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Permanent access agreements for modulators in Northern Ireland and Wales
Like the England deal, 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.
We are expecting a similar access agreement from Scotland in the coming days.
We’re delighted that Northern Ireland and Wales have now formalised agreement for permanent access for the modulator therapies, helping to ensure that everyone now, and in the future, can access these life-changing medicines.
David Ramsden, Chief Executive of Cystic Fibrosis Trust
This is an important moment, but we never forget that these medicines are not a cure, and do not work for some people and we will not stop until everyone with CF can live a life unlimited.
We hope that a similar access agreement with NHS Scotland will be agreed in the coming days.
Sophie Pierce, who has CF and is a Trustee of Cystic Fibrosis Trust, from Pembrokeshire, said: “It is fantastic news that those of us currently accessing Kaftrio and those yet to be born with cystic fibrosis will be able to continue accessing such a life changing treatment. Living with CF can be incredibly tough and so this news brings with it a sense of hope as we edge closer to a better future for those affected by CF.”
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 In November 2023, NICE’s draft guidance said that while Orkambi, Symkevi and Kaftrio were clinically effective treatments, they were too expensive at the current price for them to be able to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.