Cystic Fibrosis Drugs: Orkambi

Stephen Hepburn Excerpts

Monday 10th June 2019 Westminster Hall

[Mr Stephen Hepburn (Jarrow) (Lab)]

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.