Orkambi and Cystic Fibrosis

Oliver Letwin Excerpts

Monday 19th March 2018 Westminster Hall

[Kerry McCarthy (Bristol East) (Lab)]

It is a pleasure to see you in the Chair, Mr Evans, and I hope that you received my note, saying that I would have to leave before the end of the debate—I am sorry about that.

As my hon. Friend the Member for Dudley North (Ian Austin) said, some of us very much hoped that we would not be back here making the case for Orkambi, because our being here again means that we still have not managed to persuade the Government and the National Institute for Health and Care Excellence to fund it. However, I have been absolutely overwhelmed by the fact that so many MPs have turned out today and that, as has been said, the petition gained the required 100,000 signatures within 10 days. That is phenomenal and I cannot believe that the Government will not listen to that outpouring of support.

Constituents have contacted me about their children, their grandchildren and their friends’ children—I had a lovely email from a family friend who has a little boy aged seven. I particularly want to pay tribute to Lee Partridge in Bristol for his work with the south-west parents cystic fibrosis support group. He lost his daughter Richelle in 2016 to cystic fibrosis, aged 26, and eight months later her sister Lauren died, aged 19, from the same illness. I cannot begin to imagine what that family has gone through, although I have a 13-year-old niece, Maisie, with cystic fibrosis, whom I have mentioned here before.

Maisie has been in pretty good health considering, but I have listened to Members talk about how people’s health can suddenly take a dramatic dip, with their lung function percentage going from somewhere in the 90s way down perhaps to 20, and I realise that I cannot be too optimistic. I can look at her today and say, “This is a happy, healthy child”. Apart from being hospitalised when she was first diagnosed at a few weeks old, she has had intravenous therapy only once, and that was almost a preventive measure because her lung function had dipped a little.

I have visited the children’s cystic fibrosis unit in Bristol and the adult treatment centre at Bristol Royal Infirmary, and many people spend their entire lives coming in and out of hospital, so we have been very lucky, but Maisie still has to have a cocktail of pills. When I spend time with her, her whole life is dominated by which pills she has to take. Every time she eats something she has to take her Creon. She has to have physiotherapy. She has also just been diagnosed with the very rare Wieacker-Wolff syndrome—we have suddenly discovered that one other child in the country has it. I thank the Government for supporting the deciphering developmental disorders study into 12,000 children and adults with undiagnosed conditions that has been carried out over the past couple of years, because it was only through that that we knew what was causing Maisie’s mobility problems and mild learning difficulties. The mobility problems obviously make it more difficult for the physio to be done and for her to be more independent.

[Sir Oliver Letwin (West Dorset) (Con)]

What the hon. Lady describes is a quality of life. Does she agree that, at root, what we are dealing with here is the problem that NICE measures the quality of life-adjusted years and does not measure improvements in quality of life of the kind she describes?

[Kerry McCarthy]

How do we measure the cost to the family? How do we measure Orkambi’s worth? To me, it would clearly be life changing, perhaps even life saving. When my hon. Friend the Member for Dudley North hosted his roundtable, it was reassuring to hear that Orkambi is another step forward—Kalydeco helps only 2% of people and we think that Orkambi could help 40%—and that drugs are in the pipeline, being worked on now, that may bring help to all cystic fibrosis patients in this country. It was said that it is too soon to judge whether the developments, rather than being just life-prolonging, could be life-saving, with cystic fibrosis being a condition that people live with, like diabetes, rather than one that people die from. Surely that is where we want to get to.

The overall picture is that getting one drug on the market is a stepping stone to further research, and it is about buying time, as the right hon. Member for Chingford and Woodford Green (Mr Duncan Smith) said. Maisie is now 13 and it is imperative that she get on such a drug if she turns out to be one of the children who can be helped, because, frankly, we do not have an awful lot of time before her condition might deteriorate. I cannot stress enough how important that is to us as a family. She obviously has hopes for the future. At the moment, she wants to star in musicals, which might be slightly over-ambitious. She has just got back from a horse-riding weekend in Wales.

I finish on that note of buying time. We do not have the luxury of time. We need to see action now from the Minister and we need to look at the overall cost to the health service and to the families. Maisie’s mother—my sister—is an absolute saint. She is a teaching assistant. She works with children with disabilities. She is a forest school leader. What she does in her daily life is wonderful, juggling that with a disabled child and two other children as well. If Maisie takes a turn for the worse, my sister will effectively be removed from the workforce and become a full-time carer. We ought to consider costs like that as well. Let us not look at it as a crude mathematical calculation but as what it means for people’s lives.