Having worked alongside the NHS for a number of years, and as Chair of the APPG for Rate Genetic & Undiagnosed Coniditions, I fully appreciate the importance of healthcare and understand the devastating effect that ill health can have on those that are afflicted and their families. As such, I do not underestimate the importance of exploring new treatments. Those affected by cystic fibrosis have placed a great deal of hope in a new drug, Orkambi, and quite rightly believe that particular care should be taken in assessing its effectiveness.
Although the preliminary recommendation is that this treatment is not cost effective, it is certainly worth exploring other means of making it available to NHS patients. I was interested to read a proposal from the Cystic Fibrosis Trust for the creation of an interim arrangement between the drug's manufacturer and the NHS and know that my colleagues in the Department of Health are aware of it.
The independent body which recommends whether particular drugs should be used, the National Institute for Health and Care Excellence (NICE), has taken the initial view that the benefits of Orkambi to lung function, a test of how people with cystic fibrosis are improving overall, are modest. However, NICE does accept that Orkambi helps with the management of the condition and is of particular benefit in reducing sudden hospitalisations. This will be taken into account as NICE begins its public consultation period before publishing further guidance.
While I know the preliminary recommendation will come as a real disappointment for many, I would encourage you to share your views and experiences with NICE as part of its public consultation on Orkambi which closes on 15th April. Details can be found here:
https://www.nice.org.uk/guidance/indevelopment/GID-TAG530/consultation/…
I think we all accept that the cost to the NHS must be taken into account when it comes to new drugs. It is, however, my hope that we will soon reach a point where cost effective 'gene specific' drugs will become available to help improve the lives of people with cystic fibrosis.
I am looking forward to the wider beenfit from the Accelerated Acess to Medicines Review that will be published soon.