Life-saving drug stuck in NHS appraisal

18 May 2018

Welsh cystic fibrosis patients are not getting life-saving drugs after “staffing changes”

Campaigners are lobbying NHS Wales to stock the drug Orkambi, which could extend the lives of more than a third of those with cystic fibrosis.

But NHS Wales and Orkambi manufacturer Vertex pharmaceuticals blame each other for the problem.

Vertex told the Welsh Assembly’s petitions committee, “Disappointingly, recent staffing changes in NHS Wales Procurement have meant a meeting confirmed to take place in early April to discuss those proposals was cancelled by NHS Wales. This staffing issue has effectively halted all ongoing procurement discussions.”

But Health Minister Vaughan Gething said, “The onus is on Vertex to submit their evidence for appraisal. I am at a loss to explain why Vertex have not sent any evidence – existing or new – to AWMSG [the All Wales Medical Strategy Group] for appraisal.”

It follows a petition which was started by Rhian Barrance calling for “urgent access to the drug”.

She and her friend started the petition to help get medication for her friend’s son Lorcán.

What is Cystic Fibrosis?

Cystic Fibrosis is a genetic condition which causes mucus to build up in people’s lungs, shortening their lives.

The condition is caused by a mutation in the gene which manages the outer lining  of your lungs and stomach.

In Wales, 418 people have the condition, only half of them live past 47 years of age.

Orkambi works by targeting that genetic mutation helping

But the drug is expensive and has been known to sell for $300,000.


Vertex pharmaceuticals said “We are still in negotiations with the NHS and so do not want to comment further at this time. We are hoping for a successful resolution.”

A Welsh Government spokesperson said: “In 2016, NICE was unable to recommend the use of Orkambi as its considerable cost was not in balance with its likely benefits.

We understand AWMSG contacted Vertex on 27 April urging them to submit for reappraisal including the new clinical data they have informed the petitions committee is now available but to date no response has been received.”

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