PSY87 - ECONOMIC BURDEN OF TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY

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To estimate the economic burden of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Portugal, a rare genetic disease associated with major disability and psychological burden for patients and caregivers. In a population-based cost analysis, we evaluated the cost of TTR-FAP in Portugal during year 2016. We obtained data for healthcare resource use and costs from national official sources. We estimated direct healthcare costs for drugs consumed, including disease-modifying treatments, and for primary care, outpatient, and inpatient resource use. Additionally, we estimated the costs of lost workdays due to the illness using the human capital method. We estimated €32.3 million as the cost of TTR-FAP in Portugal, regarding 1865 patients (45.8% male; mean age: 52.3±15.4 years) in 2016. Productivity losses were estimated to cost €4.4 million. Disease-modifying treatments accounted for the majority of the costs (€18.8 million, 58% of TTR-FAP costs), followed by costs with other medicines (€6.2 million, 19%) including immunosuppressive drugs, and inpatient episodes (€2.0 million, 6%), covering liver transplant related hospitalizations costs. TTR-FAP is associated with high economic burden as it affects mainly young productive adults. These data nurture public health and policy intelligence, which is required to deliver affordable care and inform preventive and treatment interventions funds allocation within the rare disease setting.