Although US drug manufacturer Vertex has reached an agreement with Equity Pharmaceuticals to import and distribute its cystic fibrosis treatment Trikafta in SA, the drug will probably be out of reach for many eligible patients, particularly public health patients, because of its cost.
Only 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.
The rare genetic disorder cystic fibrosis leads to an overproduction of mucus in patients’ lungs, but the exorbitant cost of the medication has placed Vertex at the centre of an international campaign by activists pushing it to slash the cost of the treatment, which few governments and patients can afford.
BusinessLIVE reports that the US list price of Trikafta is more than $300 000 a year.
As Vertex has not attempted to register Trikafta with the SA Health Products Regulatory Authority (SAPHRA), until now the only option for SA patients was to travel to Argentina to buy 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 non-profit organisation called the Access to Innovative Care Foundation (AICF), Vertex spokesperson Daria Munsel said.
DHMS offers a CTFR modulator benefit of up to R400 000 a year.
“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,” said Discovery Health CEO Ron Whelan.
A total of 214 members of DHMS’ comprehensive and executive plans were registered for CF, 80% of whom were likely to be eligible for treatment with triple combination CFTR modulators such as Trikafta, he added. The benefit remained available for patients who obtained the Argentinian generic, Trixacar.
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 like Trikafta were not provided at all in the state sector, said UCT paediatric pulmonologist Marco Zampoli.
About 600 patients were recorded on the SA cystic fibrosis registry, but this was an underrepresentation of the true extent of the disease, he added.
“We suspect there are quite a lot of undiagnosed patients, and sadly, a number of children who die at very young without being diagnosed.”
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.
See more from MedicalBrief archives:
SA health NGOs join in cystic fibrosis drugs battle
Court battle looms over lifesaving cystic fibrosis drug
Discovery battles for affordable cystic fibrosis drug
Emotive campaign over £105,000 per year cystic fibrosis drug