BACKGROUND: The aim of this study was to estimate the resource implications and budget impact of managing adults with Fabry disease in Norway, from the perspective of the publicly funded healthcare system. METHODS: A decision model was constructed using published clinical outcomes and clinician-derived resource utilization estimates. The model was used to estimate the annual healthcare cost of managing a cohort of 64 adult Fabry patients in an average year. RESULTS: The expected annual cost of managing 60 existing Fabry patients and four new patients in Norway each year was estimated to be NOK 55·8 million (€6·7 million). In an average year, patients receiving enzyme replacement therapy (ERT) with agalsidase alfa (Replagal(®)) at 0·2 mg kg⁻¹ or agalsidase beta (Fabrazyme(®)) at 1·0 mg kg⁻¹ are collectively expected to make 586 attendances to their family practitioner's office for their infusions, which equates to 128 eight-hour days associated with ERT. Encouraging more patients to undergo home-based infusions has substantial potential to free-up community-based resources. In comparison, the community-related benefit that can be obtained by switching from agalsidase beta (1·0 mg kg⁻¹) to agalsidase alpha (0·2 mg kg⁻¹) is marginal, and dependent on the two doses being clinically equivalent. CONCLUSION: Maximizing the proportion of adults with Fabry disease undergoing home-based infusions has the potential to release community-based resources for alternative use by non-Fabry patients, thereby improving the efficiency of the publicly funded healthcare system in Norway.
BACKGROUND: The aim of this study was to estimate the resource implications and budget impact of managing adults with Fabry disease in Norway, from the perspective of the publicly funded healthcare system. METHODS: A decision model was constructed using published clinical outcomes and clinician-derived resource utilization estimates. The model was used to estimate the annual healthcare cost of managing a cohort of 64 adult Fabry patients in an average year. RESULTS: The expected annual cost of managing 60 existing Fabry patients and four new patients in Norway each year was estimated to be NOK 55·8 million (€6·7 million). In an average year, patients receiving enzyme replacement therapy (ERT) with agalsidase alfa (Replagal(®)) at 0·2 mg kg⁻¹ or agalsidase beta (Fabrazyme(®)) at 1·0 mg kg⁻¹ are collectively expected to make 586 attendances to their family practitioner's office for their infusions, which equates to 128 eight-hour days associated with ERT. Encouraging more patients to undergo home-based infusions has substantial potential to free-up community-based resources. In comparison, the community-related benefit that can be obtained by switching from agalsidase beta (1·0 mg kg⁻¹) to agalsidase alpha (0·2 mg kg⁻¹) is marginal, and dependent on the two doses being clinically equivalent. CONCLUSION: Maximizing the proportion of adults with Fabry disease undergoing home-based infusions has the potential to release community-based resources for alternative use by non-Fabry patients, thereby improving the efficiency of the publicly funded healthcare system in Norway.
Authors: Michał Nowicki; Stanisława Bazan-Socha; Mariusz Kłopotowski; Beata Błażejewska-Hyżorek; Mariusz Kusztal; Krzysztof Pawlaczyk; Jarosław Sławek; Andrzej Oko; Zofia Oko-Sarnowska Journal: Int J Environ Res Public Health Date: 2021-08-04 Impact factor: 3.390