Future access to Kaftrio for rare mutations across the UK

The existing license for Kaftrio in the UK is for people with CF aged two and above with at least one F508del mutation in the CFTR gene. As part of the interim access arrangement, access was also granted for those with mutations that had been approved by the US Food and Drug Administration (FDA), meaning the UK has some of the most comprehensive access in the world. 

However, since the original clinical trials, evidence has been emerging that shows that some people with rarer mutations could also benefit from these medicines. A recent study in the US from the Cystic Fibrosis Foundation and academics has found that there is a potential clinical benefit of Kaftrio in another 152 rare mutations. 

Our call for equal access

In Scotland and Wales, clinicians have been able to secure access for some individuals with CF where there is evidence they could benefit, and there is some flexibility in Northern Ireland. At the moment, the same is not the case in England.

We’ve been calling on NHS England to take a similar approach to other nations of the UK. They’ve told us they will look to address this as part of discussions on a new long-term deal for access to modulators, part of the NICE appraisal process.

As these commercial discussions may continue for some time, we want the NHS in England to put an interim arrangement in place to ensure that people do not miss out on accessing medicines that could significantly improve their health.

The UK Cystic Fibrosis Medical Association (CFMA) has also highlighted concern about unequal access to modulators across the nations of the UK.

Treatment options for those who cannot take modulators

Treatment options for people not able to take current CFTR modulators, including those with rarer mutations or who are not able to tolerate the current medicines is a research priority for the Trust.

We are funding a Strategic Research Centre on gene editing, which is looking into how the mutation (mistake) in the CFTR gene that causes CF could be corrected. This research is helping to develop treatments for all people with CF, prioritising those who are not currently eligible to take modulators.

Excitingly, a number of early phase clinical trials using different genetic therapy approaches are about to open in the UK. Further research will be needed to prove that these new approaches are effective, and this will take time, but this is an important step towards effective treatments being available for everyone with CF. For more information about ongoing clinical trials please speak to your clinical team or visit the Clinical Trials Digital Hub.

Cystic Fibrosis Trust is committed to funding research that will improve lives for everyone with CF – and supporting our community every step of the way. We won’t stop until CF does.