OBJECTIVES:

Anderson-Fabry and Gaucher are genetic disorders caused by lysosomal enzyme deficiencies [1]. Enzyme replacement therapy, administered by periodic intravenous infusions in the hospital setting, controls underlying enzyme deficiency. Hospital-based infusion implies patients travels and waits at referral centres, which might interfere with their and caregivers daily life activities. Domicile infusion can improve therapy adherence and quality of life [2]. However, additional resources consumption could be required. This study aimed at comparing domicile and hospital-based infusion costs and identifying savings impact factors.

METHODS:

An Excel model was populated by semi-structured questionnaire: times and procedure protocol were retrieved from the referral centre of Rimini, Emilia Romagna Region. Unit costs were from literature. Sensitivity analysis was performed by modifying hospital fixed cost, domicile nurse working schedule and salary, specialist visit cost, number of nurse per patient and infusion duration.

RESULTS:

Base case results showed 272.13€ saving per patient in the domicile setting. Drivers of cost are hourly nurse salary (30€/h in the base case) and total journey kilometres (30km in the base case). Saving vanished if hourly operator’s salary increased by 18.9% (35.67€/h) or if kilometres of journey increased by 63% (48.9km). Hospital fixed cost (150€/day) (91% reduction), infusion duration (exceeding of 3 hours) and number of hospital nurses per patient (ratio 2:3 which might be up to ratio 2:5) did not nullify cost savings. Finally, specialist cost (50.77€/h in the basecase) had no impact on saving.

CONCLUSIONS:

Enzyme replacement therapy domicile infusion therapy saves NHS costs if implemented with specific procedure features. The model is reproducible in other centres due to the robustness of the sensitivity analysis implemented and it is a valuable decision-maker tool.