Cystic Fibrosis Drugs: Orkambi

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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?

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.

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.

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.”