OBJECTIVES: Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare, progressive, debilitating and life-threatening neurodegenerative disease. Treatment for clinical symptoms of the disease is a priority. Although a rare disease, Portugal has the largest TTR-FAP foci in the world but data on treatment costs is lacking. The purpose of this study is to estimate the ambulatory medicines costs of TTR-FAP in Portugal. METHODS: TTR-FAP patient’s medicines utilization is fully funded by National Health System in Portugal since 2001. Since March 2013 Portuguese electronic prescription system became more generalized, allowing central monitoring and validation of medicines prescription and dispensing. This database only contains medicines dispensing data in ambulatory setting. TTR-FAP anonymized patient’s utilization was requested to Administração Central do Sistema Saúde(ACSS). International Non-proprietary Names (INN) were extracted from INFARMED medicines database. Total and mean annual costs per patient are reported in euros.

RESULTS: Over the year 2014, a total of 70 286 electronic dispensing activities were imputed to 2013 TTR-FAP anonymized patients in ACSS database. The total cost with TTR-FAP medicines dispensed in ambulatory setting was 1 612 673 €. The mean annual cost per patient was 801 € (standard deviation 1 233 €). CONCLUSIONS: We can conclude that Portuguese ACSS electronic prescription database is a useful, relevant and accessible strategy to estimate medicines expenditure trough anonymized individual patient data. Access to additional individual patient data such as age/gender would enhance future studies. We can estimate that TTR-FAP disease in Portugal accounted for 1 612 673 € total cost in ambulatory medicines, with a mean annual cost per patient of 801 €.