which underlines the need for Orkambi to be available. Ultimately, it would be better value for the taxpayer and our constituents.

[Paul Scully]

My hon. Friend is absolutely right. Before I led a similar debate for the Petitions Committee roughly this time last year, I went to the Cystic Fibrosis Trust, as I did this morning, where I met and spoke to a lot of parents whose children are suffering, as he has mentioned. This morning, I met a number of adult sufferers, who I will speak further about in a minute. One of them, who is 43, described the disease—this is harsh, especially for any sufferers watching—as his lungs filling up and effectively drowning. That is pertinent to me, because my father died of mesothelioma 30 years ago, after a year of suffering. This man is 43; I cannot imagine that suffering. Early treatment for children, however, stops that build-up in the first place and allows them to keep their lung capacity higher for longer, so they can have a proper standard of living.

[Catherine McKinnell (Newcastle upon Tyne North) (Lab)]

I suspect that my fellow Petitions Committee member shares my concern that families are being put in the terrible position of having not only to deal with a devastating illness and diagnosis, but fight for a treatment that they know is available and that other people across Europe and across the world are receiving. Whether it is cystic fibrosis, Batten disease or phenylketonuria, or PKU, it is wrong that families are wasting their precious energy, which they would like to channel into looking after their children, into fighting for a treatment that could solve many of those medical issues.

[Paul Scully]

I totally agree with the hon. Lady. It is frustrating to look at access around the world; there is even an interim solution in Scotland. It would be interesting to hear from the Minister about how that may pan out. Scotland has given interim access, including for compassionate use, and further access to some sufferers who can use Orkambi with a view to reporting back in August.

It is disappointing to find that people living with CF, in their ingenuity, have had to resort to looking at a buyers’ club. The Vertex drug is patent pending in Argentina, so another company is making a copy that can be sourced for £18,000 a patient—still a lot of money—rather than £104,000. That £18,000 comes out of their pockets, however, which defeats the idea of the NHS being free at the point of need.

[Mark Tami (Alyn and Deeside) (Lab)]

It is bad enough if a child is suffering with an illness for which there are no medicines to help, but it is incredibly cruel for the patient and the family when they know there is a drug that can help their child.

[Paul Scully]

There are clearly two sides to the negotiations. The NHS has to understand that CF sufferers are not just names on a spreadsheet; they are real people. We are all here as politicians to represent our constituents, but we are humans first and politicians second. We have to remember the humanity. On the other side of the negotiations, Vertex needs to make sure that these patients—the people living with CF—are not just names on a shareholder report.

[John Spellar (Warley) (Lab)]

I thank the hon. Gentleman for his comprehensive introduction. The fundamental core of the problem is the greed of Vertex and its unreasonableness in the discussions. Should the Government not play a much stronger role in dealing with the issue of generics? That is the demand of those who are suffering, such as my constituent Nicola Johnson and her son George, whose capacity is steadily reducing. The Government need to get a move on with that alternative route.

[Paul Scully]

The right hon. Gentleman is right to say that Vertex has to be reasonable, because the UK is effectively the biggest market for a pharma company that comes in to tackle CF, for the reasons that I have mentioned. It has to understand that the issue is not all about its share price in the long term. As a free-market liberal economist, I recognise that private companies must be allowed to deliver fair profits for their shareholders, which will further research and future investment, but they are sailing close to the wind if they are profiteering from human suffering.

[Crispin Blunt (Reigate) (Con)]

I am grateful to my hon. Friend for leading the debate. I spoke in the previous debate on the subject in the main Chamber. It is right to focus on Orkambi and Vertex, but the issue is actually the process of the National Institute for Health and Care Excellence. If we cannot assess issues such as antimicrobial resistance and new antibiotics in the NICE system, or get a positive answer about them, we need to look at its assessment. I hope the Minister is open to that.

[Paul Scully]

My hon. Friend makes an interesting point. Clearly, a portfolio of drugs is coming through the system. The original drugs treated only 5% of cystic fibrosis sufferers, but now the figure is 50%. The triple therapy that is being researched can benefit up to 90%, and clinical trials show an increase of 10% in some people’s lung capacity in a single week of using the drug. It is disappointing that Vertex has not included the triple therapy in its negotiations about the portfolio. None the less, my hon. Friend is right that the NICE system needs to be reviewed to take an interesting and innovative approach to drug research in future.

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

There are those who are listening to the debate but are not experts like the families who live with this every day, but lung transplants are a common procedure. The lungs fill up and do not function, and lung transplants are often the only option. How does NICE take that into consideration? It cannot do so when it is looking at the viability and cost-effectiveness of Orkambi.

[Paul Scully]

My right hon. Friend makes a really interesting observation. This morning I saw a number of people, some of whom have had a lung transplant. Orkambi was able to get them to the point where they could have a lung transplant in the first place. I spoke to most of the people via video link, because they could not be in the same room as me due to the risk of cross-infection and aspergillosis hitting their lungs. Aspergillus gets in our lungs, and most of us just bat it off and do not have an issue with it. However, it can adversely affect these people’s lung functions, or even be fatal. That is how debilitating the disease is. What struck me about all this are the mental health issues behind it, which I will come back to later.

[Janet Daby (Lewisham East) (Lab)]

I thank the hon. Gentleman for giving us such a comprehensive overview of cystic fibrosis. A constituent of mine, Joanne, has contacted me regarding her daughter Lauren, who suffers from cystic fibrosis. Access to treatments such as Orkambi, which has already been mentioned, would be absolutely crucial to managing hers and so many other people’s conditions in our country. Hon. Members might know that 16 May marks a year since the Prime Minister called for a speedy resolution to this issue. Does the hon. Gentleman agree that we need to hear the actions that the Government can take, and are taking, to open up access to these lifesaving treatments for people such as Lauren in my constituency?

[Paul Scully]

One of the reasons why NICE was set up in the first place was to take politics out of drug development. We need to ensure that we have the balance right between the Government intervening and the clinicians—the people who can make their assessments without political interference—making their decision. None the less, we clearly must have a view.

One of the things that some people have suggested—I think it was intimated in an earlier intervention—is Crown use of patents, to allow the use of generic drugs and effectively remove patents from pharmaceutical companies. Obviously, that is in extremis. There is an inherent problem with the potential lack of investment in future research, should we start taking away patents from private companies.

[Emma Hardy (Kingston upon Hull West and Hessle) (Lab)]

The hon. Gentleman is making a very powerful speech, and I stand here today representing my constituents. Does he agree that when factoring in costs, NICE should also factor in the ongoing costs that not treating cystic fibrosis will bring? It needs to factor in the cost of the additional mental health treatment and other health treatments that will be needed, and possibly even the cost of lung transplants. This is not a simple comparison with the cost of treatment; other things need to be taken into account.

[Paul Scully]

The hon. Lady has hit on a really crucial point. The underlying thing that I took away from my meeting with the people living with CF this morning was mental health, which ran through all their situations.

I heard from Oli Rayner, who talked about the fact that he is 43 and has dedicated his whole life to staying alive; he had effectively been told that he would not make 10 years. He was then told that he would not make 20, then 30. This is a guy who has now got cystic fibrosis-related diabetes and a number of other conditions. He has had a lung transplant, and he had Orkambi to get him to that stage. The fact is that his lungs are now doing what he wants them to do, without his having to think about it. We can imagine the mental issues that he had before.

Jessica Jones told me that people with CF are very good at living. Yvonne Hughes said she felt broken. One lady, Carly Beale, told me that she had been on the original Orkambi trial. The NHS had not prepared her for when Orkambi stopped at the end of the trial. She had suddenly improved and started to get her life back—perhaps a life that she had not had in the first place—and she was not ready to have it taken away from her. She said that it is almost worse that this drug exists but she cannot get access to it. She said, “I’d rather it didn’t exist than have it dangled in front of me in expectation.”

[Kerry McCarthy (Bristol East) (Lab)]

I am really pleased that there is now cross-party working on this issue. Life expectancy is a very sensitive issue for cystic fibrosis patients and their parents. I am aware that Conservative MPs have been sending round a letter that points out—as if the letter’s recipients ought to be grateful—that life expectancy for CF patients has now increased to 40, although I think it is more like 31. It seems a little insensitive, and I hope that MPs present would report that back.

[Paul Scully]

It is an unfortunate manner of phrasing. There is no doubt about the fact that median life expectancy has increased. For someone born now, the projection is that they are likely to have a median life expectancy of 47. It is clearly an improvement, and hopefully these drugs will carry on extending that. It is a matter of wording, and I do not think any offence was intended.

[Andrew Griffiths (Burton) (Con)]

I think my hon. Friend will win this year’s prize for taking the most interventions in a debate, such is the level of interest in this debate. I know the Minister will have taken note of the cross-party interest, and I hope that Vertex is also taking note of just how much interest there is among Members of Parliament.

I am here today because Elaine and Chris Colborne came to see about their three-year-old grandchildren, James and Alex. Although it is terrible for any parent—I am one myself—to see their child suffering, it is even worse for a grandparent in some respects. They see the little child suffering, and they also see the anguish of their child—the parent who on a day-by-day basis has to live with the consequences and the pain that comes from CF. This is a hugely important debate and we agree that it is not just about Orkambi; it is actually about the pipeline of other drugs that are coming through. Even if we were to solve the problem with Orkambi, we might be in a situation in which new drugs come forward in the future. We need to tackle the issue, not just this particular drug.

[Paul Scully]

My hon. Friend is absolutely right to say that we are looking at that pipeline, which is so important. That is why this process is different from the kind that NICE is used to. I hope that NICE can be accommodating enough to suit the pipeline, and that Vertex can come back with its triple therapy, which will help so many people.

[Mr Ranil Jayawardena (North East Hampshire) (Con)]

I congratulate my hon. Friend on opening this important debate. Although we can get hung up on process, NICE, NHS England and, dare I say it, Her Majesty’s Government, is it not true that real people’s lives are at the heart of this? As I understand it, Vertex has been offered the most generous settlement in the entire history of the NHS. Is it not time that the company now took the brave decision to accept a very generous deal?

[Paul Scully]

My hon. Friend is absolutely right. Vertex has a pipeline that should help its shares—their value has doubled over the past few years, and I am sure they will do very well in future—but we must have a balance with shareholder value, so that the company can continue to invest and continue its research. As he says, this is about human beings, their quality of life and their lives.

[Ian Paisley (North Antrim) (DUP)]

The hon. Gentleman has struck a chord with the points that he has raised, which have been heard by Members of different parties. We must also make this cross-channel, and Northern Ireland cannot be left out of this arrangement. I know the chief medical officer has been in discussions with NICE and the Department to ensure that no postcode in the whole of the United Kingdom is left out of a settlement in this matter.

[Paul Scully]

The hon. Gentleman is absolutely right. Northern Ireland must surely be in there too.

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

This debate is primarily about the availability of Orkambi in England. The 540 of my constituents who signed the petition and Rebecka and Matthew Bow, who contacted me on behalf of their daughter, Sofia, are really keen to see progress in Wales. Does the hon. Gentleman agree that it is important for health across the UK that we work together to ensure that there is a co-ordinated approach with Vertex to make Orkambi available right across the United Kingdom?

[Paul Scully]

The hon. Gentleman is right. Cystic fibrosis is a rare disease that particularly affects this part of the world, so we must find a way of working together across the whole of the UK.

[James Frith (Bury North) (Lab)]

The hon. Gentleman is making a compelling case. I know that this issue matters to him and to many others in the Chamber. One of the many issues that victims of this wicked disease face is that they cannot congregate and make the case for themselves—such is the risk of infection. It is therefore incumbent on us to make a cross-party effort to amplify their cause. They face the inflexibility of NICE and the rigidity of Vertex’s pricing, but they now have the hope that the Minister will hear anew the campaign from colleagues from across the House.

Does the hon. Gentleman agree that, in this instance, per-patient pricing is not an accurate conclusion for Vertex to reach? It should take the deal on the table and begin to save lives; its indecision to date is costing lives.

[Paul Scully]

The hon. Gentleman has absolutely nailed it. That is very much the point. This is about human beings, and we are dangling something in front of them that they just cannot access. The fact that people go off to Argentina and spend their own money to get the drug is ridiculous. That is a really important point.

[Anneliese Dodds (Oxford East) (Lab/Co-op)]

rose—

[Paul Scully]

I will make two more points about mental health, and then I will let someone else intervene.

As I said earlier, Oli Rayner said that he effectively spent 10 years preparing to die. He even cold-called a vicar to plan his own funeral. He is now 43 and has a relatively clean bill of health after having a lung transplant. It is outrageous that he had to do that.

I want to raise with the Minister the case of Carlie Pleasant, who ironically works for the NHS. She has CF and has had to go to the hospital a number of times. Her HR manager has told her that she has had too many days off sick, and that she has reached the trigger point. Basically, she has been told that she may be able to make up for it if she is not off for the next couple of months. She has a husband and a young child, she is running a home and she is trying to keep her job and pay her mortgage. How much pressure does that put on her mental health?

There must be thousands of sufferers in a similar situation across the country. We are talking about a life-saving, quality-of-life drug. When we, and especially the Government, try to ensure parity of esteem between mental and physical health, we must all ensure that that balance is reflected not just by treatments but by everybody in the health system and every employer.

[Anneliese Dodds]

I am very grateful indeed to the hon. Gentleman for giving way. He is making a very persuasive case, and it is obviously supported by many of us. Returning briefly to his point about buyers’ clubs, he will be aware that many sufferers and their families have already made significant financial contributions to CF research. The Oxford group, made up mainly of parents and grandparents of people with CF, has raised more than £174,000 for research just since 2003. They are obviously very upset at the current impasse, as they have made that contribution and they now feel that they have to do it again through buyers’ groups. Does the hon. Gentleman agree that that is totally unacceptable?

[Paul Scully]

The hon. Lady is right. The fact that people have to resort to such things is ridiculous given that we have a comprehensive health system. It is about time Vertex recognised that there is plenty of time for it to make a reasonable profit on its drugs portfolio. It is not helping the situation. There are a number of people from the Cystic Fibrosis Trust in the Public Gallery, but unfortunately, as the hon. Member for Bury North (James Frith) said, many sufferers cannot be here because of the danger of cross-infection. They are hopefully watching the debate on television or on their computers.

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

The hon. Gentleman is being extremely generous in giving way. On the issue of shareholdings and profit, when I was doing research for this debate, I came across something in the Wall Street news that told me that Vertex gives a higher than average return on investment. Most companies that operate in this area give a return on investment to shareholders of 20%; last year, Vertex gave 45%.

[Paul Scully]

I am grateful to the hon. Lady for pointing that out.

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

rose—

[Paul Scully]

I am just about to conclude. My hon. Friend the Member for Burton (Andrew Griffiths) made an interesting point. In order not to top the record, I will be finishing very soon.

[Luke Hall]

It is absolutely devastating that we are having a debate in Westminster Hall again about an issue that still has not been resolved. I thank my hon. Friend for his commitment to it. He opened the previous debate, when the Chamber was completely full, and he has opened this debate fantastically well. Does he agree that the message that should go out from this debate is that a permanent deal must be agreed with Vertex as soon as possible? We cannot rely on buyers’ clubs or any other mechanism.

[Paul Scully]

My hon. Friend is absolutely right.

[Sir George Howarth (Knowsley) (Lab)]

The hon. Gentleman is making a very powerful case that we all accept. A moment or two ago, he mentioned somebody he met earlier today who suffers not only from diabetes but from cystic fibrosis. Could we imagine a situation in which someone who has type 1 diabetes and needs insulin to survive has it taken away? Although the two conditions are completely different, is there not some equivalence between the two?

[Paul Scully]

The right hon. Gentleman is absolutely right; that is a really good point. Actually, the diabetes came from the cystic fibrosis, but he is right to talk about the medication in that way.

[Alan Mak (Havant) (Con)]

My hon. Friend is making a persuasive speech. This issue is important in my Havant constituency, as it is in the constituency of my right hon. Friend the Member for Tunbridge Wells (Greg Clark), who is sitting next to me. Does my hon. Friend the Member for Sutton and Cheam (Paul Scully) agree that while we praise the work of the Cystic Fibrosis Trust, we should also commend it for the research into the condition that it does itself? I urge hon. Members from across the House to support any bids for funding that it makes to the NHS or other funding bodies so it can continue its important work in this field.

[Paul Scully]

My hon. Friend is absolutely right. I have been to the Cystic Fibrosis Trust twice, and I was absolutely blown away. It is the kind of organisation that I got into politics to help. I met Lynsey Beswick, who works there—she may be in the Public Gallery. She is a CF sufferer, and she has just climbed Snowdon. I would struggle with that, and she has reduced lung capacity. [Applause.] That is the first time I have got a round of applause in Westminster Hall; I am not sure it is to be encouraged. Lynsey, that is for you.

[John Stevenson (Carlisle) (Con)]

My hon. Friend is giving personal experiences of how this illness has affected individuals. I met Ayda’s family last week to discuss how it affected them. I have been thinking about the benefits that the introduction of the drug could bring about. One is a saving to the NHS through not needing to use other drugs. The family would need fewer visits to the hospital, which would have an effect on patient care and would free up time for the hospital to look after other patients. Does he agree that it would also bring about a significant improvement to family life, not just for the individual, but for the extended family too?

--- Later in debate ---

[Paul Scully]

My hon. Friend is absolutely right. The young son of the lady who had her Orkambi withdrawn at the end of the trial asked her, “Mum, what was the worst day of your life?” She said, “It was when that drug was taken from me.” Her son—from memory, I think he was seven—said, “You know what? That was the worst day in my life as well.” This is about the extended family and the people around the patient. A number of hon. Members have talked about the cost per patient, but it is not just about the patient; it is also about their friends, family and loved ones.

[Gillian Keegan (Chichester) (Con)]

I thank my hon. Friend for securing this debate. I met my constituent Jack when I went to visit our local hospital. It is not surprising that I met him, because he spends six months in the hospital every year. He is now running out of treatment; he has tried everything. He is on 30 tablets a day, and he is on nebulisers. He is trying treatment after treatment, and the treatments are running out. Patients like Jack need this drug urgently, because there is almost nothing left.

[Paul Scully]

My hon. Friend is absolutely right. People are desperate; this has been dangled in front of them and it is about time that we acted.

I welcome my hon. Friend the Minister to her place—it is fantastic to see her serve as well as she does. I know that Vertex and NICE will be listening to the debate, and I hope that they act. It is so important that that we get this right and get it done now, for the sake of the thousands of people across the country who look to politicians to do things. I talked about the independence of the system, but we need none the less to do everything we can to bring the parties together to ensure that they deal with the matter. As I keep saying, this is about human beings, their life expectancy and their quality of life. I look forward to the Minister’s response.

[Several hon. Members rose—]

--- Later in debate ---

[Paul Scully]

You have joined us halfway through the debate, Mr Hanson, and it is a pleasure to serve under your chairmanship.

I thank all colleagues across the Chamber for their contributions. I thank the Cystic Fibrosis Trust for pulling together such a fantastic and insightful meeting this morning, and all those who participated, including the Minister’s constituent, Lucy Baxter. Lucy described cystic fibrosis phenomenally well, capturing it in one sentence: it is like breathing through a straw and then going running. It is that tiny lung capacity that really starts to illustrate the issue that these people live through, from day to day.

We have heard a lot about NICE and possible changes for that portfolio drug—system proposals. We have heard a lot about the need for Vertex to yield somewhat in the negotiations; as the Minister said, it is the outlier. I caution colleagues as they look at the alternatives, whether Crown use, compulsory licensing or a buyers’ club. Yes, we need those weapons in our armoury for the negotiations, but there is always the possible unintended consequence of fallout—the risk for future research into rare diseases of all sorts—if we start to undermine a market that clearly, however, needs to be looked at in relation to big pharma and how it can hold the NHS over a barrel. We just have to be careful about unintended consequences.

I also thank Alasdair Mackenzie, the Community and Outreach Engagement manager for Parliament. He joined me this morning at the deliberations with the Cystic Fibrosis Trust because of his personal interest. His partner, Becky, died of cystic fibrosis just a few years ago. His insight, and his sharing of Becky’s experience, were invaluable to me in my contributions this morning.

I wish, again, to put on the record my thanks, and to give all speed to the Minister, NICE and NHS England in our negotiations with Vertex. I hope that we can bring this matter to a resolution and give that life-saving drug—that quality-of-life drug—to all those thousands of people across the country as soon as possible.

Question put and agreed to.

Resolved,

That this House has considered e-petition 231602 relating to access to Orkambi and other drugs for people with cystic fibrosis.