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Published on: April 18, 2025
[Cystic fibrosis - A look at Belgium in 2022]
P Lebecque1, O Bauraind2, M Thimmesch1
1Service de Pneumologie pédiatrique et Mucoviscidose, CHC MontLégia, Liège, Belgique.
Insights
Cystic fibrosis care in Belgium is high quality but inconsistent across centers. Delayed access to new CFTR gene modulators, despite proven effectiveness, hinders optimal patient outcomes.
Area of Science:
- Medical Research
- Public Health Policy
- Genetics
Background:
- Cystic fibrosis (CF) care presents significant financial challenges.
- Belgium's national health system funds CF care and the Belgian Cystic Fibrosis Registry.
- Recent data provide insights into the quality of CF care in Belgium.
Purpose of the Study:
- To evaluate the current quality of care for cystic fibrosis patients in Belgium.
- To assess the utilization of symptomatic treatments and access to advanced therapies.
- To identify gaps in newborn screening and gene modulator reimbursement.
Main Methods:
- Analysis of data from the Belgian Cystic Fibrosis Registry.
- Evaluation of national health system's financial support for CF care.
- Assessment of treatment accessibility and reimbursement policies.
Main Results:
- Overall high quality of CF care in Belgium, with inter-center variability.
- Heterogeneous use of primary symptomatic treatments.
- World-leading access to lung transplantation.
- Delayed implementation of neonatal screening for CF.
- Lagging reimbursement for CFTR gene function modulators, including a highly effective triple therapy.
Conclusions:
- While CF care quality is generally high, disparities exist.
- Timely access to advanced CFTR modulator therapies is crucial but currently limited by reimbursement issues.
- The high cost and lack of reimbursement for effective triple therapy negatively impact patient outcomes.
Abstract:
Cystic fibrosis care is expensive. In Belgium, its financial support is not provided by powerful charities but by the national health system, which also sponsors the Belgian Cystic Fibrosis Registry. Recent data allow to better evaluate the quality of care for patients with cystic fibrosis in our country. Overall, it is high but varies from one centre to another. Similarly, use of the main symptomatic treatments is heterogeneous. Access to lung transplantation is one of the fluidest in the world. However, Belgium was one of the last medicalised countries to implement a neonatal screening programme for cystic fibrosis. It also lags behind in regard of the reimbursement of modulators of the CTFR gene function. This is especially detrimental for the lack of reimbursement of a recent highly effective combination of three modulators. The cost of this triple therapy is opaque and far too high. However, its effectiveness is impressive and, in the long term, around 90 % of Belgian patients with cystic fibrosis are expected to greatly benefit from it.
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