Orkambi and Cystic Fibrosis

Stella Creasy Excerpts

Monday 19th March 2018 Westminster Hall

[Tulip Siddiq (Hampstead and Kilburn) (Lab)]

It is a pleasure to serve under your chairmanship, Sir Roger. I pay tribute to the hon. Member for Sutton and Cheam (Paul Scully). I usually apply to speak in Westminster Hall rather than in the main Chamber because the waiting time tends to be shorter, but I have had to wait today because of the huge turnout, which is testament to his tenacity in securing this important debate. Many constituents have emailed us about how their condition has affected their lives. I will cut straight to the chase and say that all the people who emailed me were unanimous in their desire to see Orkambi made available on the NHS.

In all the representations I have received, whether from families who have been affected by the disease or from world class medical professionals in Hampstead and Kilburn, there is one clear message: cystic fibrosis patients do not have time to waste. I put this bluntly not because I want to state the obvious about a life-limiting condition, which has been made clear by all the contributors so far, nor to suggest that Ministers object to introducing the precision medicines that could extend life chances. I say it because it is the key message that must weigh upon all our contributions today.

I presume that most Members have received a briefing from the Cystic Fibrosis Trust, which does exceptional work in advocating for those suffering from the condition. I am proud to say that I have a constituent, Ffyona Dawber, who is a trustee and is passionate about this debate and patient education more broadly. Ffyona and her colleagues at the trust have told me over and over again about the possible benefits of Orkambi. They say that medicines such as Orkambi have been proven to add years to a patient’s life. They point to the fact that Orkambi has been shown to slow decline in lung function by 42% and to cut the number of infections requiring hospitalisation by 61%. As has been quoted many times, they point to the NICE appraisal of 2016 that said that Orkambi is both “important and effective”. Given that the possible benefits of Orkambi seem to be settled, it is necessary to spell out why the Government should act now and agree a sustainable solution over the cost.

It is also important to highlight the voices of those whose futures depend on the introduction of that important drug. My hon. Friend the Member for Erith and Thamesmead (Teresa Pearce), who is sitting next to me, talked about how we have to speak up for residents and constituents who cannot come here and speak for themselves. Many who have the condition cannot travel to Parliament to witness the debate.

I echo the words of a resident of mine, Caroline Brown, who wrote to me about her treatment for cystic fibrosis. She told my office that she felt “well supported” by the NHS. She paid specific tribute to the doctors and nurses at the Royal Brompton Hospital on the Fulham Road, saying that

“they are utterly amazing. I have had the best care there and I cannot fault them.”

Her tribute to the staff at the Royal Brompton reflects those paid by other local residents, especially those whose children rely on the specialist cystic fibrosis centre at Great Ormond Street. For patients such as Caroline, my constituent, the debate over Orkambi is about enabling our world-leading medical community to focus their efforts more sharply on those with advanced symptoms, and for pharmaceutical companies to get on with investing in research for aspects of the condition where the breakthrough represented by Orkambi still evades us.

It will come as no surprise to anyone to learn how Caroline felt about the situation surrounding Orkambi. As expected, she was clear in her support for its immediate roll-out. She said:

“When I look to the future it would be very comforting to know that, if I was to need it, there would be medication there that would help me. I can’t bear the thought of knowing that there is something out there that could potentially save my life, and that I am not able to take that as I couldn’t afford it. Being symptom free for someone with Cystic Fibrosis would be life changing.”

She went on to underline the consensus that exists among cystic fibrosis sufferers. A lot of them cannot meet and be in the same room for the reasons outlined by other Members, but there is one benefit of social media—I stress one benefit—which is that it has enabled a network of people with cystic fibrosis to discuss and reflect upon the key debates in the community and to share the experience of their treatments. She knows that fellow patients are unanimous:

“They all agree it should 100% be made available on the NHS. It should be available to everyone that needs it...it is sad that money is getting in the way of people's health improving.”

[Stella Creasy (Walthamstow) (Lab/Co-op)]

My hon. Friend is making an incredibly powerful speech. May I add the voice of my six-year-old constituent? She would not benefit from Orkambi, but we must recognise that, if we do not get this right, the other treatments that might help her will be subject to similar delays. She is only six years old, but she does not need any delay. Does my hon. Friend agree that this matter is not only about Orkambi, but about how we deal with life-changing drugs and whether we understand the power of them to make such a difference?

[Tulip Siddiq]

I absolutely agree with my hon. Friend. There are lots of heart-breaking stories from people who are six and even younger who share the frustrations of the people I am speaking for today.

Caroline, my constituent, was adamant that Orkambi is just one frustration that those with cystic fibrosis have over how the current range of treatments are made available. What aggravates my constituent is the fact that cystic fibrosis is the only incurable life-threatening disease where people have to pay for their prescriptions. I greatly sympathise with her view that charging patients for medication, in light of the fact that they often undertake between three to five hours of treatment a day, does not seem to square up to the rhetoric of Ministers who pledge their support for the cystic fibrosis community. As my constituent concludes, some people have to take thousands of pills over their lifetime, so paying for them makes life very difficult. Why should cystic fibrosis be an exception when people with other diseases do not have to pay?

I will close with three questions that I want the Minister to answer. Do the Government accept responsibility for the pace at which negotiations are being conducted, and will they apologise for the anxieties that patients are experiencing as a result? Secondly, what steps are Ministers taking to ensure that pharmaceutical companies will ensure that fair and responsible pricing for a deal can always be agreed when treatments are required on the NHS? Finally, will the Minister acknowledge the importance of finding a solution that guarantees we are never put in this position again for the future pipeline of treatments for cystic fibrosis and many other life-threatening conditions?