A new class of drugs that could extend the lives of hundreds of cystic fibrosis (CF) patients in South Africa is out of reach because Vertex Pharmaceuticals, the American manufacturer, holds intellectual property (IP) monopolies on all of them – known as CFTR modulators.
The exorbitant prices it has set on elexacaftor/ivacaftor/tezacaftor (Trikafta/Kaftrio) have led to global controversy and huge inequalities of access worldwide.
Despite patenting its products in South Africa, Vertex has not engaged with the government or Health Department to ensure access, nor has it applied to the South African Health Products Regulatory Authority (Sahpra) for regulatory approval, reports Health-e News.
“Worse – they have patented their medicines in countries they are not supplying, like South Africa, blocking patients’ access to more affordable generic versions of the drugs to protect their profits,” says Cherie Nel, a South African CF patient, who has submitted papers to the Gauteng High Court seeking a compulsory licence – a form of IP flexibility – on Trikafta. A trial date is pending.
The South African Cystic Fibrosis Association (SACFA), along with Vertex Save Us and Just Treatment (global campaigners and CF patient advocates), with guidance from Rare Diseases SA (RDSA), are all fighting back against Vertex to reduce the price of Trikafta for low-middle-income countries.
A compulsory licence will allow CF patients in South Africa access to more affordable generic versions of the drug, as several of them have to access the generic CFTRS from Argentina.
One local CF patient is six-year-old Rynah du Plessis, whose every day is a struggle to breathe. She was 18-months-old when she was diagnosed with the genetic disease that makes breathing difficult.
Rynah starts her day nebulising with hypertonic saline to help her breathe a little easier. Next: physiotherapy exercises, also to clear her lungs. Her routine is the same every morning, every evening, reports Health-e News.
She also swallows a handful of vitamins daily, and with each meal, take three enzymes to help her food digest. When she’s unwell, her routine intensifies. She nebulises up to five times daily and does physiotherapy three to four times daily. If she is very sick, she can spend up to a fortnight in hospital on IV antibiotics.
Her mother, Joelene du Plessis, says Rynah could not start school until she was around four. “She had several ear infections that would lead to chest infections and was at the doctor almost every week.”
After receiving grommets just before her first birthday, they thought her condition would improve. But it did not. “I took her to her paediatrician when she started breathing strangely. An X-ray revealed something very strange inside her right lung.”
After 10 days in the hospital, she was given a CF diagnosis, confirmed by genetic tests and a sweat test.
According to SACFA, it’s a genetic disease in which affected children inherit two copies (one from each parent) of the faulty gene that causes cystic fibrosis.
The two faulty genes together are then unable to produce a protein in the body called CFTR (CF transmembrane conductance protein), which is important for regulating water and salt balance in mucous membranes.
Thick tenacious mucous build up in their organs, especially the lungs and pancreas. Symptoms include weight loss and malnutrition, bloated stomach, tummy cramps, recurrent chest infections and a bad cough. Bacterial infections frequently attack their lungs, eventually leading to permanent scarring.
Revolutionary CF treatment
Without treatment, CF patients often die in infancy, and their life expectancy is just 20 years in South Africa. Rynah is one of about 550 diagnosed cases in the country, and there are probably many more undiagnosed.
For Rynah, affordable access to Trikafta could mean a normal life expectancy.
“She could look forward to a more normal life – fewer hospitalisations in her future, fewer tablets and treatments that fill her days as a child and even more so as a young adult. It would prevent her from needing a lung transplant. It would mean health, time, less trauma and a bright future”, says her mother.
Dr Marco Zampoli, paediatric pulmonologist and head of the CF Clinic at the Red Cross Children’s Hospital, says the average treatment costs for the desperately needed drugs, which are all-inclusive, are about R15 000 to R20 000 per month.
“Trikafta is a miracle drug. It helps the body produce a better quality CFTR protein, which reverses the effects. If started early enough, it will prevent irreversible damage to the organs, especially the lungs. It is a step towards a real cure. Instead of dying at a young age, people with cystic fibrosis can look forward to a much healthier and normal life, especially if started as children,” he says.
Astronomical costs of treatment
Before Rynah was on medical aid, there was not much more her parents could do. Obtaining medical insurance meant she could now be nebulised with pulmozyme at night too. This costs R10 000 and helps manage paediatric and adult patients with CF.
“If the compulsory licence that Cheri (Nel) filed is granted, it would mean affordable access to life-changing medication that can prevent the constant damage of this invisible disease and improve the quality of life and extend the life expectancy of my child and many other patients around South Africa,” said Du Plessis.
According to Kelly du Plessis, CEO and founder of RDSA, access to treatment in both the public and private sectors remains fragmented. “Rural patients have additional challenges in terms of referrals into tertiary centres, as well as logistical challenges such as transport costs, time off work, etc. Access to effective treatment remains difficult in both settings due to high cost and budget constraints.”
Du Plessis says there has been more awareness of CF, which ultimately will lead to increased patient numbers. “Newborn-screening efforts in the private sector have proven effective in early diagnosis, but out-of-pocket costs for this screening reduce the availability to the population at large.”
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