NICE modulator appraisal

NICE modulator appraisal

Kaftrio, Orkambi and Symkevi have been permanently approved on the NHS in England, Wales, Scotland and Northern Ireland.

The NHS in England, Wales, Scotland and Northern Ireland have agreed long-term access to CF modulator drugs Kaftrio, Symkevia and Orkambi.

This follows the conclusion of commercial deals between NHS England, Wales, Scotland and Northern Ireland and the pharmaceutical company Vertex.

This information was updated on 31 July 2024.

What is the latest update?
The National Institute for Health and Care Excellence (NICE) has issued a ‘positive recommendation’ for CFTR modulators. This means the life-changing cystic fibrosis (CF) modulator drugs will be permanently made available on the NHS in England, Wales, Scotland and Northern Ireland.
This agreement includes:
- Kaftrio® (ivacaftor/tezacaftor/elexacaftor)
- Symkevi® (tezacaftor/ivacaftor)
- Orkambi® (lumacaftor/ivacaftor).
There is also a commitment by all parties to work together towards rapid access for all eligible patients for future treatments for CF. Any future medicines will be assessed by NICE.
This agreement includes both people already on modulators and those who become eligible to start modulators in future.
Is this the same across all four nations of the UK?

The NHS has confirmed that cystic fibrosis (CF) modulator drugs will be permanently made available on the NHS in England, Wales and Northern Ireland.
On July 22, we received confirmation that agreements between NHS Scotland and Vertex have also been finalised. The agreement is comparable to the one in England, Northern Ireland and Wales, and will provide access to modulator therapies for all existing and future eligible CF patients in Scotland.
What is the situation in the Crown Dependences (Guernsey, Jersey and the Isle of Man)?

The States of Guernsey Committee for Health and Social Care has confirmed that the modulators Kaftrio, Symkevi, and Orkambi, will be permanently available for eligible adults and children with CF in Guernsey.

We have had a response from the Isle of Man who are conducting a wider review process of all NICE-approved treatments and the Department of Health and Social Care have requested a costed plan by the end of the summer.

We have written to the relevant authorities regarding the situation in Jersey and they've told us they will respond in due course.
What about people with rare mutations not covered by this deal?

There is evidence that a small number of people with CF with rare mutations that are not currently eligible may benefit from trying a modulator.
These people are not covered by the deal, but NHS England have stated that they want to explore ways for anyone who might benefit from modulators to have access, if their CF care team recommends this.
We will continue to work with all stakeholders to ensure that this happens as quickly as possible.
The new agreement also means that patient access will expand as new licensing extensions are approved by either the MHRA in the UK or FDA in the United States, ensuring access in the UK can keep up with emerging evidence from the US.
What does this mean for very young children who are not yet old enough to be on modulators?

This agreement covers current and future access to modulators. Once they are old enough, children will receive modulators according to the eligibility criteria, if recommended by their CF care team.
I’ve got questions, who can help?

If you have any questions about eligibility for modulators or how this update affects your CF treatment, please speak to your CF team.
If you have questions or concerns, we are here to support you. Please contact our Helpline.
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