Cystic Fibrosis Drugs: Orkambi Debate

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Department: Department of Health and Social Care

Cystic Fibrosis Drugs: Orkambi
Kerry McCarthy Excerpts
Monday 10th June 2019

(1 year ago)

Westminster Hall
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Department of Health and Social Care
Paul Scully Portrait Paul Scully - Hansard
10 Jun 2019, 4:40 p.m.

The hon. Lady has hit on a really crucial point. The underlying thing that I took away from my meeting with the people living with CF this morning was mental health, which ran through all their situations.

I heard from Oli Rayner, who talked about the fact that he is 43 and has dedicated his whole life to staying alive; he had effectively been told that he would not make 10 years. He was then told that he would not make 20, then 30. This is a guy who has now got cystic fibrosis-related diabetes and a number of other conditions. He has had a lung transplant, and he had Orkambi to get him to that stage. The fact is that his lungs are now doing what he wants them to do, without his having to think about it. We can imagine the mental issues that he had before.

Jessica Jones told me that people with CF are very good at living. Yvonne Hughes said she felt broken. One lady, Carly Beale, told me that she had been on the original Orkambi trial. The NHS had not prepared her for when Orkambi stopped at the end of the trial. She had suddenly improved and started to get her life back—perhaps a life that she had not had in the first place—and she was not ready to have it taken away from her. She said that it is almost worse that this drug exists but she cannot get access to it. She said, “I’d rather it didn’t exist than have it dangled in front of me in expectation.”

Kerry McCarthy Portrait Kerry McCarthy (Bristol East) (Lab) - Hansard
10 Jun 2019, 4:39 p.m.

I am really pleased that there is now cross-party working on this issue. Life expectancy is a very sensitive issue for cystic fibrosis patients and their parents. I am aware that Conservative MPs have been sending round a letter that points out—as if the letter’s recipients ought to be grateful—that life expectancy for CF patients has now increased to 40, although I think it is more like 31. It seems a little insensitive, and I hope that MPs present would report that back.

Paul Scully Portrait Paul Scully - Hansard
10 Jun 2019, 4:46 p.m.

It is an unfortunate manner of phrasing. There is no doubt about the fact that median life expectancy has increased. For someone born now, the projection is that they are likely to have a median life expectancy of 47. It is clearly an improvement, and hopefully these drugs will carry on extending that. It is a matter of wording, and I do not think any offence was intended.

Break in Debate

Mr Adrian Bailey (in the Chair) Hansard

Order. Seventeen would-be speakers are listed for the debate. Back-Bench contributions will conclude at 6.58 pm to allow for the Front-Bench speeches and the proposer’s summing up. I will therefore put a six-minute time limit on individual contributions. I call Kerry McCarthy.

Kerry McCarthy Portrait Kerry McCarthy (Bristol East) (Lab) - Hansard
10 Jun 2019, 5:01 p.m.

Thank you, Mr Bailey; it is a pleasure to see you in the Chair. I thank the hon. Member for Sutton and Cheam (Paul Scully) for introducing the debate on behalf of the Petitions Committee.

As we have heard, Orkambi is a life-changing treatment that stands to benefit 4,000 cystic fibrosis patients in the UK—40% of the total number of people with the condition—and which was licensed almost four years ago. In some ways, the debate has almost moved beyond Orkambi, because other drugs now being developed would help an even greater proportion of patients. Vertex’s ongoing refusal to offer a price for Orkambi that the NHS can afford means that those patients are still unable to benefit from the treatment.

I will point out—this is important to what I will say next—that in 2017 alone, Vertex earned £2.5 billion from the sale of Orkambi, while its chief executive was paid more than £17 million. Pharmaceutical companies such as Vertex play an important role in the development of innovative medicines and money goes into that, so I entirely accept that they should make a profit as a result, but to pursue excessive reward at the cost of patients’ lives is, I believe, morally bankrupt.

I will speak about alternative approaches. A few weeks ago, I had the opportunity to meet an Argentine drug company that has come up with a generic alternative to Orkambi. I also met two parents, one of whom was fortunate enough to be in a position to be able to spend a six-figure sum on Orkambi for his young son, who was, he said, doing well on it. He was clearly in a very privileged position to be able to do that. The other father was not in that position and was looking at the buyers’ club approach that has been mentioned and that featured on “Newsnight” on Tuesday. He said that if he had to sell his house to afford to buy the drugs at the reduced price—around the £18,000-mark—he would do so, because his son is far more important to him than the place he lives.

I was introduced to the Argentine drugs company by a Bristol-based international organisation called Just Treatment. With a range of experts in the field, Just Treatment has made contact with the generic manufacturer of the drugs, which can supply a safe and effective version of Orkambi at a cost for individual CF patients of about £23,000 per patient per year—just 30% of what Vertex charges. If there were a buyers’ club, the price could come down.

The term “buyers’ club” comes from the Hollywood film, “Dallas Buyers Club”, which gave a fictionalised account of the buyers’ clubs that popped up in the ’80s and ’90s because of the lack of access to HIV treatment. The clubs enable patients to understand their right to procure more affordable generic versions of drugs that they would otherwise struggle to access.

In Argentina, a small number of people have CF, and only a couple of hundred would benefit from Orkambi, of whom 120 take the generic alternative and seem to be doing well. The longest that anyone has taken it is 18 months, so although it is early days, treatments seem to be going well. Initial discussions with the generic manufacturer suggest that once 500 patients sign up to receive the drug, the cost would drop to just 20% of that demanded by Vertex.

Clearly, whether it costs £18,000 or £23,000, the drug is still not affordable for most people, which is why I would argue that although that is a step forward for some, the Government need to step in and help patients. The most effective way they can do that is by pursuing a Crown use licence for Orkambi. A few months ago in the House, the hon. Member for North Herefordshire (Bill Wiggin) mentioned that possibility in the context of access to medicines for CF patients. In the legal provisions set out in section 55(1) of the Patents Act 1977, the Government have the power to suspend Vertex’s patent on Orkambi and overturn its monopoly. One of the Government’s primary objections to that route has been the concern that it could take years to develop a generic product, but as I have said, a generic product is now on the market, is being tested and is available for patients in the UK, although only three boxes can be provided at a time and it is still unaffordable at the price that I mentioned.

The Government should also explore the funding of a large-scale clinical trial, which would enable the NHS to use research exemptions in intellectual property rights law and allow it to use the generic version of Orkambi. That approach has been used before, with the HIV prevention medication PrEP, or pre-exposure prophylaxis, and ensured that tens of thousands of patients were able to access that drug while its expensive branded counterpart was still under patent—I would be particularly interested in Minister’s response to that. A trial could be set up in anticipation of a Crown use licence, providing data necessary to support regulatory approval of the generic version of Orkambi.

If the Government are unwilling to pursue either of those routes—I cannot see why they should be reluctant to do so—there is another alternative that goes down the buyers’ club route: providing personalised health budget payments to help patients meet the cost of the generic drug. People are absolutely fed up of the deadlock between Vertex and the NHS, so I urge the Government to look at those more imaginative solutions.

John Howell Portrait John Howell (Henley) (Con) - Hansard
10 Jun 2019, 5:09 p.m.

It is a pleasure to serve under your chairmanship, Mr Bailey. My first introduction to cystic fibrosis came before I became an MP. I wrote some newsletters and did some public relations work for the Cystic Fibrosis Holiday Fund, the main objective of which was to provide holidays and ancillary facilities to under-18s who suffer from cystic fibrosis. On the basis of medical advice that was given in 2000, we now cannot take those children away together, so the fund spends most of its time generating respite break grants and providing the Family Revitalise programme. Those initiatives are both important, but do not compare with making available Orkambi or any of the other drugs that have been mentioned.

Two families in my constituency have children with cystic fibrosis. I have spent time with both families, and have seen that largely the children are happy, normal children who enjoy all the things that other children enjoy. Hanging over them, however, is the threat of a double-lung transplant just to stay alive.

Orkambi changes lives, and we need to look at ways that we can make it available. A number of structural difficulties were identified during the conversations that I have had on the matter. The first is one of commercial incentive and risk. To compound that point, one can look at the relative strength-in-numbers of those who suffer from diabetes or from cystic fibrosis: diabetes accounts for 4 million people, while cystic fibrosis accounts for only 70,000. A major hurdle is therefore already built in for those with cystic fibrosis to overcome. We should not forget that.

The issue of the time taken, which has already been raised, goes back to criticisms of the NICE process. The criticisms that I would make fall into three types: first, NICE adopts the same evaluation process for a drug that might treat tens of millions of people as it does for a drug that treats a few hundred thousand or, indeed, a few thousand. We need to bring home to NICE that that is not a right way to proceed.

Secondly, the same evaluation process is also used whether the drug is taken for a brief period or a long one—in other words, whether it is a short use cancer-related drug or, as in the case of Orkambi, it must keep being taken over many long periods. That factor needs to be built into any evaluation of the drug as well.

The third criticism that I would make of the NICE process is that it is too focused on short-term benefits, and not on long-term benefits, which we know that Orkambi can produce. As has been mentioned, the data released by Vertex show that after 96 weeks of treatment, the rate of lung function decline reduced by 42%. That is a major long-term thing to hang on to. Furthermore, the net value of Orkambi is hard to calculate and therefore to capture accurately. A number of direct costs need to be taken into account, such as the cost of hospitalisation, and there is evidence that Orkambi starts to reduce the number of other medicines that need to be taken.

We have heard that Orkambi is available in many other countries in Europe, although I hear that the Spanish Government are having difficulties with Vertex, in the same way as we are, over the availability of the drug. The agreement that was reached with Vertex to make Orkambi available was a disappointing affair. We need to put on the pressure to ensure that that happens and that generic drugs are brought forward to be used instead. The example often cited is Ireland—both families in my constituency mentioned the situation there—and it is interesting to note that success story of the use of Orkambi. It has been very successful there, and we should all take that to heart in making progress to ensure that young people suffering from cystic fibrosis have access to this drug.

Break in Debate

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.

Break in Debate

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.

Kerry McCarthy Portrait Kerry McCarthy - Hansard

Will the Minister give way?

Seema Kennedy Hansard

Can I just make a tiny bit of progress first?

Kerry McCarthy Portrait Kerry McCarthy - Hansard

It is on this issue.

Seema Kennedy Hansard

All right, given that we have more than half an hour left.

Kerry McCarthy Portrait Kerry McCarthy - Hansard

I really welcome the fact that the Minister is saying so passionately that she feels she has a moral obligation to act. The question is: when? I say that because Vertex has been in these talks for a very long time now. How much longer will it be given before the Minister decides to look at the other options?

Seema Kennedy Hansard
10 Jun 2019, 6:56 p.m.

Well, I have already said it, so I suppose that means—

Kerry McCarthy Portrait Kerry McCarthy - Hansard
10 Jun 2019, 6:56 p.m.


Seema Kennedy Hansard
10 Jun 2019, 6:56 p.m.

Now. We will look at other options to consider what other methods we can use. As the hon. Member for Washington and Sunderland West said, Crown use licensing has other risks, so the best option is to get a deal. However, we will look at other methods that might enable families to receive the drugs they need.

Some Members have said that Orkambi is available in other countries and asked, “So why not here?” Although that is true, it is also true that other countries have faced problems in agreeing an acceptable price with Vertex; around 50% of the global cystic fibrosis population is unable to access Orkambi. It is not approved for reimbursement in Spain, which I have touched on, or in Portugal, and it is not used routinely in France. It is also of note that the Canadian equivalent of NICE has rejected the drug, saying that the benefit of Orkambi is small and uncertain.

We do not have sight of specific commercial agreements where Orkambi is approved and we do not have the same population needs, as we know, because of the specific population that the UK has; compared with other countries, the UK has a very high proportion of people with cystic fibrosis. So I am not able to make comparisons with other countries. Cystic fibrosis affects about 10,500 people in the United Kingdom—a far higher figure than in other countries. It represents 12% of the global cystic fibrosis population. The UK is a very important market for Vertex.

I thank all Members who have spoken so passionately here today. In particular, I thank the people who have joined us here in the Gallery; I pay tribute to them for all that they do to support their family members. I also thank those people, such as Lucy Baxter, who work so hard to raise awareness of this issue. I thank members of the Health and Social Care Committee, which is carrying out a very thorough and transparent inquiry into this issue, helping to shine a light on it.

As we have heard in great and moving detail today, cystic fibrosis is a devastating, life-limiting condition, and the bravery of those affected should be an inspiration to us all. Drugs that improve sufferers’ quality of life should be available where appropriate, and I urge Vertex to do everything it can to price its medicines fairly and in a way that reflects the health benefits to patients.

NHS England and NICE will, of course, continue their efforts to reach an agreement with Vertex. Access to treatment for all patients is, and always will be, a priority for this Government. My Department has a moral obligation to look at other options now, and that is what we shall do.