Fighting for life-saving drugs
Find out more about the latest updates on the NICE appraisal of Kaftrio, Orkambi and Symkevi
What are we doing to get life-saving drugs?
We have been working alongside the CF community since the first CFTR modulator was licensed in 2015 to get access to all life-saving drugs for the people who need them. Since then, the campaign has intensified, and despite resounding proof as to the effectiveness of these drugs, we faced huge challenges.
After a sustained fight by the CF community and the Trust, Orkambi and Symkevi were finally made available on the NHS at the end of 2019 and Kaftrio in August 2020. Part of the NHS deals to grant access to modulator therapies included the agreement that data would be collected on their effectiveness. Cystic Fibrosis Trust has provided evidence to the National Institute for Health and Care Excellence (NICE) on the impact of access to these treatments, including the stories of people with CF and their families. We expect NICE to publish further information on this appraisal later this year and will keep our community informed every step of the way.
We are continuing to work to ensure a smooth roll-out so that everyone with CF in the UK who is eligible for these drugs will get access.
Find out what’s happened so far in our fight for access to life-saving drugs.
What do these drugs do?
Life-saving drugs, also known as precision medicines, CFTR modulators and modulator therapies, work to tackle the underlying cause of cystic fibrosis.
Cystic fibrosis is caused by a faulty gene that affects the production of a protein called CFTR. Its production is affected by different mutations. There are over 2,000 known mutations that can cause CF, and the treatments you can take depend on the mutations you have. Find out more about the science behind what causes cystic fibrosis.
While conventional CF treatments target the symptoms of cystic fibrosis, for example antibiotics that fight infections or steroids that reduce inflammation in the airways, these drugs tackle the underlying genetic mutations that cause CF by helping to make the CFTR protein work effectively. Find out how this happens.
What drugs are currently available?
Four drugs, Kalydeco, Orkambi, Symkevi and Kaftrio, are currently available on the NHS after a long fight by the Trust and the CF community. Deals were struck in England, Wales, Northern Ireland and Scotland for Kafrio to be available on the NHS on the first day of its licensing, which was granted on 21 August 2020. Find out more about some of the life-saving drugs that are available or in the pipeline below.
We recognise that even with access to four CFTR modulator drugs, there will still be people with cystic fibrosis who are not eligible and we will not stop until everyone with cystic fibrosis has access to the most effective medicines. This is why we’re investing in a variety of research projects.
Kaftrio, Vertex's triple combination therapy
The triple combination therapy, Kaftrio (brand name Trikafta in the United States) uses a combination of ivacaftor, tezacaftor and elexacaftor, and targets mutations that approximately 90% of the CF population have. Find out more about the drug and its availability here.
Take a look at some frequently asked questions about our fight for access to life-saving drugs, with detailed information on all the different treatments.
Find out what's been happening in our campaign for life-saving drugs since it began in 2015.
In the news
Check out the latest news about cystic fibrosis, from new developments in research to nationwide campaigns, including local, national and international stories.