NICE approve permanent access to life changing cystic fibrosis drugs
This is a really significant moment as it offers certainty and hope to thousands of people affected by CF, a life-long, life-limiting condition without a cure.
In 2020, a landmark interim deal was reached between NHS England and the pharmaceutical company Vertex, which was rapidly followed by deals in Wales, Scotland, and Northern Ireland. As part of this deal, the UK Cystic Fibrosis Registry, in partnership with NHS England, NICE and Vertex collected real-world evidence, looking at the effectiveness of modulator therapies ahead of an appraisal from NICE.
In 2022 the formal process began, with many people from the CF community sharing their experiences of the medicines. In November 2023, NICE’s draft guidance said that while Orkambi, Symkevi and Kaftrio were clinically effective treatments, they were too expensive at the current price for them to be able to recommend them for use on the NHS.
That led to further discussions and negotiations and the announcement from NICE today follows the conclusion of a deal between NHS England and Vertex, which includes future license extensions issued by UK or U.S. regulators of these medicines.
As part of the process NICE’s independent committee used their updated method of appraising medicines, which gives extra weight to health benefits for treating more severe diseases like cystic fibrosis. It is one of the first times this has been used for non-cancer medicines, recognising the severity of CF.
It also includes a commitment by all parties to work together on a path towards rapid access for all eligible patients for future treatments for CF. Any future medicines would need to follow the same NICE appraisal process.
We know that Vertex is also working with the NHS authorities in Scotland, Wales and Northern Ireland and expect similar access agreements to be formalised in the coming days.
This is a fantastic moment for many people with cystic fibrosis and their families – ending uncertainty and helping to ensure that everyone who can benefit is able to access these vital medicines – now and in the future.
David Ramsden, Chief Executive of Cystic Fibrosis Trust
We should not forget though that these treatments are not a cure and simply don’t work for some people. With the support of our incredible community, clinicians, and researchers a lot has been achieved but we know there is still lots more to do.
Ludovic Fenaux, Senior Vice President, Vertex International, said, “We are delighted to have agreed extended long-term access to KAFTRIO® (ivacaftor/tezacaftor/elexacaftor), SYMKEVI® (tezacaftor/ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) for eligible CF patients in England.”
“I would like to acknowledge the collaboration of NHS England, NICE and the SMC, and also thank the CF community for their contribution in describing the value that these innovative medicines bring to patients.”
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