Carlie Pleasant’s mum and dad were told when she was born that she wouldn’t live to 15. Later, the doctors said she might make it to 30. She’s now 29 and has a husband and baby son and everything to live for – which makes her terrified that cystic fibrosis will bring it all to an end.
“I think about it every single day and there is nothing I can do,” she said. “Every day is basically feeling like I’m not in control of my life.”
Two years ago there appeared to be new hope. Pleasant has the genetic mutation that is targeted by Orkambi, a new type of drug called a CFTR modulator that for the first time works on the underlying causes of the disease, rather than the symptoms. That’s a huge breakthrough.
Orkambi represents hope for 40% of the 10,400 people with cystic fibrosis in the UK who have the same genetic fault as Pleasant. But they cannot have it on the NHS, because Vertex, the US-based manufacturer, wants more than £100,000 per patient per year for a drug that must be taken for life. NHS England says that is unaffordable.
People with cystic fibrosis have faulty genes that stop production of a protein governing the movement of salt and water between cells. It causes a build-up of mucus in the lungs, meaning it is hard for them to breathe and they are susceptible to infections.
“Winter is obviously extremely hard,” said Pleasant, who woke in the night feeling unwell and worried that she was heading back to hospital. In October she spent three weeks in isolation, cared for by staff in gowns and gloves and masks, with intravenous lines from elbow to chest to pump her full of heavy-duty antibiotics. Cystic fibrosis patients get multi-drug-resistant infections.
“I’m not allowed out without being escorted by a CF nurse in case we meet other CF patients. We are so dangerous to be around each other. It’s lonely. It’s scary.”
Are there precedents for the battle over the cost of Orkambi?
The fight for access to Orkambi – which manufacturers have set at more than £100,000 per patient per year – has much in common with the fight for the breast cancer drug Herceptin back in 2006. The underlying issues are the same – new, innovative drugs are launched at sky-high prices that pharmaceutical companies claim are necessary to recoup their costs, but healthcare providers and patients say they cannot afford.
How can a drug be so expensive?
The drugs with the highest price tags are often those for which there are fewer patients, so less profit to be made. Cystic fibrosis is a rare disease, affecting 10,400 people in the UK, where the genetic fault is most common, and about 100,000 worldwide. Drugs that cure a disease may be more expensive than those that have to be taken for many years.
How does the NHS try to control the cost of drugs?
In 2017, the NHS drugs bill was £16bn and rising at 7% a year. The NHS has actively tried to keep costs down by bringing in an appraisal mechanism, run by the National Institute for Health and Care Excellence (Nice), to ensure only drugs that are value for money are used. This has effectively capped and reduced drug prices.
Has that effort been successful in the past?
The biggest pharmaceutical companies are multinational and all of them look to world markets, making it more difficult to secure price reductions. But the NHS has been relatively successful in negotiating prices down. Cancer drug Kadcyla arrived in 2015 priced at £90,000 per patient per year. After a lot of noise and negotiation, the manufacturer, Roche, agreed an undisclosed discount for the NHS.
Orkambi is not a cure, but it offers stability, she says. “I will be able to keep well and get stronger instead of trying to fight the inevitable decline.”
Christina Walker is fighting for Orkambi for her eight-year-old son, Luis, who this week was off school again with an infection. She calls it “an immoral and unbearable situation”. She is one of the parents who have signed a letter to the prime minister and the health secretary, Matt Hancock, calling for the government to override Vertex’s patent and allow cheaper generic versions of the drug to be made.
She is also one of those behind a letter sent to the CEO of Vertex, Dr Jeff Leiden, earlier this week, calling on him to “end the threat to life from cystic fibrosis worldwide”.
“I was contacted by a Polish parent desperate for Orkambi; she told me Vertex has not applied to the authorities in Poland,” she said.
“At the same time I discovered that both Kalydeco (ivacaftor, which can help only 5% of patients) and Orkambi (ivacaftor with lumacaftor) remain unavailable in New Zealand. I know that in the US, where the company developed the drugs, patients struggle with affordability due to the level of co-pays [the excess demanded by insurers]. The situation is tragic because these are the first and only drugs to tackle the root cause of CF, extending the lives of people with the cruel, genetic condition. I watch my son suffer daily and know that he will only get worse as his disease progresses without access to modulator drugs.”
Vertex, which declined to discuss the pricing controversy with the Guardian, replied to the parents pledging its “unwavering commitment to ensuring broad and timely access to the life-changing cystic fibrosis medicines that we have already discovered and developed in our research labs and the new ones that are on their way”.
“We are at a pivotal moment for the global CF community. Within a few years, with continued investment and research, we hope to offer life-changing treatments for 90% of people with CF,” wrote CEO Jeff Leiden. Vertex had “embarked on a hugely ambitious program of investment and research, with no guarantee of success” 20 years ago and had reinvested nearly 70% of its profits in the last five years. “However, our work in CF is not done and we need to continue to invest at very high levels if we are to achieve our goal of developing such medicines for the 90% of CF patients we think we can help.
“Vertex shares the responsibility with governments and regulators to do all that we can to agree to a fair price that reflects the value of these medicines in all countries where they are needed … importantly, in the countries where access is still not agreed, our position is always the same – we are doing everything we can to work constructively with the government authorities to find workable solutions.”
In the US, Juliana Keeping has a son with cystic fibrosis, six-year-old Eli, who she says “is doing very well. He’s in kindergarten, he loves pizza and Lego”. Eli has been taking Orkambi for the last two weeks, paid for by their health insurance.
“My hope is that it staves off the decline that happens with cystic fibrosis,” she said. She wants him to gain more weight and get a little taller and “recover from colds on his own instead of needing antibiotics”.
The future looks good as long as they have insurance and can meet the extra costs of these drugs, but Keeping is campaigning for the many who cannot afford Orkambi. “I admire the science behind these medications. We are grateful that the company is making them. But they brought the drug to market basically at the same price as a mortgage on a home.”
Her friend Laura Moser, from Texas, was on Orkambi, but has had to stop it because she cannot afford the co-pay her husband’s insurance requires. Yet Moser’s parents worked throughout her childhood to raise money for research that helped fund the development of these drugs – she estimates they raised $750,000 over her lifetime.
Vertex did not pay for the earliest research. Much of the money came from donations to the Cystic Fibrosis Foundation in the US – from parents of affected children such as the Mosers – and from the federally funded National Institutes of Health. Vertex bought the patents for $3.3bn. It has already made nearly that amount back from sales.
Concerns about the ethics of setting such high prices were aired as early as 2012, when the first drug, Kalydeco, came to market at $294,000 (£257,000) a year. A group of 24 US doctors and researchers involved in the development of the drug wrote to Vertex’s CEO.
“We have invested our lives and careers toward the success of these inspiring therapeutic agents. We also write with feelings of dismay and disappointment that the triumph and honor that should be yours is diminished by the unconscionable price assigned to Kalydeco,” they said in their letter.
The UK campaigners calling on the government to set aside Vertex’s patent and allow cheaper generic production of these drugs argue that the company did not take all the risk and has already made substantial profits. “By June of last year Vertex had accrued $2.8bn in cash and cash equivalents,” they say in their briefing for the government. “Analysts conservatively estimate they will generate profits of $13bn on Orkambi and another related drug, Kalydeco, alone.
“Tens of thousands of patients in over dozens of countries around the world are currently without access to this important medicine as the company hold out for the highest possible price.”
David Ramsden, chief executive of the Cystic Fibrosis Trust, said that in Monday’s debate, “the government will have the opportunity to demonstrate how seriously it has considered all options at its disposal to achieve access to life-changing medicines for thousands of people with cystic fibrosis. They and their families are desperate for progress and despair at all those responsible for failing to deliver a deal. The wait must end!”
