Last year a South African woman took a multibillion-dollar United States pharmaceutical company to court with the aim of securing access to life-changing cystic fibrosis medicines. That case has now been dropped following a reduction in the price charged for the medicines in South Africa.
Cheri Nel, a Johannesburg-based investment banker, has dropped a potentially landmark court case against Vertex Pharmaceuticals. Nel was asking the Gauteng Division of the High Court in Pretoria to grant a compulsory license to allow generic versions of a cystic fibrosis medicine called Trikafta to be imported into South Africa. No such compulsory licenses on medicines have ever been granted in South Africa.
Trikafta, which was registered in the United States in 2019, has been hailed as a "miracle" treatment for cystic fibrosis, which causes severe damage to the lungs, digestive system and other organs in the body. The medicine is effective in treating around 90 percent of people living with the condition. It significantly improves the quality of life of people living with cystic fibrosis, eliminating many of its debilitating symptoms, while also slowing the disease's progression and extending survival.
In February 2023, when Nel launched her lawsuit against the Boston-headquartered pharmaceutical company, the only way people in South Africa could access Trikafta was by travelling to Argentina to buy it from an Argentinian company selling a generic version of the medicine.
This is because Vertex, the company that holds the patents on Trikafta in South Africa, refused to register the medicine with the South African Health Products Regulatory Authority (SAHPRA) or identify a local distributor that could import unregistered Trikafta via Section 21 authorisations - a mechanism allowing importation of unregistered medicines.
The United States list price for Trikafta is currently over $300 000 (around R5.5 million at the current rand/dollar exchange rate) per person per year, which South Africans feared they would also have to pay if or when Vertex finally started supplying its medicine in the country. Researchers in the United Kingdom have estimated that Trikafta can be produced for under $6 000 (around R110 000 at the current rand/dollar exchange rate) per person per year.
When Nel filed the case, generic Trikafta from Argentina - called Trixacar - was much cheaper than Vertex's product (but still prohibitively expensive for many) at around $60 000, or almost R1 million per person per year. But the Argentinian company selling generic Trixacar faced potential patent infringement challenges if it shipped Trixacar to South Africa. Thus, the only way to get the medicine into South Africa at the time was to travel to Argentina to collect it. People living with cystic fibrosis in South Africa learnt how to do this through an informal network or Buyers Club of people around the world that were reliant on the Argentinian product.
Launching a legal case
Nel argued that Vertex was abusing its patents in South Africa by refusing to make Trikafta available in the country on reasonable terms, while also blocking other manufacturers from supplying the medicine in the country. If successful, Nel's case would have allowed generic Trikafta to be shipped directly to South Africa, removing the need for travel to Argentina to access the medicine.
According to Nel, Vertex argued in the company's answering documents to her legal filing that, as she was the only named applicant in the case, a compulsory license for importation could only be considered for her.
Nel then worked with the South African Cystic Fibrosis Association (SACFA) to get other people living with cystic fibrosis admitted as co-applicants in the case. This process of seeking more people to join her case, she said, was time-consuming, difficult, and expensive, but more than 100 people were working towards being admitted as co-applicants before the case was dropped.
Under pressure, Vertex starts providing Trikafta in South Africa
As the case gained momentum and made headlines around the world, Vertex finally opened the door to allow some people living in South Africa to access their product.
In May 2024, Vertex identified Equity Pharmaceuticals as the local company through which Trikafta could be imported into South Africa via Section 21 authorisations. These authorisations are granted by SAHPRA to enable importation of an unregistered medicine and are meant to be used in exceptional circumstances to remedy the need for an unregistered medicine, such as when there is a shortage of the registered product.
While Vertex has not confirmed to Spotlight or stated publicly the price of Trikafta for people living in South Africa, Nel and Doctors Without Borders' Candice Sehoma told us that the company is charging around R400 000 ($22 000) for a year's supply of the medicine.
While still unaffordable for many and much higher than the estimated cost of manufacturing, the R400 000 price is drastically lower than the R5.5 million price charged in the United States and originally feared for South Africa.
It seems improbable that Vertex would have offered the much reduced price to people living in South Africa had Nel not launched the court case
Some medical schemes now paying for Trikafta
As emerged in April this year, Vertex reached an agreement with some medical schemes in South Africa to provide the medicine for people on top-end plans.
"Four private healthcare providers are currently funding Trikafta for eligible patients and we are open for conversations with more insurance companies," Vertex's spokesperson Daria Munsel confirmed to Spotlight.
The exact nature of the conversations and/or agreements between Vertex and medical schemes in South Africa however remains somewhat unclear.
Discovery Health's CEO, Dr Ron Whelan, told Spotlight it has engaged Vertex about the "benefits available" and "affordable access" of the class of medications that Trikafta falls in but there is "no specific commercial agreement in place" in South Africa.
He noted that Discovery Health Medical Scheme members on the comprehensive and executive plans have a suite of benefits available for the treatment of cystic fibrosis with medicines like Trikafta "of up to R400 000 per annum" for eligible people.
According to Vertex, uptake of its product has been swift and is already starting to make a difference in the lives of people living with cystic fibrosis in South Africa. "Over 100 South Africans with CF [cystic fibrosis] have been prescribed our triple combination treatment in just the first two months of the medicine being available," said Munsel.
The cystic fibrosis registry, an initiative which seeks to identify and collect data on the outcomes of people living with cystic fibrosis in South Africa, identified 525 people living with cystic fibrosis in the country as of December 2020. Experts believe there are many more undiagnosed cases.
Why did Nel drop the case?
Not only is Vertex's price for people in South Africa now lower than the 2023 price of Argentinian generics, but the cost of a year's supply of generic Trikafta from Argentina have increased from around $60 000 to around $100 000 due to hyperinflation in that country.
With Vertex now offering a price lower than the cost of Argentinian generics, Nel decided that her legal case was no longer the best avenue to enhance access to the medicine. The aim of the case "was to get access to the medication... to put pills in patients' mouths", she told Spotlight.
Nel said it is now probably better to redirect efforts to getting government at national or provincial levels to buy the medicine for patients in the public sector.
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"There is a lot of work still to be done... my efforts are still there, it's just being redirected," she said.
"The fact that Trikafta will now be available in South Africa at a much lower price compared to generic versions globally, certainly undercuts the legal case for a compulsory license," said Tendai Mafuma of SECTION27, a public interest law centre. The Treatment Action Campaign and Doctors Without Borders, represented by SECTION27, were admitted as friends of the court in the case.
Why won't Vertex register its product in SA?
While much has changed because of Nel's legal action, Vertex has held fast on its refusal to register Trikafta with SAHPRA.
When asked about Vertex's plans to register Trikafta in South Africa, Munsel said: "We strongly believe that this [Section 21 Authorization] is the fastest and most efficient route to sustainable access in South Africa, which does not require a regulatory filing."
While registering medicines can be onerous and time consuming, it is a routine practice required for pharmaceutical companies to operate around the world. Full registration also typically requires that safety, effectiveness and quality is more closely scrutinised than is the case with Section 21 authorisations.
Nel believes that Vertex has chosen not to register Trikafta in South Africa because of the price transparency requirements embedded in South African law. If other countries know what price South Africa is paying then they may also demand a lower price, she said.
The law requires that there is a transparent pricing system for medicines sold in the private sector, but these requirements do not extend to unregistered medicines imported through Section 21 authorisations, explained Mafuma.
Note: SECTION27 was involved in the court case that is the subject of this article. Spotlight is published by SECTION27, but is editorially independent - and independence that the editors guard jealously. Spotlight is a member of the South African Press Council.
