The decision to green light three treatments - Kaftrio, Symkevi and Orkambi - follows years of campaigning by the Cystic Fibrosis Trust and patients
The Cystic Fibrosis Trust described the decision as a “really significant moment” as it offered certainty and hope to thousands of people. Three life-changing cystic fibrosis drugs will be made more widely available on the NHS in England, the health watchdog has confirmed.
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.
As part of the process, NICE’s independent committee used 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, recognising the severity of CF. Any future medicines would need to follow the same NICE appraisal process.
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