Debate on an e-petition relating to access to the drug Orkambi for people with Cystic Fibrosis

Orkambi is the brand name of a medicine (lumacaftor–ivacaftor) that is licenced to treat cystic fibrosis in patients who have a specific genetic mutation causing the disease (called the F508del mutation). In 2016, the National Institute for Health and Care Excellence (NICE) reviewed the use of Orkambi and concluded that it could not recommend it for routine use on the basis that it could not be considered cost-effective.[1]

Since this time, there have been discussions between the pharmaceutical company that makes Orkambi, Vertex, and NHS England regarding the funding of this medicine. It is not routinely funded anywhere in the UK at present.

There has been ongoing campaigning for patient access to Orkambi. A petition on the Government and Parliament Petitions website calls on the Government to bring the negotiations between Vertex, NHS England and NICE to a resolution.[2] At the time of writing, it has been signed by over 114,000 people. The Petitions Committee has considered the petition and tabled a debate on this subject for 19 March 2018. The debate will be led by Paul Scully MP.

[1] NICE, Lumacaftor–ivacaftor for treating cystic fibrosis homozygous for the F508del mutation, July 2016

[2] UK Government and Parliament petitions, Make Orkambi available on the NHS for people with Cystic Fibrosis