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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.
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 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.
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?
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—
Mr Adrian Bailey (in the Chair)
Order. Seventeen would-be speakers are listed for the debate. Back-Bench contributions will conclude at 6.58 pm to allow for the Front-Bench speeches and the proposer’s summing up. I will therefore put a six-minute time limit on individual contributions. I call Kerry McCarthy.
Kerry McCarthy (Bristol East) (Lab)
Thank you, Mr Bailey; it is a pleasure to see you in the Chair. I thank the hon. Member for Sutton and Cheam (Paul Scully) for introducing the debate on behalf of the Petitions Committee.
As we have heard, Orkambi is a life-changing treatment that stands to benefit 4,000 cystic fibrosis patients in the UK—40% of the total number of people with the condition—and which was licensed almost four years ago. In some ways, the debate has almost moved beyond Orkambi, because other drugs now being developed would help an even greater proportion of patients. Vertex’s ongoing refusal to offer a price for Orkambi that the NHS can afford means that those patients are still unable to benefit from the treatment.
I will point out—this is important to what I will say next—that in 2017 alone, Vertex earned £2.5 billion from the sale of Orkambi, while its chief executive was paid more than £17 million. Pharmaceutical companies such as Vertex play an important role in the development of innovative medicines and money goes into that, so I entirely accept that they should make a profit as a result, but to pursue excessive reward at the cost of patients’ lives is, I believe, morally bankrupt.
I will speak about alternative approaches. A few weeks ago, I had the opportunity to meet an Argentine drug company that has come up with a generic alternative to Orkambi. I also met two parents, one of whom was fortunate enough to be in a position to be able to spend a six-figure sum on Orkambi for his young son, who was, he said, doing well on it. He was clearly in a very privileged position to be able to do that. The other father was not in that position and was looking at the buyers’ club approach that has been mentioned and that featured on “Newsnight” on Tuesday. He said that if he had to sell his house to afford to buy the drugs at the reduced price—around the £18,000-mark—he would do so, because his son is far more important to him than the place he lives.
I was introduced to the Argentine drugs company by a Bristol-based international organisation called Just Treatment. With a range of experts in the field, Just Treatment has made contact with the generic manufacturer of the drugs, which can supply a safe and effective version of Orkambi at a cost for individual CF patients of about £23,000 per patient per year—just 30% of what Vertex charges. If there were a buyers’ club, the price could come down.
The term “buyers’ club” comes from the Hollywood film, “Dallas Buyers Club”, which gave a fictionalised account of the buyers’ clubs that popped up in the ’80s and ’90s because of the lack of access to HIV treatment. The clubs enable patients to understand their right to procure more affordable generic versions of drugs that they would otherwise struggle to access.
In Argentina, a small number of people have CF, and only a couple of hundred would benefit from Orkambi, of whom 120 take the generic alternative and seem to be doing well. The longest that anyone has taken it is 18 months, so although it is early days, treatments seem to be going well. Initial discussions with the generic manufacturer suggest that once 500 patients sign up to receive the drug, the cost would drop to just 20% of that demanded by Vertex.
Clearly, whether it costs £18,000 or £23,000, the drug is still not affordable for most people, which is why I would argue that although that is a step forward for some, the Government need to step in and help patients. The most effective way they can do that is by pursuing a Crown use licence for Orkambi. A few months ago in the House, the hon. Member for North Herefordshire (Bill Wiggin) mentioned that possibility in the context of access to medicines for CF patients. In the legal provisions set out in section 55(1) of the Patents Act 1977, the Government have the power to suspend Vertex’s patent on Orkambi and overturn its monopoly. One of the Government’s primary objections to that route has been the concern that it could take years to develop a generic product, but as I have said, a generic product is now on the market, is being tested and is available for patients in the UK, although only three boxes can be provided at a time and it is still unaffordable at the price that I mentioned.
The Government should also explore the funding of a large-scale clinical trial, which would enable the NHS to use research exemptions in intellectual property rights law and allow it to use the generic version of Orkambi. That approach has been used before, with the HIV prevention medication PrEP, or pre-exposure prophylaxis, and ensured that tens of thousands of patients were able to access that drug while its expensive branded counterpart was still under patent—I would be particularly interested in Minister’s response to that. A trial could be set up in anticipation of a Crown use licence, providing data necessary to support regulatory approval of the generic version of Orkambi.
If the Government are unwilling to pursue either of those routes—I cannot see why they should be reluctant to do so—there is another alternative that goes down the buyers’ club route: providing personalised health budget payments to help patients meet the cost of the generic drug. People are absolutely fed up of the deadlock between Vertex and the NHS, so I urge the Government to look at those more imaginative solutions.
John Howell (Henley) (Con)
It is a pleasure to serve under your chairmanship, Mr Bailey. My first introduction to cystic fibrosis came before I became an MP. I wrote some newsletters and did some public relations work for the Cystic Fibrosis Holiday Fund, the main objective of which was to provide holidays and ancillary facilities to under-18s who suffer from cystic fibrosis. On the basis of medical advice that was given in 2000, we now cannot take those children away together, so the fund spends most of its time generating respite break grants and providing the Family Revitalise programme. Those initiatives are both important, but do not compare with making available Orkambi or any of the other drugs that have been mentioned.
Two families in my constituency have children with cystic fibrosis. I have spent time with both families, and have seen that largely the children are happy, normal children who enjoy all the things that other children enjoy. Hanging over them, however, is the threat of a double-lung transplant just to stay alive.
Orkambi changes lives, and we need to look at ways that we can make it available. A number of structural difficulties were identified during the conversations that I have had on the matter. The first is one of commercial incentive and risk. To compound that point, one can look at the relative strength in numbers of those who suffer from diabetes or from cystic fibrosis: diabetes accounts for 4 million people, while cystic fibrosis accounts for only 70,000. A major hurdle is therefore already built in for those with cystic fibrosis to overcome. We should not forget that.
The issue of the time taken, which has already been raised, goes back to criticisms of the NICE process. The criticisms that I would make fall into three types: first, NICE adopts the same evaluation process for a drug that might treat tens of millions of people as it does for a drug that treats a few hundred thousand or, indeed, a few thousand. We need to bring home to NICE that that is not a right way to proceed.
Secondly, the same evaluation process is also used whether the drug is taken for a brief period or a long one—in other words, whether it is a short use cancer-related drug or, as in the case of Orkambi, it must keep being taken over many long periods. That factor needs to be built into any evaluation of the drug as well.
The third criticism that I would make of the NICE process is that it is too focused on short-term benefits, and not on long-term benefits, which we know that Orkambi can produce. As has been mentioned, the data released by Vertex show that after 96 weeks of treatment, the rate of lung function decline reduced by 42%. That is a major long-term thing to hang on to. Furthermore, the net value of Orkambi is hard to calculate and therefore to capture accurately. A number of direct costs need to be taken into account, such as the cost of hospitalisation, and there is evidence that Orkambi starts to reduce the number of other medicines that need to be taken.
We have heard that Orkambi is available in many other countries in Europe, although I hear that the Spanish Government are having difficulties with Vertex, in the same way as we are, over the availability of the drug. The agreement that was reached with Vertex to make Orkambi available was a disappointing affair. We need to put on the pressure to ensure that that happens and that generic drugs are brought forward to be used instead. The example often cited is Ireland—both families in my constituency mentioned the situation there—and it is interesting to note that success story of the use of Orkambi. It has been very successful there, and we should all take that to heart in making progress to ensure that young people suffering from cystic fibrosis have access to this drug.
Dr Sarah Wollaston (Totnes) (Ind)
I thank my constituent, Cathy Meredith, who started this petition. I also thank Oli Rayner and the many others living with cystic fibrosis who, sadly, cannot be in the Public Gallery with us today because cystic fibrosis is such a curiously isolating condition—those suffering from it cannot be in the same room as others because of the risk of transmitting resistant infections.
I will start with some context on the cause of cystic fibrosis, which is a mutation in the cystic fibrosis transmembrane conductance regulator gene, affecting the production of a protein that in turn has consequences for the balance of salts and fluids moving across membranes, leading to an accumulation of thick, sticky mucus in the lungs and other organs. The point, however, is that although 10,000 people in the UK live with cystic fibrosis, it is not really a single condition: there are many mutations of the CFTR gene. That has consequences for the types of medication to which people will best respond. We need to think of cystic fibrosis not only as a rare genetic condition but as a series of much rarer conditions. That is important to note.
We now have some real hope for progress with the CFTR modulators, but we need to make that progress much more rapidly than we are. The negotiations between Vertex and NHS England have dragged on for far too long. The patients living with cystic fibrosis and their families have been lost in those discussions. We need not only to return to thinking about them, but to bear in mind the implications that go far beyond those living with cystic fibrosis.
The NHS has a responsibility to consider the wider cost of drugs, including the opportunity costs—what we cannot treat if our NHS budget is consumed completely by the ever-rising cost of drugs. For the NHS to have that responsibility is a tough message for all of us, which is why we need bodies such as NICE to make the decisions to ensure fairness for all patients who rely on NHS resources. To put that in context, the drugs budget in 2017-18 was £18.2 billion. A little more than half of that was for hospital drugs and, over the past seven years, the costs of those drugs have increased by 119%. We therefore have to bear in mind the implications of taking a free-for-all approach to drugs costs, which the Minister will not want to do.
The Government are trying to get the parties around the table. Unfortunately, the gap is huge between what Vertex continues to demand for the drugs and what the NHS is offering based on recommendations from NICE. The gap is not small; it is considerable. Other companies have come to the table to negotiate their prices, so I call on Vertex to look again at what is happening. It is absolutely disgraceful that families have to resort to such things as buyers’ clubs; the inequalities that that creates are horrific. We need Vertex to focus on what is happening.
I am also concerned about some of the points made during our Health and Social Care Committee inquiry into Vertex. For example, we asked the company directly whether drug supplies had been destroyed because they were going off date, and we were told that that was not the case and was very unlikely to happen—but it has been happening. That is wholly unacceptable.
To come back to the alternatives, the hon. Member for Bristol East (Kerry McCarthy) touched on the issue of Crown use licences, for example. One of the areas that our Committee considered was possible referral to the Competition and Markets Authority. In fact, we have now heard that that would take many years, so unfortunately the area does not look like one we can pursue further. However, given so little progress since our inquiry, the Committee wrote to all the parties involved in the negotiations—NHS England, Vertex and NICE—to ask where we are now.
The most promising idea that we should take forward to apply pressure is that of interim agreements, such as in Scotland. An interim price is agreed, further research is carried out and all parties agree to a review based on the outcomes of that further research. That is being managed in Scotland and other places, as we have heard from other speakers today, and I urge Vertex to do that here. We all recognise the need for a fair price to enable further research to take place. We all recognise that many other drugs are in the pipeline, particularly a very promising triple therapy, which NHS England has now agreed to take off the table so that it does not distort future pricing. That is a sensible thing to do at this stage, so that within the current offer we look just at the three existing treatments. At a later stage, we can come back to look at the triple therapy evidence. I urge all parties to come to an interim agreement at least, and to continue to put patients front and centre in everything they do.
Finally, I would like to touch on the political aspect, because both President Trump and US Secretary of State Azar have repeatedly referred to using their muscle in trade negotiations to increase the price that European countries would have to pay for their drugs. They have referred to the NHS “freeloading”, for example. That is very worrying. We all need to be aware of the dangers of a future trade deal and the implications that it could have on negotiations for a range of other products. I hope that those points have contributed to the debate. All parties need to focus on the people at the heart of the issue: the patients who are living with cystic fibrosis.
Mr Adrian Bailey (in the Chair)
I gave the Chair of the Select Committee a little latitude, but that may not be available to other speakers. I call Sir Mike Penning.
Sir Mike Penning (Hemel Hempstead) (Con)
We all hoped that we would not be in this debate again, a year on from the last one. It is a tribute to the House that we have come together, from across the House, for a debate, which probably should take place in the main Chamber, about what is in my opinion an immoral situation, frankly: families are still waiting for a drug that we know can extend lives and prevent a serious need for invasive and very painful surgery. The situation causes a lot of worry among extended families.
Across the Irish sea in the Republic of Ireland—a country that is poorer than us in GDP terms—and in other countries that are also poorer than us, the deal has been done. It is not just a shame but a blight on our proud NHS that we have not come to an agreement one way or the other with Vertex and that we have not made a decision about whether we will go down the Crown use licensing route if Vertex cannot do a deal with us. We could have started that way back—they said it would delay everything, but we are here now, and there are patents being developed around the world. The situation is not of benefit to Vertex—we have heard about its shares—because it has not been able to sell its product in a country with a prominent number of CF sufferers.
Why is this happening? Is it just about cost? Is it just because civil servants and Vertex do not care about the lives of those wonderful people and their children? Is it about greed, or is it about how we procure drugs, as we have heard? When NICE was brought in, it took the politician, quite rightly, out of the decision making. But we cannot be outside it, because we are here as representatives of people who are suffering in constituencies around the country. We are here to be their voice. They have done fantastically well—the campaign group is brilliant: one of the top campaigns out there—but we do not seem to get anywhere. We have to look at how NICE looks at whether something is value for money. How can we value someone’s life? How do we value someone having a lung transplant later in life? What if they are not well enough to have that lung transplant, and they die early? What about the cost on not only physical health but mental health?
Let me stretch the House’s imagination a little. I used to be the roads Minister. Understandably, everybody wants roads—they want bypasses here and there. I had a simple way of looking at them: we would look at the benefit-cost ratio and say, “If we put money into that pot, what is the benefit to the community?” It would be £1 billion for a bypass, but the community benefit would be £3 billion, for example. Clearly, the way that NICE is looking at this drug is that there is not a good cost-benefit ratio, even though we know how much benefit there would be. NICE needs to look down the other end of the telescope.
Things have changed since NICE was set up—medicine has changed and drugs have changed, as the Chair of the Health and Social Care Committee, the hon. Member for Totnes (Dr Wollaston), mentioned. There are generic drugs coming down the line that are fundamentally targeted at certain illnesses, particularly ones that people inherit and are born with. Those drugs can turn off that switch and make the situation better, but NICE was never set up to deal with them. I was a shadow Health Minister for four and a half years; we looked at how NICE could develop and where to go with it. NICE is quite fixed, but because we politicians tell it to be. The House set up NICE—those with a long enough memory will remember when we did—with the Department of Health.
The key is for NICE to look at this issue differently. We can set up trials and we can find out why Scotland has an interim agreement, but the trials are there now. How immoral is it that someone was put on Vertex and their life expectancy got better, but it was removed? We are not talking about millions of people—surely, Vertex could have addressed that. We cannot sit here or in the main Chamber next year debating exactly the same thing that we debated last year. I do not care what colour the Government are: if there is a change of Government tomorrow morning, the issue will be exactly the same. Minister, for the sake of humanity, and families and loved ones, we have to do something about this, and we need to do it now.
Thelma Walker (Colne Valley) (Lab)
It is a pleasure to serve under your chairmanship, Mr Bailey.
It is just two years ago last week that I was elected Member of Parliament for Colne Valley. Over the past two years, it has been a real privilege to serve and speak up for my constituents. One day, however, will stand out more than most for me, among many amazing and challenging days. It was Friday 31 May, when two children visited me in my constituency office. One was nine-year-old Tristan with his grandpa John, and the other was one-year-old toddler Charlie with his Mum and Dad. I had had a busy advice surgery that afternoon, but went straight to meet Charlie in one room, while Tristan waited patiently in another. They were in different rooms to avoid cross infection as they both suffer from cystic fibrosis.
They had come to see me to each hand me a letter in which they described their daily challenges living with cystic fibrosis. The letter also described how access to just one particular drug might make their daily battle with the disease a bit easier, and might result in both children living longer lives. One-year-old Charlie, along with his parents, would benefit if he could access the necessary drug from such an early age. Charlie handed over his letter to me, which I must admit was a bit soggy and chewed by the time I got it. It was a joy to be with him and his parents.
Tristan also handed me a letter. Following the death of Tristan’s father in 2016, Tristan’s grandpa and granny, John and Frances, look after Tristan as his guardians. The family have experienced more than their fair share of loss. Like Tristan, his uncle Adam suffered from cystic fibrosis, and died aged 25 in 1999. I can only imagine the immense pain and grief and incredible frustration of losing a son, yet still, nearly 20 years later, being unable to access the drugs that already exist and that would improve the life of their grandchild. I cannot express how much admiration I have for Tristan and his family, and for Charlie and his; they have campaigned with the Cystic Fibrosis Trust to improve the lives of others.
What is this drug? It is called Orkambi; it already exists and it would improve the lives of children such as Tristan and Charlie. It is made by the pharmaceutical company Vertex, and the fact that it is not already available to those who need it is shameful. There are at least 2,834 people currently dealing with cystic fibrosis in England who could benefit from Orkambi.
Why has Orkambi not been made available? The current situation is that Vertex, NHS England and the National Institute for Health and Care Excellence cannot agree a price. We know that Vertex declined a £500 million offer from NHS England, and we know that the NHS has made a new offer, which currently remains confidential. Vertex did not consider £500 million an adequate sum, but it did, allegedly, consider it okay to destroy 8,000 packs, or a 600-year supply of Orkambi, as the deadlock continued. The lives of children such as Tristan and Charlie seem to be deemed acceptable collateral damage in this boardroom-style version of healthcare provision.
I urge Vertex to consider the NHS’s current offer with the lives of children such as Charlie and Tristan at the forefront of its mind. This seems to me to be a straightforward ethical decision for Vertex, which should be willing to compromise on its profit margin in circumstances where the lives of children are at stake. Let us be clear: Vertex is not struggling to make ends meet. It is a $43 billion company. That is about the same as the GDP of Bolivia or Tunisia. Vertex’s chief executive officer received a total salary of around $19 million in 2018, making him one of the best paid CEOs in the pharmaceutical industry. As ever, the problem is not that there is not enough money, but that big business is not willing to spend it.
This situation, in which children’s lives are held to ransom, must not continue. Vertex and NHS England must agree a deal soon, or an interim solution must be found, as happened in Scotland. Children’s lives must not continue to be held hostage. Tristan’s grandpa has already lost a son to this condition. There is simply no excuse for not prolonging and improving Tristan’s life. I will continue to campaign with Tristan and his grandpa, and Charlie and his parents, until this drug is made available to them and they are able to get on with their lives.
I will finish with a direct plea from Tristan’s grandpa, John Binns:
“We don’t have time – please don’t waste it. It’s costing lives and there are too many cystic fibrosis angels already.”
Ian Austin (Dudley North) (Ind)
We have been campaigning on this issue for well over four years; I think this is the fifth or sixth debate we have had in this Chamber or the main Chamber. We have met two Prime Ministers, two Health Secretaries and several Health Ministers. We have a new Health Minister with us today. We welcome her to her place, and we hope she will be able to unlock this issue in a way that her predecessors did not. I cannot remember how many cross-party letters we have delivered to Downing Street. There have been countless meetings between MPs, Vertex and the Cystic Fibrosis Trust. However, four years after this drug was licensed, it still is not being made available to the people who need it.
Imagine how many people’s lungs have suffered irreparable damage in that period. Imagine how many people have seen not just the quality but the length of their life reduced over the past four years. I pay tribute to the brilliant people from the Cystic Fibrosis Trust who are with us today: David Ramsden, Nick Medhurst and the brilliant Lynsey Beswick, whom we heard about earlier. Most of all, I pay tribute to the army of parents and carers who work so hard on this issue all year round. We are in this room today because of the inspirational work they do. It is humbling and heartbreaking to meet them and listen to their stories.
I became involved in this issue because I met Carly Jeavons, whom the hon. Member for Sutton and Cheam (Paul Scully) spoke about. She is from Dudley, and she took part in a clinical trial for Orkambi. Before being put on that trial, she had to choose between leaving work, with all the financial hardship that would cause, and struggling on in work with her health getting worse. She had to take 90 tablets and do two hours of physiotherapy a day. Her lung function was 44%. She had to spend two weeks in hospital every three months. That is not an unusual story, by the way, for people with cystic fibrosis.
Carly 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…hospital visits have massively reduced,”
and unplanned admissions are now non-existent. Since she started having Orkambi, she has been able to go on holiday for the first time, she has got married, she has started a family and she has started a business. She makes a much bigger contribution to the economy. People talk about the cost of providing these drugs; what about the contribution that people who are given Orkambi can make to the country and the economy? Let us think about that.
I did not really understand the impact that having a condition such as cystic fibrosis has on the rest of someone’s life. I mentioned Lynsey a moment ago. I hope she does not mind me telling everyone this. She is in her 30s, and she told me that, at a time when her friends are getting married, planning families, developing their careers, starting businesses and making long-term plans, people such as her are deterred from doing those things. It was heartbreaking to listen to her tell me that, so I cannot imagine what it is like for her.
I hope people forgive me for being blunt, but people in Lynsey’s position are just not able to plan for their future in the same way because they do not know how long they have to live. Every day that people who could be helped by Orkambi are denied access to it is another day for which their lungs suffer irreparable damage. It is not like breaking a leg—a leg can be fixed and become normal again, but someone’s lungs will not recover.
I understand that Vertex wants to make a profit. Of course it does, and of course it needs to be able to fund future drug developments. But surely it would make sense to sell a lot of a drug at a slightly lower price than to sell nothing at a really high price. I am not an expert on pharmaceutical company economics, but that seems to me to be worth looking at.
What assessment has the Minister made of the case for Crown use licensing, where the Government take control of the situation and ensure that these drugs are provided? What assessment has she made of the case for the Government providing the cheaper drugs that are available elsewhere? It is a disgrace that families have to buy them from Argentina.
The Minister is new to the Department, and we really hope she will work night and day to get this sorted out. She should get NHS England, NICE and Vertex in a room and not let them out until they have come to an agreement. That is what we need to happen. Will she also meet the cross-party group of MPs who have been working on this issue, and the parents and the Cystic Fibrosis Trust, so we can discuss it in more detail than we are able to this afternoon?
The situation is urgent. The system has not worked. People with cystic fibrosis have been put in an appalling position. This really does need to be sorted out now.
Jeff Smith (Manchester, Withington) (Lab)
It is a pleasure to see you in the Chair, Mr Bailey. I congratulate the hon. Member for Sutton and Cheam (Paul Scully) and all the other Members who have made thoughtful and powerful contributions. As we heard, it is now more than a year since the Prime Minister called for a “speedy resolution” to this crisis. For years, patients and their loved ones have seen their health decline, knowing that a possible solution has been sitting on the shelf the whole time. I will use two personal stories to illustrate why this issue is so urgent and important.
My constituent Clare Dempsey has been advocating for patient access to Orkambi and the associated precision treatments on behalf of her son, Jake Wright, who is nine and was diagnosed with cystic fibrosis at birth. His story is an insight into the relentlessness of treating the symptoms of the disease. He has to take 200 tablets a week, undergo two hours of physio a day and use nebulisers morning and night. Every year, he spends two weeks in hospital in isolation, receiving intravenous antibiotics. Jake has lost 16% of his lung function and a recent CT scan of his lungs showed early signs of bronchiectasis, which can leave lungs more vulnerable to infection. Precision medicines such as Orkambi that tackle the underlying causes of CF could have prevented those irreversible changes to Jake’s health. Imagine how it must feel as a parent to know that.
There is also the associated emotional and financial pressure on families. Clare is a cancer researcher by profession, but she has had to go part time to care for Jake. Not only is the NHS losing her skills but she is unable to earn money for the family, who have had to put their house on the market. If the root causes of Jake’s cystic fibrosis go unaddressed, the level of care he will need from his family and medical professionals will only increase. We talk about cost-benefit analyses, but NICE cannot take into account that effect on families, the NHS and wider society. We need to bear that in mind carefully when talking about this issue.
I want to talk about another affected family. My constituents Sadie Lawty and her husband Steve Sanders have a three-year-old daughter, Eloise, who is a lovely young girl I have had the great pleasure of meeting. She was diagnosed with cystic fibrosis when just two weeks old. It was a bombshell to Sadie and Steve, because as far as they knew there was no family history of it. At the time, they were told there were many reasons to be hopeful, because a new family of pharmaceuticals that targeted the underlying causes of cystic fibrosis had recently been introduced. They were given hope, but their hope has been frustrated.
Orkambi has existed for the whole of Eloise’s life, and its positive effects are long established. The frustrating reality for Eloise and her family is that they are no closer to accessing it than when she was first diagnosed. As we heard in Jake’s case, there is never a day off when combating the condition. Medicines have to be administered around the clock and there are endless trips for assessments and treatments. In fact, while we are having this debate Eloise’s parents are taking her to the doctor’s for her annual review, essentially to find out how much damage has been caused by cystic fibrosis so far.
Accessing Orkambi could help sustain Eloise and offer her parents some longer-term peace of mind. It is not a wonder drug, but it is a source of hope, so it is frustrating for Eloise’s family that their hope has been dashed. Eloise is doing well at the moment; hopefully that will continue. She will start nursery soon. At three years old, she has a while before the cumulative effects of cystic fibrosis really set in, but many people cannot afford to wait much longer.
I think back to my first brush with the condition, when I had a girlfriend who was a nurse. She was looking after a very charming young man who sadly died while still in his teens. That brought home to me the personal tragedy involved with this condition. It is therefore vital that the Government take responsibility for pulling people together and finding a solution among NICE, the NHS and Vertex as quickly as possible.
There are potential ways forward. My hon. Friend the Member for Bristol East (Kerry McCarthy) outlined solutions such as Crown use licences to overturn the monopoly and access to drugs through clinical trials. The Chair of the Health and Social Care Committee, the hon. Member for Totnes (Dr Wollaston), talked about the interim arrangements in Scotland, which may provide for a way forward. Whatever that way forward is, we need to find it.
I am pleased to see the Minister in her new role and hope that, as my hon. Friend the Member for Dudley North (Ian Austin) said, she will really grip on to this issue and try to find a solution that delivers for all the people I talked about, for the people who need the drug now, for the people in the Public Gallery, and in memory of the people who died. I plea with her to get the Government to get people around the table and find a solution.
Mr Stephen Hepburn (Jarrow) (Lab)
It is a pleasure to serve under your chairmanship, Mr Bailey. Like others, I pay tribute to the hon. Member for Sutton and Cheam (Paul Scully) for bringing about the debate and allowing us once again to give the Government a jog and tell them to get this sorted out once and for all. We are all aware of cystic fibrosis. It affects all of our constituencies. It is a life-limiting condition that affects more than 10,000 people in the UK. It is a condition with no cure as of yet, and half of those who have it will die before their 31st birthday.
We are also aware that there is a drug available. Orkambi, developed by Vertex, treats not only the symptoms associated with cystic fibrosis but the underlying causes. One of these patients is four-year-old Harriet Corr from Jarrow, whose parents Emma and Chris are here today. They desperately want a better quality of life for their daughter. They contacted me because they were frustrated and upset that Harriet could not get access to this life-changing drug as it is not available on the NHS in England. An even bigger blow to them is that if they lived an hour further north, in Scotland, Harriet could qualify for it next year. Why? The NHS in England and NICE do not deem the drug cost-effective and have spent more than two years negotiating a price, without success.
It is totally unacceptable that a country with the second highest number of sufferers, and one that leads the way in cystic fibrosis outcomes, now risks falling behind many other European countries and the USA and allowing people with the condition to become physically sicker than necessary all because an agreement on the economics of treatment cannot be reached. We all know that because of that we have had five parliamentary debates, and still there has been no action from the Government. These drugs need to be on the NHS now, because every day without them is another day of suffering for the people living with the condition.
If people want to focus on the economics, the evidence is clear. As Members have outlined, this treatment prevents the condition from escalating, thus preventing more expensive treatment further down the line. It would reduce the number of times patients used the NHS and hospitals throughout the year, which would obviously be a saving to the NHS and—more importantly—a better outcome for patients and their families.
Let us look at the human aspect of the debate. Families are well aware that access to the treatment is not a cure for the condition, but it certainly helps those affected live as normal and fulfilling a life as possible in the short time they are with us. The NICE appraisal methods fail to recognise that. How can a cost be placed on a child with cystic fibrosis and their ability to manage their condition, to go to school, to play with their friends, to go to university later on in life and get a job as well as easing the burden of care on their families and carers? Why should Harriet and her family not have a worthwhile quality of life and get to do the things many of us take for granted?
Time is running out for some patients, and we are calling on the Government to act now to resolve this matter urgently. I thank Members from across the House for pushing this case forward. I also pay tribute to the campaigners, who have fought hard, organised a petition and shouted so loudly that surely this Government, in the fifth or sixth richest country in the world, can no longer ignore them.
Stephen Morgan (Portsmouth South) (Lab)
It is a pleasure to serve under your chairmanship, Mr Bailey. Kick-starting a social media campaign, meeting local politicians and working tirelessly alongside organisations such as the Cystic Fibrosis Trust—do those sound like the actions of a five-year-old? Ivy Weir is a remarkable local campaigner who I am lucky to have living in my city. With the support of Gemma, her dedicated mother, Ivy lives with cystic fibrosis. She has carved a path into uncharted territory when it comes to accessing Orkambi.
Ivy helped me send out 650 letters to all MPs asking them to take part in the #StrawfieChallenge. She has been on national news, alongside her mother—committed campaigner Gemma Weir—calling for that change. She has helped deliver hundreds of letters to 10 Downing Street, calling for Orkambi to be free on the NHS. Ivy, Gemma and other CF activists have shown immense dedication and resilience in the face of adversity; now we in Westminster must do the same.
There have been five parliamentary debates about access to these medicines, yet we still see no light at the end of the tunnel. Over a year ago, the Prime Minister called for a speedy resolution to this emergency; there has still been no progress. We have the chance to improve the lives of thousands of people across our nation. There has been enough dialogue; now we need to see action.
As we have heard today, over 10,000 people in the UK live with cystic fibrosis—one in every 2,500 babies born. In the last four years, this genetic disorder has tragically claimed the lives of 210 people. What makes these figures more harrowing is that drugs are available to relieve many of the symptoms and greatly improve the standard of living for those with CF, including improving life expectancy.
Orkambi can make a real difference. The obstacle to the widespread implementation of these drugs is the cost; I find that morally repugnant. We live in a nation that forged an NHS from the ashes of world war two, that prides itself on universal healthcare for all and that is the envy of the world, because healthcare is free at the point of delivery and based on clinical need, not ability to pay. As such, the UK is a global bastion when it comes to cystic fibrosis outcomes. However, if Orkambi and other drugs are not rapidly added to the list of drugs available on the NHS, we will lose this valued status. Letting costs stand in the way of saving lives does a great disservice to the principles the NHS is rooted in: universality, equality and fairness.
The Health and Social Care Committee has done vital work in carving out potential ways forward. Its recent calls for interim access would allow negotiations to continue, without the unnecessary suffering of those living with CF. However, the non-binding nature of the suggestions means that three months on, no such agreement has been set in motion. Where NHS England and Vertex remain at loggerheads, NHS Scotland has established a deal with the pharmaceutical company. This will create a brutal postcode lottery. The level at which people suffer from cystic fibrosis is currently determined by where they live in the UK. I am confident that this was not the NHS envisaged by its founders nearly three quarters of a century ago.
I have seen the effects on my constituents in Portsmouth at first hand; I will make every effort to ensure their concerns are raised. In 2017, I wrote to the right hon. Member for South West Surrey (Mr Hunt) in his capacity as Secretary of State for Health, urging a swift response. In 2018, I wrote to the Secretary of State for Health and Social Care, the right hon. Member for West Suffolk (Matt Hancock), on his appointment, asking him to do the same. In 2019, I wrote to the Prime Minister, asking her to intervene. Now is the time for leadership on this most pressing of matters. We must see progress and we must see it quickly.
It has been announced that Vertex Pharmaceuticals could potentially develop treatments for 90% of those who live with CF over the next five years. If solid, robust negotiating foundations are not established now, that could undermine patient access for generations. Today, I call on the Department of Health and Social Care, Vertex and the Prime Minister to make this a national priority and help to bring an end to the suffering of thousands of people across the UK.
Rachael Maskell (York Central) (Lab/Co-op)
It is a pleasure to serve under your chairmanship, Mr Bailey.
Leading up to this debate, I spoke to constituents, one of whom described the mental strain of knowing that a drug was available but could not be prescribed to their child. Another parent talked about the administration of 22 drugs, and the physio and hospital appointments that were needed, as well as the stress of their child being admitted to hospital. Their child is now 21 years old and there have been no changes, even though there is a miracle drug that could transform their life. Another constituent, Jack, who is seven years old and at the start of his journey, desperately wants hope for his future.
We are at the cutting edge of a generation of pharmaceutical breakthroughs that have the power to transform the lives of people who experience a range of medical diagnoses. It is an exciting point in our journey with medicine, and it could be transformative to patients and carers. To deny therapy is ethically abhorrent. Many Members today have said that this is about negotiation, but I say that negotiation is not the answer. We should not be wrestling over drugs time and time again; we need a completely different framework for addressing the cost of pharmaceuticals.
NICE needs an overhaul to ensure it is fulfilling its role and looking at patients’ life course. I know that it does that work, but it needs to look at its effectiveness over a patient’s life course and at the economic value it can bring not just to medicine, but to the wider economy. It also needs to ensure that the levers are in place for it to look at a portfolio of drugs, as opposed to looking at drugs one by one.
We need to understand how much the NHS can afford to pay for the benefits of new drugs, taking into account the health benefits that could be achieved with the money the NHS must find to pay for them. This assessment, based on a NICE appraisal, could inform a debate about national rebate agreements in the current pharmaceutical price regulation scheme. There are several costs involved: the manufacturing cost, the price the NHS can afford, the NICE assessment and the market price, which is driven primarily by the US market. That is a cause of frustration.
The argument among academics is that a mechanism of a national value-based rebate should reflect the difference between the amount the NHS is willing to pay for the benefits and the manufacturer’s asking price. That would provide better incentives for manufacturers to make long-term investment choices, recoup costs and deliver for the NHS. It would also provide fairer rewards for innovation; manufacturers that produced more effective drugs at affordable prices would not need to pay a rebate, but those that charged more would pay a higher rebate. That would not impact the list price for the global market, and the rebate could be spread across the portfolio to provide better opportunities for manufacturers to bring new products to market.
That would take the politics out of NICE and NHS England. Canada is currently developing something similar, to ensure that drugs reach the places they need to reach. It can be further incentivised: should a manufacturer not supply a product when its production costs mean it could do so without making a loss, it could lose its patent. That would provide the required leverage. We need to hold discussions about this not just across the sector, but with the European Medicines Agency; the impact could be greater with more countries on board.
[David Hanson in the Chair]
People will have seen reports about the generic drug from Argentina, and we have heard about it in the debate today. We cannot allow inequality in access to pharmaceuticals in our country to grow, because it creates health inequalities. People who can pay will be able to afford that drug, and they will have better health outcomes. In the light of the ethics of the debate, the Department of Health and Social Care needs to move fast, now.
We need to be alert when it comes to trade deals with the United States, which is significant to the debate. The US will clearly want to drop the carrot of big pharma in front of Ministers, but that is a serious trap. The President talked about the NHS being “on the table” in his speech about trade negotiations, and we must be alert to the real agenda. It is through big pharma that he will get access to the NHS. As we have already heard, with respect to the size of the NHS drugs budget, that is a way of controlling it. We need to be on top of that.
Instead of drug-by-drug negotiation, we need a framework leading automatically to the leverage required to procure medicines. That will make such a difference. It will remove the uncertainty about different pharmaceuticals, as we move forward through the exciting developments that are taking place. Let us take the politics out of the process and enable NHS England and NICE to do their jobs, even under a new contract. I ask the Minister to do her job and to put a new framework in place.
Laura Smith (Crewe and Nantwich) (Lab)
It is a pleasure to serve under your chairmanship in this hugely important debate, Mr Hanson. I thank all the constituents who signed the petition, and those in the Public Gallery.
I am not surprised that my constituency featured so high up on the list of respondents, because my constituent, Elle Morris, became the face of a poster campaign this year pleading for life-changing drugs to be available to UK sufferers of cystic fibrosis. I was lucky and privileged to know Elle, who was from Nantwich, and to have her visit us here in Parliament. She sadly passed away in her parents’ arms at Great Ormond Street Hospital for Children on 3 January 2018.
Elle had undergone a successful double lung transplant in July 2017, but complications set in when microbacteria, which originally infected her lungs, got into her bones. Sadly, despite treatment, which included the removal of part of her sternum and rib cage, she passed away. Now her image is being used in a campaign pleading for others to get access to the potential life-prolonging treatment drug, Orkambi. Her face has been displayed on four of the country’s largest digital screens, in Birmingham, the south-east, south London and Manchester, promoting #OrkambiNow. It goes without saying that we in Crewe and Nantwich are exceptionally proud of everything that Elle achieved in her short life, and all that she continues to achieve. Her campaign group, Elle’s Wishes, documented her brave journey, including her support, alongside the successful campaign run by the Daily Mirror, for opt-out organ donation.
Ahead of today’s debate I asked Elle’s mother Becky, a friend of mine, whether there was anything specific that she wanted me to mention to the Minister. Becky is an amazing credit to her daughter and continues to campaign tirelessly for CF sufferers. She said:
“Orkambi becoming available is so important to Elle’s memory and it would prevent future heartbreak and suffering to other CF patients and their families. It’s that step closer to maintaining health, and preventing more damage to a sufferer until a cure can be found. Organ donation is the only option currently and the cost is huge, and as we found not always successful as it suppressed the immune system which is vital for CF sufferers to keep strong and built up. If it was your child, would you do anything possible to keep them alive and as healthy as can be? Is there really a cost between a child and their health and their life? Elle was a true CF warrior and fought so hard. She appreciated life so much, the simple things like the countryside, school, spending time with her friends. She loved her home and her cups of tea. She could have potentially still been enjoying those things and looking forward to her 14th birthday in January 2020 but instead she didn’t reach her 12th birthday. Please, please, as a bereaved mother missing her beautiful daughter every day consider doing everything you can to help those that still have hope. Why should they suffer? Yours Becky Whitfield.”
I have many other constituents, as we all have, who are fighting CF or supporting family members who are suffering. It is frankly not good enough that Orkambi, which we all know can help to save lives, is not currently available on the NHS unless compassionate grounds are given. We have debated the subject before, and we know that cystic fibrosis affects about 10,400 children and adults in the UK, that Orkambi will change cystic fibrosis care for the better, and that it works for eight in 20 people. About 50% of individuals with cystic fibrosis in the UK have the genetic mutation that Orkambi can tackle, 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 other countries including Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Greece, and the United States. I agree with the Health and Social Care Committee that it is hugely disappointing that NHS England and Vertex are yet to come to an agreement. I ask the Minister to explain her understanding of the position that has been reached. The Scottish Medicines Consortium has managed to agree a figure, and we must do the same.
It is disgusting that, while children such as my constituent die from their condition, it is reported that a pharmaceutical giant such as Vertex can charge the NHS an unaffordable price for a life-changing drug and get a huge increase in its revenue. Vertex reported that its net income more than doubled in the last quarter from $158 million to $337 million. It said that the jump in profits was
“largely driven by the strong growth in total CF product revenues”.
It is frankly disgraceful that we live in a world so driven by profit-making and the market that such companies can hold lives to ransom in that way. I challenge Vertex to waive confidentiality so as to give evidence for its claim to have made
“the best offer in the world”
to the NHS.
The NHS has made its proposals public. That big pharmaceutical firm must do the same if it is to have any chance of having the public’s confidence that it is behaving in an ethical way. Failing to do so only helps to build the case against the continuation of the marketised pharmaceuticals industry in its current form. Perhaps it is time for Government to reconsider their role in the sector and to intervene, with legislation or by other means, to prevent such situations, where profit margins become the primary concern, rather than the lives of children who suffer from CF, like Elle.
Karl Turner (Kingston upon Hull East) (Lab)
It is a pleasure to see you in the Chair, Mr Hanson. I am grateful to be called to speak in this incredibly important debate on behalf of my young constituent, Oliver Ward, who is seven years of age. I raised the issue in Prime Minister’s questions last week, and I intend, if I get the opportunity, to raise it with the Prime Minister again before she leaves No. 10 Downing Street.
I want to pay tribute to Oliver and to his mum, Emma Gadie, who has campaigned tirelessly on CF. In particular, she has raised the issue of the battle with Vertex for Orkambi. She says her little boy is her hero, and has described his daily routine, which includes taking up to 23 pills a day, and having incredibly rigorous physio just to be as normal as he can be during the day.
It seems to me that the situation is a perfect example of predatory capitalism. Vertex has a turnover of $45 billion, and the chief executive rakes in something of the order of £15 million a year—I think I am right in saying that he has £100 million in share sales to his name—and yet he is holding the NHS to ransom. People are literally dying while the business behaves in an intolerable way. I saw a tweet recently in which Vertex was celebrating its 30th birthday, but some sufferers do not make it beyond 31. It is utterly despicable. I try to be non-partisan and non-party political about it, but it is about time the Secretary of State got into a room with Vertex, NHS England and NICE and sorted it out.
The Prime Minister said on 16 May last year that she expected a “speedy resolution” to the situation. Frankly, the Secretary of State, the right hon. Member for West Suffolk (Matt Hancock), should spend more time sorting out this incredibly important issue than travelling around the country trying to ingratiate himself with Tory party members in order to get himself into 10 Downing Street. This is appalling. It is not going away—hon. Members on both sides of this House are not going to let it go away—and the Minister must take action following this debate.
Clive Lewis (Norwich South) (Lab)
It is a pleasure to serve under your chairmanship, Mr Hanson. I add my thanks to the hon. Member for Sutton and Cheam (Paul Scully) for securing this debate.
I will focus on two key things: asking the Minister a series of questions on potential remedies for this situation, but also using my time to speak about Rachael and Ethan, a mother and child in my constituency who are forced to live with the realities of cystic fibrosis every day, and the adverse impact that the actions of the company Vertex are having on their lives. Ethan is 11 years old. He has a big smile and loves pizza, trampolining and spending time with his friends, but sadly that is where the similarity to other 11-year-olds ends, since every day his life and that of his mum Rachael are dominated by the strain of dealing with cystic fibrosis.
To put this in context, because his lung function has declined to just 54%, in the past four weeks alone Ethan has coped with two separate full-day hospital admissions, 14 days of intravenous antibiotics, which are administered by his mum twice a day at home and take 45 minutes each time, and two sessions with a psychologist to help him to overcome needle phobia brought about by years of blood tests. All that is on top of his regular daily cocktail of medication and a physio session of a minimum of 22 minutes every day. Yet none of those treatments are designed to cure Ethan. They are simply designed to treat his symptoms—symptoms that are expected to get worse the older he gets. They attempt to slow down the irreversible lung damage that will slowly cause him to suffocate. He knows that. In 2017, half of all people who died with cystic fibrosis were under the age of 31, as has already been stated. It is a statistic that his mum Rachael is only too aware of as she spends the majority of her time caring for her son.
We know that Vertex drugs could change the lives of cystic fibrosis sufferers and their families, since they fix the underlying genetic mutations that cause the condition, but, as we have heard today, Vertex is more interested in 45% investor returns than in 20%. That seems to be its priority. Although the chronic underfunding of the NHS is a contributing factor in this story, it is by no means the only reason why a deal has not been reached. The unforgivable actions of Vertex Pharmaceuticals, which admittedly does important work in research and development relating to the treatment of cystic fibrosis, mean that it continues to put patients’ lives at risk as it seeks to extract the highest possible price from our NHS. When Alexander Fleming created penicillin, he had it publicly patented so that it was accessible to all, and it became a revolution in modern medicine. Should we not be legislating for pharmaceutical companies to do something similar and put patients, not profits, at the centre of their development?
I have a number of questions for the Minister. How do we approach this matter systematically, so that we are not back here time and time again, as we have heard? The reality is that we have a socialised—dare I say socialist—healthcare system, which treats people’s health on a collective basis, based not on their ability to pay, but on their need. That is quite revolutionary. However, that healthcare system operates in an international pharmaceutical industry based on rapacious profiteering. How do we square that circle? I suggest the answer lies at the European level, not the US level. Ultimately, the EU’s being one of the biggest healthcare markets in the world gives the European Union immense clout in imposing its will on international pharmaceutical companies; that is one of the reasons I want to stay in it.
My other question is a philosophical one: what is a fair price for years of investment and research? Who determines what is a fair price—our collective democracies, which enable these companies to exist, or a handful of corporate executives whose primary motivation is to maximise profit? Who determines that price? At the moment, I do not think that the balance is correct.
Finally, has the Minister considered the use of compulsory licensing, allowing a UK company or other company to make cheap generic copies? That is allowed under World Trade Organisation rules; the definition of an emergency is up to the host country that needs to use it, and from initial research it is something that could be used and has been used before. I know that is not something she would want to do initially, but ultimately, would she consider it if Vertex refuses to move?
I will leave hon. Members with a quote from Ethan, who had his 11th birthday on 5 June. He said:
“I want to live a long life, because then I get to see some things and do things, so please fund The Triple”.
Jim Shannon (Strangford) (DUP)
I congratulate the hon. Member for Sutton and Cheam (Paul Scully) on setting the scene so well for us, and all the right hon. and hon. Members who have made such fantastic contributions on a subject in which we all have a deep interest.
This is an issue that I have spoken on many times in this Chamber and indeed outside it. I have received emails from constituents with photos of their children, begging me to do something to give these precious little ones a better quality of life. If ever we needed to be reminded of the importance of this for the children, as every hon. Member has said, that is such a reminder.
Let us be clear: cystic fibrosis is not only, tragically, a life-limiting disease, but a disease that massively impacts on the quality of life and the life experiences of the sufferers and their families, because the families live the children’s tragedy as well. Cystic fibrosis is one of the UK’s most common life-threatening inherited diseases. It is caused by a defective gene carried by one person in 25, usually without their knowing it. That is more than 2 million people in the UK, and if two carriers have a baby, the child has a one in four chance of having cystic fibrosis.
Around 10,400 people in the UK have cystic fibrosis; that is one in every 2,500 babies born. It affects some 100,000 people in the world. According to the most recent report from the UK Cystic Fibrosis Registry, based on people with CF who were recorded as alive from 2013 to 2017, half of people born with cystic fibrosis in 2017 were expected to live to at least 47, but the 132 people with CF who died in 2017 had a median age of 31. That is a massive difference and we cannot ignore it.
Parents are begging me, begging us, begging this House to ensure that those years are of the best possible quality. After numerous trials, some carried out with Northern Ireland constituents, Orkambi seems to be a drug that does exactly that for many people—enabling the best possible quality of life. My most recent correspondence from Richard Pengelly, the permanent secretary for health in Northern Ireland, outlined clearly that he does not have the power to do what we all need him to do and what he wants to do:
“Let me say that I share your disappointment that the progress in making this drug more widely available has not advanced as we had hoped. At the heart of this matter is the inability of the manufacturer Vertex to come to agreement with the relevant UK Health Technology Assessment bodies.”
Mr Gregory Campbell (East Londonderry) (DUP)
When we have the most senior civil servant in Northern Ireland, along with NHS England and virtually everyone else who has any dealings with the issue, saying, “Look, we need action, we are powerless to move,” does that not throw the ball firmly back into the Government’s court to resolve the matter with the company?
Jim Shannon
I thank my hon. Friend for those words. This is not an easy subject for the Minister to respond to, but it is one that has captured the interest of us all on behalf of our constituents, and we need the Government to grasp that and move it forward to the next place. We look for that.
If the Republic of Ireland is able to come to some arrangement with Vertex, if the Scottish Parliament is able to do similar and if, according to the background information, it is possible to go to Argentina and buy a year’s course of drugs for one patient at £23,000, compared with £104,000 for a year’s supply here, that tells me that something can be done if we had the willpower to do it, as my hon. Friend the Member for East Londonderry (Mr Campbell) has said. We can look around at our UK neighbours and look toward Scotland, whose Government have reached an agreement with Vertex.
The permanent secretary went on to say:
“In the absence of this positive NICE determination, the Health and Social Care Board…may take into account guidance produced by other appropriate HTA”—
health technology assessment—
“bodies based in other UK countries such as the Scottish Medicines Consortium…when making decisions about access to new drugs.”
I say to the Minister that I have made a comment about the Republic of Ireland, but I also make a comment about Scotland, because I think that the process enables us to use what Scotland has done as an example for us elsewhere.
The permanent secretary continued:
“The Department is aware that Vertex have re-applied to the SMC for consideration of approval for Orkambi, and that in the meantime there is currently limited access to the drug in Scotland via their PACS”—
peer approved clinical system—
“Tier 2 scheme, which is broadly analogous to our Individual Funding Request Process.
If Orkambi is approved by the SMC, details of the funding models in place, which are currently bound by commercial confidentiality, will be shared with the other UK countries. This will allow for us to have full access to the evidence and costs associated with this therapy and will inform any further decisions on access.”
The hon. Member for Colne Valley (Thelma Walker) referred to the destruction of some medications. Whatever the reason for that was—whether they had run out of time or whatever—I think it is disgraceful that people have destroyed some medicines rather than letting them be used by the general public, by those who need them. If that is not unacceptable in this day and age, I do not know what is. It is absolutely disgraceful; it really annoys me.
It is simply terrible that we are in a position where our hand are tied. I say again to the Government: look to your Scottish counterparts. I firmly believe that we can and must do more from this place and that that must start with acknowledging that the NICE guidelines do not currently take into account the differences, when it comes to pricing, between treatments for rare diseases and a new antibiotic strain. We need a new form of assessment for rare diseases and I would like to see that taking place as soon as possible to ensure that the mummy of my two-year-old constituent, who is asking me for this drug in order to give her child as normal a life as possible, can look forward to securing the best for her child. That is what every Member has said here today on behalf of their constituents.
Again according to the background information that I have, in May 2019 the Government said in response to a parliamentary question that discussions between Vertex, NHS England and NICE were ongoing. You know something? They have been ongoing for more than a year. Let’s get them sorted. Time is passing fast. I am joining colleagues in asking the Department to make the administrative changes necessary to end the Orkambi stalemate with NICE and to put in place a body designed specifically to address rare disease patients and their needs. We acknowledge that NICE does a tremendous job in ensuring that safe, cost-effective medicines are available on prescription, but we need a different set-up for those whose illnesses are very different and for the sake of my constituents and those represented by other MPs who have spoken today. I am asking that those decisions be taken and the changes made to enable Richard Pengelly, the permanent secretary at the Department of Health in Northern Ireland, to do what he knows he needs to do and allow the prescription of Orkambi to those whose lives would be radically altered and enhanced by it. It would give them life-changing opportunities. As others have said, give those children a chance.
David Hanson (in the Chair)
The previous Chair set a time limit on speeches of six minutes, which has been successful, in that all right hon. and hon. Members have been able to take part in the debate, but it leaves us with a maximum of one hour and 10 minutes for the Front-Bench speakers to respond. Front-Bench speakers, who have more than the normal 10 minutes, can be more flexible and take interventions should they so wish.
The other point I wish to make is that because the winding-up speeches have started early, some Members who have spoken in the debate are not present for these speeches and they should be. If you are watching on television, please return to the Chamber now. This is an important debate; you have contributed to it and you should be present for the Front-Bench responses.
Marion Fellows (Motherwell and Wishaw) (SNP)
It is a pleasure to serve under your chairmanship, Mr Hanson. I thank the hon. Member for Sutton and Cheam (Paul Scully) for setting out in his opening speech facts that a lot of us did not know and facts that some of us did. It was a very useful start to this wide-ranging and well attended debate. I will just ask this question, though: how many times do we have to debate this very serious subject?
Again, I have to declare a personal interest. I am a cystic fibrosis carrier. My late husband was, too. My children are carriers, and my granddaughter, Saoirse Grace, has cystic fibrosis—mutations F508del and D1152H. I do not understand to any great degree what the last part of that actually means, but I do know that she is not the worst sufferer of cystic fibrosis. She has the best kind, if you like. She is pancreatic sufficient, and for that we are always grateful. Saoirse will not directly benefit from Orkambi, but along with 90% of people with cystic fibrosis in the UK, she will benefit from the triple therapy coming down the line. Those therapies will deliver unprecedented improvements in acute lung health and reductions in pulmonary exacerbations—a key driver of decline.
Approximately 900 people in Scotland live with cystic fibrosis. NHS Scotland estimates that one in 24 Scots has a CFTR mutation, which, if carried by both parents, would lead to a child being born with cystic fibrosis. England has the highest prevalence of cystic fibrosis in the world, with 1% of the world’s population but 12% of the world’s CF population.
There should be no postcode lottery for treatment. The UK Government should follow the Scottish Government’s lead on cystic fibrosis treatment. For people in England to receive Orkambi, NHS England would need to pay Vertex £500 million over five years and £l billion over the next 10 years. However, in a debate on CF and Orkambi last year, it was stated that the costs of Orkambi can be offset by reduced hospital admissions, and other benefits should also offset the cost, as has been stated in the Chamber today. Vertex has stated that its offer to NHS England for the provision of its CF drugs represents the lowest price for Vertex’s portfolio of CF drugs in any country in the world. I point out that I am not here to make a case for Vertex; I am here to make a case for people with CF who need this drug.
The Scottish Government recently announced that NHS Scotland and Vertex Pharmaceuticals had reached a deal to provide interim access to Orkambi and Symkevi, through a system known as peer approved clinical system tier 2, while the Scottish Medicines Consortium reviews the relevant evidence for Orkambi. PACS tier 2, introduced by the Scottish Government in June 2018, involves an individual application for patient access to drugs not recommended by the SMC and not routinely available on the NHS.
The Scottish National party believes that the UK Government need to do more to facilitate interim access to Orkambi and other drugs to treat cystic fibrosis and to get NICE to re-evaluate making the drug more widely available for people living with the illness. In England, NICE said Orkambi was too expensive for the NHS in 2016, and since then Vertex and NICE have been unable to reach an agreement that will bring these drugs to patients. The UK Government are showing a lack of flexibility by only urging Vertex Pharmaceuticals to fully re-engage with the NICE appraisal process and to accept the offer that the NHS made in July 2018. Vertex has said that the methods used by the Scottish Government to obtain the drugs reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases such as cystic fibrosis, and that it is hopeful that, through that process, all eligible patients in Scotland could have access to its medicines soon. If Orkambi and Symkevi are accepted by the SMC, which makes decisions independently of the Scottish Government, eligible patients in Scotland could have access to these precision CF medicines in 2019.
In the interim, Vertex and the Scottish Government have agreed a confidential discount that would be applied to approved PACS tier 2 applications. The Scottish Government also asked Vertex to provide access to the medicines at a discounted price to the list price while they finished the contract negotiations, and Vertex said, “Of course.” Vertex stated that it would be willing to do exactly the same thing in England.
There is agreement across the Chamber that Orkambi, Symkevi and Vertex’s triple therapy should be available UK-wide. I have no desire to get into the argument between NICE and Vertex in England. I want England to do what Scotland has done. I want children across the UK to get the benefits of these drugs. As has been said, this problem will exist whatever view we hold of Government at the UK level. However, political will must be exercised in the process of getting those with cystic fibrosis the drugs that will improve their lives and futures.
Finally—I will not take too long, Mr Hanson—I give my heartfelt thanks to all the parents and guardians of CF sufferers, and to those affected by cystic fibrosis, for their continuous campaigning and awareness-raising. It is awful that they have to keep doing that. I ask the Minister, please do not let their work be in vain.
Mrs Sharon Hodgson (Washington and Sunderland West) (Lab)
It is a pleasure to serve under your chairmanship, Mr Hanson, in this very important debate. I thank the hon. Member for Sutton and Cheam (Paul Scully) for opening this debate on behalf of the Petitions Committee and for his excellent opening speech, in which he took many interventions. I thank all hon. Members for their passionate contributions. I counted more than a dozen speeches by Back Benchers, but I lost count, because I was distracted by the mouse that joined us. Given that it has been such a busy day in the main Chamber, this debate has shown how important this issue is to the House, as well as to all our constituents who have signed this important e-petition.
Access to drugs for patients with cystic fibrosis is an issue that is incredibly important to us all. I congratulate, in particular, my hon. Friends the Members for Bristol East (Kerry McCarthy) and for Jarrow (Mr Hepburn), and the hon. Member for Dudley North (Ian Austin) on their tireless campaigning on this issue, along with patients, campaigners and charities, such as the Cystic Fibrosis Trust. I commend the trust for its expert briefings and support to patients and their families over many years.
Finally, I thank the 108,144 people who signed this e-petition, 310 of whom live in my constituency. As has been said, we debated a similar e-petition in March 2018, and there have already been five parliamentary debates about access to such medicines. I hoped, as others did, that by now cystic fibrosis patients would have access to the drugs that they need and deserve. Unfortunately, that has not been the case.
Just over a year ago, on 16 May 2018, in response to a question from my hon. Friend the Member for Erith and Thamesmead (Teresa Pearce), the Prime Minister called for a “speedy resolution” to the crisis. However, the only speedy thing has been the deterioration of the health of cystic fibrosis sufferers while they wait for a resolution of the crisis.
More than 10,000 people in the UK live with cystic fibrosis, and it is thought that around 50% of CF patients could benefit from Orkambi; that is more than 5,000 people. Although that is a lot of people, in NHS terms it is a small cohort. And yet Orkambi is still not available to patients, despite being licensed for use in the UK since 2015.
The UK is currently a world leader in cystic fibrosis outcomes, but that is changing. People with cystic fibrosis are physically sicker than they would be if these medicines were available to them. NHS England continues to make offers to Vertex, including the largest ever financial commitment in its 70-year existence, but that was rejected. Since then, an even better offer has been made, but again Vertex has been unwilling to accept it.
I know that that is frustrating for patients and their families, who have waited years for access to these life-saving drugs. We are all frustrated on their behalf. As my hon. Friend the Member for Colne Valley (Thelma Walker) Valley mentioned, in March it was reported in the news that nearly 8,000 packs of Orkambi had been destroyed because they were past their sell-by date. That would have particularly stung patients and their families. Those drugs were valued by Vertex at £104,000 per patient per year. With my limited maths skills, I reckon that means that more than £60 million of drugs were destroyed by Vertex—drugs that could have been given to patients.
It is an outrage that Vertex would destroy so many packs of a life-saving drug while in a cost dispute with NHS England. So many patients could have benefited from those drugs. It was spiteful of Vertex to watch those drugs go out of date so they would have to be destroyed. In the midst of all that, Vertex reported a 40% rise in its revenues, with net income doubling in the previous quarter. I am sure I am not alone in feeling shocked and angry at that.
By refusing to play fair with NHS England, Vertex is holding lives at ransom, and patients and their families are the ones left suffering. Therefore, I was not surprised by the feature on “Newsnight” last week about parents and families establishing a cystic fibrosis buyers’ club to buy the generic drug Lucaftor from Argentina. The stress and frustration that families face because of Vertex mean that they now feel they have no other option but to take matters into their own hands.
Laura Smith
It is great to hear my hon. Friend’s response. It seems perverse to me that the interests of big pharmaceutical companies can hold such enormous sway in this country, to the extent that cystic fibrosis sufferers can be left without their treatment—treatment that has the potential to prolong their lives significantly.
Mrs Hodgson
I agree with my hon. Friend. I hope that the Minister will have some ideas about how this drug company can be held to account and not be allowed to continue in this way. I hope the Minister agrees that the situation should never have been allowed to get to this stage.
Lucaftor has the same active ingredients as Orkambi, and the Argentinian pharmaceutical company Gador is offering a price of £23,000 per patient per year, which drops to £18,000 if patients and their families can get together a group of more than 500 patients to purchase Lucaftor as a collective. That is significantly lower than the £104,000 Vertex wants for Orkambi. I say “want” deliberately—it is not the cost, but what Vertex wants. Of course, for many patients in the UK, Lucaftor will still be way too expensive to access, so it is not a feasible alternative at all. That is why NHS England and Vertex need to come to a conclusion that puts cystic fibrosis patients first, and ensures that they have access to the life-saving drugs they need and deserve.
Kerry McCarthy
I thank my hon. Friend for all her support on this issue. I agree that the issue with the Argentinian solution is that in a buyers’ club where people have to pay privately, the drugs will still be out of reach for many people. However, the fact that Gador is offering this drug for so much less than Vertex is charging for a similar product means that the NHS could, if it decided to trial the drug, buy it for 4,000 patients who would benefit from Orkambi. Therefore, no one would have to pay for it privately. The NHS could fund it, but at much less than Vertex is asking for. I ask the Minister: why is that not the solution?
Mrs Hodgson
I was going to come on to that, but if a point is worth making once, it is worth making twice. I will make it to the Minister as well, so she will have plenty of time to think about it.
As we all agree, patients and their families should not be put in the position—as some are—of having to pay thousands of pounds for their treatment. Family income should not determine who lives and who dies. That is why the NHS was founded—so that all could have access to the same excellent treatment, regardless of means. That was true 70 years ago when the NHS was formed, and it is still true today.
As the hon. Member for Sutton and Cheam pointed out, our NHS is there for us all and should not be held to ransom by a pharmaceutical company, but neither should access be denied because of unfit processes and systems in the NHS. Over the years, as a shadow public health Minister, I have met many patient groups, including those with cystic fibrosis, who are missing out on life-changing medicines because their condition is not rare enough and is therefore not deemed by NICE to be cost-effective. We need an appraisal process that is fit for purpose and that will capture rare diseases such as cystic fibrosis effectively.
Without drugs such as Orkambi, patients and their families are being harmed physically and psychologically. Every day without the drugs that patients need makes their condition worse and threatens their lives. What steps will the Minister take to ensure that patients with rare diseases have access to the medicines that they need and deserve? It is about access not just to Orkambi, but to other precision medications such as Symkevi and the next generation of cystic fibrosis drugs that could help patients who are suffering.
Vertex recently announced the headline results for its fourth cystic fibrosis medicine, a triple combination therapy that could radically transform the lives of nine in 10 people who live with cystic fibrosis in the UK, delivering unprecedented improvements in acute lung health. That is amazing news, but patients fear that they will never be able to access this ground-breaking drug. I urge Vertex to put patients first and consider the real-life impact of this cost dispute on patients and their families.
Vertex and NHS England must come to an agreement urgently, because patients have already waited far too long. If an arrangement cannot be made soon, will the Minister personally step in and pursue the alternatives that my hon. Friend the Member for Bristol East mentioned, such as a Crown use licence or a clinical trial? Cystic fibrosis patients need urgent access now to the drug that they have been denied for three years. It is time the Government considered all alternatives.
The Parliamentary Under-Secretary of State for Health and Social Care (Seema Kennedy)
It is always a particular pleasure to serve under your chairmanship, Mr Hanson. I thank my hon. Friend the Member for Sutton and Cheam (Paul Scully) for opening the debate on behalf of the Petitions Committee. I pay tribute to the more than 100,000 people who signed the petition, and I thank all right hon. and hon. Members who have spoken in the debate; I am sure that they will be rushing back for the wind-ups.
I have been very touched by the stories that we have heard today and the compassion shown by my hon. Friend and all hon. Members in speaking about cystic fibrosis and its physical effects, emotional effects and effects on mental health for those who live with it and for their families. It is a debilitating condition, and I know how absolutely desperate sufferers and families are for access to treatments.
I recognise the great work undertaken by the Cystic Fibrosis Trust and its strong voice in supporting families and bringing cystic fibrosis to the attention of parliamentarians. I also pay tribute to my young constituent Lucy Baxter, who was on “BBC Breakfast” this morning and who lives with cystic fibrosis. She spoke to me very soon after I became a Member of Parliament and is an absolute inspiration to me and to the whole cystic fibrosis community.
Today’s debate has been heartfelt and passionate. The stories that we have heard clearly make the case that Orkambi and other drugs for people with cystic fibrosis should be available on the NHS at a price that is fair and affordable. The Government and I share that view. As the Chair of the Health and Social Care Committee, the hon. Member for Totnes (Dr Wollaston), set out so clearly, we must remember that the NHS must use its budget fairly for the good of all patients. That is why we rightly have a system whereby experts, not politicians, determine the fair price for medicines, based on robust evidence. That system has helped many thousands of patients to benefit from rapid access to effective new medicines.
Karl Turner
Forgive me, but I genuinely do not know the answer to this—I find it completely confusing. If the Republic of Ireland and Scotland can get an interim agreement, why cannot we sort this out for patients here in England?
Seema Kennedy
I will talk about the interim measures, but I think the more important thing that we need to grip is having a permanent solution for everybody living with cystic fibrosis.
Throughout the negotiations, which are rightly being led by NHS England, the Government have been crystal clear that Vertex must re-engage with the NICE process. I know that hon. Members have questions about that process, and I will try to address some of the points that have been raised. The hon. Member for York Central (Rachael Maskell) raised more detailed points for me to consider; if I do not address them, I will write to her with more detail, but there are some points about the NICE process that I will address later in my remarks.
Jim Shannon
Would it be possible for the Minister to give her Department’s and her own opinion on the buyers’ group that is seeking to buy similar drugs from Argentina for cystic fibrosis? Have the Government given that possibility any consideration?
Seema Kennedy
I shall turn later in my speech to that specific avenue that some sufferers have gone down.
Dr Wollaston
The Minister referred to the importance of Vertex engaging with NICE. Does she share my concern that when I wrote to Vertex and NICE about the failure to make progress, Vertex assured me that it had contributed “substantial new evidence” on the three products in question, yet I subsequently heard from NICE that it had received only
“an overview of the clinical evidence”,
rather than genuine engagement? Will she join me in calling on Vertex to properly engage with the process, so that we can get the full evidence base on which to make these decisions?
Seema Kennedy
I agree with the Chair of the Health and Social Care Committee and urge Vertex to re-engage with the NICE process. To date, unfortunately, it has continued to refuse to accept the process or has suggested unacceptable conditions on the NICE value assessment of its product, which would render the outcome meaningless. That comes despite NHS England’s latest proposals offering to reimburse Vertex ahead of a positive NICE recommendation, which for a deal of this size is unprecedented; agreeing to implement real-world data collection, as the Committee has called for, to help Vertex to demonstrate the value of its medicines; and offering significantly increased prices in comparison with their offer last July.
NICE has a 20-year history and is internationally renowned and independent. Its methods and processes for the development of its guidance have been in place for 20 years, but it recognises that it needs to evolve. It continues to review its procedures to ensure that they remain fit for purpose; it is now undertaking a review of its technology appraisal methods in line with the commitment in the 2019 voluntary scheme, and it encourages all stakeholders to engage. NICE has recommended 75% of the drugs for rare diseases—some of which I will touch on later—that have been assessed through its technology appraisal programme for the eligible patient population.
Last week, the Association of the British Pharmaceutical Industry made it clear that
“NICE is the cornerstone of NHS efforts to ensure the price being charged by a company represents the value being delivered.”
Commenting on the current situation, it said that
“the APBI would always encourage companies to fully engage with NICE at all stages of the process.”
Furthermore, it commented on the current structure of NHS England’s proposed deal with Vertex, saying that
“the structure of the offer represents exactly the sort of flexibility the industry has been calling for, for some time.”
However, Vertex is willing to accept only its own valuation of Orkambi; I draw your attention, Mr Hanson, to comments directed at Vertex by Members from across the House, including those made by the shadow Minister, the hon. Member for Washington and Sunderland West (Mrs Hodgson).
Jeremy Lefroy (Stafford) (Con)
I am grateful to the Minister for giving way and I apologise for not being here at the beginning of the debate, because of important statements in the main Chamber.
My constituent William Smith, who is a pupil at Cooper Perry Primary School, has cystic fibrosis. Along with his entire class, he presented me with the facts of his case and with letters to the Government, and they made it quite clear that they expect not only the Government but Vertex to come to a decision on this matter. Is it not absolutely vital that a company such as Vertex should engage with the Government given that the lives and futures of people such as William Smith, my 10-year-old constituent, are at stake?
Seema Kennedy
My hon. Friend makes a very good point. Members from across the House have told very moving stories of their constituents, the lives they lead, and the stresses and the strains put on them by the lack of an agreement on this matter. However, other drug companies are developing medications for rare diseases, and agreements have been reached on those. I will turn to them very shortly.
We can look at what happened in Spain earlier this year, when Vertex did not accept the terms of Spain’s health outcome-related proposal. The Spanish proposal, which is similar to the recent NHS England offer, is based on the ongoing collection and interpretation of real world data. Why is that not acceptable to Vertex? I also note that dialogue between Spain and Vertex has been ongoing for three years, which is similar to the situation here in England.
We will never walk away, but Vertex must now agree to engage with NICE and we urge it to accept all the flexibilities that NHS England has put on the table. There is nothing unusual about Vertex that means that this is not the right thing for it to do. Recently, we have seen deals reached as part of the NICE appraisal process, including that for ocrelizumab, which is an innovative multiple sclerosis drug, that for Spinraza, which is for people with spinal muscular atrophy, and that for axicabtagene ciloleucel chimeric antigen receptor t cell, or CAR-T, treatments.
Given that Vertex remains an extreme outlier in both pricing and behaviour, it is no wonder that patients and families have been looking at alternative solutions to secure access to this drug, and we have heard about the buyers’ club. Hon. Members have also talked about Crown use licensing, and the hon. Member for Bristol East (Kerry McCarthy) talked about large-scale clinical trials.
Unless Vertex changes its approach and behaves responsibly, I have a moral obligation to look at these other options. Of course NHS England and NICE will carry on the negotiations, because a negotiated outcome is the desired option. However, I have no alternative but to look at these other options on the table.
Clive Lewis
I thank the Minister for giving way. I raised the issue of compulsory licensing, which the Government have within their armoury. Of course it would be a measure of last resort, but given that we are dealing with people’s lives, the quality of their lives and a company that is quite simply being intransigent, and greedy, surely that option should be considered. That would send a message to those pharmaceutical companies—that global pharma industry—that if they are going to be greedy and put people’s lives at risk, despite being made fair offers, this option could be used by our Government.
Seema Kennedy
I thank the hon. Gentleman for his intervention. As my hon. Friend the Member for Sutton and Cheam said in his opening speech, which was very well made, we recognise the importance of British pharmaceutical companies and that companies invest hugely in developing new drugs. However, as the other examples of drugs for rare diseases that I have given show, it is possible to go through the NICE appraisal process and reach an agreement with NHS England. As one hon. Member who is no longer in their place said, this is an offer for a long-term agreement.
Vertex is an outlier, and I would like to put that on the record.
Seema Kennedy
Yes, and I hope that I will be able to remember the hon. Lady’s question.
Mrs Hodgson
At this point I should clarify, for the benefit of the campaigners who I have spoken to about the Crown use licensing option, that it is not an immediate solution from their point of view; I understand that it would take at least a couple of years. If an agreement can be reached, there would be an immediate outcome. That is why the campaign is called Orkambi Now; it is about trying to get the drug now. Although the Crown use licensing option would be an option to consider if nothing else can be found, it would not give the sufferers and their families the drugs as quickly as we would like.
Seema Kennedy
As always, the shadow Minister makes an excellent point. Crown use licensing is not something that any Government would consider lightly. It is very rarely used in health. It has probably not been used—my officials will correct me if I am wrong—since the 1970s.
The ideal thing is to get a deal, and deals have been done with other pharmaceutical companies; that is the point I want to make. As I have said, Vertex is an outlier in this regard, but that does not mean that I do not have an obligation to look at other options. I will do that.
Kerry McCarthy
I really welcome the fact that the Minister is saying so passionately that she feels she has a moral obligation to act. The question is: when? I say that because Vertex has been in these talks for a very long time now. How much longer will it be given before the Minister decides to look at the other options?
Seema Kennedy
Now. We will look at other options to consider what other methods we can use. As the hon. Member for Washington and Sunderland West said, Crown use licensing has other risks, so the best option is to get a deal. However, we will look at other methods that might enable families to receive the drugs they need.
Some Members have said that Orkambi is available in other countries and asked, “So why not here?” Although that is true, it is also true that other countries have faced problems in agreeing an acceptable price with Vertex; around 50% of the global cystic fibrosis population is unable to access Orkambi. It is not approved for reimbursement in Spain, which I have touched on, or in Portugal, and it is not used routinely in France. It is also of note that the Canadian equivalent of NICE has rejected the drug, saying that the benefit of Orkambi is small and uncertain.
We do not have sight of specific commercial agreements where Orkambi is approved and we do not have the same population needs, as we know, because of the specific population that the UK has; compared with other countries, the UK has a very high proportion of people with cystic fibrosis. So I am not able to make comparisons with other countries. Cystic fibrosis affects about 10,500 people in the United Kingdom—a far higher figure than in other countries. It represents 12% of the global cystic fibrosis population. The UK is a very important market for Vertex.
I thank all Members who have spoken so passionately here today. In particular, I thank the people who have joined us here in the Gallery; I pay tribute to them for all that they do to support their family members. I also thank those people, such as Lucy Baxter, who work so hard to raise awareness of this issue. I thank members of the Health and Social Care Committee, which is carrying out a very thorough and transparent inquiry into this issue, helping to shine a light on it.
As we have heard in great and moving detail today, cystic fibrosis is a devastating, life-limiting condition, and the bravery of those affected should be an inspiration to us all. Drugs that improve sufferers’ quality of life should be available where appropriate, and I urge Vertex to do everything it can to price its medicines fairly and in a way that reflects the health benefits to patients.
NHS England and NICE will, of course, continue their efforts to reach an agreement with Vertex. Access to treatment for all patients is, and always will be, a priority for this Government. My Department has a moral obligation to look at other options now, and that is what we shall do.
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.