Summary. Introduction. The evolving epidemiological landscape of transthyretin amyloid cardiomyopathy (ATTR-CM), particularly in its wild-type form, highlights a progressive increase in diagnoses. This trend calls for a critical reflection on the economic sustainability of tafamidis treatment, given the expanding eligible patient population and the lack of price renegotiation. Methods. A retrospective study was conducted on patients treated at an Asl (2023-2025). Persistence and adherence to the treatment were evaluated. A linear regression model estimated future incidence and economic impact. Results. Among 17 patients, persistence was 58.82%, adherence (>80%) 100%. Incidence rose from 15.2 to 35.73 cases/million (2018-2025). The 5-year cost per patient exceeded € 815,000. Based on the ATTR-ACT trial NNT (7.46), 2.3 deaths may be prevented, though this benefit may be overestimated. Discussion. The rising incidence was not followed by a price revision. Use in older, frail patients and the absence of dynamic pricing agreements raise questions about cost-effectiveness and equitable access.
[Tafamidis between real-world evidence and registration trials: towards a price renegotiation with a view to sustainability and appropriateness]
Lupi, Tiziano
Primo
;Santoleri, Fiorenzo;
2025-01-01
Abstract
Summary. Introduction. The evolving epidemiological landscape of transthyretin amyloid cardiomyopathy (ATTR-CM), particularly in its wild-type form, highlights a progressive increase in diagnoses. This trend calls for a critical reflection on the economic sustainability of tafamidis treatment, given the expanding eligible patient population and the lack of price renegotiation. Methods. A retrospective study was conducted on patients treated at an Asl (2023-2025). Persistence and adherence to the treatment were evaluated. A linear regression model estimated future incidence and economic impact. Results. Among 17 patients, persistence was 58.82%, adherence (>80%) 100%. Incidence rose from 15.2 to 35.73 cases/million (2018-2025). The 5-year cost per patient exceeded € 815,000. Based on the ATTR-ACT trial NNT (7.46), 2.3 deaths may be prevented, though this benefit may be overestimated. Discussion. The rising incidence was not followed by a price revision. Use in older, frail patients and the absence of dynamic pricing agreements raise questions about cost-effectiveness and equitable access.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
