Access to cystic fibrosis drugs approved in England

By Catherine Snowdon, Health reporter

Getty X-ray showing mucus in the lungsGetty

Cystic fibrosis causes thick mucus to build up in the lungs

A deal has been reached to provide NHS patients with continued access to crucial drugs that treat the life-limiting condition cystic fibrosis.

Officials had previously disputed the price being charged by Vertex, the pharmaceutical company that makes the treatments.

After a period of negotiation, the National Institute for Health and Care Excellence (NICE) has now given approval for the modulator drugs – Kaftrio, Symkevi and Orkambi – to be made available on the NHS in England.

It is thought patients in Wales, Scotland and Northern Ireland will soon see similar agreements announced.

Cystic Fibrosis can cause patients to die before reaching their 40s, as mucus clogs and damages their lungs. The modulator drugs are revolutionary in that they treat the root cause, by bypassing the genetic errors responsible for the disease.

It is thought about 11,000 people have the condition in the UK.

Updated method

In 2020, an interim deal was reached between NHS England and Vertex, which was followed by deals in Wales, Scotland, and Northern Ireland. This temporary agreement included the need for a formal appraisal by NICE.

In November 2023, that process resulted in draft guidance which caused dismay among patients and their families, as it deemed the drugs too expensive to continue to provide.

The treatments are thought to cost the NHS well over £100,000 a year per patient.

Negotiations ensued with NICE’s independent committee using 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, and allowed the committee to pay more for the drugs.

The deal means all existing and future eligible cystic fibrosis patients should be able to access the treatments.

‘Fair price’

Ludovic Fenaux, senior vice-president at Vertex International, said the company was “delighted” and thanked patients for “their contribution in describing the value that these innovative medicines bring to patients”.

The deal also includes a commitment by all parties to work together on a path towards rapid access for all eligible patients for future treatments for the condition.

A spokesperson for NICE said the organisation “is grateful to NHS England, Vertex and the patient groups for working so tirelessly to provide the right evidence to allow a fair price to be agreed” for the NHS and taxpayers.

David Ramsden, chief executive of Cystic Fibrosis Trust, called the deal a “fantastic moment”.

“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,” he added.

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