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South Africans take on big pharmaceutical companies to gain access to ‘wonder’ drug for cystic fibrosis

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South Africans take on big pharmaceutical companies to gain access to 'wonder' drug for cystic fibrosis

VSheri Nel has a direct message to the multi-billion dollar pharmaceutical company Vertex: “Anyone who dies today – it’s your fault. » Vertex makes a “wonder drug” called Trikafta that can transform the lives of people with cystic fibrosis.

The drug gives them a normal life expectancy, rather than facing the risk of dying as young adults, and a life that is no longer blighted by frequent lung infections and hospitalizations.

When US regulators approved Trikafta for the first time in October 2019, “everyone was over the moon – it was a big party,” says Nel. “But then, as time went on, we realized, ‘oh shit, it’s not that easy to get’.”

Nel, 39, is filing a lawsuit against Vertex, which is headquartered in Boston in her native South Africa, accusing the company of patent abuse and violating patients’ human rights under the state’s constitution. country.

Drugs costs $326,000 (£255,000) per year for each patient, “something no South African can afford,” she says. “Maybe even people in industrialized countries can’t afford it. »

Vertex, which reported last year revenue of $9.87 billionmainly for its cystic fibrosis products, is also the subject of criticism regarding the lack of access to drugs or their cost in other countries.

Britain’s treatment watchdog, the National Institute for Health and Care Excellence (Nice), said the drug was probably too expensive to deliver value for money to the NHS. Only two middle-income countries have access, and no low-income countries have access.

People with cystic fibrosis (CF) have genetic mutations that mean their bodies don’t properly make or direct the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which helps salt and water move in and out. 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 get rid of 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 article published in the Journal of Cystic Fibrosis found that of approximately 162,000 people living with the disease worldwide, only 12% were receiving a triple combination of medications.

Under international laws, pharmaceutical companies have exclusive rights to manufacture and market their patented medicines for up to 20 years. The idea is to encourage research into new treatments by giving companies a solid chance of recouping that investment.

However, Nel’s lawsuit accuses Vertex of failing to meet a South African legal obligation to make patented medicines “available to the Republic on reasonable terms”. She wants the creation of a “compulsory license” for the drug, allowing generic manufacturers to produce it at a fraction of the cost of Vertex.

The company has not applied for official registration of Trikafta with South Africa’s treatment watchdog, and although patients can apply for special permission to introduce it, Nel says the price is too high. high for this to be a real option.

Cheri Nel has taken over Vertex Pharmaceuticals with the aim of introducing Trikafta, a cystic fibrosis drug, to South Africa. Photograph: Madeleine Cronjé/The Guardian

“Even if they signed up, if they don’t lower the price significantly, the product is still not available on reasonable terms. They will still be guilty of patent abuse – that’s the allegation I made in the court documents,” she says.

Nel hopes that the country’s approximately 500 cystic fibrosis patients will join his legal battle as co-plaintiffs – around 100 of whom have already come forward to express their support. Half of South Africa’s patients with the disease are children.

Vertex filed an 800-page response to Nel’s arguments in court, signaling its intent to fight on several grounds.

“It makes the blood boil,” Nel says, emphasizing that time is not on patients’ side.

“People need to have access to this medicine. With each day that passes without medication, their lungs deteriorate further, and some of this damage is permanent. So you need to get people to start taking this medication as early in their lives as possible.

In a statement to the Guardian, a Vertex spokesperson said: “We believe in the importance of respecting intellectual property rights to drive innovation and address unmet medical needs across the world.

“At Vertex, we have worked tirelessly for more than 20 years to design, discover and develop cystic fibrosis drugs to treat the underlying cause of the disease. Patents provide the incentives needed to stimulate investment in research and development in areas where medical needs are unmet.

Since last August, Nel has been taking a generic version of the drug made by another manufacturer in Germany. Argentinawhere Vertex does not have a patent.

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 this private use is low.

“It’s been absolutely phenomenal,” she says. “I wake up in the morning now (and) I don’t have sinus headaches anymore. I don’t cough much anymore. I don’t need to take a lot of headache medication to treat my sinuses.

“I don’t need to be so nervous about catching germs because it’s under control and my lung function literally improved within 48 hours.”

The drug’s effectiveness left her with a love/hate relationship with Vertex, she said.

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“You love them for inventing science, and now there is hope for people, but you hate them for how they exploited the situation and how they made really, really great profits.”

Nel is part of an informal buyers’ club, organized on WhatsApp, where cystic fibrosis patients in South Africa organize trips to Argentina to buy generic drugs. There were no direct flights until the end of last year, so the journey via Dubai took 35 hours.

This generic version costs about $6,000 a box, she says, and she can make it last up to two months by stretching out the doses. “It’s still a lot,” said Nel. “But it’s a little more affordable.” This is not the case where you have to sell all your assets just to get medicine.

She says she never intended “for this to become 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 his trial to recover part of its costs.

She says she’s lucky that cystic fibrosis hasn’t limited her life as much as it does for many people. But she has to go to the hospital to be treated for infections once a month on average, which generally requires around two weeks of antibiotics.

As the affidavits from her co-candidates come in, she says, “I think we’re going to see some terrible, heartbreaking, heartbreaking stories. I mean, kids can’t go to school. It can make you live a completely abnormal life.

A parent who signed the lawsuit told Nel’s lawyers that her daughter had to have part of her lung removed “and she’s six or seven years old,” Nel said.

Janco Koorts, now seven years old, with his mother Tanya Muller-Koorts. Janco was diagnosed with cystic fibrosis at the age of two. Photograph: Madeleine Cronjé/The Guardian

Tanya Koorts is another parent who plans to join the lawsuit. Her seven-year-old son Janco was diagnosed with cystic fibrosis when he was two years old.

“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 taking a generic form of the drug from Argentina, which it describes as “a miracle”. His lung function increased and the salt levels in his sweat – a key indicator of his illness – 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,” Koorts says. “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 breathe deeply, which he’s never been able to do.

Janco Koorts with a box containing the generic form of the drug. Photograph: Madeleine Cronjé/The Guardian

Koorts says she was motivated to join the cystic fibrosis community. “Every breath counts,” she says. “I will fight for them. They deserve to live.

The Vertex spokeswoman said the company intended to offer its cystic fibrosis drugs to eligible patients in South Africa and was in the final stages of confirming access to Trikafta “on a named patient base,” which she described as “the quickest and most efficient route to entry.” in South Africa, given the country’s consistently difficult reimbursement system for rare diseases.

She said Vertex had an agreement with a local distributor and was in advanced discussions with private insurers, hoping that eligible patients would have access “very soon.”

The spokesperson added: “In addition to working flexibly with health systems around the world, bringing our CF portfolio to patients in more than 60 countries, in 2022 we launched a pilot program drug donation for CF patients in some low-income countries. Currently, the pilot program will provide Trikafta free of charge to people with cystic fibrosis in 12 countries on four continents.

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