Three life-changing cystic fibrosis drugs will be made available on the NHS in England, the health watchdog has confirmed. The green light – for the treatments Kaftrio, Symkevi and Orkambi – follows years of campaigning by the Cystic Fibrosis Trust and patients. Describing it as a “really significant moment”, the charity said it offers certainty and hope to thousands of people affected by cystic fibrosis, a life-long, life-limiting condition without a cure.
The treatments were hailed around the world as a “groundbreaking” moment for patients when the drugs manufacturer Vertex Pharmaceuticals developed them. It meant that cystic fibrosis – which often causes patients to die before their 40s, as mucus clogs and damages their lungs – could be treated at the root cause for the first time, by bypassing the genetic errors responsible for the disease. But these treatments were thought to cost the NHS well over £100,000 a year per patient.
The newest of the so-called modulator drugs, Kaftrio, which has been described as a ‘miracle’ treatment for children as young as two years old living with cystic fibrosis, has proven effective for about 90 per cent of CF patients. But with more than 11,000 in the UK, it would, at list price, have cost the NHS more than £1bn a year. Last November, the National Institute for Health and Care Excellence (NICE) deemed the three drugs too expensive in its draft guidance, despite being clinically effective treatments.
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