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In November 2023, that process resulted in draft guidance which caused dismay among patients and their families, as it deemed the drugs too expensive to continue to provide.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