Cystic fibrosis medicines now available on the NHS in England

NHS England has reached a commercial agreement with Vertex Pharmaceuticals to allow NHS patients in England access to all three of their cystic fibrosis medicines.

Vertex Pharmaceuticals is the licence holder for the cystic fibrosis treatments ivacaftor/lumacaftor (Orkambi), tezacaftor/ivacaftor (Symkevi) and ivacaftor (Kalydeco). Orkambi, which was first licensed in the EU in 2015, is indicated for the treatment of cystic fibrosis in adults and children aged two years and older who are homozygous for the F508del mutation in the CFTR gene, which around 50% of people with cystic fibrosis have, amounting to approximately 5000 patients in the UK. Symkevi and Kalydeco can also be used in cystic fibrosis patients with certain other mutations in the CFTR gene.

For a long time, eligible patients have been unable to access these treatments due to NHS England being unable to reach a deal with the company on the cost of the medicines. The list price of Orkambi is approximately £104,000 per year per patient, and a 2016 NICE technology appraisal did not recommend it because it was deemed not to be a cost-effective use of NHS resources. However, following long drawn out negotiations between NICE, NHS England and Vertex, a confidential agreement on the price of all three cystic fibrosis treatments has finally been reached. An additional condition of the agreement is that Vertex will submit its full portfolio – including a forthcoming triple therapy (elexacaftor/tezacaftor/ivacaftor) – to NICE for comprehensive technology appraisal.

In September, a five-year agreement was also reached between the Scottish government and Vertex, to make Orkambi and Symkevi available to NHS patients in Scotland at a confidential discount.

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