Published — 1 June, 2019


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

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, and I very much hope a positive solution can be found.

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. I believe, therefore, that it would not be appropriate for Ministers to interfere in its important work.

You may be pleased to know 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.

I am aware that a debate was held on 4th February to discuss crown use licensing and how it might be used in the case of Orkambi. I understand that my colleagues in the Department of Health and Social Care are keeping all options on the table. However, they are clear that the quickest and best solution for patients is for Vertex to accept the NHS’s offer – the largest ever offer of its kind in the history of the NHS. I and my Ministerial colleagues urge Vertex to accept this very generous offer.

Thank you again for taking the time to contact me.