OBJECTIVES: Imiglucerase dosing for the treatment of Gaucher type 1 disease indicates that a patient needs 60 IU/Kg infusion every 14 days. Depending on the patient’s weight, the use of 400 IU vials per infusion can result in drug waste. With a vial use redistribution maintaining an average dosage of 60 IU/Kg per infusion in a monthly basis approach, waste can be minimized or eliminated. The objective is to calculate the potential waste reduction and cost savings resulting from this redistribution.

METHODS: A model was developed to compare two scenarios. The first one corresponded to standard 400 IU vial use based upon 60 IU/Kg infusion every 14 days. The second scenario reassigns the number of vials per infusion in a month, with an average dosage of 60 IU/Kg per infusion. For example, a 50kg patient would receive 7 and 8 vials respectively in the two infusions for the month. The comparison allowed to calculate the potential savings for the healthcare system.

The study modelled a cohort of 147 patients with Gaucher type 1 disease with different weights, which determines vial use and expected drug waste. Costs for Imiglucerase acquisition were obtained from national public databases.

RESULTS: The comparison between the two scenarios shows that the reduction in drug waste allows for potential savings of up to 20% of the annual treatment value, depending on the patient’s weight. Results for the cohort show potential savings of around 6%.

CONCLUSIONS: The dosing approach based on differential vial use per infusion, represents an opportunity for the minimization of waste in enzyme replacement therapies for Gaucher disease, optimizing drug use and resources for the financing of orphan diseases in healthcare systems, guaranteeing the correct dosage for patients.