Roadmap to Global Cystic Fibrosis Care

Cystic fibrosis (CF) is a life-limiting inherited disorder that affects approximately 160,000 people worldwide. While substantial progress has been achieved in high-income countries (HICs) through early diagnosis, multidisciplinary care, and advanced therapies, most people with CF (pwCF) living in low- and middle-income countries (LMICs) face persistent barriers to diagnosis and treatment, resulting in poor outcomes and reduced survival.

This manuscript outlines a comprehensive roadmap for advancing global CF care by highlighting the current challenges, successful models of care, and actionable strategies to bridge the gap between HICs and LMICs.

We examined the literature and global registries and analyzed outcomes from recent collaborations between CF centers in HICs and LMICs. We identified essential building blocks-including capacity building, training, integration of registries, multidisciplinary clinical care, equitable access to essential medications, and advocacy. The goal for advocacy is to increase awareness about the disease in LMICs.

Significant disparities exist in access to timely diagnosis, specialized care teams, CFTR modulator therapies, and patient registries. Collaboration efforts between HICs and LMICs focused on care team education, local adaptation of international guidelines, cross-training, telemedicine implementation, and family engagement have shown meaningful improvements in CF care in some of the LMICs. Advocacy efforts are critical in increasing access to breakthrough therapies and newborn screening in resource-limited settings.

CF is a global disease that requires a unified and equitable global response. By fostering strong partnerships, investing in workforce and system development, prioritizing equity and advocacy, the global community can substantially reduce disparities in CF outcomes. Coordinated international efforts will be crucial to ensure every person with CF, regardless of demographics, has access to quality care and the opportunity for improved survival and quality of life.