NICE Appraisals: Rare Diseases Treatments — House of Commons, 2019-03-21
Topic: Health
Kerry Gillian McCarthy (Labour, Centre-left): I will be brief because I have spoken on this topic many times, specifically about cystic fibrosis, but before I get on to that I congratulate my hon. Friend the Member for Blaydon (Liz Twist) on her speech about PKU. I popped into the PKU drop-in event a few months ago purely by accident when I was doing one of those classic MP things where we are asked to go here, there and everywhere to be photographed holding up a sign or wearing a silly hat. I knew nothing about PKU, but I talked to an 11-year-old boy there about his diet. I am somebody who chooses to have a fairly restrictive diet in that I do not eat meat, dairy or fish, but I look for protein alternatives, and I was struck by the sheer difficulty that the boy has in functioning and by the fact that he is unable to enjoy the normal life of an 11-year-old. My heart went out to him, and I do not need to add any more, because my hon. Friend made such an excellent speech We have also had excellent speeches about the difficulties of obtaining Spinraza. I do not claim to be an expert, as some other Members are, but constituents have been in touch with me with similar problems. I would therefore support any efforts to get the drug approved, because that would make a huge difference to their lives As I said, I am here to talk about cystic fibrosis, and I have a personal interest in that my 14-year-old niece Maisie has CF. She must be the most mentioned niece in Parliament, because she gets name-checked quite a lot. Before I talk about Maisie, I want to mention the recent evidence to the Health and Social Care Committee from Oli Rayner, who lives with cystic fibrosis. He has just turned 40 and had a lung transport around 18 months ago, so he is at the healthier end of the spectrum, and he talked about being quite lucky until he reached his thirties, when his health started to deteriorate However, many cystic fibrosis patients are not as lucky. My constituent Lee Partridge lost both his daughters within eight months when they were both in their late teens, and it is common for people to start to deteriorate when they reach their teens. Luckily, my niece is very much at the healthier end of the spectrum, but that does not influence my judgment; I want to make the case for Orkambi to be available not just for her when it is needed, but for everybody else with the condition I have been trying to get my head around quality-adjusted life years, how the calculations are made, whether NHS England is doing the right thing and whether NICE is using the right sorts of calculations compared with other countries. NICE says the calculations factor in societal benefits, but it is not clear how they do that The hon. Member for Reigate (Crispin Blunt), who made an excellent speech, spoke about the cost of hospital treatment to society as a whole. If a person has to go into hospital several times a year, there is the cost of care, but there is also the cost of lost work time, the cost of care by family members and even the cost of transportation to hospital if they live in a rural area. There are so many factors. Although NICE says it takes those things into account, I cannot quite see them. Was it Hugh Dalton or Clement Attlee who was called a "desiccated calculating machine"? Anyway, the system seems to be based on desiccated calculations It may be incredibly naive of me, but why cannot the system just think of the impact on people’s lives and of the hell they are going through? It is incredibly difficult for teenagers to come to terms with discovering that they have a life-limiting condition, and there is also the issue of the availability of mental health support and counselling It is striking that everyone who gave evidence to the Health and Social Care Committee said that the drugs work. There is no question but that this would be of benefit to people. Kalydeco, the first drug, helped only 5% of patients, and it was funded in part because not many people would benefit and, therefore, the costs were lower. We now have Orkambi, and there will soon be a successor that will benefit about 40% of patients. For the future we are looking at triple therapies that will cover about 90% of the cystic fibrosis community. If 90% of people can benefit, of course the overall cost will go up, but the decision should not be made on the basis that we fund one thing because not many people will benefit I am concerned that Vertex and other companies would be deterred from exploring further therapies and treatments if they cannot get a commercial deal. The line that jumps out of the Health and Social Care Committee’s letter to Vertex, which it wrote shortly after the meeting, is: "Vertex appears to have decided on the pricing of its therapies on the basis of the return it wants to make, rather than the value which they bring. NHS England is right to continue to take the wider patient population for whom it is responsible into account." The Minister has previously responded to such debates, so can we have an update on where the conversations have got with Vertex, including the Secretary of State’s meeting? There are so many cystic fibrosis patients waiting to hear the outcome. Last time I looked, the petition was up to about 80,000 signatures. Can we try to get some answers soon?