Cystic Fibrosis Drugs: Orkambi Debate

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John Howell

Main Page: John Howell (Conservative) - Henley)

Cystic Fibrosis Drugs: Orkambi

John Howell Excerpts
Monday 10th June 2019

(1 year, 3 months ago)

Westminster Hall
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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.

Dr Sarah Wollaston (Totnes) (Ind) Hansard
10 Jun 2019, 5:13 p.m.

I thank my constituent, Cathy Meredith, who started this petition. I also thank Oli Rayner and the many others living with cystic fibrosis who, sadly, cannot be in the Public Gallery with us today because cystic fibrosis is such a curiously isolating condition—those suffering from it cannot be in the same room as others because of the risk of transmitting resistant infections.

I will start with some context on the cause of cystic fibrosis, which is a mutation in the cystic fibrosis transmembrane conductance regulator gene, affecting the production of a protein that in turn has consequences for the balance of salts and fluids moving across membranes, leading to an accumulation of thick, sticky mucus in the lungs and other organs. The point, however, is that although 10,000 people in the UK live with cystic fibrosis, it is not really a single condition: there are many mutations of the CFTR gene. That has consequences for the types of medication to which people will best respond. We need to think of cystic fibrosis not only as a rare genetic condition but as a series of much rarer conditions. That is important to note.

We now have some real hope for progress with the CFTR modulators, but we need to make that progress much more rapidly than we are. The negotiations between Vertex and NHS England have dragged on for far too long. The patients living with cystic fibrosis and their families, have been lost in those discussions. We need not only to return to thinking about them, but to bear in mind the implications that go far beyond those living with cystic fibrosis.

The NHS has a responsibility to consider the wider cost of drugs, including the opportunity costs—what we cannot treat if our NHS budget is consumed completely by the ever-rising cost of drugs. For the NHS to have that responsibility is a tough message for all of us, which is why we need bodies such as NICE to make the decisions to ensure fairness for all patients who rely on NHS resources. To put that in context, the drugs budget in 2017-18 was £18.2 billion. A little more than half of that was for hospital drugs and, over the past seven years, the costs of those drugs have increased by 119%. We therefore have to bear in mind the implications of taking a free-for-all approach to drugs costs, which the Minister will not want to do.

The Government are trying to get the parties around the table. Unfortunately, the gap is huge between what Vertex continues to demand for the drugs and what the NHS is offering based on recommendations from NICE. The gap is not small; it is considerable. Other companies have come to the table to negotiate their prices, so I call on Vertex to look again at what is happening. It is absolutely disgraceful that families have to resort to such things as buyers’ clubs; the inequalities that that creates are horrific. We need Vertex to focus on what is happening.

I am also concerned about some of the points made during our Health and Social Care Committee inquiry into Vertex. For example, we asked the company directly whether drug supplies had been destroyed because they were going off date, and we were told that that was not the case and was very unlikely to happen—but it has been happening. That is wholly unacceptable.

To come back to the alternatives, the hon. Member for Bristol East (Kerry McCarthy) touched on the issue of Crown use licences, for example. One of the areas that our Committee considered was possible referral to the Competition and Markets Authority. In fact, we have now heard that that would take many years, so unfortunately the area does not look like one we can pursue further. However, given so little progress since our inquiry, the Committee wrote to all the parties involved in the negotiations—NHS England, Vertex and NICE—to ask where we are now.

The most promising idea that we should take forward to apply pressure is that of interim agreements, such as in Scotland. An interim price is agreed, further research is carried out and all parties agree to a review based on the outcomes of that further research. That is being managed in Scotland and other places, as we have heard from other speakers today, and I urge Vertex to do that here. We all recognise the need for a fair price to enable further research to take place. We all recognise that many other drugs are in the pipeline, particularly a very promising triple therapy, which NHS England has now agreed to take off the table so that it does not distort future pricing. That is a sensible thing to do at this stage, so that within the current offer we look just at the three existing treatments. At a later stage, we can come back to look at the triple therapy evidence. I urge all parties to come to an interim agreement at least, and to continue to put patients front and centre in everything they do.

Finally, I would like to touch on the political aspect, because both President Trump and US Secretary of State Azar have repeated referred to using their muscle in trade negotiations to increase the price that European countries would have to pay for their drugs. They have referred to the NHS “freeloading”, for example. That is very worrying. We all need to be aware of the dangers of a future trade deal and the implications that it could have on negotiations for a range of other products. I hope that those points have contributed to the debate. All parties need to focus on the people at the heart of the issue: the patients who are living with cystic fibrosis.