NICE modulator appraisal – what happens now?

Summary

• The NICE guidance on Orkambi, Symkevi and Kaftrio that was published in November 2023 is draft and not a final decision. 
• Future access to these treatments is protected for anyone already taking a modulator treatment, whatever the final outcome. 
• People with CF can continue to start on modulator treatments while the NICE process is ongoing. 
• The appraisal could continue well into 2024. We are expecting another update from NICE at the end of January.

On 14 December 2023, the NICE committee met for the second time to discuss the appraisal of Orkambi, Symkevi and Kaftrio. They referenced the huge number of contributions from the CF community, and it was clear the voices and experiences of the community have been heard. Thank you to everyone who submitted their views to NICE.

The committee also acknowledged that the appraisal process and the way in which the draft decision was communicated least year had caused significant worry for the CF community and referenced the uncaptured benefits of these treatments for people with CF and their families.

Following the meeting, NICE confirmed that no one will lose their current access to the modulator treatments and indicated that they will be liaising with key stakeholders to determine the most appropriate next steps.

We are expecting another update at the end of January and are hopeful that all parties will now work swiftly to end the uncertainty and ensure all those who could benefit from these medicines in the future can do so. 

We’ll continue to keep the CF community informed as the appraisal progresses, and we won’t stop working to make sure that everyone with CF have the treatments they need to live a long and healthy life.

If you’re feeling anxious or worried, please contact our Helpline.  

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Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects around 11,000 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.

Since 1964, we've supported people with cystic fibrosis to live longer, healthier lives - and we won’t stop until everyone can live without limits imposed by CF.