OBJECTIVES:

Gaucher Disease is a rare disease in Colombia with a low prevalence. Nonetheless, healthcare treatment costs related to it remain high, representing a burden on the health system. This study developed a Budget Impact Analysis of including Enzyme Replacement Therapy - ERT (Imiglucerase, Taliglucerase, Velaglucerase) and Substrate Reduction Therapy - SRT (Eliglustat) for type I Gaucher Disease (GDI) from the health system perspective over a 3-year time horizon in enrolled Colombian population.

METHODS:

Population was calculated from the count of people with a confirmed diagnosis in administrative records. Required health services were defined from the standard expected care in a typical GDI patient, while costs were estimated from databases of medications and medical procedures. Three possible scenarios were included: a base case scenario where there is a predominance of treatment with Imiglucerase; a second scenario where there is a predominance of treatment with Eliglustat; and a third scenario where the impact on weight change is analyzed.

RESULTS:

Estimated costs of including GDI treatments in the base case scenario reach up to USD 190,630,825 over the 3-year time horizon with Imiglucerase representing 64.73% of treatment mix. Treatment related costs represent 99.76% of total GDI budget impact. This reflects the consequences changes in treatment predominance could have on expected budget impact, with an increase of USD 874,167,450 (458.57%) due to a change of 34.20% predominance of Eliglustat in the treatment mix. On the other hand, using lower and upper weight limits result in a budget impact of USD 185,919,378 (2.47% decrease with respect to base case scenario) and USD 197,827,867 (3.78% increase with respect to base case scenario), respectively.

CONCLUSIONS:

Therapy of choice and its frequency affect the most on budget impact in the health system. Almost all GDI cost burden is due to medications and treatment mix.