Kaftrio, one year on

Blog - Lynsey Beswick -

Lynsey Beswick, Public Affairs and Advocacy Manager at the Trust, reflects on the year since the landmark deal for access to Kaftrio in England was announced amidst the COVID-19 pandemic.

Today marks the one-year anniversary that NHS England and Vertex announced a deal for a commercial agreement for access to Kaftrio, the first triple combination therapy for the treatment of cystic fibrosis (CF). The announcement was a significant and exciting moment towards access to the drug on the NHS for thousands of people living with CF.  

It followed news of a positive recommendation for the treatment from the European Medicine Agency (EMA), paving the way for a formal a European licence of the drug in August 2020. The deal also triggered further agreements in the subsequent weeks across the UK in Wales, Northern Ireland, Scotland, and the Crown Dependencies.

Kaftrio is a modulator therapy currently licensed for use in the UK for those age 12 and over with at least one copy of the F508del mutation. It has helped to transform the lives of many by treating the underlying cause of the condition. The deal meant that many more thousands of people with CF who had been unable to benefit from previous modulator therapies including Kalydeco, Orkambi and Symkevi could finally access these treatments for the very first time on the NHS.

The breakthrough followed years of relentless campaigning alongside the CF community, supporters, and parliamentarians to fight for access to new medicines, that included parliamentary debates, protests and a health committee inquiry. We want to take this time of reflection to thank once again everyone who took the time to support and champion the cause over the years.

A message from David Ramsden

David Ramsden from the Cystic Fibrosis Trust said: Today marks one year since NHS England and Vertex announced a deal that paved the way for access to Kaftrio across the whole of the UK.  This followed years of campaigning by the incredible cystic fibrosis community and means that thousands of people living with CF are now taking this truly game-changing medicine.

As we mark this day, we re-commit to doing all we can to ensure that everyone who can benefit from Kaftrio gets rapid access to it and that we will continue to invest in research to find treatments for those who can’t. We will not stop.

Lucy's story

Lucy Baxter, who has CF, reflects on the moment she found out she heard about the Kaftrio agreement and how this has positively impacted her life. She recalls:

“When I found out the Kaftrio deal was announced, I saw it on Facebook in my room and started crying. I rushed downstairs and told my mum and we both hugged and cried together. It was a life changing moment and it made me feel that my future was secured. One year on, I am still so thankful and don’t take it for granted how lucky I am to be able to have this treatment. I feel so much better and healthier."

We won’t stop

One year on the Trust remain committed to ensuring that Kaftrio is made available through the NHS to everyone with CF who can benefit from it.

In May 2021, we welcomed the news that an application for a licence extension of Kaftrio for children aged 6-11, with at least one F508del mutation, has been submitted to the EMA and MHRA regulatory bodies for consideration. This is an important next step in the fight for wider access to CF medicines across the UK.

No one left behind

This treatment won’t work for everyone. And we won’t stop our work to ensure there are effective treatment for everyone with cystic fibrosis. We will continue to invest money in life saving research to find treatments for all. You can read more about our research that leaves no one behind, and you can also read this blog about further work we are doing around transplant. We are holding a CF live event next month to talk about this work in further detail. Keep an eye out for more details on this event here.

Support

We appreciate that many people may have mixed emotions about this day, including those who cannot benefit from Kaftrio or the other currently licensed modulators. If you would like further support, our Helpline is available 0300 373 1000. You can also access online support resources here.

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