Orkambi and Cystic Fibrosis

Bob Stewart Excerpts

Monday 19th March 2018 Westminster Hall

[Paul Scully]

The right hon. Gentleman’s contribution comes to the nub of the issue. I will scrap most of my speech, which I had not written anyway, and dash around. There are two issues. We can talk about the Government getting involved in pushing NHS England and Vertex together to make a sensible deal in this case, but I come back to the point that this is an analogue system in a digital age. I will try to do it justice, but it is a case of inequality. About 400 patients have access to Kalydeco, which I mentioned earlier. They have a particular mutation to which Kalydeco responds. Around 3,000 patients would be eligible to access Orkambi. However, the point about cystic fibrosis is that, because it is a genetic disease, it cannot be caught, so we know pretty well the number of people we will need to treat over the next few years. There are around 10,400 sufferers in this country, which is extraordinary, because there are only 70,000 sufferers around the world.

Cystic fibrosis is a pernicious disease, and I have not even started talking about its effect on the children of the people I have met. However, it is not big enough to require an international epidemic-style solution, and it is not small enough to be a rare disease. It fits somewhere in the middle.

[Bob Stewart (Beckenham) (Con)]

Nobody can catch cystic fibrosis, but as my hon. Friend mentioned, two sufferers in the same room together can really affect one another. The bugs from their lungs can transfer, presumably through the air.

[Paul Scully]

Absolutely. A lot of the people I have met have aspergillus, which is a mould-based infection. It is around in the air in many homes, which for us does not matter, but for sufferers can cause a severe reaction and a severe loss of lung capacity.

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[Bob Stewart (Beckenham) (Con)]

Being born with cystic fibrosis is just the bad luck of the draw. Until I started researching cystic fibrosis to speak today, I had no idea that one in 25 of us carries a faulty gene that causes it. Nor did I realise that people were born with the condition and cannot catch it. The Cystic Fibrosis Trust tells me that about 10,400 people have cystic fibrosis in this country.

It is bad luck if both parents have a gene that might generate cystic fibrosis in their child. Most potential parents carrying a generating cystic fibrosis gene are totally unaware that they could have a baby cursed by the condition. I gather that the chance of having a baby with cystic fibrosis when both parents have the gene is about one in four. That was the case with Trudy and Gary Davey, my constituents, whose son suffers from the condition. Trudy is the daughter of my very close friend, Maggie Hopgood. Maggie’s grandson Zac had the standard blood test just after his birth, and he has been on cystic fibrosis medication ever since—that is for life.

Cystic fibrosis is a rotten condition to have. Most often it attacks our lungs. Sufferers get a mucus build-up in their lungs, and lungs do not work well when they are drowning, but it can be worse than that. Our pancreas is tasked with producing enzymes that break down food in our stomachs, but that organ can also often fill up with mucus, ruining what it is meant to do. Cystic fibrosis does not stop its rampage there. It can also cause diabetes and bone disease. Cystic fibrosis is literally a pain to live with. It is always life-threatening, and it is certainly long-life restricting.

I come to the main point of this debate, which is to get cystic fibrosis sufferers, such as seven-year-old Zac Davey, better access to Orkambi. Orkambi seems to make a huge difference to the lifestyles and lives of sufferers. I gather that it works on about 50% of cystic fibrosis sufferers and slows the decline in their lung function by 42%, which is quite a result.

As we have heard, Orkambi is not normally available on the NHS except in extreme compassionate cases. Of course, the reason for that is expense. Per patient, Orkambi can cost more than £100,000 a year. On the plus side, more widespread use of Orkambi is likely to reduce the requirement for hospital treatment among sufferers—there is a spin-off in reduced NHS costs. The drive must be to reduce the costs of Orkambi. That requires action from Vertex, the US-based company that produces it, and from the Government and the NHS. I gather that negotiations have been ongoing for a while. We are talking about people’s lives and their life expectancy. It must be sorted and it can be. Hopefully this debate, having raised the matter for the third time in Parliament, as we have heard, will add impetus to the resolution of the problem, and Orkambi will be made more readily available to the many who have sore need of it such as my friend’s grandson.

[Sir Roger Gale in the Chair]

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[Patricia Gibson (North Ayrshire and Arran) (SNP)]

The development of the treatment offered by Orkambi has offered renewed hope to those living with cystic fibrosis and their families. Orkambi is suitable to treat around 50% of those with cystic fibrosis in the UK and, as we have heard today, that treatment can now be extended to those from aged six years, as opposed to the original licence, which was to treat those aged 12 and over.

We have heard today at some length about the controversy over the cost of the drug. As a Scottish MP, I was heartened by the Scottish Government Cabinet Secretary for Health and Sport, Shona Robison MSP, who strongly encouraged the manufacturer Vertex to take forward discussions with the patient access scheme assessment group secretariat. Those conversations continue. It is easy, and perhaps obvious, to point out the high cost of the drug—we have heard much about it—but who in this Chamber would not stretch every sinew for their child or loved one to access the drug if they could benefit from it? That is why parents and relatives of those living with cystic fibrosis have been campaigning so hard. What else can they do?

We have heard about the cost of the drug, but as we have also heard, the costs can be offset by reduced hospital admissions. NHS funding will always be under pressure as our technology advances. One consequence is that the cost of treatment is always likely to be driven upwards. Costs are important, and we have to be mindful of them, but I ask hon. Members, “If your child or loved one could benefit from a treatment, however expensive, what would you not do to help them to access it?” That is what drives those campaigning for access to Orkambi.

The campaign has been effective and moving, as we have heard. We must keep listening and empathising with what those people are saying, because we cannot just talk about pounds and pence when it comes to treatment. We are dealing with real lives, which must guide progress. Progress must be made—we all hope that happens sooner rather than later.

[Bob Stewart]

My point is very straightforward. It seems grossly unfair that some people can get hold of this drug and some people cannot. We ought to level the playing field.

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[Bob Stewart]

On my good friend’s excellent point, I wonder how much it costs to put a child in hospital for a month. I am pretty sure it is not far off £100,000.

[Crispin Blunt]

My hon. Friend leads me to my next point. How do we wish to value better and longer life for those with cystic fibrosis? How do we wish to value the savings to the NHS of reducing the 9,500 hospital admissions and the 100,000-plus hospital bed days a year? How do we wish to value the societal and economic benefits of helping young people grow up to play a fuller part in their education and employment, and the benefits to their families and care givers? How do we wish to value and reward the risk and innovation that goes into researching and developing breakthrough medicines?

NICE applies rigid health economics methodologies through its standard technology appraisal, which is designed not for rare diseases and specialised services but for primary care medicines that treat large populations with well-known diseases. Are we content for NICE to apply a threshold for valuing life that has not changed since it was established in 1999, even though healthcare inflation has almost tripled what we spend on healthcare?

My right hon. Friend the Member for Chingford and Woodford Green (Mr Duncan Smith) referred to social impact bonds and payment by results. I do not envy those who have to make those evaluations, such as the Minister and NICE. The costs fall on NICE and the health service budget, and the benefits are often felt elsewhere, not least in patients’ quality of life. Our society and the Government have to become more sophisticated about early investment. We must be able to measure the savings that come from having fewer hospital admissions and from the greater contribution to society that people who suffer cystic fibrosis will make if we improve their quality of life and reduce the degradation of their lungs.

Does the Minister accept that, although NICE has a specific evaluation process for highly specialised technologies for ultra-rare diseases, it is missing a framework for other rare diseases and precision medicines that treat sub-groups of rare diseases? He will no doubt point to initiatives such as the accelerated access review and the sector deal for the life sciences industry. I welcome those schemes in so far as they aim to address some of the access challenges, but they count for little if there is not a willingness to find innovative and flexible approaches to introducing innovation in the NHS. If we get this right, the UK has huge opportunities better to serve NHS patients and attract industry investment in clinical trials.

Vertex, the manufacturer of Orkambi, recently proposed a portfolio arrangement to NHS England, as we have heard from many colleagues, whereby all its current and future cystic fibrosis medicines could be made available to eligible patients at a fixed cost to the NHS, irrespective of the number of patients treated. Vertex wants to work with NHS England and NICE to put a long-term arrangement in place, as it has already done in Ireland. As the company introduces new medicines and line extensions, patients will get rapid access to the most suitable products for them upon regulatory approval, and the NHS will have budget certainty. Vertex expects to develop therapies that will treat 90% of cystic fibrosis patients within seven years.

In the light of the company’s proposal and the strength of feeling expressed in the petition and by colleagues in this debate, will the Minister look at mandating NHS England and NICE to prioritise discussions with Vertex to find an innovative and sustainable funding solution? It gives me hope that the offer that Vertex found unacceptable last Friday included a possible portfolio approach. It failed simply because NICE was not prepared to increase the resources it already pays for existing drugs, not including Orkambi. That plainly meant that the offer could only be unacceptable to Vertex, given that it is proposing new treatments that are going to treat 10 times as many patients as are being treated by the drugs currently available.

This is urgent. As my constituent, Sharon Cranfield, said to me:

“Each day of delay is additional delay of ‘irreversible lung damage’”

for those with treatable cystic fibrosis.

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[David Linden]

Absolutely; I know that my hon. Friend is a passionate champion of her constituents in Glasgow Central and she is right to put that on the record.

Numerous other constituents have come to me, including Lee Bennie from Garrowhill. Again, she made the case that we need Orkambi now. Over the course of the weekend, I had the privilege of spending some time talking to a friend’s brother, Ross Moore. He is not a constituent of mine, but he has access to Orkambi through the compassionate access scheme. Ross is a remarkable young man who is incredibly articulate. I was quite struck by how frank he is; he has lived with cystic fibrosis for so long and I was very moved by the way in which he could explain why he has access to Orkambi and why he thinks that other people should have access to it.

Ross was moved on to Orkambi only in October last year because his lung function had dropped below 45%. He was quite honest in saying that the first six to 10 weeks were very tough; for some people that can be make or break, but he has got through that. My hon. Friend the Member for Glasgow Central (Alison Thewliss) has mentioned people’s ability to try to put that effort into physio, and Ross said that it is a very arduous process. He does physio for two to three hours a day. When speaking to him, I was struck that he already has access to Orkambi, and he does not want to pull up the ladder behind him. He said to me before I came into this Chamber, “Go in there and explain the benefit of Orkambi; we need to make sure that people understand that for those who do not have access to it already we have got to have that access, because it is only through that research and that ability to use it that they will have it.”

[Bob Stewart]

Does that mean that the hon. Gentleman’s constituent will have Orkambi for life, or will it be taken off him later?

[David Linden]

While I was having that conversation with Ross, that was at the back of my mind. I am very glad that he answered before I managed to ask it, because it is very difficult to ask somebody, “Will you have access to this for the rest of your life?” Thankfully, in Ross’s case, he will.

My hon. Friend the Member for North Ayrshire and Arran (Patricia Gibson) outlined very eloquently the situation in Scotland; for the purposes of time I do not want do that. I had a phone call when I left Glasgow airport this afternoon from my constituent, Helen Seery, who lives in Carmyle. Her son, Layton, was born just 22 months ago and has cystic fibrosis. The 20-week scan showed that he would have cystic fibrosis; he was born prematurely at just 28 weeks. His lungs are naturally weaker as a result of being a premature baby. My constituent Helen made the point about what a difference it makes in Ireland, where people have access to Orkambi at five and six years old. I will visit Layton in Carmyle next week. I am aware that it is a challenge for Helen to bring somebody into the house due to the risk to Layton.

It is important that we stand up and make speeches and that we will send the Hansard to the constituent afterwards, but the most important thing for me is going back to Helen and telling her and Layton that I stood up and said that I think that Orkambi should be available in Carmyle, as well as Carlisle. That is the message that we have to leave with the Minister. Constituents are absolutely clear that we need Orkambi now.

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[Marion Fellows]

I agree with the hon. Gentleman. We are talking about people whose lives are shortened and people whose lives are deeply affected. These people could be of such benefit to society as a whole. It is difficult for me to think beyond my granddaughter, and it is also difficult to listen to stories of what might happen. I know she is lucky, but I also know and am passionately concerned about other children who are even more affected by CF than she is. We owe it to them and to people who will inherit CF in future.

I have to be a carrier of the CF gene, and my husband must also be a carrier. We were so lucky that none of our children were affected. It is a terrible, silent disease. The fact that CF sufferers cannot even get together for comfort and mutual support—it all has to be done through parents’ groups—is even more heartbreaking.

[Bob Stewart]

I am deeply affected by listening to the story of the hon. Lady’s granddaughter. When did the hon. Lady and her husband know that they were carriers? That is pretty important.

[Marion Fellows]

I probably am a carrier—unfortunately, for another reason, we have not been tested yet. That is another issue that we will deal with. It would help future research. Actually, after my daughter was tested, she said to me, “Mum, you and dad must go out and buy a lottery ticket—you must be the luckiest people around,” because I have three children and none of us has CF.

The Scottish Government are encouraging patient representatives to participate in Scottish Medicine Consortium meetings. That must be a good driver to let clinicians and other medical people see with their own eyes the difficulties undergone by people who cannot access these drugs.

Sir Roger, you have been most considerate, and I will now sum up. There is an overarching need for a change in the way in which new drugs are approved for use. Cost-benefit analysis needs to be used to calculate the true costs. For example, we should look at reductions in hospital care costs and include the benefits to the economy from the use of these drugs—especially Orkambi, which is our exemplar for now. Of course, CF suffers must be able to play a full part in society and, as we have heard, they could do so if they could access drugs.

Orkambi could be just the start of a whole line of drugs that will benefit CF suffers. No one can cure the disease at the moment, but such drugs will lead to so many more better and productive lives. Yes, there are issues around pricing new drugs. Pharmaceutical companies are not there for charitable purposes, and we all know and understand how expensive it is to create new drugs, but there must be a better way of pricing the cost of new drugs at their introduction across years of their use rather than huge up-front costs.

I was asked by the Scotland parents support group to demand that the Minister takes responsibility for the negotiations with Vertex, and that he brings NHS England and Vertex Pharmaceuticals to the table. Deals and negotiations should never be conducted by email. I am happy to do so, and I wait with interest for his response.