OBJECTIVES : The ATTR-ACT trial showed that tafamidis reduced all-cause mortality and heart failure hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The aim of this study was to estimate the impact of tafamidis on survival and quality-adjusted life years (QALY).

METHODS : A multi-state cohort Markov model was developed to simulate the disease course of ATTR-CM throughout a lifetime. For survival extrapolation, survival curves were fitted by treatment arm by New York Heart Association (NYHA) class I/II (68% of patients) and NYHA class III (32% of patients) cohorts using the individual patient-level data from both the ATTR-ACT and the corresponding long-term extension study. Univariate and multivariate sensitivity analyses were conducted.

RESULTS : The predicted mean survival for the total population (NYHA class I/II+III) was 6.73 years for tafamidis and 2.85 years for standard of care (SoC), resulting in an incremental mean survival of 3.88 years (95% CI 1.32-5.66). Of the 6.73 life-years, patients on tafamidis spend on average 4.82 years in NYHA class I/II while patients on SoC spend on average 1.60 life-years in these classes. The combination of longer survival in lower NYHA classes produced a QALY gain of 5.39 for tafamidis and 2.11 for SoC, resulting in 3.29 incremental QALYs (95% CI 1.21-4.74) in favor of tafamidis.

CONCLUSIONS : Based on the results of the disease simulation model, tafamidis is expected to more than double the life expectancy and QALYs of ATTR-CM patients compared to SoC. Longer-term follow-up data from the ATTR-ACT extension study data will further inform these findings.