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Access to Orkambi

Volume 645: debated on Tuesday 17 July 2018

Motion made, and Question proposed, That this House do now adjourn.—(Mims Davies.)

We are living in an era when public confidence in a mainstream political class is at an all-time low. Too often the public suspect that we choose the low ground when they yearn for us to build common cause in pursuit of the high ground. Tonight we have a chance, in a small way, to prove them wrong, by using this debate to show the relevance and humanity of Parliament in the cause of human dignity and human life. I pay tribute to colleagues here, especially the hon. Members for Dudley North (Ian Austin), for South Cambridgeshire (Heidi Allen), for Erith and Thamesmead (Teresa Pearce), for Bury North (James Frith) and for York Central (Rachael Maskell), the right hon. Member for Hemel Hempstead (Sir Mike Penning) and the hon. Member for Strangford (Jim Shannon), for championing this issue so passionately and effectively over a long period of time. I also want to place on record our appreciation for the tremendous work of organisations, including the Cystic Fibrosis Trust, which ensure that the voices of people with cystic fibrosis and their families are heard and heard loudly.

I thank the hon. Gentleman for being so generous. The fact that so many Members have stayed behind tonight is an indication of the importance of the issue. It was my privilege to attend an event at Belfast City Hall this month, standing side by side with CF sufferers and their families calling for Orkambi to be made available. Does the hon. Gentleman agree that medication that is proven to improve the quality of life for CF sufferers must be made available regardless of postcode? The Department must again ask the National Institute for Health and Care Excellence to enter into negotiations with a pharmaceutical company to provide this drug and allow CF children to progress and CF adults to achieve a good quality of life. Once again, I congratulate him on bringing this issue to the House. It is very important.

I agree entirely with the hon. Gentleman. It is absolutely vital that we see an engagement process that leads to action. I will develop that argument as I make my contribution here this evening.

The hon. Gentleman mentioned many organisations. I wish to pay tribute to Scotland Parents CF Support Group, which has been very generous in helping me to understand and bring this matter to people’s notice. This is a UK-wide issue. I know that we are discussing NHS England, but this is also an issue in Scotland. Hopefully, if we can get those in England to get this drug, it will have a domino effect in Scotland, thereby saving and improving the lives of people with CF.

I think that, in this context, we can all unite behind the concept that we want a resolution that is UK-wide if at all possible. Clearly, that would require different organisations to be involved, but if we can set a precedent here, we could make rapid progress, and that can only help all parts of the United Kingdom. May I make some progress, and then I will give way to other hon. Members?

All of us who have the privilege of serving in this House are mothers or fathers, brothers or sisters, grandparents, uncles or aunts, godparents, friends or neighbours. It could very easily be one of our family members or friends who is diagnosed with cystic fibrosis at birth and who struggles with a life of perpetual illness, frequent hospitalisation and a daily multitude of drugs. Worse still, they could be living with the fear of premature death at an age when many young people are getting married, starting a family or taking their career to a new level.

I ask hon. Members to imagine that their family member or friend was denied access to a drug that could improve their quality of life and prolong their life for many years—a drug that was readily available in 12 other countries. No one in this Chamber today would accept no for an answer, or remain silent as the NHS and a major drugs company traded increasingly public blows as to who is to blame for unnecessary suffering and potential loss of life. If the situation is not good enough for our loved ones, it should not be good enough for anybody else’s.

I first became aware of Orkambi when a young constituent from Prestwich attended my constituency surgery with her mother. Many Members will have met constituents in a similar situation. Alex Darkin is 10 years old and suffers from cystic fibrosis. She is a remarkable girl, whose courage and positivity are truly inspirational. Alex started this year with 80% lung function—a number that scared her mother, Emma, because Alex’s lung function was over 100% a few months previously. These days, Emma would give anything to see the number 80 again. Alex’s lung function continues to drop and is now around the 54% to 56% mark.

Alex has physio and takes a large amount of medication just to manage her condition. She brought all that medication to my surgery and I found myself looking at a young person who has to go through the routine of taking that medication every day; that, in itself, is a massive challenge. Alex has intravenous antibiotics every three months, and her daily life is inevitably dominated by her drug and physiotherapy regime. Very sadly, she now has irreparable lung damage. A consultant recently advised that doctors are running out of options with regard to medication, and explained that if Alex were a year older her parents would be able to apply for Orkambi on compassionate grounds, as her lung function had deteriorated more than 25% in less than three months. Emma and Alex contacted me because they could not afford to wait another year to get this drug on compassionate grounds. Surely a truly compassionate society would ensure that this drug was available to all who needed it.

I congratulate the hon. Gentleman on securing this debate. I think that we all tried to get this debate, which is why we are all here. One of the reasons I said to Mr Speaker earlier that I was not happy was that I wanted us to get on to this debate. We have all heard stories in our constituencies, as I am sure you have, Madam Deputy Speaker. As a country of this wealth, we should be ashamed. People in countries with nowhere near the wealth of this country have been given these drugs to improve their quality of life. We are here to right that shame.

The right hon. Gentleman speaks for everybody present this evening and probably many who are not.

There is inevitably sadness, but there should be real anger because this situation is causing human misery to many people who have enough challenges in their lives without not being able to resolve this situation. It would not be right for any of us to pretend that these decisions are easy. NICE does a good job in very challenging circumstances, on the whole. In no way should we or do we seek to undermine its work.

The hon. Gentleman is being very generous in giving way. As he is reflecting on NICE and its process, does he agree that there should be a change in the way it measures or calculates quality of life when it comes to rare rather than ultra-rare diseases? If the condition was classed as ultra-rare, we may well have got the go-ahead.

I think that most people would accept that now is the time for a review of the criteria that are applied and the processes that are undertaken. Any organisation needs to be continually improving. We do not condemn NICE. In fact, we acknowledge that it has got many difficult decisions right, but it has also got some wrong. Time and again, the reason that it has got those decisions wrong—arguably—is that the criteria it is applying and the criteria it has been given by the Department of Health are out of date and flawed. I think we would all want to see a review of NICE’s remit and the way in which it carries out its work over a reasonable period of time. It would be helpful if the Minister would respond to that point when he concludes the debate.

My hon. Friend is making a very important speech. As I know from being a physio who worked with cystic fibrosis patients, when making an assessment of the cost of treating cystic fibrosis, we must look at the social cost, the economic cost and the immense cost of keeping somebody alive. Does he agree that Orkambi would be a solution to that, let alone addressing the human cost?

I entirely agree—my hon. Friend makes absolutely the right point. When there is an incomplete assessment in looking at value for money versus outcomes, that will lead to flawed decisions that are incredibly difficult to justify. We have a mismatch between the basis on which NICE is expected to make these decisions and appropriate processes. Instead of everybody hinting, “Yes, of course there’s a need to review NICE—of course that would be a good thing”, we would like to hear from the Minister a timescale as part of the response to the specific issue of Orkambi in terms of NICE’s roles and responsibilities.

Now is an opportunity because we have a new Secretary of State. I have already contacted him about this issue. The way that NICE is making its decisions is affecting not only drugs like Orkambi but an awful lot of drugs that people in our constituencies desperately need. Does the hon. Gentleman agree that now is absolutely the right time to grasp this situation on Orkambi and challenge NICE about its decision-making criteria? Let us change the way it is looking at the value of quality of life and do that urgently, using Orkambi as a starting point.

I entirely agree. It is always very important, when a new Minister begins a role, that they have a very clear sense of a positive agenda on which they want to achieve change. This will be a very appropriate issue for the new Secretary of State to adopt and to drive forward.

Orkambi would not be suitable for my constituent Holly, who is eight years old, but other drugs that have been manufactured would be more suitable for her. Does my hon. Friend agree that if NICE had more flexibility in looking at pipeline deals, that would help many more people such as my constituent Holly?

Of course I agree entirely. It must be very frustrating for Holly and her family to find themselves in this situation. There really is no excuse for delaying the beginning of a review. Members know full well how long these reviews can take, so let us get on with it. I think we are united in a belief that this is absolutely essential as part of the lessons that we need to learn from this situation.

The hon. Gentleman alluded to having spoken to families of people who were on a large cocktail of drugs and the costs associated with that. Let us not say that it is just down to costs; I appreciate that NICE might well be using the wrong process. This cocktail of drugs adds up to a significant cost, and there can be a dramatic saving if they can come off some of those drugs, as well as losing the side-effects that come with them.

I agree entirely. We sometimes spend vast amounts of public money reacting to a problem, and the rhetoric is all about prevention and early intervention, but we end up doing the opposite. This is a very good example of that.

I congratulate my hon. Friend on securing this debate. Does he agree that there is a human cost to this gridlock for cystic fibrosis sufferers, who are victims of this disease and this disagreement? Frankly, NHS England should get in a room with Vertex and they should stay in there until they come out with an agreement to end this gridlock, so that cystic fibrosis sufferers do not see this debate just as yet another conversation but as delivering change and transformation of their lives.

My hon. Friend clearly wants to finish my speech off for me. Of course, he is absolutely right to make that point. The most important thing to see from this debate is action, not words. I will go on to talk about what those actions should be.

I am very grateful. This is a really frustrating process, and the Government have to find a way of bringing it to a conclusion. I agree that NHS England and Vertex have to get back into negotiations, which should not stop until this is resolved. Does my hon. Friend agree that it might be an idea for the Secretary of State to get Sir Simon Stevens and Jeff Leiden, the chief executive of Vertex, in a room—

The right hon. Gentleman could lock the door, but first he has to get them in there so that negotiations resume and are not concluded until they resolve this issue, because it really does have to be sorted out.

On that point, does my hon. Friend agree that it was worrying to read the word “final” in NHS England’s response to Vertex yesterday? It cannot be final. I really hope the Minister is listening to this. [Interruption.] The word “final” was in there. [Interruption.] Well, he is the Minister. The word “final” cannot be used until it is finally resolved. That is when it will be final.

I agree entirely with my hon. Friend, and I will develop his argument as I continue my speech.

A quote from Emma, Alex’s mum, sums up many of the frustrations of the constituents represented here today. She says, very movingly:

“Alex has a real love for life and all she wants to do is live a long and happy live without having to fight to breathe.”

That powerful statement says it all.

Since March, when we last debated this issue, NHS England and Vertex have met on four occasions. I welcome the fact that, during those negotiations, NHS England accepted the principle of a funding agreement that supports a portfolio approach to current and future drugs. That is undoubtedly a step forward and a positive response to the contributions parliamentarians made in the initial debate, but it is clear that these negotiations have not produced an agreement that is acceptable to both parties. Indeed, the public recrimination suggests that the negotiations had all but broken down. It remains to be seen whether NHS England’s written offer, made on the eve of this debate, is a basis for progress, but the early indications are not encouraging.

It would be inappropriate for anyone to make judgments about the validity of either party’s case without having full access to the information, which remains confidential for reasons of commercial sensitivity. I know I speak for all parliamentarians when I express concern about the fact that, as my hon. Friend the Member for Dudley North said, NHS England described its offer as “final”. It could lead to a stalemate that once again lets down patients and their families. To be clear, I respect the need for an objective process that delivers appropriate medication and value for money for the taxpayer. If NICE did not exist, it would have to be invented, but—this point has been made time and again tonight—the shortcomings of the current assessment processes are well documented. That is why NHS England’s involvement in this instance is to be welcomed.

Equally, Vertex has the right to seek financial remuneration at a level that maintains its viability and supports the development of the company. Sadly, I am unconvinced that more of the same will lead to an agreement that will enable Orkambi and successor drugs to be made available to people with cystic fibrosis. Consequently—my hon. Friend the Member for Bury North suggested this—I urge the Secretary of State for Health and Social Care to facilitate an urgent meeting with the chief executive officers of NHS England and Vertex. In that meeting, they should seek to resolve any outstanding differences, and agree a rapid timescale for the roll-out of Orkambi across the country and an approach for other relevant drugs. People with cystic fibrosis have been waiting far too long.

The hon. Gentleman is well aware that health is a devolved matter in Northern Ireland, but we do not have a Health Minister because we have not had a functioning Assembly for 18 months. Will he join me in urging the Minister to liaise with the permanent secretary in the Department of Health in Northern Ireland to reassure the many cystic fibrosis suffers and their families, who are desperately anxious, that we have a drug available? The health service must enter into final discussions to reach the outcome we all want.

I agree entirely. The absence of a functioning Executive is a source of continued regret to those of us how care passionately about Northern Ireland. Progress has been made, but there is now a stalemate. It is incredibly important that, in the absence of an Executive, Ministers ensure that any solution that is reached also benefits people with cystic fibrosis in Northern Ireland.

The UK Government need to commit to a proactive role to help secure a deal as soon as possible that will ensure access to these new treatments. They must fulfil their own stated ambition for NHS patients to be

“among the first in the world to get life-changing treatments.”

Vertex, the pharmaceutical company, must be fair and responsible with pricing to ensure that a deal on access to new treatments can be agreed—and agreed rapidly. The Government must consider looking towards the future pipeline of treatments for cystic fibrosis to ensure that a robust and fair system is in place for appraising high-cost new medicines to avoid similar issues about access to medicines with other companies and drugs in the future. The time for words is over; on behalf of our constituents, tonight we demand action.

I say from the outset that there are 22 people in the Chamber for this Adjournment debate. Normally, it is me, the proposer of the debate and the hon. Member for Strangford (Jim Shannon). I say to the hon. Member for Bury South (Mr Lewis) that it is nice for other people to be here with us. I will say at the start that I will not take any interventions. A lot of Members have come to listen to this debate, and they want to hear the Minister. There are things that I as the Minister need to put on the record, and I am going to put them on the record before we finish at 8.5 pm.

I thank the hon. Gentleman for securing this debate and for giving me the opportunity to speak once more on an important issue that matters to me and my constituents as much as it does to his. May I again place on the record the sterling work undertaken by the Cystic Fibrosis Trust? Its support for cutting-edge research, campaigns to drive up standards of care and support for the CF community is fabulous. We are lucky to have it.

Once more, we have heard hon. Members make compassionate—that is a very good word—pleas urging an immediate resolution in the ongoing discussions between NHS England and Vertex to make Orkambi available on the NHS in England. To the hon. Member for North Down (Lady Hermon), who raised this in an intervention, I say that, absolutely, I will make sure my officials are talking to the permanent secretary—the very hard-working and probably overworked permanent secretary—at Stormont, because we need to make sure we have a joined-up approach. I wholeheartedly agree with everyone’s calls for exactly that: every effort must be made to ensure that precision medicines are made widely available to cystic fibrosis patients.

Many people have raised concerns this evening and in many other places about NICE’s process for the assessment of drugs for rarer diseases. I said this in the last debate in Westminster Hall and I will say it again: it is a key commitment of this Government to ensure that people with comparatively rarer conditions, such as CF, get the same quality, safety and efficacy in medicines as those who have more common conditions. I assure the House that where a company is willing to set a fair price for a drug, NICE’s technology appraisal process has been proven to be suitable for the assessment of drugs for rare diseases.

I said I would not give way, and I will not do so.

I thought the hon. Member for Bury South made a good point when he said if NICE did not exist, we would have to invent it. I agree. Indeed, NICE has been able to recommend a number of drugs for patients with rare diseases through its technology appraisal process, including pirfenidone for pulmonary fibrosis and mifamurtide—why they cannot produce easier names, I will never know—for a rare form of bone cancer, to name but two.

I know that it is distressing, to put it mildly, for patients and their families, as well as campaigners, when NICE is not able to recommend a treatment. Unfortunately, there will continue to be occasions when NICE is not able to a recommend one for routine use on the NHS. As we know, NICE has not so far been able to recommend the use of Orkambi for treating CF, because the benefits were not sufficient to justify its considerable cost. At list price, the cost per quality-adjusted life year of Orkambi is many times higher than the upper end of the threshold used by NICE in deciding whether to recommend a treatment. NICE does not stick rigidly to a cost per QALY threshold, and it applies plenty of flexibility in deciding whether to recommend treatments where the decision is a borderline one. However, it must be stressed that the cost per QALY is not an arbitrary measure, but a tool to assess what the impact would be on other NHS patients if the NHS were to spend money on a particular treatment.

I want to talk about the discussions with Vertex. As Members know, NHS England has been in intensive discussions with Vertex to encourage it to lower the cost of Orkambi to a level that would allow NHS England to fund its use without adversely impacting other patients. I and the Under-Secretary of State Health, Lord O’Shaughnessy, who sits in the other place—he leads on this area for us—have been keeping an extremely close eye on these discussion, as I promised the House in the Westminster Hall debate I would do. As Members will remember, we wrote to Vertex in April, urging it to commit to pricing that is responsible and proportionate. I have the personal assurance from NHS England that it is committed to seeking a way forward in these negotiations, and I think it has shown flexibility.

Sadly—I am not ratcheting up the rhetoric or name-calling, as this is just fact—Vertex has been unwilling to price responsibly thus far, which has, as we have heard today, meant that patients have missed out on two years of treatment. Vertex claims that it has made the NHS

“the best offer in the world”—

but it has yet to substantiate that claim. I would challenge Vertex to waive confidentiality—which it can do: I cannot—so that we can all see, in the interests of transparency, the kind of prices it is trying to charge the NHS and, as a result, our constituents.

As mentioned this evening by the hon. Member for Bury South, NHS England wrote to Vertex yesterday to set out a proposed five-year deal with an option to extend, which provides the potential for Vertex to secure revenues from the NHS in the region of £500 million over the next five years. NHS England has made the content of this proposal public, to give patients and taxpayers the opportunity to make a judgment about the fairness of this offer. The proposed deal, if accepted, would guarantee immediate and expanded access, as clinically appropriate, to the two licensed medicines, Orkambi and Kalydeco—as was said earlier, Orkambi is not the only show in town. It would also provide immediate access for Symdeco from the date it is licensed for use in the UK, which is expected in coming weeks.

If Vertex is not willing to accept the deal, it must return to discussions with NICE and go through that process. Let me be clear that I truly hope it does not come to that, but until Vertex adjusts its prices, NICE will not be able to recommend the drug’s use. I repeat that we have put a figure on the table. The ball is in Vertex’s court. It has made a preliminary response today, saying that it is good to see that NHS England is negotiating. NHS England has been negotiating throughout. It is not Ministers who are negotiating on this.

That is kind of the Minister and I thank him for his serious response to the debate.

We said the last time we met that we did not want Ministers to keep an eye on the issue: we wanted Ministers to be actively engaged in it. The Minister gave us an assurance that his colleague would be actively engaged, not keeping an eye on it. Today, we repeat that we do not want NHS England and the drug company left in a room alone to work this out, otherwise we will be back here in six months’ time. We expect the Secretary of State to be in the room with the chief executive of NHS England and the drug company to bang heads together and come up with a satisfactory conclusion. Will the Minister respond directly to that point and give us an assurance that there will be political involvement and engagement in the negotiations, because we have waited for too long?

When I say “keeping an eye”, I do not mean it like keeping an eye on the football scores on a Saturday afternoon. It is just my turn of phrase. Ministers are keeping very close tabs on this and making sure that NHS England is in no doubt that we want it to come to a conclusion with Vertex. But it takes two to tango, as we are seeing in another negotiation right now. We need Vertex to tango. The ball is in its court, and it is time to return it.

I stand by what I said at the last debate: it is vital that we go through the right process here. There is a process. I do not think for one minute that the Opposition are suggesting that if they were in government, Ministers would be making these decisions like Roman emperors, as that would be totally wrong. It is an important principle that the NHS must ensure that healthcare services get the best value for patients, and that is the approach NHS England is rightly taking.

NHS England has proven that it can strike innovative deals, working with the life sciences sector to make treatments available for patients, while securing fair value for taxpayers—our constituents. That includes hundreds of patients with multiple sclerosis who now benefit from a treatment called cladribine and the use of pertuzumab for breast cancer. It is important for me to be clear with hon. Members that Ministers should not make a decision on behalf of NHS England, which has the clinical expertise and the powers to purchase medicines.

I put it on record again that I urge Vertex to accept the proposal presented by NHS England, or it will only make those living with cystic fibrosis suffer. Yes, of course there are discussions among the new ministerial team. I spoke to the new Secretary of State just this evening in the Lobby. He is taking an incredibly close interest in this issue, less than a week into the job, and I pay tribute to him for that. I ask Vertex to recognise NHS England’s flexible approach. It would represent the largest ever financial commitment of its type in the 70-year history of the NHS. I certainly hope that they can reach a deal. Many, including in my own Winchester constituency, are counting on them.

Question put and agreed to.

House adjourned.