Orkambi and Cystic Fibrosis

Liz Kendall Excerpts

Monday 19th March 2018 Westminster Hall

[Liz Kendall (Leicester West) (Lab)]

The hon. Gentleman is being very generous with his time. One hundred and eighteen of my constituents signed the petition, so I am very grateful for the debate. He speaks with great passion about the huge challenges facing people living with cystic fibrosis. I agree that NHS England and the drug company need to get together to resolve this issue as quickly as possible. However, does he agree that the bigger issue is about ensuring that NICE has the proper processes in place to make sure that new drugs are agreed as quickly as possible? Does he also agree that the Government should review that, to make sure, not only with this issue but with others, that people have confidence in the process and that that follows the right principles and guidelines?

[Paul Scully]

I thank the hon. Lady for that intervention, and I totally agree with her.

I will finish on the issue of living with cystic fibrosis by saying that in addition to having physio and nebulisers, sufferers often have to take a huge cocktail of drugs, as the hon. Member for South Antrim said. The cupboard that I saw for Grace was bigger than one of my kitchen cupboards and it was just full of medicines. There have to be savings in that respect. To return to the hon. Lady’s point, it is really important that we have a system that is fit for purpose. We can sit there and put sticking plasters, as it were, on all these things, but that is what we need. When we are talking about the health service as a whole, it is not always about the big headlines that we argue about across the Chamber. It is about efficiency and ensuring that things work in the modern age. For modern diseases, there need to be modern treatments and so on.

--- Later in debate ---

[James Frith (Bury North) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Evans. My constituent Graham Little got in touch with me. His wife Claire has cystic fibrosis. Until that moment, I was not aware of the potential life-changing drug available to sufferers of cystic fibrosis. Graham and his father-in-law Brad raised with me the status of the precision medicine Orkambi. The result of today’s debate could make the difference and give them decades more with their wife and daughter, Claire, and give her decades more with her children. I cannot imagine how that must feel.

We can either observe the steady death caused by cystic fibrosis—it has been compared to drowning—or reach for the life jacket, which is primed, ready for use and in all sizes. Our country has the largest number of cystic fibrosis patients anywhere in the world, so we ask the Government what they are going to do for our cystic fibrosis sufferers. If they will not make Orkambi available, what are they going to do? The transformative, life-prolonging impact of the drug is tantamount to life-saving. Used early enough, it can ensure that people fulfil their potential. It can prolong life and lung capacity, enabling those with the disease to contribute to the economy and wider society, as well as providing extra decades with family and loved ones.

The Government should consider the case for a commissioning body for rare diseases. It is not right that the judgment criteria used to determine funding on globally common diseases are universally applied when the uncommon traits of a rare disease bring untypical symptoms and a different economic argument. We risk overlooking the incredible efficacy of the drugs now available.

The power in numbers is not much solace to a cancer sufferer, but such is the profile of cancer treatment that we are all assured of the progress being made there. The same cannot be said for the rare diseases argument, however. We have a job to do in making the case for rare diseases. I hope the Minister will listen to the case being made for a medicine and treatment decision-making body for rare diseases, as well as consider how we bring NICE into the modern age, 20 years after it was established.

[Liz Kendall]

My hon. Friend will know that the Government review NICE once every three years. Does he agree that the next review, which will start in July this year, must look seriously at how NICE makes its decisions? Does he also agree that the results of that review should come out quickly? Last time, it took about a year and a half.

[James Frith]

I completely agree with my hon. Friend. We need to see far more nimble-footed decision making by the decision-making body, as per my call for a specific dedicated body for rare diseases. Parents in my constituency make the case about the plight of those with rare diseases. There is a constant sense of those enduring rare diseases being overlooked.

We come to the remaining agents at the table. Just this weekend, NHS England has refused to agree to the deal that Vertex proposed last month. I do not think the wording of the refusal was constructive, but I am not surprised that the NHS was unwilling to sign up in principle to the deal, which amounts to a futures prospectus from Vertex off the back of a sure thing with Orkambi. Patient groups are at the table, and I commend the work that the Cystic Fibrosis Trust is doing on behalf of CF sufferers.

We need compromise and urgency from all. Nobody should expect Vertex to surrender its drugs, but neither should we accept a failure to reach an agreed price. I say to Vertex that the pipeline might need to be for another time. When I met Vertex, I cautioned that its “portfolio of drugs” approach may be cost-prohibitive to a decision, and that appears to have been the case. We need Orkambi for patients today. It is not the time to test the innovative decision making of NHS England. Vertex should take in good faith that a fair price to the world’s largest population of cystic fibrosis sufferers will set it up well for future developments. A price somewhere between what it was offering in its recent pipeline deal and a single purchase deal is where we are now. I ask Vertex to please stay at the table. We keep in mind its proposal of having Orkambi readily available, swiftly and easily, for all. We urge focus on enabling that. The rest will surely follow.

At the table, there is a ministerial chair that needs filling by a Minister who is willing to lean in to the debate and signal their support for prescribing Orkambi on the NHS; willing to lead and lean in to the deal-making part of the job to transform lives; willing to look at the costs and to help with the price; and willing to align the political will to the possibility of life for those with cystic fibrosis. Treatment for cystic fibrosis has moved from science fiction to science fact, so the Minister is required to act and step in. The Government have a choice to make. They can ignore our arguments, or, having heard them, choose to change their mind. In changing their mind, they can change lives and lifetimes. I urge the Minister to do that and make real-life change for cystic fibrosis sufferers a reality. He would have support from Members across the House, as has been demonstrated by the magnificent strength of feeling in the contributions and arguments made today.