Why an agreement on a drug called Orkambi matters

Over the years many constituents have contacted me about diseases and conditions they suffer from. And unless I or someone in my family has suffered from the same condition, they will always know more about it than I ever will, and the names of the drugs that make the most difference.

In 2010 some constituent friends asked me to join a fund raising walk for their son Patrick’s condition - Cystic Fibrosis. I knew nothing about it then. It’s quite a rare condition, but the UK has the second highest prevalence for cystic fibrosis of any country in the world and about 10,000 British nationals live with it. In essence if you have cystic fibrosis the glands that produce mucus and sweat can get easily blocked, making it very hard to breathe and digest: and your pancreas can get blocked. Only half the sufferers live beyond 40, although improved treatment does mean longer lives are possible.

Off we went up a mountain in the west of Ireland, learning a bit more about Patrick’s health and the condition in general from the Cystic Fibrosis Trust charity who came with a big group from Gloucester. As a result I’ve kept a gentle eye out for news which might make things better for cystic fibrosis sufferers. Over a year ago Patrick’s family and other sufferers in Gloucester contacted me about a new drug from the UK pharmaceutical company Vertex, called Orkambi. The issue was about the drug’s availability on the NHS: new drug research takes years, costs a lot of money and the drug then expensive to access. NHS negotiations invariably take time.

So I was delighted yesterday when the Health Secretary Matt Hancock announced NHS England has secured a definitive agreement with Vertex Pharmaceuticals. This makes available all three of their UK-licensed cystic fibrosis (CF) medicines (Orkambi, Kalydeco and Symkevi) to sufferers on the NHS (completely free of charge to the user). Around 5,000 people (or half our total sufferers) can now take up these treatments, without limit on patient numbers, and clinicians can start prescribing these drugs within 30 days from now.

This drug won’t directly help all CF sufferers. But the agreement with Vertex means that the company will submit its full portfolio, including in due course its ‘Triple Therapy’ to NICE for comprehensive appraisal. The Triple Therapy is the drug most likely to make a huge difference to Patrick and others with less common genes, and it’s now only a matter of time before this too becomes available. So this is immediate good news for half our country’s sufferers and a huge leap forward for others from an agreement on access to Vertex’s other drug development.

It’s been a long journey. The NHS has long committed to offering CF sufferers the best treatment available to help ease their symptoms and make the condition easier to live with, and now NHS England and Vertex have worked together to arrive at a deal to make the best new CF treatments available. The price agreed is a very good one for taxpayers, which means the NHS can afford to go on providing access to other ground breaking medicines.

Recent examples include access to CAR-T therapy (which can cure some children and adults with blood cancers), pembrolizumab (for lung cancer), a deal to eliminate Hepatitis C and a deal to make nusinersen (for spinal muscular atrophy) available. The NHS England team works with the best pharmaceutical companies to make new and promising treatments available to all, and has once again shown that science and research can help alleviate even the most difficult conditions. Which is why Orkambi matters.

Please do share this with anyone you know who has cystic fibrosis or suffers from rare conditions. Will you (or they) let me know let me know what other conditions you have that still await ground breaking new drugs?

Let me know your thoughts  via richard.graham.mp@parliament.uk

Best regards