Budget Impact Analysis Of Migalastat Incorporation vs. Enzyme Replacement Therapies For Fabry Disease: Validation of a Cost-Assessment Tool In Brazilian Private Healthcare System
Author(s)
Joao Paulo Dos Reis Neto, PhD, MD1, JULIANA BUSCH, MD2.
1ceo, Diretor-Presidente da Capesesp, Rio de Janeiro, Brazil, 2CAPESESP, Rio de Janeiro, Brazil.
1ceo, Diretor-Presidente da Capesesp, Rio de Janeiro, Brazil, 2CAPESESP, Rio de Janeiro, Brazil.
OBJECTIVES: This study aims to assess the budgetary impact of incorporating migalastat for Fabry disease compared to enzyme replacement therapies (ERTs). Using a budget impact analysis (BIA) model, the research evaluates the cost implications over a 5year horizon testing a calculator designed for this purpose.
METHODS: BIA was conducted from the payer perspective, incorporating epidemiological data, treatment costs, and Fabry diagnosis stratified by age and GLA mutation eligibility for migalastat. The model used static prevalence and cost for ERT and migalastat. A validated calculator enabled customization of inputs such as prevalence, costs, and adherence was developed and validated through pilot testing. Key parameters included Fabry prevalence (0.003%) and migalastat eligibility (35%). Migalastat prices considered standard dose and regimen with price proposed by the manufacturer (15% discount), and for available ERTs, the average annual cost based on dose, frequency of use and cost of administration. Annual costs were set at US$158,298 for ERT and US$143,469 for migalastat. Sensitivity analysis was performed.
RESULTS: The model projected an incremental shift in treatment distribution, with 50% of Fabry patients eligible based on 46 million beneficiaries from private system for migalastat transitioning from ERT. Over the 5-year period, population increased marginally due to a demographic growth rate, and the total cost of treatment in the baseline (ERT-only) scenario was estimated at US$321 million. Introducing migalastat with negotiated price reduced total costs to US$312 million, yielding a cumulative negative budget impact of US$9 million.
CONCLUSIONS: Although ERTs are well-established treatment options, lifelong biweekly infusions place a significant burden on patients and healthcare systems. The incorporation of migalastat offers an oral therapeutic alternative which may improve treatment adherence and clinical outcomes representing a cost-saving alternative for eligible patients with Fabry disease. This study highlights the importance of BIA models and tools, such as the calculator tested, to inform healthcare policy decisions.
METHODS: BIA was conducted from the payer perspective, incorporating epidemiological data, treatment costs, and Fabry diagnosis stratified by age and GLA mutation eligibility for migalastat. The model used static prevalence and cost for ERT and migalastat. A validated calculator enabled customization of inputs such as prevalence, costs, and adherence was developed and validated through pilot testing. Key parameters included Fabry prevalence (0.003%) and migalastat eligibility (35%). Migalastat prices considered standard dose and regimen with price proposed by the manufacturer (15% discount), and for available ERTs, the average annual cost based on dose, frequency of use and cost of administration. Annual costs were set at US$158,298 for ERT and US$143,469 for migalastat. Sensitivity analysis was performed.
RESULTS: The model projected an incremental shift in treatment distribution, with 50% of Fabry patients eligible based on 46 million beneficiaries from private system for migalastat transitioning from ERT. Over the 5-year period, population increased marginally due to a demographic growth rate, and the total cost of treatment in the baseline (ERT-only) scenario was estimated at US$321 million. Introducing migalastat with negotiated price reduced total costs to US$312 million, yielding a cumulative negative budget impact of US$9 million.
CONCLUSIONS: Although ERTs are well-established treatment options, lifelong biweekly infusions place a significant burden on patients and healthcare systems. The incorporation of migalastat offers an oral therapeutic alternative which may improve treatment adherence and clinical outcomes representing a cost-saving alternative for eligible patients with Fabry disease. This study highlights the importance of BIA models and tools, such as the calculator tested, to inform healthcare policy decisions.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA59
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Genetic, Regenerative & Curative Therapies, Rare & Orphan Diseases