Cystic fibrosis drug campaign makes no progress in year

Child with cystic fibrosis in a hospital bed Image copyright Getty Images

It is a year on from the delivery of more than 1,000 letters to Downing Street, many of them from children, pleading for access to a cystic fibrosis drug, called Orkambi. But there is still no decision on whether it will be made available on the NHS.

At the time, in May 2018, Prime Minister Theresa May said there was an ongoing dialogue with the drug company Vertex and she hoped for a "speedy resolution to the negotiations".

Yet, despite months of talks, no agreement has yet been reached.

More than 10,000 people in the UK have the debilitating genetic lung condition cystic fibrosis.

For about half of them, a drug called Orkambi could make a big difference - but the NHS says it is too expensive to fund.

Sir Andrew Dillon is chief executive of the National Institute for Health and Care Excellence (NICE), which last month celebrated its 20th anniversary as the body responsible for deciding which drugs offer value for money for the NHS.

He said in a recent BBC interview: "In virtually all cases we have managed to find a way through. So I am hopeful that in continuing to talk to Vertex, we can persuade them of the need to think carefully and change their expectations of what the NHS should pay so we can get these new treatments available to patients."

Orkambi has been licensed for use in the UK since 2015 - but is only available to a very small number of cystic fibrosis patients, on compassionate grounds, at the company's discretion.

The Cystic Fibrosis Trust says the drug has been shown to improve lung health by up to 42% and reduce hospitalisations by 61%.

Image caption Annabel's mum says it is "heartbreaking" her daughter cannot be treated with Orkambi

The official list price of the drug is around £105,000 per patient per year. Vertex says that, in practice, the price negotiated with healthcare systems is always lower than that.

It is available to patients in 10 countries, but NICE says the price quoted by Vertex is too high for the NHS in England.

'Clock ticking away'

Four-year-old Annabel has cystic fibrosis. Like other patients, mucus can build up in her lungs and she is vulnerable to chest infections. Her mother Liz Brennan has to organise a complex combination of treatments for Annabel every day.

She cannot understand why her daughter is unable to get Orkambi on the NHS.

She said: "It's heartbreaking. It feels like a clock ticking away - every opportunity that she could have this medication and stop the clock on her CF.

"It's scary as a parent to think what could happen. "

The issue has now got to Westminster, with demonstrations by patients and the issue raised at Prime Minister's Questions. The Health Select Committee is investigating.

There have been high-level talks between the company, NICE and NHS England, with Health Secretary Matt Hancock involved as well.

So far no conclusion has been reached.

'I've turned a corner'

There are separate negotiations with the Scottish government and its regulator the Scottish Medicines Consortium. The administrations in Wales and Northern Ireland tend to follow decisions made by NICE.

Mike Boyle is one of the few cystic fibrosis patients who is given Orkambi by the manufacturer on compassionate grounds. That is because his condition has got a lot worse.

He still has to use an inflatable vest to free up his lungs, but he says the drug has transformed his life:

"I can see how it's made a difference to me. I was desperate to get it really.

"I'm always a positive person with cystic fibrosis. I always try and fight - my saying is 'I don't let CF win'.

"I control CF, not CF control me, as best as I can.

"That's been really hard over the last two-and-a-half years really.

"But now I feel that I've turned the corner and this drug has let me have my life back again."

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