Modelling the resource implications of managing adults with Fabry disease in Norway favours home infusion.
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.


CONCLUSIONS
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.
Catalyst Health Economics Consultants, Northwood, Middlesex, UK. julian.guest@catalyst-health.co.uk
Julian F
JF
Guest
Trond
T
Jenssen
Gunnar
G
Houge
Willy
W
Aaseboe
Camilla
C
Tøndel
Einar
E
Svarstad
D000328:Adult; D002017:Budgets; D015331:Cohort Studies; D000795:Fabry Disease / Q000188:drug therapy / Q000191:economics*; D017048:Health Care Costs; D015276:Health Care Rationing; D006295:Health Resources / Q000191:economics / Q000458:organization & administration / Q000706:statistics & numerical data*; D006699:Home Care Services / Q000191:economics; D018718:Home Infusion Therapy / Q000191:economics*; D006801:Humans; D007527:Isoenzymes / Q000191:economics / Q000627:therapeutic use; D018803:Models, Economic; D009664:Norway / Q000453:epidemiology; D011994:Recombinant Proteins; D040841:Resource Allocation / Q000191:economics; D000519:alpha-Galactosidase / Q000191:economics / Q000627:therapeutic use
D007527:Isoenzymes; D011994:Recombinant Proteins; C000627036:agalsidase alfa; D000519:alpha-Galactosidase; C459420:agalsidase beta