OBJECTIVES: Transthyretin-associated amyloid cardiomyopathy (ATTR-CM) is a rare condition, caused by cardiac infiltration of amyloid fibrils, leading to organ dysfunction and, consequently, progressive heart failure. The hereditary form of the disease usually manifests itself after 47 years of age, with a median survival of 2.5 years after diagnosis for patients without treatment. The wild form affects almost exclusively older people, with a mean age at diagnosis of 74 years and a median survival from diagnosis in untreated patients of approximately 3.5 years (depending on the stage of the disease). The aim of this analysis is to compare utility and incremental costs between tafamidis and symptomatic treatment.

METHODS: A cost-effectiveness analysis was developed from the perspective of the Brazilian public healthcare system, using a Markov model to follow up patients over 60 years old, with ATTR-CM in functional classes II or III in a lifetime time horizon, considering the transition through different states of health. The outcomes evaluated were life years gained (LY) and quality-adjusted life years gained (QALY).

RESULTS: The results showed that tafamidis meglumine provides significant gains in effectiveness (LY = 3.28 and AVAQ = 2.55) when compared to the symptomatic treatment, with the need for incremental costs, resulting in an incremental cost-effectiveness ratio (ICER) per LY of BRL 373.1 thousand and RCEI/AVAQ of BRL 478.6 thousand.

CONCLUSIONS: The clinical evidence and the result of the cost-effectiveness showed that tafamidis provides gains for patients with ATTR-CM. It’s incorporation in the Brazilian public healthcare would be extreme important to patients, because it is the only specific treatment available for ATTR-CM.