24 October 2019

NHS England has announced today (Thursday 24 October) that life-saving cystic fibrosis drugs will be made fully available on the NHS. 

An agreement has been secured with Vertex Pharmaceuticals for Orkambi, Symkevi and Kalydeco, which means around 5000 people may now take up these treatments. 

There is no cap on patient numbers, and each and every patient in England who might benefit can now get these treatments, free on the NHS. 

Clinicians should be able to begin prescribing these drugs within 30 days.

Royal Papworth Hospital is a regional centre for adults with cystic fibrosis, treating 351 patients from the East of England and beyond.

Dr Helen Barker, Consultant in Respiratory Medicine at Royal Papworth Hospital NHS Foundation Trust, said: “This is amazing news for cystic fibrosis patients. For a little while we have been able to provide these drugs to our sickest patients on compassionate grounds and have seen amazing results.

“This has meant fewer sick days, fewer hospital interventions and we have people alive today who otherwise may not have been.

“I feel so grateful that these drugs have been developed and are now being funded and we will shortly have them in our hand. This is the start of better lives for our patients with CF.”

Alex Stobbs, 29, is a patient at Royal Papworth and has been on Symkevi for compassionate use since January 2018. He said: “I had a really bad 2017 when I was in hospital all summer. I was initially given Orkambi but had strong side effects, so I was then put on Symkevi which has been brilliant.

“For the past two years there is no doubt it has helped to stabilise my lung function at around 35%, ensuring that I’ve been out of hospital for greater lengths of time than otherwise would have been the case. I’m still on the transplant waiting list, but my lung function would be much lower if it wasn’t for Symkevi. 

“There has been so much passionate campaigning and awareness-raising for these drugs over so many years now you start to wonder whether it would actually happen. 

“Understandably, added to the huge social media presence to make the issue known across the UK, there has been plenty of emotion from patients and families. At the same time you are fully aware that there are finer details of the agreement that need to be sorted out.

“This incredible news doesn’t just give hope today, but also gives all patients hope that similar deals for future advances in drug development are possible in the future.” 

Read more about NHS England’s agreement with Vertex.