Orkambi and Cystic Fibrosis

Marie Rimmer Excerpts

Monday 19th March 2018 Westminster Hall

[Ms Marie Rimmer (St Helens South and Whiston) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Evans. I congratulate the hon. Member for Sutton and Cheam (Paul Scully) on securing the debate. For good reason, the issue has galvanised popular opinion.

As we all know, cystic fibrosis is an extremely serious, debilitating and life-limiting—yet thankfully rare—diagnosis. As we have heard, in February, Vertex proposed a portfolio approach whereby all current and future cystic fibrosis medicines, including Orkambi, would be made available to eligible patients through a fixed budget cap to the NHS.

I was extremely disappointed to hear NHS England’s response on Friday that there will not be further funding for Orkambi. As it is a treatment that is proven to improve and prolong the lives of 50% of sufferers, that decision is a cruelty to cystic fibrosis sufferers across the country. The portfolio approach has been accepted and agreed in the Republic of Ireland, Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Greece and the US, but sadly, discussions here—with the world’s best health service—have broken down.

CF causes a build-up of mucus in the lungs that means that patients require physiotherapy, almost from birth, to keep them breathing properly. CF patients are more susceptible to lung infections and can suffer from malnutrition owing to the effect of the condition on the pancreas. When NICE made its initial evaluation of Orkambi as a treatment for the devastating condition, the evidence that it slows lung deterioration by 52% and reduces hospital admissions was not available, and therefore not costed. NICE is simply not fit for purpose any longer; it cannot evaluate a portfolio approach and precision drugs. For NHS England to turn its back on the small group of patients that suffers with this rare, debilitating illness and to ask Vertex to reapply to NICE is shocking. If it did reapply, it would get exactly the same answer—so why do it? Does NHS England understand that? Does NICE understand that? Are we going to send these seriously affected families around and around?

Vertex now has a broader pipeline of 18 drugs that will follow Orkambi in the next seven years. If the portfolio is agreed, it will advance on to those medicines. Will the Minister work towards a more flexible approach? Simply, he must get involved to benefit the small group of patients for whom a much better quality of life is in sight, but just out of reach, otherwise they will never get the treatment that they deserve and that they should be getting.

The parents of two constituents, Christian Jones and George Barton, contacted me. Christian’s mother, Hayley, wrote to me and said that Orkambi

“is not a cure but it is life altering, as it lowers admissions and reduces lung deterioration by 42%.”

She believes that Christian’s other medications will only increase unless he can access the drug. She also wrote:

“This genetic condition is awful to live with as a parent, wondering whether you will bury your child before they reach 20. I can’t begin to know how it must be for my son and other patients when they can’t breathe properly or speak without coughing or how his lungs must burn.”

She has nothing but praise for the NHS, in particular Whiston Hospital in my constituency where Christian has been treated since he was just weeks old. He is now two. In trials, Orkambi has been shown to help children aged between six and 11 to improve their oxygen uptake by up to 10%.

Orkambi would also be of immeasurable benefit to my constituent George Barton. George’s mother, Louise, wrote to me when she heard that the NHS’s provision of Orkambi was going to be debated in this place. George is eight years old and, like Christian and most other CF sufferers, was diagnosed as a young baby. CF is progressive, so George’s lungs are beginning to degenerate. Every day, he takes 30 to 40 tablets, has an hour of physio and takes two nebulised antibiotics. Louise told me that when he catches an infection, he can quickly become so seriously ill that he needs extra physiotherapy and intravenous antibiotics three times a day for a full two weeks. At times, George has to stay in Whiston Hospital, or, because he and Louise live nearby, the nurses come out and take care of him at home. In fact, when Louise first wrote to me, she was in hospital with George on a two-week stay. She wrote that although

“the damage to his lungs is becoming more serious, Orkambi can slow this down”.

George, Christian and thousands of other CF patients need that niche treatment, and there is the political will in this place to fight for them. Only about 3,000 people would benefit from the drugs. I urge the Minister to get involved, to facilitate further discussions between NHS England and Vertex, and to push for a flexible solution. Orkambi is a different type of drug that we cannot evaluate at this time. This is not only about licensing Orkambi, but the promise of those 18 drugs still to come, which Vertex will advance on to. NICE simply cannot evaluate and I urge the Minister to intervene.