OBJECTIVES: To compare the total costs associated with treating patients with transthyretin amyloid polyneuropathy (ATTR-PN) in Spain.

METHODS: An Excel cost-calculator was developed to itemize all costs related to treatment with tafamidis, patisiran and inotersen in the context of ATTR-PN. The Summary of Product Characteristics (SmPCs) and input from five Spanish ATTR-PN experts were used to inform drug acquisition, pre-treatment, administration, concomitant medication, disease management, monitoring, adverse events, and travel/work loss inputs. Results are reported as total costs per-patient per-year.

RESULTS: The only drug requiring pre-treatment and IV administration is patisiran, resulting in a cost of €11,282.59. The need for patisiran- and inotersen-treated patients to visit hospitals for administration or monitoring was associated with increased patient travel time and loss of productivity compared to those treated with tafamidis. The average number of clinic visits and resulting annual costs were lower for tafamidis (8.2, €238.33) than for patisiran (21.4, €823.15) and inotersen (14.8, €428.71). Similarly, productivity loss due to treatment showed 4.5, 23.6, and 13.4 weekly hours lost and incurred costs per year of €3,345.71, €13,938.29 and €10,110.72 in those treated with tafamidis, patisiran and inotersen, respectively. The costs for managing adverse events in those exposed to inotersen was €2,340.43, which was greater than in those exposed to tafamidis (€1,597.08) or patisiran (€1,596.98). Drug acquisition costs according to public ex-factory prices with mandatory deduction (4%) were €129,737, €427,250, and €291,076 for tafamidis, patisiran (mean patient weight of 70.87 kg), and inotersen, respectively. Overall, the total annual per-patient costs was lowest for patients treated with tafamidis (€137,944), followed by inotersen (€307,981), and patisiran (€457,921).

CONCLUSIONS: This is the first analysis that considered costs beyond drug acquisition among patients with ATTR-PN treated with tafamidis, patisiran, or inotersen. Treating patients with tafamidis would result in substantially lower costs and lower patient burden than with patisiran or inotersen.