In-depth: How cystic fibrosis is being treated in SA while breakthrough meds remain out of reachCystic fibrosis (CF) is a debilitating and often deadly disease. Until recently, the only treatments for its symptoms were difficult to administer and time-consuming. IMAGE: Nick Youngson CC BY-SA 3.0 Pix4free.org

In-depth: How cystic fibrosis is being treated in SA while breakthrough meds remain out of reach

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Legal action is underway in South Africa to improve access to a game-changing new class of medicines called CFTR modulator therapies for treatment of cystic fibrosis (CF). Where available, CFTR modulator therapies—which must be taken daily as oral medicines—are transforming CF from a debilitating, life-shortening illness into a chronic, manageable condition.

While existing CFTR modulator therapies don’t work for all people with CF, they are effective for around ninety percent of CF patients. When started early, they can prevent progressive and accumulative lung damage in people with CF. And, even when started late in people with advanced lung disease, they can sometimes prevent the need for a lung transplant – a gruelling and risky procedure.

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Professor Marco Zampoli, a paediatric pulmonologist from the University of Cape Town, says that some patients overseas who have started CF modulator therapies “have been taken off the lung transplant list… you are basically rescuing people from the grave”. But he adds that it is far better to initiate CFTR modulator therapy early before accumulative lung damage occurs.

Yet, despite the acute need for these medicines, no CFTR modulator therapies have been registered for use in South Africa. The company that developed CFTR modulator therapies, Vertex Pharmaceuticals, has sought patents on these medicines in South Africa but has not pursued their registration in the country.

While Vertex Pharmaceuticals has not yet indicated what price it would charge for its CFTR modulator therapies if supplied to South Africa, in other countries the company is charging upwards of R5 million annually for the medicines, which must be taken as a life-long treatment. This price is unaffordable for the vast majority of people in South Africa and, according to researchers writing in the Journal of cystic fibrosis, far more than the cost of manufacturing the medicines.

In an effort to overcome Vertex’s patents and allow generic companies to manufacture and supply CFTR modulator therapies in South Africa at more affordable prices, a woman with CF in South Africa is taking Vertex to court. Cheri Nel, together with the South African Cystic Fibrosis Association, are seeking the granting of a compulsory license by South African courts to allow for generic CFTR modulators to be manufactured and made available in South Africa. (Spotlight previously reported on both that court case and on how CF is diagnosed in South Africa.)

Treating the symptoms of CF

In the absence of registered and reasonably priced CFTR modulators in South Africa, the only way to treat CF in the country is to prevent and treat its symptoms. (A small group of CF patients in South Africa are importing generic CFTR modulators from Argentina, but the cost of doing this is prohibitive. Read more here).

Because CF impacts many different organs and bodily functions, preventing and treating its symptoms typically requires the use of multiple different medicines and time-consuming therapies. And, because of gaps in available care from the public health sector and in coverage from private medical insurers, managing CF and its symptoms places a large financial burden on people with CF and their families.

“A lot of families do their individual fundraising. You can look on Backabuddy, you can look on GivenGain – all the South Africans are busy fundraising at the moment to cover their CF-related costs and medical expenses,” says Belinda Nell, a South African CF activist working to facilitate access to CFTR modulator therapies.

While there are many barriers to receiving optimal CF care in South Africa – including cost and long distances to treatment centres – a proactive CF community comprising patients, their families, and CF clinicians are working together closely to address these challenges.

“The parents and the patients that are involved are quite remarkable [and] the group of doctors that treat them is also reasonably small. There are probably eight to ten doctors with a special interest in treating CF in all of South Africa. So, in that way, it’s a very small community and we are plugged into patient advocacy groups,” says Professor Greg Calligaro, head of the adult CF clinic and lung transplantation services at Groote Schuur Hospital.

Where can a person with CF in South Africa get care?

In the absence of government guidelines, the South African Cystic Fibrosis Association (SACFA) has developed and published guidelines for management and treatment of cystic fibrosis across South Africa’s public and private health sectors.

Because treating CF requires specialised knowledge as well as monitoring and treatment of multiple organs and bodily functions, the guidelines recommend that all CF patients in the country receive care from a multi-disciplinary team at a specialised CF clinic.

Specialised CF clinics are located in several public (mostly academic) hospitals, private hospitals, and hospitals working in public-private partnerships in Johannesburg, Pretoria, Cape Town, Durban, Gqeberha, and Bloemfontein.

SACFA’s guidelines recommend that people with CF visit a specialised CF clinic at least three to four times a year, but acknowledge that many patients living far away from CF clinics will be unable to do this. As a minimum standard of care, the guidelines recommend that CF patients living far from CF clinics visit a specialised clinic at least once a year and that their care is shared with a local healthcare provider close to where they live.

graphic of hospitals in gauteng that provide CF care
Facilities in Gauteng that provide treatment and care for cystic fibrosis. From www.sacfa.org.za

What is the distribution of CF care across the public and private sectors?

The 2020 national CF registry reported that forty percent of people with CF in South Africa are receiving care exclusively in the public sector. Yet, for many people with CF in South Africa, their care is not exclusively located at a public or private facility but overlaps both sectors.

Patients with private medical insurance that allow them to be hospitalised in private hospitals may seek ongoing care from CF specialists working in public sector CF clinics and patients without medical insurance that predominantly use the public sector for care may buy medicines that are unavailable in the public sector out-of-pocket from private pharmacies.

“At Red Cross Children’s Hospital we have a lot of private patients who come there for outpatient care because the expertise is there, but [if they need hospitalisation] they will go to a private hospital,” says Zampoli.

facilities in WC providing CF care
From www.sacfa.org.za

SACFA’s Kimberly Keyzer explains that a lot of patients start their CF care in a public facility but when financially feasible, move to a hospital plan that allows for hospitalisation in a private facility, and then – if possible – to a more expensive, comprehensive medical aid plan that also provides coverage for CF medicines.

Tanya Koorts, the mother of a five-year-old boy with cystic fibrosis who was previously reliant on the public sector for his care, says that CF patients and their families receiving care in public hospitals face difficult conditions.

“When these kids get admitted, the people next to these children – it’s mostly mothers, aunts, grandmothers – they need to sleep on the floor,” says Koorts. “Sometimes they don’t have enough food for each day. That person needs to be emotionally and mentally taken care of as well because they are in that room 24 hours a day for two weeks.”

Koorts adds that children with cystic fibrosis need to follow a high-fat, high-calorie diet, but that public hospitals do not provide this type of food to hospitalised children with CF.

Keyzer says that after being approached for help from Charlotte Maxeke Hospital’s paediatric ward for assistance in ensuring that children with CF receive the food they need, SACFA is now providing food parcels to Charlotte Maxeke Hospital.

“We buy the food, we give it over to the doctors, and then they distribute it accordingly to the patients,” says Keyzer.

graph of number of patients with CF
From www.sacfa.org.za

A basket of medicines and interventions

As CF affects multiple organs in the body, it requires a broad range of different treatments. These treatments include medicines to help break down thick mucus that builds up in one’s lungs, medicines to prevent and treat infections, and medicines to enable the digestion of food.

Ongoing care for CF also involves daily physical therapy, typically administered by patients’ parents and family members, adherence to a high-fat, nutrient-dense diet, and daily nebulisation to open the airways and administer medication.

Access to CF medicines in the public and private health sector

Keyzer told Spotlight that, when financially feasible, many people with CF seek private medical insurance because it helps facilitate access to important CF medicines that are not available, or continuously in stock, in the public sector.

Zampoli, however, notes that people with private medical insurance are not always better off than those seeking care in the public sector – given the high costs of top-tier medical plans which cover CF medicines, and the uncovered costs or co-payments that patients with private medical insurance must often foot for high-priced CF treatment.

“It’s almost better not to be on medical aid if you have CF and you cannot afford the highest top of the range [coverage], because then you get billed for the treatments which are expensive,” says Zampoli.

Because CF is not a prescribed minimum benefit (PMB) condition – a condition that all private medical schemes in South Africa are required to provide a minimum standard of coverage for – there is no standardised level of coverage guaranteed for people with private medical insurance. As a result, CF patients are often denied coverage for essential treatments from their medical insurers, says Nell.

The consensus guidelines for management of CF published by SACFA include an extensive list of medicines that are used to treat CF.

graphic of what happens in body of person with cystic fibrosis
What happens in the body with cystic fibrosis.

Medicine to enable digestion of food

People with CF are often severely under-nourished because the sticky mucus in their digestive systems impedes the production of enzymes required to digest food. CREON is a pancreatic enzyme replacement therapy that many people with CF must take every time they eat in order to digest their food.

While CREON is available in the public sector for patients with CF, shortages of the medicine in the public sector are common, according to Nell and Keyzer. In the private sector, 100 capsules of CREON cost between R1 000 and R2 500 depending on the dosage needed.

Medicine that helps break down mucus

Many people with CF must adhere to long-term treatment to break down the sticky mucus that builds up in their lungs, impeding their lung function and increasing their susceptibility to infections.

Pulmozyme, which is inhaled using a nebuliser, was specifically developed as a mucus-thinning agent for people with CF. A month’s supply of Pulmozyme in the private sector costs around R11 700 or double that for adult patients that must use it twice per day.

Pulmozyme is not available in the public sector, but hypertonic saline solution is, which, according to Caligaro, is also effective in clearing one’s airways and thinning mucus but uses a different mechanism of action.

Medicine to ward off infections

People with CF typically require long-term, prophylactic antibiotic treatment to prevent infections to which they are particularly susceptible. One of the more common medicines used for this is tobramycin, which is inhaled twice daily using a nebuliser.

A month of tobramycin solution for inhalation in the private sector costs between R28 000 and R36 000 depending on the brand used. This special formulation of tobramycin is unavailable in the public sector and, according to Calligaro, public sector patients are typically given the intravenous form of tobramycin as an alternative “which they then break open and mix with saline to nebulise”.

He adds, however, that rarely patients cannot inhale the intravenous form of tobramycin due to hypersensitivity reactions and need access to the more expensive formulation developed specifically for inhalation.

child with nebuliser
The public health sector in South Africa does not supply public sector healthcare users with the required nebulisers for treating cystic fibrosis. PHOTO: Nurelsa Abdullah/Flickr

A critical medical device

As many of the medications used by people with CF must be inhaled via nebulisation, people with CF also require nebulisers for ongoing, at-home use. Calligaro explains that the type of nebulisers required for delivery of medication to CF patients are not the types of nebulising devices commonly sold in commercial pharmacies, but more expensive, specialist medical devices.

The public sector, however, does not supply public sector users with the required nebulisers for CF treatment. SACFA is filling this gap where it can, procuring nebulisers for CF patients that are unable to afford the devices themselves.

“It’s normally taken case by case when a doctor comes to us saying we have a patient that requires a nebuliser,” says Keyzer, adding that sometimes nebulisers get passed down from one patient to another and then, rather than a whole new device, they just need certain part replacements.

While nebulisation – or nebbing – is a critical part of ongoing CF management, the time requirements of daily nebulisation in addition to daily physical therapy, are burdensome on patients. This challenge is further compounded by loadshedding, which can interrupt the electricity supply required for nebulisation.

Availability of lung transplant operations

While medicines and physical therapy are used to protect the lungs of people with CF, the damage done to people’s lungs is accumulative. When facing life-threatening, advanced lung disease, some patients pursue a lung transplant. Lung transplant operations are available to both public and private sector patients, but access is limited.

Patients with private medical insurance can receive lung transplants from Milpark Hospital in Johannesburg or from Groote Schuur Hospital in Cape Town. Patients without private insurance who are reliant on government services can only receive lung transplants from Groote Schuur.

The geographical locations of sites providing lung transplants in South Africa, as well as low rates of organ donation in the country, are often barriers to lung transplantation in the country. Calligaro explains that lungs remain viable for transplant outside the body for only around six hours and so, practically, only people who can reach transplant sites within a few hours of organs becoming available can receive transplants.

And, as with other CF services, private medical aids won’t always pay for lung transplants. Calligaro explains that while all solid organ transplants should be covered as PMBs, private medical insurers will sometimes try to deny coverage for lung transplants, as the organ is not specifically mentioned on the PMB list.

“This is a very expensive procedure. If you can’t guarantee that everything’s going to be covered, then it becomes quite crippling,” says Calligaro.

The massive potential of CFTR modulators

If made available at affordable prices, the introduction of CFTR modulator therapies could transform the care of CF patients in South Africa. For patients that are eligible to receive these treatments, CFTR modulator therapies could help decentralise care by reducing the frequency of required visits to specialised CF clinics. The medicines would also reduce the need for ongoing, time-consuming, and expensive treatment of CF symptoms – as well as the need for lung transplants.

Yet, in order for the potential benefits of CFTR modulator therapies to be met in South Africa, they will have to be available at affordable prices, budgeted for, be consistently available from the Department of Health, and fully covered by private medical insurers.

For now, in the absence of these life-changing drugs and in the face of other ongoing shortcomings in available CF care and insurance coverage, cystic fibrosis patients, their families, and clinicians are largely left on their own to plug the gaps in available care.

This is part 3 of a Spotlight special series on cystic fibrosis in South Africa. Click here to read Part 1 and here to read Part 2.