Deal announced for triple-therapy in England
Today’s announcement by Simon Stevens, Chief Executive of NHS England, applies to people with cystic fibrosis (CF) in England, aged 12 and over with two copies of the F508del mutation, or one copy of F508del and one copy of a 'minimal function mutation'.
In addition, clinicians will be free to start prescribing the triple combination therapy and other drugs in the portfolio for certain patients with rare mutations that are covered by the U.S. Food & Drug Administration’s licensing decisions.
The announcement could also pave the way for similar deals in Scotland, Wales and Northern Ireland, pending ongoing individual discussions.
The news follows the announcement of a positive recommendation for the treatment by the European Medicines Agency last week, and it will be made available in England.
The deal is a huge step forward in a hard-fought campaign for access to life-saving drugs in which the Trust and the CF community have stood shoulder to shoulder. A letter from NHS England to the Trust confirming the news recognised the ongoing roll of the Trust and the UK CF Registry in supporting the data collection aspects of the agreement.
Speaking at the Health and Social Care Select Committee this morning, Simon Stevens stated that:
“… NHSE have signed a commercial agreement with Vertex Pharmaceuticals to make available their new triple therapy, the very day it gets its European license. And furthermore, we have also agreed a flexibility so that clinicians will be able to use that not only within its European license and any other future indications but also other drugs in the portfolio for rare mutations that might be covered by licensing by the US FDA as well. And then the last piece of good news here, is that in negotiating, NHSE has also inserted “tag-along” rights for Scotland, Wales, and NI so that should those devolved administrations want to benefit from this agreement. This is a very significant day for cystic fibrosis patients that really tackles the underlying cause of the disease by helping lungs work effectively.”
You can watch the clip in full here.
David Ramsden, Chief Executive of the Cystic Fibrosis Trust, said: “It is fantastic news that a deal has been done between NHS England and Vertex Pharmaceuticals and Kaftrio will now be available to thousands of people across England in the coming weeks. This will truly save lives.
“This is a great day, but we know that there is more to do and we will not stop until everyone with CF across the UK has access to life saving drugs.”
Alexandra Andrews, aged 45, from Nottingham, has been taking the drug since May 2020 on compassionate grounds due to her poor health. She said: “It’s been amazing. It’s improved my quality of life no end. I can actually do more of the little things in life. I can exercise again and I feel healthier. This drug has made a massive difference and it’s helped me to feel better mentally too.
“I can now have a conversation without feeling out of breath, without struggling to breath. It’s like a miracle drug and it’s given me a new lease of life."
The Trust will continue to campaign for access to life-saving drugs for everyone who can benefit, and is investing in cutting-edge research for everyone living with CF, regardless of their genotype. Donate now to support this vital work, and find out more about what licensing the triple-therapy means for you.