What happens next with the NICE modulator appraisal?

Update on 19 December: The latest update from NICE confirms that no one will lose their current access to the modulator treatments and indicates that commercial negotiations between NHS England and Vertex are the likely next step.  We are expecting another update at the end of January and are hopeful that all parties will now work swiftly to end the uncertainty for all of those who could benefit from these medicines in the future.

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 next NICE committee meeting is on 14 December where the consultation submissions will be discussed. 

• There could be a number of different outcomes following this meeting and the appraisal could continue into 2024. We don’t expect to hear a final decision on 14 December.  

On 3 November 2023, the National Institute for Health and Care Excellence (NICE) published draft guidance on Orkambi, Symkevi and Kaftrio. It said that while they are clinically effective treatments, with important benefits for people with CF, they are currently too expensive to be able to recommend for use on the NHS.  

It is important to remember that this was draft guidance and not a final decision on whether the NHS would continue to provide these treatments. We welcome the reassurance from the NHS that all people with CF who are currently eligible can be confident about their long-term access to these life-changing drugs and that the NICE appraisal process does not affect anyone who is currently taking a modulator treatment. 

Cystic Fibrosis Trust has responded to the consultation — which closed on 24 November — highlighting experiences of people within the CF community, including those who responded to our survey earlier in the year. We made sure we told NICE how important it is that everyone who could benefit is able to access these drugs in the long term, and we were really clear that these drugs are changing the lives of people with CF. Many people within the CF community also shared their views on the impact of the modulator drugs. 

In our submission we gave the committee further experiences of people with CF to demonstrate the importance of these benefits, which include:  

• reduced treatment burden
• improved gastrointestinal symptoms  
• reduction in antibiotic use  
• increased health stability  
• improved parent and carer mental wellbeing. 

We also expressed our concern that: 

• key benefits of these medicines will not have been captured in the way NICE measure cost-effectiveness 
• the appraisal process and the way in which the draft decision was communicated caused significant worry, concern, and anxiety for the CF community 
• the way in which NICE measures the effect of transformative medicines on the quality of life for people with CF is insufficient. 

What happens next? 

The NICE committee will meet again on Thursday 14 December to discuss the responses to the draft guidance from stakeholders and the CF community. We’ve spoken to NICE, who confirmed that they have had a significant response from the CF community and that all responses will be shared with the committee.  

We don’t know what will happen after the committee meeting but at the moment we don’t expect an immediate announcement from NICE on 14 December. It may be that while discussions are ongoing behind closed doors, we might not hear much for some time. We are asking NICE for further clarity on when we can expect to hear more in 2024 and will continue to share any updates. 

While the appraisal is ongoing, people with CF can continue to start on modulators and the NICE process does not affect anyone already taking modulators. This does mean there is still some uncertainty for those who are not already on modulators, including for parents of very young children, and we will continue to do all we can to ask everyone involved to resolve this quickly and end this worry and uncertainty.  

This is an anxious and concerning time for the CF community but it’s important to emphasise that the draft decision is not a final decision from NICE.  

We are urging Vertex, NICE and the NHS to work together to find a solution and we don’t think that the community needs to take action in the form of protests or petitions as of right now. We are hopeful that there will be a swift resolution to this uncertainty. If this changes at all, we will be doing everything we can to ensure that everyone has access to life changing treatments. 

Cystic Fibrosis Trust will 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. This means that while we're working on making our voice and your voices heard in this process, we're also still focusing on important research, support, and care for everyone with cystic fibrosis. 

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

Call 0300 373 1000 or 020 3795 2184, Monday–Friday 10am–4pm 

Email [email protected] 

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