Orkambi and Cystic Fibrosis

Debate between Paul Sweeney and Sharon Hodgson

Monday 19th March 2018 Westminster Hall

[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.

Organ Donation (Deemed Consent) Bill

Debate between Paul Sweeney and Sharon Hodgson

2nd reading: House of Commons
Friday 23rd February 2018 Commons Chamber

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

I thank my hon. Friend the Member for Coventry North West (Mr Robinson) for securing this very important debate, for introducing this very important Bill and for his powerful and moving opening speech. I would also like to thank the right hon. Member for Chesham and Amersham (Dame Cheryl Gillan), the hon. Members for Mid Worcestershire (Nigel Huddleston), for North Devon (Peter Heaton-Jones), for Dudley South (Mike Wood), for Chippenham (Michelle Donelan) and for Hendon (Dr Offord), and my hon. Friends the Members for Ealing, Southall (Mr Sharma), for Barnsley Central (Dan Jarvis), for St Helens South and Whiston (Ms Rimmer) and for Lincoln (Karen Lee) for their excellent speeches.

In particular, I pay huge tribute to my constituency neighbour, my hon. Friend the Member for Sunderland Central (Julie Elliott). She spoke so bravely and movingly about her daughter Rebecca, who as we heard has been on dialysis for a year awaiting a kidney transplant. I hope from the bottom of my heart—I am sure we all do—that her wait is over very soon and she is successful in receiving that gift of life from a wonderful donor.

This has been an excellent debate and an example of this House at its best, as it often is on Fridays during debates on private Members’ Bills. I would like to thank hon. Members who have previously brought this issue to our attention over the past decade or so, including my hon. Friends the Members for Mitcham and Morden (Siobhain McDonagh), for Newport West (Paul Flynn) and for Barnsley Central.

I commend the Daily Mirror for its fantastic campaign to raise awareness of organ donation since the case of Max Johnson, who we have all heard so much about this morning. He was then a nine-year-old boy in need of a new heart. I understand he is now 10, which is fantastic. I want to thank the hon. Member for North Devon for telling us all about Keira Ball, Max’s donor, who I understand saved four lives. I thank her very, very brave family for taking that brave decision on that most awful of days. I also thank the more than 13,000 people who have now signed the Change.org petition.

I also commend the scriptwriters of “Coronation Street”—of which, it has to be said, I am a huge fan as a northerner—for covering this issue so well. I note that the character Carla Connor this week received a kidney from her half-brother and that all is going well. At their best, soaps can play a huge part in helping to inform the public on such issues. I hope the storyline will touch on the importance of being on the organ donation register. Finally, I pay tribute to the thousands of people who have already participated in the Government’s public consultation on organ donation. I encourage others to do so, if they have not done so already, to let their voices be heard.

The topic of organ donation is understandably an emotional one, but I am pleased that so many people are now engaging in this debate and that we have the opportunity to discuss it in the House today. This debate and the publicity around it may encourage families up and down the country to have that important discussion about organ donation before the inevitable happens. There is no doubt that these discussions need to be had and that we need more organ donors in England. Almost 25 million people are on the organ donation register, but according to the NHS blood and transplant service, 7,000 people are waiting on the list for new organs. For them, it really is a life or death situation, so it is important that as many people as possible sign up to the organ donation register.

Over the past five years, almost 5 million people have joined the register, and in 2016-17 we saw the highest ever deceased donor rates in England. More than 50,000 people are living with a functioning transplant—Max is one of them—thanks to organ donation and transplantation in the UK. These are welcome developments, but we still have a long way to go. We currently lag behind other western countries. Tragically, around 1,000 people die every year—that is three a day—while waiting for a transplant. To save those lives, we need more people on the organ donation register making those decisions with their family’s knowledge, so that when the time comes, more lives can be saved.

[Mr Sweeney]

My hon. Friend makes an excellent case by citing statistics in England, but this is a cross-border issue as well. A great strength of our national health service is that no matter what part of the UK someone comes from, they can benefit from an organ transplant. If someone in Dumfries needs a kidney donation and the donor is from Carlisle, there will be no barrier or border on the route to getting access to that transplant. That is why MPs from all parts of the UK should support organ donation changes in all parts of the UK. That includes the Scottish National party; it is just a shame that SNP Members are not here today. Does my hon. Friend agree?

[Mrs Hodgson]

Yes, and I had not noticed that nobody from the Scottish National party is here. I do not know what the situation is in Scotland, but we still want people there to be organ donors. I am sure that Rebecca, the daughter of my hon. Friend the Member for Sunderland Central, would not refuse a kidney, whether from Scotland, Wales, Northern Ireland or anywhere, so that is a very good point.

[Mr Sweeney]

Will my hon. Friend recognise the great strides that the Labour party in Scotland has made in trying to bring in legislation on opt-out organ donation there? It is unfortunate that the Scottish National party blocked the progress of a Bill from Anne McTaggart MSP in the last session of the Parliament, but there is still hope, because a private Member’s Bill is progressing through the Scottish Parliament. We hope to have Labour and cross-party support to see such legislation progress in Scotland, as well as in Wales and England.

[Mrs Hodgson]

Excellent. I am really grateful to my hon. Friend for updating us all on the situation in Scotland because, as I said, I was not aware of it. I commend that Bill and hope that our SNP friends up in Scotland will act on and progress it as soon as possible.