Health lobbyists are demanding that the Competition Commission reopen its investigation into the manufacturers of Trikafta, for the treatment of cystic fibrosis, which is expected to be added to the WHO’s Essential Medicines List but will be unaffordable for half of all patients in South Africa.
Last week, the SA Cystic Fibrosis Association (SACFA) and the Health Justice Initiative (HJI), handed over information to the commission, demanding it reopen its investigation into the drug’s manufacturer, Vertex Pharmaceuticals, for, among other things, abuse of dominance, excessive pricing of its product and anti-competitive conduct.
The health activists say the company’s tactics continue to place its blockbuster treatment out of reach for many SA patients, reports Business Day.
The commission abandoned its probe into Vertex in December 2024, saying at the time that it was satisfied patients could access Trikafta. The commission had initiated a complaint against Vertex in 2022 over allegedly exclusionary practices and excessive pricing for four of its cystic fibrosis drugs, including Trikafta.
In December, it said it had decided not to refer Vertex to the Competition Tribunal because it had appointed Equity Pharmaceuticals to import Trikafta “at prices that enable cystic fibrosis patients to access treatment”. Financial assistance was also available through a patient-assistance programme, it said at the time.
But only half of the 420 cystic fibrosis patients who are eligible for Trikafta are currently able to obtain it, according to the health activists. Most (203) of them belong to medical schemes administered by Discovery Health that offer the benefit on a discretionary basis.
These include Discovery Health Medical Scheme (DHMS), which is open to anyone who can afford its premiums, and some of Discovery Health’s other client schemes, which restrict membership to specific employer groups.
The number of DHMS patients using Trikafta has surged since Vertex appointed Equity Pharmaceuticals to import the drug in May 2024, rising from 27 to 197 in July 2025, according to Discovery Health CEO Ron Whelan.
However, access requires joining DHMS’ comprehensive or executive options, which are the most expensive plans it offers, and new members face a 24-month waiting period, during which they pay premiums but cannot access benefits.
The 24-month waiting period was imposed to protect DHMS from members moving to the scheme for the sole purpose of seeking access to high-cost treatments such as Trikafta, Whelan said.
Cover for medical scheme members who qualify for Trikafta is limited to R33 000 a month or R400 000 a year and the balance is covered by the Access to Innovative Care Foundation. The foundation does not disclose information about its programmes on its website and directs patients to make inquiries via their doctors.
Patients who receive financial support for Trikafta through the foundation are required to sign non-disclosure agreements, according to HJI executive director Fatima Hassan.
“Vertex is using its monopoly power to bully patients into silence while profiteering from their desperation. This is exactly the kind of abusive conduct our competition laws are designed to prevent,” she said.
Where it started
In 2014, reports The Lancet, Belinda Nell’s sister, Jen, died at the age of 32 after being comatose and connected to a ventilator in the intensive care unit for three weeks in South Africa. She died from cystic fibrosis.
Four years later, Belinda’s other sister, Lorryn, became oxygen-dependent with a lung function of just 15%. She, too, had cystic fibrosis. Her survival was precarious.
But there was hope: a new miracle drug was transforming lives in wealthy countries. However, its cost was astronomical, and it was not available in South Africa.
Nell applied to Vertex, the producer of Trikafta, for compassionate access, but the application was denied. After networking with parents of children with cystic fibrosis, medical professionals and lawyers, it was clear the only way to obtain the treatment Lorryn needed was to get a generic version of Trikafta.
Vertex does not hold a valid patent in Argentina, which has enabled a pharmaceutical company there, Gador, to produce a generic version, Trixacar, at a significantly cheaper price than the original.
However, Vertex’s patents in other countries restrict the import of the generic, which has limited its reach.
In December, 2021, Lorryn received three months’ supply of Trixacar from a mother who had travelled to Argentina to also buy the drugs for her child. But she was already in the end stages of cystic fibrosis.
“She was just starting to ‘live’ before she died in October, 2022 due to a severe lung infection and hyperammonaemia. She had just turned 35. She was robbed of a normal life,” Belinda said.
Genetic condition
The genetic disease cystic fibrosis damages the lungs, pancreas, and other organs. People with 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 build-up of thick, sticky mucus in the lungs, which can obstruct airways and lead to significant breathing difficulties.
Worldwide, around 162 428 people are estimated to have cystic fibrosis, with about 65% of those having been diagnosed. In recent years, a new class of medicines known as CFTR modulator therapies have revolutionised treatment by addressing the underlying cause of the disease.
This has significantly extended the life expectancy of patients who have access to the drugs, meaning they no longer face the likelihood of dying young and suffering frequent lung infections and hospital admissions.
Trikafta, taken as three tablets a day (elexacaftor–ivacaftor–tezacaftor) is the most powerful and widely used of Vertex’s five cystic fibrosis medications that are suitable for about 90% of patients. It has a list price of US$322 000 annually in the USA, and Vertex, which holds a monopoly on this class of medicines, recorded $11.02bn in product revenue in 2024, largely driven by Trikafta sales.
The drug is transforming lives in countries including Australia, the UK and Canada, where it has been made available largely through formal reimbursement agreements. But experts say Vertex is abusing the monopoly to maximise profits and, in doing so, has created huge global inequalities in access.
“You could treat the world for about two months of their sales,” said Andrew Hill, Senior Visiting Research Fellow in the Department of Pharmacology and Therapeutics at the University of Liverpool, UK, whose research found that the drugs could be made for about $5 000 per year.
“If you have cystic fibrosis and you live in a poor country, you’re lucky to live to 10 years old. It’s just tragic. Their chances of dying are worse than with HIV because drug companies are making those drugs available at affordable prices. There’s another way of doing things but access isn’t something Vertex seems to understand.”
A spokesperson for Vertex told The Lancet: “Our initial focus has been to achieve regulatory approval and reimbursement for our medicines in countries with the highest cystic fibrosis prevalence…our medicines (are) currently treating more than 75 000 patients worldwide, representing the vast majority of known patients diagnosed with CF who are eligible for a CFTR modulator therapy.”
Campaign triumphs in UK
At three-weeks-old, Gayle Pledger’s daughter was diagnosed with CF. By the time she was 11, she had started on a downward trajectory where her health continued to deteriorate.
Pledger helped lead a UK-wide campaign for access to Kaftrio (another name for Trikafta) after a four-year-long battle between the NHS and Vertex, which ultimately led to access for all eligible patients with cystic fibrosis. Although the deal saved her daughter’s life, she has used the experience to campaign for global access.
“Our system in the UK means we don’t pay anything – it’s incredible and shows how lucky we are that we don’t have to worry where the next box of drugs is coming from,” she said. “Other mums are going through the same thing and are completely helpless. But people together are powerful and there are things we can do.”
She has supported families across the world to challenge Vertex’s patents and push governments to take action. She has helped to secure access to Trikafta by threatening Vertex’s patents in countries including Brazil, Lithuania, Poland, and most recently, Chile.
“We know that rare diseases don’t have the patient numbers, so I realise it’s justified to have slightly higher prices for treatments, (but) we need a global access plan to allow access for all. That’s the solution,” she said. “Vertex is selling lives. You can’t just treat people in the richest countries.”
Earlier this year, Pledger travelled to the WHO’s headquarters with another campaigner to deliver a presentation to the WHO Expert Committee on the Selection and Use of Essential Medicines.
“I feel that if it’s listed on WHO's Essential Medicines List (EML), it puts the drug up the priority list and will put pressure on governments and Vertex, and may help, along the line, to get generic drugs for some countries,” she said.
WHO’s decision is due any day, Hill said, adding: “We believe it will be made an essential medicine. It’ll be a recognition that a drug needs to be used in every country where the disease exists. It’s an important milestone.”
The Vertex spokesperson said they were “working with urgency to expand access to additional countries, including lower-middle-income countries”.
The company said it has a donation programme running in 14 lower-middle-income countries, including Egypt, India, Kenya, Nepal and Uganda, where “hundreds of patients…are now regularly receiving donated medicine”.
Critics of donation programmes argue that they are not a long-term solution. “They’re totally unsustainable, there’s no transparency and no guarantees about longevity,” Pledger said. “It’s just a smokescreen.”
Transformed lives
Professor Marco Zampoli is a paediatric pulmonologist and head of the country’s largest paediatric cystic fibrosis clinic at the Red Cross War Memorial Children’s Hospital in Cape Town, which tracks about 100 children with the disease.
For about half of his patients, he has seen their lives transformed by CFTR modulator therapies; the other half are not so fortunate. “It is awful,” he said. “I see the disparity: those on treatment thriving, and the others deteriorating all because they’re poor.”
In 2023, a South African patient with cystic fibrosis took Vertex to court, requesting that a compulsory licence be granted to allow generic versions of Trikafta to be made and imported into the country.
At the time, the only way people in South Africa could access Trikafta was by travelling to Argentina – similar to how Nell’s sister obtained the generic. This was because Vertex had not sought formal registration for Trikafta with the country’s treatment watchdog or to identify a local distributor that could import unregistered Trikafta through a mechanism allowing importation of unregistered medicines.
However, in 2024, the country obtained limited access to Trikafta, with Vertex identifying a local pharmaceutical company through which Trikafta could be imported into South Africa via Section 21 authorisations and reimbursement agreements with some health insurance companies.
“So now we have a situation where half of cystic fibrosis patients with private health insurance are getting treatment, but the other half are not. Private health insurance is just too expensive. That’s why its addition to WHO’s EML would be a major milestone because there will be an obligation for national Health departments to find a way with Vertex to make the drugs available,” said Zampoli.
To try to help as many children as possible, he collects donations from patients who have surplus drugs and hands them out to those in need in combination with another readily available and affordable drug, clarithromycin.”
Memorandum and abuse
In lodging their recent appeal with the Competition Commission to re-open its investigation into Vertex, SACFA and the HJI said they had compelling evidence supporting the need for this.
Their documentation points out that about 420 of South Africa’s 505 diagnosed CF patients are clinically eligible for Trikafta, yet half still can’t access it, despite the Commission’s December 2024 claim that “access to the medication is secured”.
The memorandum, titled Access for the Privileged, presents testimony including that where financially privileged patients are getting Trikafta, it is a game-changer for their health, but they are doing so through high-end medical schemes or self-funding.
Many patients are unable to join the programme Vertex set up last year because they:
* Cannot get treatment via their medical schemes (treatment is excluded);
* Don’t belong to medical schemes;
* Cannot self-fund due to the astronomical costs; or
* Use the public sector for high-quality CF care, but because the state can’t afford to buy the treatment, they can’t benefit from it.
The submission reveals that:
* Vertex’s “access programme” effectively excludes public health sector patients and private sector patients on lower end medical schemes, unless they can also contribute about R33 333 per month themselves, towards Trikafta; and
* Many families are switching to expensive top-tier medical schemes to become eligible, then face a 24-month waiting period, delaying access further (CF is not yet a Prescribed Minimum Benefit).
Secret deals and suppressed voices
The memorandum details how Vertex's “access programme” in 2024 operates through secretive and often unenforceable undertakings, including:
* Patients and doctors must sign confidentiality clauses preventing them from speaking publicly about the programme or about the role of multiple local providers in implementing the programme.
* The programme can be terminated at any time with no legal guarantees for continued access and funding for existing and new applicants.
* Only about 13 medical schemes are included in the programme so far, and only their most expensive plans provide coverage and/or subsidised funding.
* Vertex has refused to register Trikafta with SAHPRA, avoiding certain price transparency requirements, relying for years on repeat, special, exemption procedures under Section 21 of the Medicines Act instead.
Demands for accountability
The groups are calling for state licensing, financial penalties and regulatory sanctions against Vertex, saying it has delayed access by design, and through its global and local strategy of excessive pricing, enabled gross profiteering while also failing to register its product.
The submission demonstrates that South Africa’s situation fits a global pattern of Vertex’s anti-competitive behaviour that has been documented by the Right to Breathe Campaign and others, and calls on the commission to compare Vertex’s behaviour in SA with that of other countries, where access was also unjustifiably delayed or denied.
Countries like Russia have issued compulsory licences, while Brazil, Uruguay, Turkey, and the UK have now secured public sector access after threatening similar licensing actions (with undisclosed prices).
The organisations want the Competition Commission to:
* Immediately reopen its investigation into Vertex’s anti-competitive conduct.
* Impose financial and other penalties against Vertex.
* Take urgent action to ensure affordable access for all patients and regulatory action to compel generic entries through licensing.
* Lower the price so everyone can benefit from Trikafta.
* Ensure Vertex also takes steps to register Trikafta with SAHPRA.
Competition Commission spokesperson Siyabulela Makunga said the commission’s prior intervention and initial undertakings by Vertex had resulted in significant progress to expand access to Trikafta in the past year, but in light of the new information it had received it would “reassess the issue of price transparency and further explore cost-effective access to cystic fibrosis treatment”.
It would engage with stakeholders, including the national Health Department, the Council for Medical Schemes and Vertex to determine how broader access to Trikafta could be enabled, he said.
The Lancet article – Costs prohibiting access to cystic fibrosis drug (Open access)
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