Patients with cystic fibrosis (CF) will now have continued access to crucial drugs to treat their life-limiting condition. The National Institute for Health and Care Excellence (NICE) has given approval for the modulator drugs – Kaftrio, Symkevi and Orkambi - to be made available at a fair price on the NHS, a reversal of a previous decision by the health regulatory body to halt access to the drugs.
The move follows a dispute about the price being charged by Vertex, the pharmaceutical company that makes the treatments. NICE had earlier said that despite transforming patients' lives, the medicines ‘were too costly for any new or future CF patients to have’.
NHS England’s director of specialised commissioning, John Stewart, said: ‘The NHS has a strong track record of offering people with cystic fibrosis life-changing treatments with 8,000 people now benefitting – and today’s deal gives families security knowing that they will be guaranteed access to these drugs for years to come.’
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CF is a life-limiting genetic condition that clogs the lungs with thick mucus and can cause patients to die before reaching their 40s. There are over 11,000 people with the condition in the UK.
In 2019, the modulator drugs became available to treat the condition by bypassing the genetic errors responsible for the disease. But in 2023, NICE deemed the drugs too expensive to continue providing as it was estimated to cost the NHS over £100,000 a year per patient.
After a series of negotiations between NICE’s independent committee and Vertex, a fair price has been agreed on for the NHS and taxpayers. According to NICE, the parties also committed to work together to ensure rapid access for all eligible patients for future treatments for the condition.
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: ‘This is a fantastic moment for many people with cystic fibrosis and their families – ending uncertainty and helping to ensure that everyone who can benefit is able to access these vital medicines – now and in the future.’