Scandal of Cystic Fibrosis Medications: "It's made us our lives"

Sam Deakin, 28, was virtually confined to the home before being prescribed to Orkambi in November 2017 and was sometimes struggling to stay upright. She was in the hospital and on the list for a lung transplant. Now she has regained strength and is even able to do pole dance at the gym.

The Nottingham barmaid said, "When I get an infection before I start a treatment, it quickly becomes dangerous and even deadly.

"I often went to the hospital, on intravenous antibiotics and oxygen. I stayed at home with my two dogs because I was not good enough to go out.

"Three days ago, pictures of my phone appeared on my phone a year ago, I was crying and I had to sit down to prepare my dinner because I did not have the necessary energy to get up, now I'm fit again, I can walk dogs.

"It was a lifeline and I had no life before," she said.

Sam wants other patients with a severe genetic lung disorder to understand it.

Now, the National Institute for Excellence in Health and Care has not approved Orkambi, which costs the NHS £ 105,000 for each patient. It is distributed only for humanitarian reasons.

However, many countries in Europe, including the Netherlands, Germany, Italy, Austria, France and the Republic of Ireland, routinely prescribe this drug .

The fact that the national NICE did not approve it – for reasons of profitability and the absence of long-term data – gave rise to a bitter dispute with Orkambi's manufacturer, Vertex.

It is estimated that this drug could help thousands of people with cystic fibrosis in the UK.

But providing Orkambi at the price of Vertex would cost the NHS about £ 540 million a year. The NHS offers a fifth of what Vertex charges.

In another case, 15-year-old Joe Barnes, who was about to die, improved a lot with the drug.

Joe, who participates in GCSEs this year, saw his weight drop to four and a half stones. But he took seven pounds in seven months, he was prescribed Orkambi.

His mother, Lorraine, is now campaigning for people with the disease to be systematically treated.

Lorraine, of Bothamsall, Nottinghamshire, who has an older son with CF, said, "Joe is in school to take exams, and last year he could not manage a full day of illness.

"It is essential that this drug be approved for people like Joe, who are struggling to live a normal life – too many lives have been lost in the meantime."

An NHS England spokeswoman said: "The Vertex groups are extremely extreme in terms of price and behavior, and families are asking this company to make Orkambi affordable and accessible."

Of the 10,400 British families affected by cystic fibrosis, many have signed a petition calling Vertex and the NHS to sign an agreement.