Cheri Nel has a blunt message for the multibillion-dollar pharmaceutical company Vertex: “Any person that dies from today – that’s on you.” Vertex makes a “miracle drug” called Trikafta that can transform the lives of people with cystic fibrosis.
The medication gives them a normal life expectancy, rather than facing the likelihood of dying as young adults, and lives that are no longer blighted by frequent lung infections and hospital admissions.
When US regulators first approved Trikafta in October 2019 “everyone was over-the-moon happy – it was a big celebration”, says Nel. “But then as time goes by, we realised, ‘oh shucks, this is not so easy to get’.”
Nel, 39, is bringing a lawsuit against the Boston-headquartered Vertex in her native South Africa accusing the company of patent abuse and of violating patients’ human rights under the country’s constitution.
The drug costs $326,000 (£255,000) a year for every patient, “which no South African person can afford”, she says. “Maybe not even people in first-world countries can afford that.”
Every day that goes by without medication, their lungs deteriorate further – and some of it is permanent damageCheri Nel
Vertex, which last year reported revenue of $9.87bn, primarily from its cystic fibrosis products, also faces criticism over a lack of access to the drugs or their cost in other countries.
The UK’s treatments watchdog, the National Institute for Health and Care Excellence (Nice), has said the drug is likely to be too expensive to provide value for money for the NHS. Only two middle-income countries have access to it, and no low-income countries do.
People with cystic fibrosis (CF) have genetic mutations that mean their body does not properly make or direct the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which helps salt and water move into and out of cells. This leads to a buildup of thick, sticky mucus in the lungs, making them extremely vulnerable to infection. Many need hours of daily physiotherapy to clear it; some will eventually need a lung transplant.
Trikafta, known as Kaftrio in the UK, contains three compounds designed to target and correct the CFTR protein in patients with a particular genetic mutation. It is sometimes called a triple-combination drug. A 2022 paper published in the Journal of Cystic Fibrosis found that of an estimated 162,000 people living with the condition globally, only 12% were receiving triple-combination drugs.
Under international laws, drug companies have exclusive rights to manufacture and market their patented drugs for up to 20 years. The idea is to incentivise research into new treatments by offering companies a solid chance to recoup that investment.
However, Nel’s lawsuit accuses Vertex of failing to meet a South African legal requirement to make patented medication “available to the Republic on reasonable terms”. She is seeking the creation of a “compulsory licence” for the drug, allowing generic manufacturers to produce it at a fraction of Vertex’s cost.
The company has not sought formal registration for Trikafta with South Africa’s treatment watchdog, and although patients can seek special approval to bring it in, Nel says the price is too high for that to be a real option.
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“Even if they registered, if they don’t come down significantly with the price, it is still not available on reasonable terms. They will still be guilty of patent abuse – that’s the allegation that I made in the court papers,” she says.
Nel hopes that all of the country’s 500 or so CF patients will join her legal battle as co-applicants – about 100 have already come forward expressing support. Half of the patients with the disease in South Africa are children.
Vertex has filed an 800-page response to Nel’s court submission, signalling its intent to fight on multiple grounds.
“It makes your blood boil,” says Nel, pointing out that time is not on the patients’ side.
“People need access to this medication. Every single day that goes by without the medication, their lungs deteriorate further, and some of it is permanent damage. So you need to get people to start on this medication as soon as possible in life.”
In a statement to the Guardian, a Vertex spokesperson said: “We believe in the importance of upholding intellectual property rights to drive innovation in tackling the unmet medical needs across the world.
“At Vertex, we have worked tirelessly for over 20 years to design, discover and develop CF medicines to treat the underlying cause of the disease. Patents provide the necessary incentives to drive research and development investments in areas of unmet medical needs.”
Each breath matters. I will fight for the cystic fibrosis community. They deserve to liveTanya Koorts
Since last August Nel has been taking a generic version of the drug made by a different manufacturer in Argentina, where Vertex does not have a patent.
By importing and using the drug she is “technically guilty of patent infringement”, Nel believes – but hopes the risk of Vertex suing her for that private use is low.
“It has absolutely been phenomenal,” she says. “I wake up in the mornings now [and] I don’t have a sinus headache. I don’t cough a lot any more. I don’t have to take a lot of headache medication to deal with my sinuses.
“I don’t have to be so nervous about picking up germs because it’s under control, and my lung function literally went up within 48 hours.”
The drug’s efficacy has left her with a love/hate relationship with Vertex, she says.
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“You love them for coming up with the science, and now there’s hope for people, but hate them for how they have been exploiting the situation and how they’ve been making really, really super profits.”
Nel is part of an informal buyers’ club, organised on WhatsApp, where CF patients in South Africa organise trips to Argentina to buy generic supplies. There were no direct flights until the end of last year so the journey via Dubai was a 35-hour trip.
That generic version costs about $6,000 a box, she says, which she can make last for up to two months by stretching the doses out. “It’s still a lot,” says Nel. “But it’s a little bit more affordable. It’s not a point where you have to sell all the property that you have just to get medication.”
She says she never intended “for it to become this big lawsuit” but adds that Vertex had turned down requests to allow patients in South Africa to import the cheaper generic version.
“The legal fees are astronomical,” Nel says. She is crowdfunding for her court case to recoup some of her costs.
She says she is lucky that cystic fibrosis has not limited her life as much as it does for many people. But she has to go to hospital to get treatment for infections once a month on average, typically necessitating a fortnight on antibiotics.
As the affidavits from her co-applicants arrive, she says: “I think we are going to see some terrible, heartbreaking, gut-wrenching stories. I mean, children are unable to go to school. It can make you have a completely abnormal life.”
One parent who has signed up to the lawsuit explained to Nel’s lawyers that her daughter has had to have a portion of her lung removed “and she’s six or seven”, says Nel.
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Tanya Koorts is another parent who intends to join the lawsuit. Her seven-year-old son, Janco, was diagnosed with cystic fibrosis when he was two.
“We nearly lost him. He fought so hard for every breath,” she says. “It is difficult for me to look at photos of the time of his diagnosis.”
Every morning, the first thing she would do on waking was to check he was breathing. Janco is now on a generic form of the medication from Argentina, which she describes as “a miracle”. His lung function has increased and salt levels in his sweat – a key indicator of the condition – have gone down.
“Where he used to get tired very quickly, he can now play the whole time with his friends. He’s got lots of energy,” says Koorts. “He also noticed the change in his body. He says his lungs feel lighter. I think what he is trying to say is that he can take deep breaths, which he never could do.”
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Koorts says she was motivated to join by the cystic fibrosis community. “Each breath matters,” she says. “I will fight for them. They deserve to live.”
The Vertex spokesperson said the company intended to bring its CF medicines to eligible patients in South Africa and was in the final stages of confirming access to Trikafta “on a named patient basis”, which she described as “the fastest and most efficient route to access in South Africa, given the country’s systemically challenging reimbursement system for rare diseases”.
She said Vertex had an agreement with a local distributor and was in advanced discussions with private insurers, expecting eligible patients to have access “very soon”.
The spokesperson added: “In addition to working flexibly with healthcare systems around the world, which has brought our CF portfolio to patients in over 60 countries, in 2022 we initiated a medicine-donation pilot programme for CF patients in certain lower-income countries. At present, the pilot programme will provide Trikafta at no cost to people with CF in 12 countries across four continents.”
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