In a Bid to Breathe:

Over the past year, the University of Cape Town’s (UCT) Health Economics Unit has been working closely with Breathtaking Fundraising NPC, a South African non-profit organisation dedicated to supporting people living with cystic fibrosis. Together, we are taking a significant step forward: conducting the first South African study to evaluate the real-world impact and cost of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulator therapy: a groundbreaking medication that targets the root cause of cystic fibrosis rather than just treating its symptoms.

The challenge:

Cystic fibrosis is a life-altering genetic condition that affects the lungs and digestive system. Although it is well-documented in high-income countries, its significant burden in South Africa remains under-researched. According to the 2023 South African Cystic Fibrosis Registry, just over 500 people are officially registered with cystic fibrosis across the country, but the actual number is likely much higher.

Until recently, treatment in South Africa focused primarily on managing symptoms through nebulised therapies, antibiotics, physiotherapy, and nutritional support. Still, the burden on the health system and families remains unacceptably high. However, the arrival of CFTR modulator therapies, such as Trikafta (a triple combination of elexacaftor, tezacaftor, and ivacaftor), has changed the landscape dramatically. These drugs improve lung function, reduce infections, and significantly enhance quality of life for people living with cystic fibrosis worldwide. However, the problem remains access. The extremely high cost of these medicines means that only a fraction of South African patients can benefit from them, with about half of eligible South Africans now receiving Trikafta. Even those with private medical insurance face significant financial and bureaucratic hurdles, further driving inequality in cystic fibrosis care between rich and poor. This inequality is what the UCT-Breathtaking Fundraising NPC collaboration aims to address.

The study:

The current research team comprises Dr Lucy Cunnama, Kelechi Nnene, and Jack Kline from the Health Economics Unit at UCT, Maré Smit from Breathtaking Fundraising NPC, Prof. Brenda Morrow (UCT Department of Paediatrics and Child Health), and Prof. Marco Zampoli (Paediatric Pulmonology, Red Cross War Memorial Children’s Hospital). The project aims to generate localised evidence on the costs and benefits of CFTR modulator therapy in South Africa.

The proposed mixed-methods study will combine a quantitative analysis of healthcare cost data from the funders’ perspective, along with qualitative insights from cystic fibrosis patients who are already receiving Trikafta, or its generic version, Trixacar. By bringing these together, we will flesh out quantitative cost insights with qualitative information on patients’ quality of life in order to understand both the economic and personal value of the CFTR modulators. Our proposal has been reviewed by the Departmental Research Ethics Committee and is now under review by the Health Sciences Research Ethics Committee. We are confident that this evidence will influence health policy, support advocacy, and strengthen the case for broader access to these life-changing medications.

For the benefit of all South Africans living with cystic fibrosis and their families:

While CFTR modulators have transformed care in high-income countries, access in South Africa remains a pressing challenge. People living with CF in South Africa deserve equitable access to these medicines, and the findings from this study will support advocacy for improved access. For families, the potential benefits are not just financial, but a deeply personal matter. People with cystic fibrosis who have started modulator therapy describe groundbreaking improvements in quality of life, and this positively influences their families and caregivers. 

How You Can Get Involved:

Breathtaking Fundraising NPC was created by and for the cystic fibrosis community, and this project seeks to continue that spirit of collaboration. There are several ways to participate and support this endeavour:

1. Share your story:
   People with cystic fibrosis who are taking Trikafta or Trixacar can help by contributing to Breathtaking Fundraising NPC’s de-identified registry. Your experiences help researchers understand how these therapies are changing lives.
2. Spread awareness:
   There is strength in numbers – help us reach more families, clinicians, and policymakers by sharing this blog and following Breathtaking Fundraising NPC updates on social media.
3. Support the research:
   Donations to Breathtaking Fundraising NPC directly fund awareness campaigns and community engagement, and they help ensure the study’s findings are widely disseminated.

Looking Ahead:

Our goal is not only to conduct rigorous research, but to communicate openly with donors, clinicians, patients and families. The proposed project timeline runs through early 2026, after which the team will release a health economics report. This report will be shared with the cystic fibrosis community, advocacy groups, and health policymakers. Our findings could play a critical role in shaping future access strategies for modulator therapies in South Africa.

This is a move towards health equity, and giving every person living with cystic fibrosis in South Africa the same opportunity to breathe easier and live fuller lives. As we continue this journey, we invite you to be part of the story. Together, we can ensure that scientific progress translates into real-world change for those who need it most.

Fill in this form to get onto our database if you or a loved one are living with Cystic Fibrosis. To contribute financially toward the funding for this report or ask any questions, please email projects@breathtakingnpc.co.za
Follow our progress on upcoming blog updates.