OBJECTIVES:

Fabry disease is known to have a significant impact on renal function. Evidence suggests that treatment with agalsidase beta reduces the progression of renal disease in Fabry patients. The objective of this study is to develop an economic analysis of the impact of treatment on renal disease progression in a Colombian population.

METHODS:

A cost-analysis model was developed using the annualized eGFR of agalsidase beta treated vs untreated Fabry patients. Simulation scenarios were performed depending on the starting stage of renal disease for a hypothetical cohort of 100 patients.

The average yearly cost of treatment per patient with kidney disease stages 1-4 and 5 (dialysis) were USD$32.9 and USD$6,631, respectively. All costs are expressed in USD$ for a 25-year time horizon with a 5% yearly discount rate using an exchange rate of COP$4,800 per USD$1.

RESULTS:

When the cohort starts in stage 1-2 renal disease, the expected cost of treated and untreated patients would be USD$44,560 and USD$1,465,716 respectively (96.9% costs avoided).

Starting in stage 3a renal disease, the expected cost of treated and untreated patients would be USD$47,034 and USD$4,464,899 respectively (98.9% costs avoided).

For stage 3b renal disease, the expected cost of treated and untreated patients would be USD$699,349 and USD$6,299,247 respectively (88.9% costs avoided).

Finally, when starting on stage 4 renal disease, the expected cost of treated and untreated patients would be USD$5,809,602 and USD$8,526,629 respectively (31.87% costs avoided).

CONCLUSIONS:

These results suggest that reduced progression of renal disease with agalsidase beta treatment translates into reduced overall costs for the management of this complication associated with Fabry disease. Early initiation of treatment provides the best clinical outcomes for patients, which are associated with the lowest accumulated costs when initiated in stage 1-2, and greater percentage of costs avoided when treatment is initiated at stages 1-2 and 3a.