Cystic Fibrosis Drugs: Orkambi

Laura Smith Excerpts

Monday 10th June 2019 Westminster Hall

[Laura Smith (Crewe and Nantwich) (Lab)]

It is a pleasure to serve under your chairmanship in this hugely important debate, Mr Hanson. I thank all the constituents who signed the petition, and those in the Public Gallery.

I am not surprised that my constituency featured so high up on the list of respondents, because my constituent, Elle Morris, became the face of a poster campaign this year pleading for life-changing drugs to be available to UK sufferers of cystic fibrosis. I was lucky and privileged to know Elle, who was from Nantwich, and to have her visit us here in Parliament. She sadly passed away in her parents’ arms at Great Ormond Street Hospital for Children on 3 January 2018.

Elle had undergone a successful double lung transplant in July 2017, but complications set in when microbacteria, which originally infected her lungs, got into her bones. Sadly, despite treatment, which included the removal of part of her sternum and rib cage, she passed away. Now her image is being used in a campaign pleading for others to get access to the potential life-prolonging treatment drug, Orkambi. Her face has been displayed on four of the country’s largest digital screens, in Birmingham, the south-east, south London and Manchester, promoting #OrkambiNow. It goes without saying that we in Crewe and Nantwich are exceptionally proud of everything that Elle achieved in her short life, and all that she continues to achieve. Her campaign group, Elle’s Wishes, documented her brave journey, including her support, alongside the successful campaign run by the Daily Mirror, for opt-out organ donation.

Ahead of today’s debate I asked Elle’s mother Becky, a friend of mine, whether there was anything specific that she wanted me to mention to the Minister. Becky is an amazing credit to her daughter and continues to campaign tirelessly for CF sufferers. She said:

“Orkambi becoming available is so important to Elle’s memory and it would prevent future heartbreak and suffering to other CF patients and their families. It’s that step closer to maintaining health, and preventing more damage to a sufferer until a cure can be found. Organ donation is the only option currently and the cost is huge, and as we found not always successful as it suppressed the immune system which is vital for CF sufferers to keep strong and built up. If it was your child, would you do anything possible to keep them alive and as healthy as can be? Is there really a cost between a child and their health and their life? Elle was a true CF warrior and fought so hard. She appreciated life so much, the simple things like the countryside, school, spending time with her friends. She loved her home and her cups of tea. She could have potentially still been enjoying those things and looking forward to her 14th birthday in January 2020 but instead she didn’t reach her 12th birthday. Please, please, as a bereaved mother missing her beautiful daughter every day consider doing everything you can to help those that still have hope. Why should they suffer? Yours Becky Whitfield.”

I have many other constituents, as we all have, who are fighting CF or supporting family members who are suffering. It is frankly not good enough that Orkambi, which we all know can help to save lives, is not currently available on the NHS unless compassionate grounds are given. We have debated the subject before, and we know that cystic fibrosis affects about 10,400 children and adults in the UK, that Orkambi will change cystic fibrosis care for the better, and that it works for eight in 20 people. About 50% of individuals with cystic fibrosis in the UK have the genetic mutation that Orkambi can tackle, so the approval of Orkambi for use on the NHS could benefit about 5,200 people living with cystic fibrosis. Orkambi has been shown to slow decline in lung function by 42% and cut the number of infections requiring hospitalisation by 61%. It gives patients not only more control over their lives but a greater quality of life.

Orkambi is available for patients in other countries including Austria, Denmark, France, Germany, Luxembourg, the Netherlands, Italy, Greece, and the United States. I agree with the Health and Social Care Committee that it is hugely disappointing that NHS England and Vertex are yet to come to an agreement. I ask the Minister to explain her understanding of the position that has been reached. The Scottish Medicines Consortium has managed to agree a figure, and we must do the same.

It is disgusting that, while children such as my constituent die from their condition, it is reported that a pharmaceutical giant such as Vertex can charge the NHS an unaffordable price for a life-changing drug and get a huge increase in its revenue. Vertex reported that its net income more than doubled in the last quarter from $158 million to $337 million. It said that the jump in profits was

“largely driven by the strong growth in total CF product revenues”.

It is frankly disgraceful that we live in a world so driven by profit-making and the market that such companies can hold lives to ransom in that way. I challenge Vertex to waive confidentiality so as to give evidence for its claim to have made

“the best offer in the world”

to the NHS.

The NHS has made its proposals public. That big pharmaceutical firm must do the same if it is to have any chance of having the public’s confidence that it is behaving in an ethical way. Failing to do so only helps to build the case against the continuation of the marketised pharmaceuticals industry in its current form. Perhaps it is time for Government to reconsider their role in the sector and to intervene, with legislation or by other means, to prevent such situations, where profit margins become the primary concern, rather than the lives of children who suffer from CF, like Elle.

--- Later in debate ---

[Mrs Sharon Hodgson (Washington and Sunderland West) (Lab)]

It is a pleasure to serve under your chairmanship, Mr Hanson, in this very important debate. I thank the hon. Member for Sutton and Cheam (Paul Scully) for opening this debate on behalf of the Petitions Committee and for his excellent opening speech, in which he took many interventions. I thank all hon. Members for their passionate contributions. I counted more than a dozen speeches by Back Benchers, but I lost count, because I was distracted by the mouse that joined us. Given that it has been such a busy day in the main Chamber, this debate has shown how important this issue is to the House, as well as to all our constituents who have signed this important e-petition.

Access to drugs for patients with cystic fibrosis is an issue that is incredibly important to us all. I congratulate, in particular, my hon. Friends the Members for Bristol East (Kerry McCarthy) and for Jarrow (Mr Hepburn), and the hon. Member for Dudley North (Ian Austin) on their tireless campaigning on this issue, along with patients, campaigners and charities, such as the Cystic Fibrosis Trust. I commend the trust for its expert briefings and support to patients and their families over many years.

Finally, I thank the 108,144 people who signed this e-petition, 310 of whom live in my constituency. As has been said, we debated a similar e-petition in March 2018, and there have already been five parliamentary debates about access to such medicines. I hoped, as others did, that by now cystic fibrosis patients would have access to the drugs that they need and deserve. Unfortunately, that has not been the case.

Just over a year ago, on 16 May 2018, in response to a question from my hon. Friend the Member for Erith and Thamesmead (Teresa Pearce), the Prime Minister called for a “speedy resolution” to the crisis. However, the only speedy thing has been the deterioration of the health of cystic fibrosis sufferers while they wait for a resolution of the crisis.

More than 10,000 people in the UK live with cystic fibrosis, and it is thought that around 50% of CF patients could benefit from Orkambi; that is more than 5,000 people. Although that is a lot of people, in NHS terms it is a small cohort. And yet Orkambi is still not available to patients, despite being licensed for use in the UK since 2015.

The UK is currently a world leader in cystic fibrosis outcomes, but that is changing. People with cystic fibrosis are physically sicker than they would be if these medicines were available to them. NHS England continues to make offers to Vertex, including the largest ever financial commitment in its 70-year existence, but that was rejected. Since then, an even better offer has been made, but again Vertex has been unwilling to accept it.

I know that that is frustrating for patients and their families, who have waited years for access to these life-saving drugs. We are all frustrated on their behalf. As my hon. Friend the Member for Colne Valley (Thelma Walker) Valley mentioned, in March it was reported in the news that nearly 8,000 packs of Orkambi had been destroyed because they were past their sell-by date. That would have particularly stung patients and their families. Those drugs were valued by Vertex at £104,000 per patient per year. With my limited maths skills, I reckon that means that more than £60 million of drugs were destroyed by Vertex—drugs that could have been given to patients.

It is an outrage that Vertex would destroy so many packs of a life-saving drug while in a cost dispute with NHS England. So many patients could have benefited from those drugs. It was spiteful of Vertex to watch those drugs go out of date so they would have to be destroyed. In the midst of all that, Vertex reported a 40% rise in its revenues, with net income doubling in the previous quarter. I am sure I am not alone in feeling shocked and angry at that.

By refusing to play fair with NHS England, Vertex is holding lives at ransom, and patients and their families are the ones left suffering. Therefore, I was not surprised by the feature on “Newsnight” last week about parents and families establishing a cystic fibrosis buyers’ club to buy the generic drug Lucaftor from Argentina. The stress and frustration that families face because of Vertex mean that they now feel they have no other option but to take matters into their own hands.

[Laura Smith]

It is great to hear my hon. Friend’s response. It seems perverse to me that the interests of big pharmaceutical companies can hold such enormous sway in this country, to the extent that cystic fibrosis sufferers can be left without their treatment—treatment that has the potential to prolong their lives significantly.

[Mrs Hodgson]

I agree with my hon. Friend. I hope that the Minister will have some ideas about how this drug company can be held to account and not be allowed to continue in this way. I hope the Minister agrees that the situation should never have been allowed to get to this stage.

Lucaftor has the same active ingredients as Orkambi, and the Argentinian pharmaceutical company Gador is offering a price of £23,000 per patient per year, which drops to £18,000 if patients and their families can get together a group of more than 500 patients to purchase Lucaftor as a collective. That is significantly lower than the £104,000 Vertex wants for Orkambi. I say “want” deliberately—it is not the cost, but what Vertex wants. Of course, for many patients in the UK, Lucaftor will still be way too expensive to access, so it is not a feasible alternative at all. That is why NHS England and Vertex need to come to a conclusion that puts cystic fibrosis patients first, and ensures that they have access to the life-saving drugs they need and deserve.