(367) Measuring Burden of Disease for Canadians, both Adult and Pediatric, with Cystic Fibrosis (CF) and their Caregivers - A Systematic Literature Review

Background: National cystic fibrosis registries are an excellent resource to describe the demographic and clinical characteristics of persons with CF (PwCF); however, they do not capture how the disease impacts the lives of PwCF and their caregivers and thereby the true direct and indirect socio-economic burdens of CF. Even with the availability of highly effective modulator therapies, there remains no cure for CF and treatment regimens are costly and labor intensive for both PwCF and their caregivers. The aim of this study was to systematically review the literature to summarize the socio-economic burden of CF and identify gaps in our knowledge.

Methods: A systematic search of PubMed, Cochrane Library, and Google Scholar was conducted in March 2022 in accordance with PRISMA guidelines. We reviewed studies that included social and/or economic costs of CF with respect to direct and indirect healthcare and non-healthcare determinants. All costs were adjusted to 2021 and expressed in USD.

Results: A total of 32 CF studies that reported on the burden of CF were included and categorized based on these three perspectives: healthcare/payers (n=17); societal (n=8); and patient/caregivers (n=7). Among the studies utilizing the healthcare/payer perspective, annual adjusted costs of CF per patient ranged from $4,552 to $143,635. These costs were primarily influenced by CF-related complications, exacerbations, disease severity, genotype, and CFTR modulator use. Studies utilizing the societal perspective identified annual adjusted costs per patient from $23,776 to $64,876. These studies highlighted the role of absenteeism, caregiver presence, and advancing age as primary drivers of disease burden. Qualitative studies utilizing a patient/caregiver perspective reported increased treatment time, significant psychological burden, and decreased caregiver productivity within the CF population. Fourteen of the 32 studies were conducted in North America and the majority were conducted more than a decade ago. Furthermore, the majority of these studies did not incorporate other societal aspects of disease burden (i.e., absenteeism vs. presenteeism, quality of life, long-term vs. short-term disability leaves, out-of-pocket expenses, and principal caregiver costs).

Conclusions: We observed significant heterogeneity in study design, methodology, and population demographics, and there were only a few studies that measured both the direct and indirect medical and non-medical impacts of CF. These findings highlight a major gap in reliable and robust estimates of CF disease burden.

Acknowledgements:

References: