CF Modulator information

Over the past decade, CFTR modulator therapies have significantly transformed the management of cystic fibrosis (CF) by targeting the underlying molecular defect rather than solely treating clinical manifestations. Several modulators are now approved and have demonstrated substantial improvements in lung function, nutritional status, quality of life, and survival among individuals with eligible CFTR genotypes.

Despite these advances, access to CFTR modulators remains uneven across countries and regions. Variability persists in regulatory approval pathways, reimbursement policies, eligibility requirements, and the timing of market availability. Consequently, although a large proportion of people with CF worldwide may be eligible for modulator therapy, access in routine clinical practice is still not universal.

Access to CFTR modulators is commonly influenced by:

• National and regional health technology assessment procedures
• Reimbursement and pricing negotiations
• Eligibility restrictions based on age, disease severity, or specific CFTR variants
• Differences in availability between public and private healthcare systems

As a result, substantial inequities in access continue to exist both across and within countries. These disparities may contribute to differences in health outcomes and may also complicate the interpretation and comparison of real-world CF data between healthcare settings.

The European Cystic Fibrosis Society Patient Registry (ECFSPR) systematically collects standardized longitudinal data on people with CF from participating countries. The registry includes information on treatment exposure, including the use of CFTR modulators, together with relevant clinical outcomes. Consequently, ECFSPR data provide an important opportunity to:

• Monitor temporal trends in CFTR modulator uptake
• Characterize variations in access across countries and regions
• Evaluate real-world effectiveness and long-term population-level outcomes

By documenting treatment exposure and associated outcomes in routine clinical practice, the ECFSPR supports clinicians, researchers, and policymakers in understanding the evolving impact of CFTR modulators while also identifying persistent challenges related to equitable access to these transformative therapies.