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Cystic Fibrosis Drugs: Orkambi
Exerpts for Mrs Sharon Hodgson
Monday 10 June 2019

(7 months, 2 weeks ago)

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Westminster Hall
Department of Health and Social Care
Marion Fellows Portrait Marion Fellows (Motherwell and Wishaw) (SNP) - Hansard
10 Jun 2019, 6:23 p.m.

It is a pleasure to serve under your chairmanship, Mr Hanson. I thank the hon. Member for Sutton and Cheam (Paul Scully) for setting out in his opening speech facts that a lot of us did not know and facts that some of us did. It was a very useful start to this wide-ranging and well attended debate. I will just ask this question, though: how many times do we have to debate this very serious subject?

Again, I have to declare a personal interest. I am a cystic fibrosis carrier. My late husband was, too. My children are carriers, and my granddaughter, Saoirse Grace, has cystic fibrosis—mutations F508del and D1152H. I do not understand to any great degree what the last part of that actually means, but I do know that she is not the worst sufferer of cystic fibrosis. She has the best kind, if you like. She is pancreatic sufficient, and for that we are always grateful. Saoirse will not directly benefit from Orkambi, but along with 90% of people with cystic fibrosis in the UK, she will benefit from the triple therapy coming down the line. Those therapies will deliver unprecedented improvements in acute lung health and reductions in pulmonary exacerbations—a key driver of decline.

Approximately 900 people in Scotland live with cystic fibrosis. NHS Scotland estimates that one in 24 Scots has a CFTR mutation, which, if carried by both parents, would lead to a child being born with cystic fibrosis. England has the highest prevalence of cystic fibrosis in the world, with 1% of the world’s population but 12% of the world’s CF population.

There should be no postcode lottery for treatment. The UK Government should follow the Scottish Government’s lead on cystic fibrosis treatment. For people in England to receive Orkambi, NHS England would need to pay Vertex £500 million over five years and £l billion over the next 10 years. However, in a debate on CF and Orkambi last year, it was stated that the costs of Orkambi can be offset by reduced hospital admissions, and other benefits should also offset the cost, as has been stated in the Chamber today. Vertex has stated that its offer to NHS England for the provision of its CF drugs represents the lowest price for Vertex’s portfolio of CF drugs in any country in the world. I point out that I am not here to make a case for Vertex; I am here to make a case for people with CF who need this drug.

The Scottish Government recently announced that NHS Scotland and Vertex Pharmaceuticals had reached a deal to provide interim access to Orkambi and Symkevi, through a system known as peer approved clinical system tier 2, while the Scottish Medicines Consortium reviews the relevant evidence for Orkambi. PACS tier 2, introduced by the Scottish Government in June 2018, involves an individual application for patient access to drugs not recommended by the SMC and not routinely available on the NHS.

The Scottish National party believes that the UK Government need to do more to facilitate interim access to Orkambi and other drugs to treat cystic fibrosis and to get NICE to re-evaluate making the drug more widely available for people living with the illness. In England, NICE said Orkambi was too expensive for the NHS in 2016, and since then Vertex and NICE have been unable to reach an agreement that will bring these drugs to patients. The UK Government are showing a lack of flexibility by only urging Vertex Pharmaceuticals to fully re-engage with the NICE appraisal process and to accept the offer that the NHS made in July 2018. Vertex has said that the methods used by the Scottish Government to obtain the drugs reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases such as cystic fibrosis, and that it is hopeful that, through that process, all eligible patients in Scotland could have access to its medicines soon. If Orkambi and Symkevi are accepted by the SMC, which makes decisions independently of the Scottish Government, eligible patients in Scotland could have access to these precision CF medicines in 2019.

In the interim, Vertex and the Scottish Government have agreed a confidential discount that would be applied to approved PACS tier 2 applications. The Scottish Government also asked Vertex to provide access to the medicines at a discounted price to the list price while they finished the contract negotiations, and Vertex said, “Of course.” Vertex stated that it would be willing to do exactly the same thing in England.

There is agreement across the Chamber that Orkambi, Symkevi and Vertex’s triple therapy should be available UK-wide. I have no desire to get into the argument between NICE and Vertex in England. I want England to do what Scotland has done. I want children across the UK to get the benefits of these drugs. As has been said, this problem will exist whatever view we hold of Government at the UK level. However, political will must be exercised in the process of getting those with cystic fibrosis the drugs that will improve their lives and futures.

Finally—I will not take too long, Mr Hanson—I give my heartfelt thanks to all the parents and guardians of CF sufferers, and to those affected by cystic fibrosis, for their continuous campaigning and awareness-raising. It is awful that they have to keep doing that. I ask the Minister, please do not let their work be in vain.

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.

Kerry McCarthy Portrait Kerry McCarthy - Hansard
10 Jun 2019, 6:39 p.m.

I thank my hon. Friend for all her support on this issue. I agree that the issue with the Argentinian solution is that in a buyers’ club where people have to pay privately, the drugs will still be out of reach for many people. However, the fact that Gador is offering this drug for so much less than Vertex is charging for a similar product means that the NHS could, if it decided to trial the drug, buy it for 4,000 patients who would benefit from Orkambi. Therefore, no one would have to pay for it privately. The NHS could fund it, but at much less than Vertex is asking for. I ask the Minister: why is that not the solution?

Mrs Sharon Hodgson Portrait Mrs Hodgson - Hansard
10 Jun 2019, 6:35 p.m.

I was going to come on to that, but if a point is worth making once, it is worth making twice. I will make it to the Minister as well, so she will have plenty of time to think about it.

As we all agree, patients and their families should not be put in the position—as some are—of having to pay thousands of pounds for their treatment. Family income should not determine who lives and who dies. That is why the NHS was founded—so that all could have access to the same excellent treatment, regardless of means. That was true 70 years ago when the NHS was formed, and it is still true today.

As the hon. Member for Sutton and Cheam pointed out, our NHS is there for us all and should not be held to ransom by a pharmaceutical company, but neither should access be denied because of unfit processes and systems in the NHS. Over the years, as a shadow public health Minister, I have met many patient groups, including those with cystic fibrosis, who are missing out on life-changing medicines because their condition is not rare enough and is therefore not deemed by NICE to be cost-effective. We need an appraisal process that is fit for purpose and that will capture rare diseases such as cystic fibrosis effectively.

Without drugs such as Orkambi, patients and their families are being harmed physically and psychologically. Every day without the drugs that patients need makes their condition worse and threatens their lives. What steps will the Minister take to ensure that patients with rare diseases have access to the medicines that they need and deserve? It is about access not just to Orkambi, but to other precision medications such as Symkevi and the next generation of cystic fibrosis drugs that could help patients who are suffering.

Vertex recently announced the headline results for its fourth cystic fibrosis medicine, a triple combination therapy that could radically transform the lives of nine in 10 people who live with cystic fibrosis in the UK, delivering unprecedented improvements in acute lung health. That is amazing news, but patients fear that they will never be able to access this ground-breaking drug. I urge Vertex to put patients first and consider the real-life impact of this cost dispute on patients and their families.

Vertex and NHS England must come to an agreement urgently, because patients have already waited far too long. If an arrangement cannot be made soon, will the Minister personally step in and pursue the alternatives that my hon. Friend the Member for Bristol East mentioned, such as a Crown use licence or a clinical trial? Cystic fibrosis patients need urgent access now to the drug that they have been denied for three years. It is time the Government considered all alternatives.

The Parliamentary Under-Secretary of State for Health and Social Care (Seema Kennedy) Hansard
10 Jun 2019, 6:42 p.m.

It is always a particular pleasure to serve under your chairmanship, Mr Hanson. I thank my hon. Friend the Member for Sutton and Cheam (Paul Scully) for opening the debate on behalf of the Petitions Committee. I pay tribute to the more than 100,000 people who signed the petition, and I thank all right hon. and hon. Members who have spoken in the debate; I am sure that they will be rushing back for the wind-ups.

I have been very touched by the stories that we have heard today and the compassion shown by my hon. Friend and all hon. Members in speaking about cystic fibrosis and its physical effects, emotional effects and effects on mental health for those who live with it and for their families. It is a debilitating condition, and I know how absolutely desperate sufferers and families are for access to treatments.

I recognise the great work undertaken by the Cystic Fibrosis Trust and its strong voice in supporting families and bringing cystic fibrosis to the attention of parliamentarians. I also pay tribute to my young constituent Lucy Baxter, who was on “BBC Breakfast” this morning and who lives with cystic fibrosis. She spoke to me very soon after I became a Member of Parliament and is an absolute inspiration to me and to the whole cystic fibrosis community.

Today’s debate has been heartfelt and passionate. The stories that we have heard clearly make the case that Orkambi and other drugs for people with cystic fibrosis should be available on the NHS at a price that is fair and affordable. The Government and I share that view. As the Chair of the Health and Social Care Committee, the hon. Member for Totnes (Dr Wollaston), set out so clearly, we must remember that the NHS must use its budget fairly for the good of all patients. That is why we rightly have a system whereby experts, not politicians, determine the fair price for medicines, based on robust evidence. That system has helped many thousands of patients to benefit from rapid access to effective new medicines.

Break in Debate

Seema Kennedy Hansard
10 Jun 2019, 6:53 p.m.

I thank the hon. Gentleman for his intervention. As my hon. Friend the Member for Sutton and Cheam said in his opening speech, which was very well made, we recognise the importance of British pharmaceutical companies and that companies invest hugely in developing new drugs. However, as the other examples of drugs for rare diseases that I have given show, it is possible to go through the NICE appraisal process and reach an agreement with NHS England. As one hon. Member who is no longer in their place said, this is an offer for a long-term agreement.

Vertex is an outlier, and I would like to put that on the record.

Mrs Sharon Hodgson Portrait Mrs Hodgson - Hansard

Will the Minister give way on that point?

Seema Kennedy Hansard
10 Jun 2019, 6:54 p.m.

Yes, and I hope that I will be able to remember the hon. Lady’s question.

Mrs Sharon Hodgson Portrait Mrs Hodgson - Hansard

At this point I should clarify, for the benefit of the campaigners who I have spoken to about the Crown use licensing option, that it is not an immediate solution from their point of view; I understand that it would take at least a couple of years. If an agreement can be reached, there would be an immediate outcome. That is why the campaign is called Orkambi Now; it is about trying to get the drug now. Although the Crown use licensing option would be an option to consider if nothing else can be found, it would not give the sufferers and their families the drugs as quickly as we would like.

Seema Kennedy Hansard

As always, the shadow Minister makes an excellent point. Crown use licensing is not something that any Government would consider lightly. It is very rarely used in health. It has probably not been used—my officials will correct me if I am wrong—since the 1970s.

The ideal thing is to get a deal, and deals have been done with other pharmaceutical companies; that is the point I want to make. As I have said, Vertex is an outlier in this regard, but that does not mean that I do not have an obligation to look at other options. I will do that.