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Permanent access agreements for modulators in Scotland
Like the other deals, 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 and future license extensions issued by UK or U.S. regulators of these medicines. Any future medicines would need to follow the same appraisal process.
I’m delighted that the NHS in Scotland and Vertex have agreed a deal to ensure long-term access to life-changing modulator treatments for people with cystic fibrosis. This is a really important moment for people with cystic fibrosis in Scotland, and follows many years of campaigning. Similar deals in England, Northern Ireland and Wales were agreed last month.
David Ramsden, Chief Executive of Cystic Fibrosis Trust
While these treatments have transformed the lives of many people with CF, they don’t work for all and are not a cure. We won’t stop until everyone with CF can live a life unlimited.
Joanna Barrett, Trustee of Cystic Fibrosis Trust, from Scotland, said: "It’s fantastic news that all eligible people with cystic fibrosis in Scotland are guaranteed access to modulator therapies, now and in the future. Treatments like Kaftrio have made an enormous difference to the lives of people with CF and while there’s more to do to make sure all people with CF can live a life unlimited, this is an enormously important milestone in making that happen."
A brief history of the process
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 In November 2023, NICE’s draft guidance said that while Orkambi, Symkevi and Kaftrio were clinically effective treatments, they were too expensive at the current price for them to be able to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 announcements we have heard both last week from England, and this week from Northern Ireland and Wales.
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.