The campaign against cystic fibrosis does not progress in the year

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

At the time, in May 2018, the Prime Minister, Theresa May, had declared that a dialogue was going on with the drug maker Vertex and that she was hoping for a "quick settlement of the negotiations".

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

In the UK, more than 10,000 people have cystic fibrosis, a debilitating genetic lung disease.

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

Sir Andrew Dillon is Executive Director of the National Institute for Health and Wellness Excellence (NICE), which celebrated the 20th anniversary of its anniversary as the body responsible for the selection of medicines offering the best value for the NHS.

In a recent interview with the BBC, he said: "In almost all cases we have managed to find a way out, so I hope that by continuing to talk to Vertex, we can convince them of the need to think carefully and change their expectations regarding the NHS should pay for us to make these new treatments available to patients. "

Orkambi has been licensed in the United Kingdom since 2015 – but it is only available for a very small number of cystic fibrosis patients, for humanitarian reasons, at the company's discretion.

The Cystic Fibrosis Trust says the drug improves lung health by almost 42% and reduces hospitalizations by 61%.

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

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

"The clock is spinning"

Annabel, four years old, has cystic fibrosis. Like other patients, mucus can build up in the lungs and is vulnerable to chest infections. Her mother Liz Brennan has to organize a complex combination of treatments for Annabel each day.

She does not understand why her daughter fails to get Orkambi in the NHS.

She said, "It's heartbreaking. It sounds like a clock – every opportunity she can have this medicine and stop the stopwatch of her FK.

"As a parent, it's scary to think about what might happen."

The question has now arrived in Westminster, with patient demonstrations and the issue raised in questions to the Prime Minister. The Health Selection Committee is investigating.

High level discussions took place between the company, NICE and NHS England, with health secretary Matt Hancock, also involved.

Until now, no conclusion has been reached.

I've turned a corner & # 39;

Separate negotiations have been initiated with the Scottish Government and its regulatory body, the Scottish Medicines Consortium. The administrations of Wales and Northern Ireland tend to follow the decisions made by NICE.

Mike Boyle is one of the few cystic fibrosis patients to receive Orkambi from the manufacturer for humanitarian reasons. This is because his condition has worsened.

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

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

"I'm still a positive person with cystic fibrosis, I always try to fight – I say," I do not let cystic fibrosis win. "

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

"It's been really difficult for two and a half years.

"But now, I feel that I've turned the corner and that this medication has allowed me to regain my life."