Draft guidance from the National Institute for Health and Care Excellence (NICE) has said that three drugs used in the treatment of cystic fibrosis (CF) should not be recommended for NHS use.

The three drugs are Kaftrio (ivacaftor/tezacaftor/elexacaftor), Orkambi (lumacaftor/ivacaftor) and Symkevi (ivacaftor/tezacaftor), which are all manufactured by Vertex Pharmaceuticals.

In the draft guidance, published on 3 November 2023, NICE said that, despite the positive effects of treatment, the drugs were above the acceptable range of NHS resources in terms of cost effectiveness.

However, patients already receiving treatment with the drugs would not be affected by the new recommendation.

Kaftrio, Orkambi and Symkevi are CF transmembrane conductance regulator (CFTR) modulators, which clinical trials have 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.

In October 2019, NHS England announced that patients would have access to Orkambi, Symkevi and Kalydeco (ivacaftor, another CF drug), following an agreement with Vertex Pharmaceuticals to provide the drugs to the NHS at a reduced cost. A further agreement with Vertex to make Kaftrio available to NHS patients was struck in 2020.

In guidance published in 2016, and before NHS England’s pricing agreement with Vertex, NICE did not recommend Orkambi for treatment of CF.

Commenting on the draft guidance, David Ramsden, chief executive of the Cystic Fibrosis Trust, said: “NICE’s initial recommendation that the modulator drugs Kaftrio, Orkambi and Symkevi are highly effective for people with CF, but just too expensive to be available on the NHS, is disappointing news.

“It is important to emphasise that those already taking any of the modulator drugs are not affected by the NICE process because of the agreements already in place, but this update creates uncertainty for those not yet on treatment.

“Vertex, NICE, and the NHS must now urgently work together to find a solution to make these treatments available for all those who could potentially benefit,” he said.

According to NICE, the 2023 prices for a 28-day supply are £8,346 for Kaftrio (plus £7,000 for ivacaftor, which should be taken as part of the treatment regimen), £6,294 for Symkevi (plus the same £7,000 for ivacaftor) and £8,000 for Orkambi.

The 2022 UK Cystic Fibrosis Registry’s annual report, published in September 2023, estimated that around 7,950 people in the UK are being treated with CFTR modulators.

A spokesperson for the Department of Health and Social Care said: “CF can be a devastating illness, which is why we have invested over £1.1bn of funding into research of rare diseases.

“It is vital that patients have access to new and innovative medicines like these, but the NHS must use its budget fairly and for the good of all patients,” they added.

The draft guidance is currently open for public consultation, which is due to close on 24 November 2023. The final NICE guidance is expected to be published on 20 March 2024.

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