Cystic Fibrosis Drugs: Orkambi
Dr Sarah Wollaston Excerpts
Monday 10th June 2019

(1 year ago)

Westminster Hall
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Department of Health and Social Care
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.

Mr Adrian Bailey (in the Chair) Hansard
10 Jun 2019, 5:20 p.m.

I gave the Chair of the Select Committee a little latitude, but that may not be available to other speakers. I call Sir Mike Penning.

Break in Debate

Seema Kennedy Hansard
10 Jun 2019, 6:47 p.m.

I shall turn later in my speech to that specific avenue that some sufferers have gone down.

Dr Wollaston Hansard
10 Jun 2019, 6:47 p.m.

The Minister referred to the importance of Vertex engaging with NICE. Does she share my concern that when I wrote to Vertex and NICE about the failure to make progress, Vertex assured me that it had contributed “substantial new evidence” on the three products in question, yet I subsequently heard from NICE that it had received only

“an overview of the clinical evidence”,

rather than genuine engagement? Will she join me in calling on Vertex to properly engage with the process, so that we can get the full evidence base on which to make these decisions?

Seema Kennedy Hansard
10 Jun 2019, 6:49 p.m.

I agree with the Chair of the Health and Social Care Committee and urge Vertex to re-engage with the NICE process. To date, unfortunately, it has continued to refuse to accept the process or has suggested unacceptable conditions on the NICE value assessment of its product, which would render the outcome meaningless. That comes despite NHS England’s latest proposals offering to reimburse Vertex ahead of a positive NICE recommendation, which for a deal of this size is unprecedented; agreeing to implement real-world data collection, as the Committee has called for, to help Vertex to demonstrate the value of its medicines; and offering significantly increased prices in comparison with their offer last July.

NICE has a 20-year history and is internationally renowned and independent. Its methods and processes for the development of its guidance have been in place for 20 years, but it recognises that it needs to evolve. It continues to review its procedures to ensure that they remain fit for purpose; it is now undertaking a review of its technology appraisal methods in line with the commitment in the 2019 voluntary scheme, and it encourages all stakeholders to engage. NICE has recommended 75% of the drugs for rare diseases—some of which I will touch on later—that have been assessed through its technology appraisal programme for the eligible patient population.

Last week, the Association of the British Pharmaceutical Industry made it clear that

“NICE is the cornerstone of NHS efforts to ensure the price being charged by a company represents the value being delivered.”

Commenting on the current situation, it said that

“the APBI would always encourage companies to fully engage with NICE at all stages of the process.”

Furthermore, it commented on the current structure of NHS England’s proposed deal with Vertex, saying that

“the structure of the offer represents exactly the sort of flexibility the industry has been calling for, for some time.”

However, Vertex is willing to accept only its own valuation of Orkambi; I draw your attention, Mr Hanson, to comments directed at Vertex by Members from across the House, including those made by the shadow Minister, the hon. Member for Washington and Sunderland West (Mrs Hodgson).