Disparities in Access to Cystic Fibrosis Therapy Across Countries

Abstract

Cystic fibrosis has been transformed by the development of CFTR modulator therapies, with substantial improvements in survival and quality of life. However, access to these therapies remains profoundly unequal worldwide. The greatest benefits have been realized in high-income countries, while people with cystic fibrosis in low- and middle-income countries and underserved populations within high-income settings continue to face limited access and poorer outcomes. Underdiagnosis is a major contributor to these disparities, as limited newborn screening, restricted access to sweat testing, and incomplete genetic characterization directly limit treatment eligibility and registry inclusion. Beyond diagnosis, disparities are driven by differences in genetic variant distribution, pricing and reimbursement policies, regulatory processes, and health system capacity. This review examines how these interrelated factors shape global access to therapies, with particular emphasis on CFTR modulators. Emerging strategies-including differential pricing, licensing mechanisms, regulatory adaptation, international collaboration, and health system strengthening-are discussed. Achieving equitable access will require coordinated action across diagnostic, economic, and policy domains to ensure that advances in cystic fibrosis care benefit patients regardless of geographic or socioeconomic context.