Johannesburg investment banker Cheri Nel has dropped a potentially landmark court case against Vertex Pharmaceutical in which she sought to secure access to lifesaving cystic fibrosis medicines, after a reduction in the price charged for the drug in South Africa.
Nel has asked the Gauteng High Court (Pretoria) to grant a compulsory licence to allow generic versions of the medicine, called Trikafta, to be imported to South Africa.
No such compulsory licences on medicines have ever been granted in South Africa, writes Catherine Tomlinson for Spotlight.
Trikafta, registered in the US in 2019, has been hailed as a “miracle” treatment for cystic fibrosis, being effective in treating around 90% of people who have the condition. It significantly improves their quality of life, 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 obtain Trikafta was by travelling to Argentina to buy it from a company there that sells a generic version.
This is because Vertex, which holds the patents on Trikafta in this country, refused to register the medicine with the SA 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 US price for Trikafta is currently more than per person per year, which South Africans feared they would also have to pay if, or when, Vertex finally started supplying its medicine here.
Researchers in the UK have estimated that Trikafta can be produced for under $6 000 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. But the Argentinian company faced potential patent infringement challenges if it shipped Trixacar to SA.
To get the medicine here, cystic fibrosis patients in South Africa would go via an informal network or Buyers Club of people worldwide who 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 here.
If successful, her case would have allowed generic Trikafta to be shipped directly to South Africa, removing the need to go all the way to Argentina for it.
In its response, Vertex said that as she was the only named applicant in the case, a compulsory licence for importation could only be considered for her.
Nel then worked with the SA Cystic Fibrosis Association (SACFA) to get other people afflicted with the disease to be admitted as co-applicants. 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.
Vertex starts providing Trikafta in SA
As the case gained momentum and made global headlines, Vertex finally opened the door to allow some South Africans to access its product.
In May 2024, Vertex identified Equity Pharmaceuticals as the local company through which Trikafta could be imported to SA via Section 21 authorisations. These are granted by SAHPRA to enable importation of an unregistered medicine 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 South Africa, Nel and Candice Sehoma from Doctors Without Borders said the company was charging $22 000 for a year’s supply of the drug.
While still unaffordable for many, and much higher than the estimated cost of manufacturing, this is drastically lower than what is charged in the United States and what was originally feared for South Africa.
It seems improbable that Vertex would have offered the much reduced price to people in South Africa had Nel not launched the court case.
Some 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 spokesperson Daria Munsel confirmed to Spotlight.
The exact agreements between Vertex and medical schemes in South Africa, however, remain somewhat unclear.
Discovery Health CEO Dr Ron Whelan told Spotlight it had discussed with Vertex the “benefits available” and “affordable access” of the class of medications under which Trikafta falls, but there is “no specific commercial agreement in place”.
Discovery Health Medical Scheme members on the comprehensive and executive plans have a suite of benefits available for cystic fibrosis treatment, and medicines like Trikafta, “of up to R400 000 per annum”, he added.
Vertex said uptake has been swift.
“More than 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 that identifies and collect data on the outcomes of cystic fibrosis patients in South Africa, identified 525 people 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 South Africans now lower than the 2023 price of Argentinian generics, but the cost of a year’s supply of that South American generic has 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 her legal case was no longer necessary. The aim of the case “was to get access to the medication… to put pills in patients’ mouths”, she told Spotlight.
She said it was now probably better to redirect efforts to getting the government at national or provincial levels to buy the medicine for patients in the public sector.
“There is still work to be done… my efforts …are just being redirected.”
Why won't Vertex register its product in SA?
While much has changed because of Nel’s legal action, Vertex has stuck by its refusal to register Trikafta with SAPHRA.
On plans to register Trikafta in South Africa, Munsel said: “We strongly believe that this (Section 21 authorisation) 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 are more closely scrutinised than is the case with Section 21 authorisations.
Nel believes Vertex has chosen not to register Trikafta in this country because of the price transparency requirements embedded in South African law. If other countries know what price South Africa is paying, they may also demand a lower price, she said.
The law requires 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.
See more from MedicalBrief archives:
SA to import costly cystic fibrosis drug, but few can afford it
Court battle looms over lifesaving cystic fibrosis drug