US drug manufacturer Vertex has reached an agreement with Equity Pharmaceuticals to import and distribute its cystic fibrosis treatment Trikafta in SA, but the drug’s high price tag means it is likely to remain out of reach for many eligible patients.
Only one medical scheme — Discovery Health Medical Scheme (DHMS) — now offers a benefit with cover for this class of drug, known as Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators, and even these patients may face co-payments for Trikafta.
Trikafta has transformed the treatment of cystic fibrosis, a rare genetic disorder that leads to an overproduction of mucus in patients’ lungs. But its high cost has placed Vertex at the centre of an international campaign by activists pressing it to lower the cost of the treatment, which few governments and patients can afford. The US list price of Trikafta is over $300,000 a year, and the product generated more than 90% of Vertex’s $9.87bn in sales revenue in 2023.
As Vertex has not sought to register Trikafta with the SA Health Products Regulatory Authority (Sahpra), until now the only option for SA patients was to travel to Argentina to purchase personal supplies of a cheaper generic version of the drug, branded Trixacar. The agreement with Equity means the drug can now be imported directly from Vertex on a patient-by-patient basis, using a Section 21 authorisation from Sahpra.
Vertex declined to disclose the price of Trikafta in SA, but said a “certain medical scheme will be funding a percentage of the treatment costs”. Patients can obtain assistance for co-payments from a nonprofit organisation called the Access to Innovative Care Foundation (AICF), Vertex spokesperson Daria Munsel said.
Munsel declined to answer Business Day’s question about whether Vertex provided funding to the AICF, saying only that the company worked with various organisations around the world to facilitate access, and provide other forms of support for patients and their families. “We do not list all the organisations we support,” she said.
DHMS offers a CTFR modulator benefit of up to R400,000 a year. The CEO of its administrator Discovery Health, Ron Whelan, said Vertex had “made a commitment to best endeavours on working with the available funding benefit”.
“We are hopeful that we get an affordable solution but are not clear on whether there will be a co-payment and what the extent of the co-payment will be,” Whelan said.
A total of 214 members of DHMS’s comprehensive and executive plans were registered for cystic fibrosis, 80% of whom were likely to be eligible for treatment with triple combination CFTR modulators such as Trikafta, Whelan said. The benefit remained available for patients who obtained the Argentinian generic, Trixacar, he said.
Schemes are under no legal obligation to cover costly cystic fibrosis treatments as the disease is not included in the prescribed minimum benefit package that all medical schemes must provide to all members. And while many schemes did cover treatment on an individual basis, newer treatments such as Trikafta were not provided at all in the state sector, UCT paediatric pulmonologist Marco Zampoli said.
“We will continue to advocate to access for everyone. This is a life-changing medicine: Trikafta is to cystic fibrosis what ARVs are to HIV. It is the difference between a good healthy life and death,” Zampoli said.
About 600 patients were recorded on the SA cystic fibrosis registry, but this was an underrepresentation of the true extent of the disease, he said.
“We suspect there are quite a lot of undiagnosed patients, and sadly a number of children who die at a very young age without being diagnosed,” he said. Researchers estimated only about 10% of the babies born each year with cystic fibrosis were diagnosed, he said.
Cheri Nel, the cystic fibrosis patient at the forefront of a legal challenge against Vertex’s patents in SA, said the case was continuing. “This announcement is a step in the right direction but it is not the real deal for state patients or those not on Discovery,” she said.
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