An Analysis of Cystic Fibrosis Care Across the Globe
Introduction
Cystic fibrosis (CF) is a genetic disorder that affects various organ systems, with the most significant impact on the lungs and digestive system. It is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This essay aims to provide a comprehensive analysis of the global perspectives on CF care, focusing on the disparities in care across different countries and regions.
Global Perspectives on Cystic Fibrosis Care
The future of CF care is being shaped by a global perspective that focuses on five key areas:
Changing Epidemiology of Cystic Fibrosis: The demographics of CF patients are changing, with more adults than children now living with the disease. This shift has implications for the types of care and support that patients need.
Future Challenges of Clinical Care and Its Delivery: As treatments improve and people with CF live longer, new challenges are emerging in the delivery of care. These include the need for more adult services, mental health support, and care for complex, multi-system disease.
Building of Cystic Fibrosis Care Globally: There is a need to build CF care capacity in countries where the disease is less common or newly recognized. This includes training healthcare professionals, establishing patient registries, and advocating for access to therapies1.
Novel Therapeutics: The development of new therapies, particularly modulator therapies that target the underlying genetic defect in CF, is transforming patient outcomes. However, these therapies are expensive and not yet available to all patients.
Patient Engagement: Engaging patients in their own care is a key aspect of improving outcomes. This includes shared decision-making, self-management education, and psychosocial support.
Disparities in Cystic Fibrosis Care
The quality and accessibility of CF care vary significantly across the globe. In countries where health expenditure per capita is low, patients with CF experience more deprivation-related health disparities than all other individuals in the population. Children with CF from more disadvantaged populations show worse lung function, more impaired growth, and higher rates of mortality than children from more affluent areas.
In countries with a high gross domestic product, most patients with CF are now aged 18 years, whereas in countries not in the EU, the average age of patients is much lower. This disparity is likely due to suboptimal therapy. Data from the European Cystic Fibrosis Society Patient Registry showed significant differences in the clinical status of patients with CF across European countries. These disparities in disease outcome result from delayed diagnosis, lack of specialized care, and limited access to medications.
Regional Differences in Cystic Fibrosis Care
In the United States, there is a nationwide network of more than 130 Cystic Fibrosis Foundation-accredited care centers that provide expert care and specialized disease management. However, this level of care is not universally available. For instance, in countries that were not part of the EU in 2003, the number of CF patients would rise by 84% if these non-EU countries had similar demographic profiles to EU countries.
In western Europe, North America, and Australia, where most of the nearly 90,000 patients with CF worldwide live, survival of patients with CF has improved significantly over the past decades. This improvement is a result of better nutrition, frequent clinical evaluations, early identification and treatment of complications, and aggressive and intensive interventions by physicians and other healthcare workers who are specially trained in the management of CF.
Conclusion
The global perspective on CF care is evolving, with a focus on addressing the changing needs of patients, building care capacity, developing new therapies, and engaging patients in their care. However, significant disparities exist in the quality and accessibility of CF care across different countries and regions. As research progresses and awareness increases, it is hoped that these disparities will be addressed, leading to improved outcomes for all individuals with CF.
References
1: Cystic Fibrosis Foundation
2: Journal of Translational Medicine
3: CHEST
4: CORDIS
5: The Lancet Respiratory Medicine Commission
