Introduction:

Tafamidis has been approved for the treatment of cardiomyopathy associated with transthyretin amyloidosis (CM-ATTR). However, its high-cost warrants in-depth evaluation of its efficacy and cost-effectiveness.

Objective:

To review the efficacy (defined as the impact on mortality and morbidity due to heart failure) and cost-effectiveness of tafamidis for the management of CM-ATTR.

Methods:

Systematic review of the literature including studies that evaluated the use of tafamidis in CM-ATTR. The quality of the included studies was assessed independently by three authors.

Results:

A total of 16 studies were included. The use of tafamidis was associated with a significant reduction in mortality, and a statistically significant reduction in heart failure-related hospitalization. Follow-up imaging reveals that tafamidis delays, but does not stop or regress, the damage induced by CM-ATTR. Although the clinical impact of tafamidis on mortality is high, its elevated cost resulted in a lack of cost-effectiveness for both screening and treatment.

Conclusion:

The use of tafamidis has a significant impact on mortality associated with CM-ATTR; However, its high cost prevents both screening and treatment from being cost-effective.

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