Orkambi and Cystic Fibrosis

Thank you for contacting me about the availability of Orkambi (lumacaftor-ivacaftor). I know many families affected by cystic fibrosis have placed a great deal of hope in this drug and believe particular care should be taken to assess its effectiveness.

I am aware that, following a public consultation, the National Institute for Health and Care Excellence (NICE) published final guidance in July 2016 which did not recommend Orkambi for the treatment of cystic fibrosis. NICE concluded that, compared to the current standard of care, the benefit Orkambi offered did not justify its considerable cost. However, I understand that dialogue between NHS England and Vertex - Orkambi's manufacturer - remains ongoing.

I recognise that these are very difficult decisions to make. I can assure you that NICE only publishes final guidance on the use of a drug after very careful consideration of the available evidence and after thorough consultation. The Government believes, therefore, that it would not be appropriate for Ministers to interfere in its important work.

I understand that NICE plans to review its guidance on the prescription of Orkambi for cystic fibrosis in 2019. However, I know that NHS England and Vertex are continuing to negotiate over the cost of Orkambi for cystic fibrosis and my ministerial colleagues - though unable to intervene in this process - wish to see Orkambi approved for use at an appropriate cost to the NHS.

You can read the Minister's reply to the recent debate here: https://hansard.parliament.uk/commons/2018-03-19/debates/41D2017B-E026-49FC-B94E-BCDB566AF1D6/OrkambiAndCysticFibrosis