Orkambi and Cystic Fibrosis

Alison McGovern Excerpts

Monday 19th March 2018 Westminster Hall

[Ian Austin]

That is a really important point and one that I hope the Minister will take into account. We should be looking not just at the cost of providing Orkambi, but at the savings that that would make in other areas. I want to develop that point in a few minutes.

What I did not understand before speaking to people with cystic fibrosis was the toll that not knowing whether they will be given these life-saving drugs takes on their mental health. I am talking about the worry that it causes them and their families and the stress and fear that it puts them through. Something else that I did not understand before meeting Carly was the impact that having a condition that reduces life expectancy has on the rest of someone’s life. Lynsey Beswick, who many hon. Members will recall was at the roundtable a few weeks ago, explained that very well. She is in her 30s and told me that, at a time when her friends are getting married, planning families, developing their careers and starting businesses—making long-term plans—people such as her are deterred from doing those things. They just cannot plan for their futures in the same way because, to put it bluntly, they do not know how long they have to live.

Since having Orkambi, Carly has been able to go on holiday abroad for the first time with her family. She has married. She has started a business. Let us think about that. She has started a business, so she is employing people and making a much bigger contribution to the economy. People talk in these debates about the cost of providing these drugs. Let us talk also about the contribution that people who are given Orkambi can make to society. Let us think not just about the cost, but about the contribution they make, the businesses they can start, the jobs they can create and the taxes that will be paid. Let us think about that as well. Let us think about the contribution that providing Orkambi can make to our economy.

What is worse, the longer people live without access to drugs such as Orkambi—I had not really appreciated this—the worse their lungs become. Every day that access to this drug is delayed is another day on which the lungs of people with cystic fibrosis are damaged. I want to repeat that point, because I really want people to think about this: every day that people who could be helped by Orkambi are denied it is another day on which their lungs suffer irreparable damage.

That damage will not be put right. It is not like some medical conditions whereby the patient is given a drug and they are cured, restored and put back to how they were originally. That is not the case here. That damage will not be put right when—or if—they eventually get this drug. The damage that has been done is permanent. Every day this treatment is delayed limits the lives of people it could help. I really want the Minister and others to understand that point, because I must confess that I had not fully understood it until I chaired that roundtable with the Cystic Fibrosis Trust and Vertex a few weeks ago.

[Alison McGovern (Wirral South) (Lab)]

My hon. Friend is making a brilliant and important speech. I am here because of my constituent Ava and her family. Ava loves horse riding and her family want her to have the opportunity to live her life as an ordinary, healthy seven-year-old. That is all they want. Orkambi could provide her with that opportunity. Is not it people such as Ava and the people my hon. Friend has mentioned whom we have to keep at the forefront of our minds?

[Ian Austin]

My hon. Friend is completely right. This is what politics is about. What are we here for? We are here to listen to people in our constituencies. It is our job to come here and speak up on their behalf, which is exactly what she has just done.

I want the Minister to look really carefully at the way in which NICE works. This is not a criticism of NICE, the Government or anyone else, but new drugs are being developed and technological changes are happening so rapidly that I want to ask whether the way in which drugs are assessed, licensed and approved still works. My central question is: how are Ministers going to ensure that these ground-breaking drugs and new developments are made available much quicker?

In 2016, NICE was not able to recommend the use of Orkambi due to uncertainty about its long-term value, impact and cost-effectiveness. Vertex submitted a fresh proposal last month. On Friday, NHS England said:

“Following advice from Nice, the NHS has asked this particular drug company to review its proposed pricing. Unless this happens, further progress at this time is frankly unlikely.”

What sort of hope does a blunt statement such as “frankly unlikely” give people with cystic fibrosis? How does it give us any confidence that new drugs such as Symdeko, which is due to have its marketing authorisation confirmed by the European Medicines Agency in the coming works, will be approved as well?