cheri Nel has a blunt message for the billion-dollar company Vertex: “Anyone who dies from today – that is your responsibility.” Vertex makes a ‘miracle drug’ called Trikafta that could transform the lives of people with cystic fibrosis.
The medication gives them a normal life expectancy, rather than risking dying as young adults, and lives no longer blighted by frequent lung infections and hospital admissions.
When US regulators first approved Trikafta in October 2019 “everyone was over the moon – it was a big party,” says Nel. “But as time went on, we realized, ‘Oh, this isn’t that easy to get’.”
Nel, 39, is filing a lawsuit against Boston-based Vertex in her native South Africa, accusing the company of patent abuse and violating patients’ human rights under the country’s constitution.
The drugs costs $326,000 (£255,000) per year for each patient, “Which no South African can afford,” she says. “Maybe even people in first world countries can’t afford that.”
Vertex, which reported last year sales of $9.87 billionmainly from its products for cystic fibrosis, is also facing criticism over a lack of access to the drugs or their costs in other countries.
The UK’s treatment watchdog, the National Institute for Health and Care Excellence (Nice), has said the drug is probably too expensive to deliver value for money to 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 produce or control the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which allows salt and water to move in 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 physical therapy every day to get rid of this; some will eventually need a lung transplant.
Trikafta, known in Britain as Kaftrio, contains three compounds designed to target and correct the CFTR protein in patients with a certain genetic mutation. It is sometimes called a triple combination drug. A document from 2022 published in the Journal of Cystic Fibrosis found that of the estimated 162,000 people living with the condition worldwide, only 12% received triple combination drugs.
Under international law, pharmaceutical companies have the exclusive right to produce and market their patented drugs for 20 years. The idea is to stimulate research into new treatments by offering companies a solid opportunity to recoup that investment.
However, Nel’s lawsuit accuses Vertex of failing to comply with a South African legal obligation to make patented medicines “available to the Republic on reasonable terms”. She is seeking the introduction of a “compulsory license” for the drug, which would allow generic manufacturers to produce it at a fraction of Vertex’s cost.
The company has not applied for formal registration for Trikafta with the South African treatment watchdog, and while patients can request special permission to insert it, Nel says the price is too high for that to be a viable option.
“Even if they have registered, the price is still not available on reasonable terms, even if they have not dropped significantly. They will still be guilty of patent abuse – that is the allegation I made in the lawsuits,” 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 people have already come forward to express their support. Half of the patients with the disease in South Africa are children.
Vertex has filed an 800-page response to Nel’s lawsuit, indicating he plans to fight on multiple grounds.
“It makes your blood boil,” says Nel, pointing out that time is not on the patient’s side. The.
“People need access to this medicine. Every day that goes by without the medication, their lungs continue to deteriorate, and some of it is permanent damage. So you have to ensure that people start taking this medicine as soon as possible in their lives.”
In a statement to the Guardian, a spokesperson for Vertex said: “We believe in the importance of upholding intellectual property rights to drive innovation in addressing unmet medical needs around the world.
“At Vertex, we have worked tirelessly for over two decades to design, discover and develop CF medicines to treat the underlying cause of the disease. Patents provide the necessary incentives to stimulate research and development investments in areas of unmet medical need.”
Since last August, Nel has been using a generic version of the drug, made by another manufacturer Argentinawhich Vertex does not have a patent on.
By importing and using the drug, she is ‘technically guilty of patent infringement’, Nel believes – but hopes that the risk of Vertex suing her for that private use is small.
“It was absolutely phenomenal,” she says. “I wake up in the morning now (and) I don’t have a sinus headache. I don’t cough much anymore. I don’t have to take a lot of headache medications to treat my sinuses.
“I don’t have to be so nervous about picking up germs because it’s under control and my lung function has literally improved within 48 hours.”
The drug’s effectiveness has left her with a love-hate relationship with Vertex, she says.
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“You love them because they come up with the science, and now there is hope for people, but you hate them because of the way they exploited the situation and how they really made super profits.”
Nel is part of an informal buyers club, organized on WhatsApp, where CF patients in South Africa organize trips to Argentina to buy generic supplies. Until the end of last year there were no direct flights, making the journey via Dubai a 35-hour journey.
That generic version costs about $6,000 per box, she says, and can last up to two months by stretching out the doses. “It’s still a lot,” says Nel. “But it is a little more affordable. It’s not a point where you have to sell all the real estate you have just to get medicine.
She says she never intended “for it to become such a big lawsuit”, but adds that Vertex had rejected requests to allow patients in South Africa to import the cheaper generic version.
“The legal costs are astronomical,” says Nel. She is crowdfunding for her lawsuit to recoup some of its costs.
She says she’s lucky that cystic fibrosis hasn’t limited her life as it has for many people. But she has to go to hospital on average once a month to be treated for infections, which usually require fourteen days of antibiotics.
As the affidavits from her co-applicants come in, she says, “I think we’re going to see some horrible, gut-wrenching, gut-wrenching stories. I mean, kids can’t go to school. It can cause you to live a completely abnormal life.”
One parent who signed the lawsuit explained to Nel’s lawyers that her daughter had to have part of her lung removed “and she’s 6 or 7,” Nel says.
Tanya Koorts is another parent who wants to join the lawsuit. Her seven-year-old son Janco was diagnosed with cystic fibrosis at the age of two.
‘We almost lost him. He fought so hard for every breath,” she says. “It’s hard for me to look at photos from when he was diagnosed.”
Every morning the first thing she did when she woke up was check to see if he was breathing. Janco is now using a generic form of the drug from Argentina, which she describes as “a miracle”. His lung function has increased and the salt content in his sweat – a key indicator of the condition – has decreased.
“Where he used to get tired very quickly, he can now play with his friends all the time. He has a lot of energy,” says Koorts. “He also noticed the change in his body. He says his lungs feel lighter. I think what he’s trying to say is that he can take a deep breath, which he could never do.
Koorts says she was motivated to join the cystic fibrosis community. “Every breath is important,” she says. “I will fight for them. They deserve to live.”
The Vertex spokesperson said the company planned to bring its CF medicines to eligible patients in South Africa and was in the final stages of confirming access to Trikafta “based on the patient’s name ”, 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 gain access “very soon.”
The spokesperson added: “In addition to flexibly working with healthcare systems around the world, bringing our CF portfolio to patients in more than 60 countries, we launched a pilot program in 2022 to donate medicines for CF patients in certain lower income countries. Currently, the pilot program will offer Trikafta free of charge to people with CF in twelve countries on four continents.”