Abstract
Background. Fabry Disease is a rare genetic disorder with an estimated prevalence of 1-5/10,000 subjects. Two enzyme replacement therapies (ERTs) are indicated for the treatment of the disease: agalsidase alpha and agalsidase beta. The aim of this economic evaluation was to estimate the time-management and economic consequences of the use of the two therapies in the Italian context from the perspective of the Italian National Health Service (INHS). Understanding the time management burden and the treatment costs will be useful to Italian hospital directors/administrators and pharmacists.
Materials and methods. We considered the dosage of the two treatments based on the weight of the subjects affected by Fabry Disease and the relative cost of administration adopting the National Health Service perspective in an annual time horizon. From an organizational point of view, the infusion time required per patient was estimated (40 minutes for agalsidase alfa and between 90 and 385 minutes for agalsidase beta).
Results. The annual cost per treatment with agalsidase alfa is between € 120,332 and € 240,361, and between € 160,341 and € 240,360 for agalsidase beta. Considering the average weight per gender reported in the Fabry Outcome Survey, this economic evaluation suggests a lower cost related to the use of agalsidase alfa compared to agalsidase beta between € 0 and € -40,009. In terms of infusion time, the use of agalsidase alfa would reduce the infusion time between -55 minutes and -345 minutes. Considering a caseload of 20 subjects at a single hospital and a proportion of subjects managed at home of 50%, we estimate an annual number of lower infusion-related hours between 217 and 1,495 hours.
Conclusions. This economic evaluation suggests that the use of agalsidase alfa compared to agalsidase beta correlates with advantages in both economic and time-management terms, allowing optimi zation and higher efficiency in the treatment of subjects affected with Fabry Disease.