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Three cystic fibrosis (CF) drugs have been approved for use on the NHS by the National Institute for Health and Care Excellence (NICE), after not initially recommending them in draft guidance.
In final guidance published on 24 July 2024, NICE recommended NHS use of vacaftor–tezacaftor–elexacaftor (Kaftrio) plus ivacaftor (Kalydeco), tezacaftor–ivacaftor (Symkevi) plus ivacaftor, and lumacaftor–ivacaftor (Orkambi), having reached a reimbursement agreement with the manufacturer Vertex.
NICE recommendations apply in England, Wales and Northern Ireland, and the drugs have also been approved for use within NHS Scotland, following collaboration between the Scottish Medicines Consortium (SMC) and NICE.
The drugs are CF transmembrane conductance regulator (CFTR) modulators. In clinical trials, they have been shown to improve lung function, growth and weight gain. They have also been shown to reduce the number of lung infections more than standard treatment.
The final NICE guidance states: “When considering the condition’s severity, and its effect on quality and length of life, the most likely cost-effectiveness estimates for IVA–TEZ–ELX, LUM–IVA and TEZ–IVA are within what NICE considers an acceptable use of NHS resources. So, they are recommended.”
The approval comes after a consultation on draft guidance, published in November 2023, which originally said the drugs should not be recommended for use in the NHS. A summary of comments on the consultation, published by NICE alongside the final guidance, said that the original negative recommendation “caused a huge amount of disappointment, anxiety and concern in the CF community”.
As part of the guidance development process, an external assessment group (EAG) developed its own economic model for the three drugs, which differed from that originally supplied by the manufacturer.
However, documentation published with the final guidance says: “In response to the draft guidance consultation, the company adopted an updated version of the EAG’s model. The committee concluded that the EAG’s updated model was suitable for decision making.”
In a statement prepared on 20 June 2024, Vertex announced that it had reached “an extended long-term reimbursement agreement with NHS England”.
“Vertex is working with the NHS authorities in Scotland, Wales and Northern Ireland to finalise similar access agreements as soon as possible,” it added.
Ludovic Fenaux, senior vice president at Vertex International, said that he “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”.
Commenting on the SMC recommendation, David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “[I am] 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 CF. This is a really important moment for people with CF in Scotland, and follows many years of campaigning.
“Similar deals in England, Northern Ireland and Wales were agreed [in June 2024].
“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.”
