Societal costs and quality of life associated with arginase 1 deficiency in a European setting – a multinational, cross-sectional survey

Olofsson S, Löfvendahl S, Widén J, Rudebeck M, Lindgren P, Stepien K M, Arnoux J-B, Luz Couce Pico M, Leão Teles E, Jacobson L.

Arginase 1 deficiency (ARG1-D) is a ultrarare disease with manifestations that cause mobility and cognitive impairment that progress over time and may lead to early mortality.

Diseases such as ARG1-D have a major impact also outside of the health care sector and the aim of this study was to estimate the current burden of disease associated with ARG1-D from a societal perspective.

The study was performed as a web-based survey of patients with ARG1-D and their caregivers in four European countries (France, Portugal, Spain, United Kingdom). This study shows that ARG1-D is associated with a high societal cost and significant impact on health-related quality-of-life (HRQoL). Earlier diagnosis and better treatment options that can postpone or withhold progression may therefore have a potential for improved HRQoL and savings for the patient, caregiver, and society.

Read more and download the article

Also read the article:  

Disease burden among patients with Arginase 1 deficiency and their caregivers: A multinational, cross-sectional survey

Journal of Medical Economics, 27(1),1146–1156
DOI: https://doi.org/10.1080/13696998.2024.2400856