The National Institute for Healthcare and Medical Technology Assessment (NIHO) has decided not to approve the categorization of Elfabrio (pegunigalzidáza alfa) for the treatment of patients with Fabry disease. This decision comes after a thorough evaluation of the drug’s clinical efficacy, safety, and cost-effectiveness within the Slovak healthcare context.
Assessment Criteria and Findings
NIHO’s evaluation focused on whether Elfabrio met the legal criteria for cost-effectiveness. The assessment revealed that the proposed reimbursement rate of €1,551 per package did not align with the maximum acceptable reimbursement of €1,445, representing a significant discount requirement of 6.9% compared to the initially requested amount. This discrepancy raised concerns about the drug’s cost-effectiveness, especially considering the high annual treatment costs for Fabry disease patients.
Recommendations for Future Consideration
NIHO suggested that the manufacturer reconsider the reimbursement price to meet the cost-effectiveness criteria. Additionally, the institute recommended specifying treatment for adult patients and defining enzymatic activity levels to better target the drug’s application. This approach aims to ensure that healthcare resources are allocated efficiently, preventing potential budget strains and maintaining the quality of care for other medical areas.
• Elfabrio did not meet NIHO’s cost-effectiveness threshold at the proposed price.
• A recommended price adjustment of up to €1,445 per package is necessary for approval.
• Targeting adult patients and defining enzymatic activity can enhance treatment specificity.
Elfabrio, approved by the European Medicines Agency in February 2023, is intended for long-term enzyme replacement therapy in Fabry disease patients. Despite demonstrating similar efficacy to existing treatments, its high cost poses significant economic challenges for the Slovak healthcare system.
The rejection of Elfabrio’s categorization underscores the critical balance between medical innovation and economic sustainability. It highlights the necessity for pharmaceutical companies to align pricing strategies with national cost-effectiveness standards to facilitate broader access to new treatments. For patients, this decision may limit the immediate availability of Elfabrio, urging continued dialogue between stakeholders to address treatment needs while managing healthcare budgets effectively.
Elfabrio’s future in Slovakia hinges on the manufacturer’s willingness to adjust pricing and refine target patient criteria. This case exemplifies the rigorous scrutiny new therapies undergo to ensure they provide valuable health outcomes without imposing undue financial burdens on the healthcare system.
Ultimately, NIHO’s decision reflects a commitment to maintaining healthcare quality and accessibility, urging continued collaboration to find viable solutions for treating rare and costly diseases like Fabry.
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