Labour MP Ian Austin will lead a parliamentary debate tomorrow, calling on the NHS to pay for the drug. His campaign was triggered by a petition signed by 114,000 people and organised by parents of children with cystic fibrosis.
Experts believe Orkambi, which stops production of mucous that clogs sufferers’ lungs, plus a pipeline of drugs in development, could save child sufferers from life-threatening damage and allow them to have a normal life expectancy.
NHS England and the National Institute for Health and Care Excellence (Nice) said last night Orkambi, available in the Republic of Ireland, Austria, Germany and the US, was not cost effective.
But campaigners say the latest research shows the drug slows the lung damage that causes premature death.
Hannah Plowman, 20, was given the drug on compassionate grounds last November when her lung function plummeted and she was put on the transplant waiting list.
But within a week of starting treatment her lungs improved dramatically and she now no longer needs a transplant and goes to the gym four times a week. Hannah, from Poole, Dorset, said: “It’s an absolute miracle. Everyone is amazed.”
Cystic fibrosis, which affects more people in the UK than anywhere else in the world apart from the US, kills half its sufferers before they reach 31.
Sister drug Kalydeco, which is currently funded by the NHS, can be used to treat fi ve per cent of cystic fibrosis sufferers and manufacturer Vertex has offered the NHS a deal to fix the cost of Orkambi for 10 years and give patients access to newer and better drugs being developed for the same price package.
But NHS negotiators want Orkambi, which costs around £100,000 a year per patient, for no more than the total it pays for Kalydeco, even though Orkambi covers a mutation that affects 40 per cent of sufferers.
Vertex said: “We are astonished and dismayed by NHS England’s initial response to our portfolio approach. It amounts to a refusal to make any additional funding available for Orkambi or future medicines.”
Dudley North MP Mr Austin said: “I was persuaded by what I saw with my own eyes. I heard first hand from one of my constituents whose life was transformed by taking part in trials for Orkambi.
“Her hospital visits have halved, she has been able to do things the rest of us take for granted like go on holiday and has even been able to set up her own business.”
NHS England said: “Following advice from Nice, the NHS has asked this particular drug company to review its proposed pricing. Unless this happens, further progress at this time is frankly unlikely.”
Nice said: “The decision was based on the evidence and price proposal provided by Vertex at the time.
“A consideration of a review of the guidance can be brought forward where there is new evidence that is likely to impact on the current recommendations.”