Cystic Fibrosis Drugs: Orkambi Debate

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Department: Department of Health and Social Care

Cystic Fibrosis Drugs: Orkambi
Ian Austin Excerpts
Monday 10th June 2019

(1 year ago)

Westminster Hall
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Department of Health and Social Care
Thelma Walker (Colne Valley) (Lab) Hansard
10 Jun 2019, 5:26 p.m.

It is a pleasure to serve under your chairmanship, Mr Bailey.

It is just two years ago last week that I was elected Member of Parliament for Colne Valley. Over the past two years, it has been a real privilege to serve and speak up for my constituents. One day, however, will stand out more than most for me, among many amazing and challenging days. It was Friday 31 May, when two children visited me in my constituency office. One was nine-year-old Tristan with his grandpa John, and the other was one-year-old toddler Charlie with his Mum and Dad. I had had a busy advice surgery that afternoon, but went straight to meet Charlie in one room, while Tristan waited patiently in another. They were in different rooms to avoid cross infection as they both suffer from cystic fibrosis.

They had come to see me to each hand me a letter in which they described their daily challenges living with cystic fibrosis. The letter also described how access to just one particular drug might make their daily battle with the disease a bit easier, and might result in both children living longer lives. One-year-old Charlie, along with his parents, would benefit if he could access the necessary drug from such an early age. Charlie handed over his letter to me, which I must admit was a bit soggy and chewed by the time I got it. It was a joy to be with him and his parents.

Tristan also handed me a letter. Following the death of Tristan’s father in 2016, Tristan’s grandpa and granny, John and Frances, look after Tristan as his guardians. The family have experienced more than their fair share of loss. Like Tristan, his uncle Adam suffered from cystic fibrosis, and died aged 25 in 1999. I can only imagine the immense pain and grief and incredible frustration of losing a son, yet still, nearly 20 years later, being unable to access the drugs that already exist and that would improve the life of their grandchild. I cannot express how much admiration I have for Tristan and his family, and for Charlie and his; they have campaigned with the Cystic Fibrosis Trust to improve the lives of others.

What is this drug? It is called Orkambi; it already exists and it would improve the lives of children such as Tristan and Charlie. It is made by the pharmaceutical company Vertex, and the fact that it is not already available to those who need it is shameful. There are at least 2,834 people currently dealing with cystic fibrosis in England who could benefit from Orkambi.

Why has Orkambi not been made available? The current situation is that Vertex, NHS England and the National Institute for Health and Care Excellence cannot agree a price. We know that Vertex declined a £500 million offer from NHS England, and we know that the NHS has made a new offer, which currently remains confidential. Vertex did not consider £500 million an adequate sum, but it did, allegedly, consider it okay to destroy 8,000 packs, or a 600-year supply of Orkambi, as the deadlock continued. The lives of children such as Tristan and Charlie seem to be deemed acceptable collateral damage in this boardroom-style version of healthcare provision.

I urge Vertex to consider the NHS’s current offer with the lives of children such as Charlie and Tristan at the forefront of its mind. This seems to me to be a straightforward ethical decision for Vertex, which should be willing to compromise on its profit margin in circumstances where the lives of children are at stake. Let us be clear: Vertex is not struggling to make ends meet. It is a $43 billion company. That is about the same as the GDP of Bolivia or Tunisia. Vertex’s chief executive officer received a total salary of around $19 million in 2018, making him one of the best paid CEOs in the pharmaceutical industry. As ever, the problem is not that there is not enough money, but that big business is not willing to spend it.

This situation, in which children’s lives are held to ransom, must not continue. Vertex and NHS England must agree a deal soon, or an interim solution must be found, as happened in Scotland. Children’s lives must not continue to be held hostage. Tristan’s grandpa has already lost a son to this condition. There is simply no excuse for not prolonging and improving Tristan’s life. I will continue to campaign with Tristan and his grandpa, and Charlie and his parents, until this drug is made available to them and they are able to get on with their lives.

I will finish with a direct plea from Tristan’s grandpa, John Binns:

“We don’t have time – please don’t waste it. It’s costing lives and there are too many cystic fibrosis angels already.”

Ian Austin (Dudley North) (Ind) Hansard
10 Jun 2019, 5:33 p.m.

We have been campaigning on this issue for well over four years; I think this is the fifth or sixth debate we have had in this Chamber or the main Chamber. We have met two Prime Ministers, two Health Secretaries and several Health Ministers. We have a new Health Minister with us today. We welcome her to her place, and we hope she will be able to unlock this issue in a way that her predecessors did not. I cannot remember how many cross-party letters we have delivered to Downing Street. There have been countless meetings between MPs, Vertex and the Cystic Fibrosis Trust. However, four years after this drug was licensed, it still is not being made available to the people who need it.

Imagine how many people’s lungs have suffered irreparable damage in that period. Imagine how many people have seen not just the quality but the length of their life reduced over the past four years. I pay tribute to the brilliant people from the Cystic Fibrosis Trust who are with us today: David Ramsden, Nick Medhurst and the brilliant Lynsey Beswick, whom we heard about earlier. Most of all, I pay tribute to the army of parents and carers who work so hard on this issue all year round. We are in this room today because of the inspirational work they do. It is humbling and heartbreaking to meet them and listen to their stories.

I became involved in this issue because I met Carly Jeavons, whom the hon. Member for Sutton and Cheam (Paul Scully) spoke about. She is from Dudley, and she took part in a clinical trial for Orkambi. Before being put on that trial, she had to choose between leaving work, with all the financial hardship that would cause, and struggling on in work with her health getting worse. She had to take 90 tablets and do two hours of physiotherapy a day. Her lung function was 44%. She had to spend two weeks in hospital every three months. That is not an unusual story, by the way, for people with cystic fibrosis.

Carly told me:

“Orkambi has changed my life. I quickly became well enough to start to live a more normal life again as a working mum. My health has remained stable…hospital visits have massively reduced,”

and unplanned admissions are now non-existent. Since she started having Orkambi, she has been able to go on holiday for the first time, she has got married, she has started a family and she has started a business. She makes a much bigger contribution to the economy. People talk about the cost of providing these drugs; what about the contribution that people who are given Orkambi can make to the country and the economy? Let us think about that.

I did not really understand the impact that having a condition such as cystic fibrosis has on the rest of someone’s life. I mentioned Lynsey a moment ago. I hope she does not mind me telling everyone this. She is in her 30s, and she told me that, at a time when her friends are getting married, planning families, developing their careers, starting businesses and making long-term plans, people such as her are deterred from doing those things. It was heartbreaking to listen to her tell me that, so I cannot imagine what it is like for her.

I hope people forgive me for being blunt, but people in Lynsey’s position are just not able to plan for their future in the same way because they do not know how long they have to live. Every day that people who could be helped by Orkambi are denied access to it is another day for which their lungs suffer irreparable damage. It is not like breaking a leg—a leg can be fixed and become normal again, but someone’s lungs will not recover.

I understand that Vertex wants to make a profit. Of course it does, and of course it needs to be able to fund future drug developments. But surely it would make sense to sell a lot of a drug at a slightly lower price than to sell nothing at a really high price. I am not an expert on pharmaceutical company economics, but that seems to me to be worth looking at.

What assessment has the Minister made of the case for Crown use licensing, where the Government take control of the situation and ensure that these drugs are provided? What assessment has she made of the case for the Government providing the cheaper drugs that are available elsewhere? It is a disgrace that families have to buy them from Argentina.

The Minister is new to the Department, and we really hope she will work night and day to get this sorted out. She should get NHS England, NICE and Vertex in a room and not let them out until they have come to an agreement. That is what we need to happen. Will she also meet the cross-party group of MPs who have been working on this issue, and the parents and the Cystic Fibrosis Trust, so we can discuss it in more detail than we are able to this afternoon?

The situation is urgent. The system has not worked. People with cystic fibrosis have been put in an appalling position. This really does need to be sorted out now.

Jeff Smith Portrait Jeff Smith (Manchester, Withington) (Lab) - Hansard
10 Jun 2019, 5:38 p.m.

It is a pleasure to see you in the Chair, Mr Bailey. I congratulate the hon. Member for Sutton and Cheam (Paul Scully) and all the other Members who have made thoughtful and powerful contributions. As we heard, it is now more than a year since the Prime Minister called for a “speedy resolution” to this crisis. For years, patients and their loved ones have seen their health decline, knowing that a possible solution has been sitting on the shelf the whole time. I will use two personal stories to illustrate why this issue is so urgent and important.

My constituent Clare Dempsey has been advocating for patient access to Orkambi and the associated precision treatments on behalf of her son, Jake Wright, who is nine and was diagnosed with cystic fibrosis at birth. His story is an insight into the relentlessness of treating the symptoms of the disease. He has to take 200 tablets a week, undergo two hours of physio a day and use nebulisers morning and night. Every year, he spends two weeks in hospital in isolation, receiving intravenous antibiotics. Jake has lost 16% of his lung function and a recent CT scan of his lungs showed early signs of bronchiectasis, which can leave lungs more vulnerable to infection. Precision medicines such as Orkambi that tackle the underlying causes of CF could have prevented those irreversible changes to Jake’s health. Imagine how it must feel as a parent to know that.

There is also the associated emotional and financial pressure on families. Clare is a cancer researcher by profession, but she has had to go part time to care for Jake. Not only is the NHS losing her skills but she is unable to earn money for the family, who have had to put their house on the market. If the root causes of Jake’s cystic fibrosis go unaddressed, the level of care he will need from his family and medical professionals will only increase. We talk about cost-benefit analyses, but NICE cannot take into account that effect on families, the NHS and wider society. We need to bear that in mind carefully when talking about this issue.

I want to talk about another affected family. My constituents Sadie Lawty and her husband Steve Sanders have a three-year-old daughter, Eloise, who is a lovely young girl I have had the great pleasure of meeting. She was diagnosed with cystic fibrosis when just two weeks old. It was a bombshell to Sadie and Steve, because as far as they knew there was no family history of it. At the time, they were told there were many reasons to be hopeful, because a new family of pharmaceuticals that targeted the underlying causes of cystic fibrosis had recently been introduced. They were given hope, but their hope has been frustrated.

Orkambi has existed for the whole of Eloise’s life, and its positive effects are long established. The frustrating reality for Eloise and her family is that they are no closer to accessing it than when she was first diagnosed. As we heard in Jake’s case, there is never a day off when combating the condition. Medicines have to be administered around the clock and there are endless trips for assessments and treatments. In fact, while we are having this debate Eloise’s parents are taking her to the doctor’s for her annual review, essentially to find out how much damage has been caused by cystic fibrosis so far.

Accessing Orkambi could help sustain Eloise and offer her parents some longer-term peace of mind. It is not a wonder drug, but it is a source of hope, so it is frustrating for Eloise’s family that their hope has been dashed. Eloise is doing well at the moment; hopefully that will continue. She will start nursery soon. At three years old, she has a while before the cumulative effects of cystic fibrosis really set in, but many people cannot afford to wait much longer.

I think back to my first brush with the condition, when I had a girlfriend who was a nurse. She was looking after a very charming young man who sadly died while still in his teens. That brought home to me the personal tragedy involved with this condition. It is therefore vital that the Government take responsibility for pulling people together and finding a solution among NICE, the NHS and Vertex as quickly as possible.

There are potential ways forward. My hon. Friend the Member for Bristol East (Kerry McCarthy) outlined solutions such as Crown use licences to overturn the monopoly and access to drugs through clinical trials. The Chair of the Health and Social Care Committee, the hon. Member for Totnes (Dr Wollaston), talked about the interim arrangements in Scotland, which may provide for a way forward. Whatever that way forward is, we need to find it.

I am pleased to see the Minister in her new role and hope that, as my hon. Friend the Member for Dudley North (Ian Austin) said, she will really grip on to this issue and try to find a solution that delivers for all the people I talked about, for the people who need the drug now, for the people in the Public Gallery, and in memory of the people who died. I plea with her to get the Government to get people around the table and find a solution.