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Cystic Fibrosis Drugs: Orkambi
Exerpts for Laura Smith
Monday 10 June 2019

(7 months, 2 weeks ago)

Read Full debate
Westminster Hall
Department of Health and Social Care
Rachael Maskell Portrait Rachael Maskell (York Central) (Lab/Co-op) - Hansard
10 Jun 2019, 6 p.m.

It is a pleasure to serve under your chairmanship, Mr Bailey.

Leading up to this debate, I spoke to constituents, one of whom described the mental strain of knowing that a drug was available but could not be prescribed to their child. Another parent talked about the administration of 22 drugs, and the physio and hospital appointments that were needed, as well as the stress of their child being admitted to hospital. Their child is now 21 years old and there have been no changes, even though there is a miracle drug that could transform their life. Another constituent, Jack, who is seven years old and at the start of his journey, desperately wants hope for his future.

We are at the cutting edge of a generation of pharmaceutical breakthroughs that have the power to transform the lives of people who experience a range of medical diagnoses. It is an exciting point in our journey with medicine, and it could be transformative to patients and carers. To deny therapy is ethically abhorrent. Many Members today have said that this is about negotiation, but I say that negotiation is not the answer. We should not be wrestling over drugs time and time again; we need a completely different framework for addressing the cost of pharmaceuticals.

NICE needs an overhaul to ensure it is fulfilling its role and looking at patients’ life course. I know that it does that work, but it needs to look at its effectiveness over a patient’s life course and at the economic value it can bring not just to medicine, but to the wider economy. It also needs to ensure that the levers are in place for it to look at a portfolio of drugs, as opposed to looking at drugs one by one.

We need to understand how much the NHS can afford to pay for the benefits of new drugs, taking into account the health benefits that could be achieved with the money the NHS must find to pay for them. This assessment, based on a NICE appraisal, could inform a debate about national rebate agreements in the current pharmaceutical price regulation scheme. There are several costs involved: the manufacturing cost, the price the NHS can afford, the NICE assessment and the market price, which is driven primarily by the US market. That is a cause of frustration.

The argument among academics is that a mechanism of a national value-based rebate should reflect the difference between the amount the NHS is willing to pay for the benefits and the manufacturer’s asking price. That would provide better incentives for manufacturers to make long-term investment choices, recoup costs and deliver for the NHS. It would also provide fairer rewards for innovation; manufacturers that produced more effective drugs at affordable prices would not need to pay a rebate, but those that charged more would pay a higher rebate. That would not impact the list price for the global market, and the rebate could be spread across the portfolio to provide better opportunities for manufacturers to bring new products to market.

That would take the politics out of NICE and NHS England. Canada is currently developing something similar, to ensure that drugs reach the places they need to reach. It can be further incentivised: should a manufacturer not supply a product when its production costs mean it could do so without making a loss, it could lose its patent. That would provide the required leverage. We need to hold discussions about this not just across the sector, but with the European Medicines Agency; the impact could be greater with more countries on board.

[David Hanson in the Chair]

People will have seen reports about the generic drug from Argentina, and we have heard about it in the debate today. We cannot allow inequality in access to pharmaceuticals in our country to grow, because it creates health inequalities. People who can pay will be able to afford that drug, and they will have better health outcomes. In the light of the ethics of the debate, the Department of Health and Social Care needs to move fast, now.

We need to be alert when it comes to trade deals with the United States, which is significant to the debate. The US will clearly want to drop the carrot of big pharma in front of Ministers, but that is a serious trap. The President talked about the NHS being “on the table” in his speech about trade negotiations, and we must be alert to the real agenda. It is through big pharma that he will get access to the NHS. As we have already heard, with respect to the size of the NHS drugs budget, that is a way of controlling it. We need to be on top of that.

Instead of drug-by-drug negotiation, we need a framework leading automatically to the leverage required to procure medicines. That will make such a difference. It will remove the uncertainty about different pharmaceuticals, as we move forward through the exciting developments that are taking place. Let us take the politics out of the process and enable NHS England and NICE to do their jobs, even under a new contract. I ask the Minister to do her job and to put a new framework in place.

Laura Smith (Crewe and Nantwich) (Lab) Hansard
10 Jun 2019, 6 p.m.

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.

Karl Turner Portrait Karl Turner (Kingston upon Hull East) (Lab) - Hansard
10 Jun 2019, 6:07 p.m.

It is a pleasure to see you in the Chair, Mr Hanson. I am grateful to be called to speak in this incredibly important debate on behalf of my young constituent, Oliver Ward, who is seven years of age. I raised the issue in Prime Minister’s questions last week, and I intend, if I get the opportunity, to raise it with the Prime Minister again before she leaves No. 10 Downing Street.

I want to pay tribute to Oliver and to his mum, Emma Gadie, who has campaigned tirelessly on CF. In particular, she has raised the issue of the battle with Vertex for Orkambi. She says her little boy is her hero, and has described his daily routine, which includes taking up to 23 pills a day, and having incredibly rigorous physio just to be as normal as he can be during the day.

It seems to me that the situation is a perfect example of predatory capitalism. Vertex has a turnover of $45 billion, and the chief executive rakes in something of the order of £15 million a year—I think I am right in saying that he has £100 million in share sales to his name—and yet he is holding the NHS to ransom. People are literally dying while the business behaves in an intolerable way. I saw a tweet recently in which Vertex was celebrating its 30th birthday, but some sufferers do not make it beyond 31. It is utterly despicable. I try to be non-partisan and non-party political about it, but it is about time the Secretary of State got into a room with Vertex, NHS England and NICE and sorted it out.

The Prime Minister said on 16 May last year that she expected a “speedy resolution” to the situation. Frankly, the Secretary of State, the right hon. Member for West Suffolk (Matt Hancock), should spend more time sorting out this incredibly important issue than travelling around the country trying to ingratiate himself with Tory party members in order to get himself into 10 Downing Street. This is appalling. It is not going away—hon. Members on both sides of this House are not going to let it go away—and the Minister must take action following this debate.

Break in Debate

Mrs Sharon Hodgson Portrait Mrs Sharon Hodgson (Washington and Sunderland West) (Lab) - Hansard
10 Jun 2019, 6:31 p.m.

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 Hansard
10 Jun 2019, 6:37 p.m.

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 Sharon Hodgson Portrait Mrs Hodgson - Hansard
10 Jun 2019, 6:37 p.m.

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.