° CMother and father of Arley Plessant were informed when she was born that she would not live until 15. Later the doctors said she could reach 30. She is now 29 and has a husband and son and everything to live for – which makes you terrified that cystic fibrosis will come to an end.
"I think about it every day and I can not do anything," she said. "Everyday I feel as if I do not control my life."
Two years ago there was a new hope. The genetic mutation that is directed to Orkambi, a new type of medicine called the CFTR modulator that works for the first time on the underlying causes of the disease rather than the symptoms, is pleasing. This is a huge breakthrough.
Orkambi presents hope for 40% of 10,400 people with cystic fibrosis in the UK who have the same genetic error as Pleasant. But they can not use it at the NHS because American producer Vertex wants more than 100,000 pounds a year for a medicine that has to be taken for life. NHS England says it's inaccessible.
People with cystic fibrosis have defective genes that stop the production of protein that regulates the movement of salt and water between the cells. It causes accumulation of mucus in the lungs, which means they are hard to breathe and are susceptible to infections.
"The winter is obviously extremely difficult," said Plezant, who woke up at night and worried that he was returning to the hospital. In October she spent three weeks in isolation, taking care of staff in dresses and gloves and masks, with intravenous lines from elbows to chest to pump her with heavy antibiotics. Cystic fibrosis patients receive multi-drug resistant infections.
"I'm not allowed to go out without being accompanied by a nurse if I meet other CF patients. We are so dangerous to be around each other. It's lonely. It's scary. "
Orkambi is not light but offers stability, she says. "I will be able to do well and become stronger instead of trying to fight the inevitable decline."
Christina Walker struggles for Orkambi for her eight-year-old son, Louis, who was again out of school this week with an infection. She calls it an "immoral and intolerable situation." She is one of the parents who signed a letter to Prime Minister and Health Secretary Matt Hancock, urging the government to overcome Vertex's patent and allow cheaper generic versions of the drug.
She is also one of those behind a letter sent to Vertex CEO Dr. Jeff Leiden earlier this week, urging him to "end the life threatening world of cystic fibrosis."
"I contacted a Polish parent, desperate for Orkambi; she told me that Vertex did not turn to the authorities in Poland, "she said.
"At the same time, I discovered that Calideco (ivacaftor, which can help only 5% of patients) and Orcambi (ivacaftor with luminacaftor) remain inaccessible in New Zealand. I know that in the US where the company is developing drugs, patients are struggling with accessibility due to the level of joint payments [the excess demanded by insurers]The situation is tragic because they are the first and only drugs that deal with the major cause of CF, extending the lives of people with a cruel, genetic disease. I see my son suffer daily, and I know he will only get worse when his illness progresses without access to modulator drugs. "
Vertex, who declined to discuss the Guardian pricing dispute, told parents that they had promised "a firm commitment to providing widespread and timely access to cystic fibrosis drugs that have already been discovered and developed in our research laboratories and new ones that are on the way ".
"We are at the heart of the global CF community. Within a few years, with continued investment and research, we hope to offer treatment that will change the lives of 90% of CF patients, "Executive Director Jeff Leiden writes. Vertex has "taken an extremely ambitious investment and research program without a guarantee of success" 20 years ago and has re-invested nearly 70% of its profits over the past five years. "However, our CF work has not been done and we need to continue investing at very high levels if we want to achieve our goal of developing such drugs for 90% of patients with CF that we think we can help.
"Vertex shares responsibility with governments and regulators to do their best to agree on a fair price that reflects the value of these medicines in all countries where they are needed … it is important that in countries where access is still inconsistent , our position is always the same – we do our best to work constructively with state authorities to find working solutions. "
In the United States, Juliana Heping has a son with cystic fibrosis, six-year-old Eli, who says she is doing very well. He is in kindergarten, loves pizza and Lego. " In the last two weeks, Eli has taken Orkambi, paid for by health insurance.
"I hope she will repel the fall that happens with cystic fibrosis," she said. She wants to get more weight and become a little taller and "recover from colds instead of needing antibiotics".
The future looks good as long as there is insurance and can meet the extra costs of these medicines, but maintaining campaigns is for many who can not afford Orkambi. – I admire the science behind these drugs. We are grateful that the company is doing so. But they brought the medicine on the market basically at the same price as a home mortgage. "
Her girlfriend, Laura Mozer of Texas, was in Orkambi, but she had to stop him because she could not afford to pay for a co-payment that required her husband's insurance. However, Mozer's parents have worked during their childhood to raise money for research that has helped finance the development of these drugs – she says, they have raised $ 750,000 in their lives.
Vertex does not pay for earliest research. Much of the money comes from donations from the Cystic Fibrosis Foundation in the US – from parents of affected children such as Mosers – and from federally funded National Health Institutes. Vertex bought the patents for $ 3.3 billion. It has already made almost that amount back from sales.
The worries about the ethics of setting such high prices were broadcast as early as 2012 when the first drug, Kalydeco, came to the market at a price of $ 294,000 (£ 257,000) a year. A team of 24 US doctors and researchers involved in drug development wrote to Vertex's Chief Executive Officer.
"We have invested our lives and careers in the success of these inspirational therapeutic agents. We also write with a sense of anxiety and disappointment that the triumph and honor that must be yours is reduced by the unreasonable price assigned to Calideco, "they say in their letter.
UK campaigns calling on the government to revoke Vertex's patent and allow cheaper generic production of these drugs claim that the company has not taken the full risk and has already made significant profits. "Until June last year, Vertex has accumulated 2.8 billion dollars in cash and cash equivalents," they said in a briefing on the government. "Analysts conservatively estimate that they will generate a $ 13 billion profit for Orkambi and another related drug, Kalydeco.
"Tens of thousands of patients in over a dozen countries around the world currently have no access to this important drug as the company maintains the highest possible price."
David Ramsden, CEO of Cystic Fibrosis Trust, said in a debate on Monday, "the government will have the opportunity to demonstrate how seriously it has considered all the options at its disposal to access drugs that change the lives of thousands of people with cystic fibrosis, and they and their families are desperate for progress and despair of everyone who is responsible for failing to make a deal.