Orkambi and Cystic Fibrosis

Paul Sweeney Excerpts

Monday 19th March 2018 Westminster Hall

[Bim Afolami]

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

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

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

[Bim Afolami]

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

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

--- Later in debate ---

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

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

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

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

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

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

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

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

[Mr Sweeney]

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

[Marion Fellows]

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

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

--- Later in debate ---

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

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

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

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

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

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

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

[Mr Sweeney]

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

[Mrs Hodgson]

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

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

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

In response to the online petition, the Government responded:

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

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