Cystic Fibrosis Drugs: Orkambi Debate

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Mr Stephen Hepburn

Main Page: Mr Stephen Hepburn (Independent) - Jarrow)

Cystic Fibrosis Drugs: Orkambi

Mr Stephen Hepburn Excerpts
Monday 10th June 2019

(1 year, 2 months ago)

Westminster Hall
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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.

Mr Stephen Hepburn (Jarrow) (Lab) Hansard
10 Jun 2019, 5:44 p.m.

It is a pleasure to serve under your chairmanship, Mr Bailey. Like others, I pay tribute to the hon. Member for Sutton and Cheam (Paul Scully) for bringing about the debate and allowing us once again to give the Government a jog and tell them to get this sorted out once and for all. We are all aware of cystic fibrosis. It affects all of our constituencies. It is a life-limiting condition that affects more than 10,000 people in the UK. It is a condition with no cure as of yet, and half of those who have it will die before their 31st birthday.

We are also aware that there is a drug available. Orkambi, developed by Vertex, treats not only the symptoms associated with cystic fibrosis but the underlying causes. One of these patients is four-year-old Harriet Corr from Jarrow, whose parents Emma and Chris are here today. They desperately want a better quality of life for their daughter. They contacted me because they were frustrated and upset that Harriet could not get access to this life-changing drug as it is not available on the NHS in England. An even bigger blow to them is that if they lived an hour further north, in Scotland, Harriet could qualify for it next year. Why? The NHS in England and NICE do not deem the drug cost-effective and have spent more than two years negotiating a price, without success.

It is totally unacceptable that a country with the second highest number of sufferers, and one that leads the way in cystic fibrosis outcomes, now risks falling behind many other European countries and the USA and allowing people with the condition to become physically sicker than necessary all because an agreement on the economics of treatment cannot be reached. We all know that because of that we have had five parliamentary debates, and still there has been no action from the Government. These drugs need to be on the NHS now, because every day without them is another day of suffering for the people living with the condition.

If people want to focus on the economics, the evidence is clear. As Members have outlined, this treatment prevents the condition from escalating, thus preventing more expensive treatment further down the line. It would reduce the number of times patients used the NHS and hospitals throughout the year, which would obviously be a saving to the NHS and—more importantly—a better outcome for patients and their families.

Let us look at the human aspect of the debate. Families are well aware that access to the treatment is not a cure for the condition, but it certainly helps those affected live as normal and fulfilling a life as possible in the short time they are with us. The NICE appraisal methods fail to recognise that. How can a cost be placed on a child with cystic fibrosis and their ability to manage their condition, to go to school, to play with their friends, to go to university later on in life and get a job as well as easing the burden of care on their families and carers? Why should Harriet and her family not have a worthwhile quality of life and get to do the things many of us take for granted?

Time is running out for some patients, and we are calling on the Government to act now to resolve this matter urgently. I thank Members from across the House for pushing this case forward. I also pay tribute to the campaigners, who have fought hard, organised a petition and shouted so loudly that surely this Government, in the fifth or sixth richest country in the world, can no longer ignore them.

Stephen Morgan Portrait Stephen Morgan (Portsmouth South) (Lab) - Hansard
10 Jun 2019, 5:49 p.m.

It is a pleasure to serve under your chairmanship, Mr Bailey. Kick-starting a social media campaign, meeting local politicians and working tirelessly alongside organisations such as the Cystic Fibrosis Trust—do those sound like the actions of a five-year-old? Ivy Weir is a remarkable local campaigner who I am lucky to have living in my city. With the support of Gemma, her dedicated mother, Ivy lives with cystic fibrosis. She has carved a path into uncharted territory when it comes to accessing Orkambi.

Ivy helped me send out 650 letters to all MPs asking them to take part in the #StrawfieChallenge. She has been on national news, alongside her mother—committed campaigner Gemma Weir—calling for that change. She has helped deliver hundreds of letters to 10 Downing Street, calling for Orkambi to be free on the NHS. Ivy, Gemma and other CF activists have shown immense dedication and resilience in the face of adversity; now we in Westminster must do the same.

There have been five parliamentary debates about access to these medicines, yet we still see no light at the end of the tunnel. Over a year ago, the Prime Minister called for a speedy resolution to this emergency; there has still been no progress. We have the chance to improve the lives of thousands of people across our nation. There has been enough dialogue; now we need to see action.

As we have heard today, over 10,000 people in the UK live with cystic fibrosis—one in every 2,500 babies born. In the last four years, this genetic disorder has tragically claimed the lives of 210 people. What makes these figures more harrowing is that drugs are available to relieve many of the symptoms and greatly improve the standard of living for those with CF, including improving life expectancy.

Orkambi can make a real difference. The obstacle to the widespread implementation of these drugs is the cost; I find that morally repugnant. We live in a nation that forged an NHS from the ashes of world war two, that prides itself on universal healthcare for all and that is the envy of the world, because healthcare is free at the point of delivery and based on clinical need, not ability to pay. As such, the UK is a global bastion when it comes to cystic fibrosis outcomes. However, if Orkambi and other drugs are not rapidly added to the list of drugs available on the NHS, we will lose this valued status. Letting costs stand in the way of saving lives does a great disservice to the principles the NHS is rooted in: universality, equality and fairness.

The Health and Social Care Committee has done vital work in carving out potential ways forward. Its recent calls for interim access would allow negotiations to continue, without the unnecessary suffering of those living with CF. However, the non-binding nature of the suggestions means that three months on, no such agreement has been set in motion. Where NHS England and Vertex remain at loggerheads, NHS Scotland has established a deal with the pharmaceutical company. This will create a brutal postcode lottery. The level at which people suffer from cystic fibrosis is currently determined by where they live in the UK. I am confident that this was not the NHS envisaged by its founders nearly three quarters of a century ago.

I have seen the effects on my constituents in Portsmouth first hand; I will make every effort to ensure their concerns are raised. In 2017, I wrote to the right hon. Member for South West Surrey (Mr Hunt) in his capacity of Secretary of State for Health, urging a swift response. In 2018, I wrote to the Secretary of State for Health and Social Care, the right hon. Member for West Suffolk (Matt Hancock), on his appointment, asking him to do the same. In 2019, I wrote to the Prime Minister, asking her to intervene. Now is the time for leadership on this most pressing of matters. We must see progress and we must see it quickly.

It has been announced that Vertex Pharmaceuticals could potentially develop treatments for 90% of those who live with CF, over the next five years. If solid, robust negotiating foundations are not established now, that could undermine patient access for generations. Today, I call on the Department of Health and Social Care, Vertex and the Prime Minister to make this a national priority and help to bring an end to the suffering of thousands of people across the UK.