TAFAMIDIS

The PBAC did not recommend changes to the risk sharing arrangement (RSA) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). The PBAC agreed that a change to the RSA expenditure caps may be appropriate, but not to the degree proposed. The PBAC considered that the revised financial estimates and prevalence data presented in the submission were uncertain. The PBAC recognised that newer diagnostic techniques (i.e. bone scanning) have increased ATTR-CM diagnosis, and expanded the population treated with tafamidis above the existing patient estimates. The PBAC raised concerns that the expanded population may differ from the population that was originally recommended for PBS listing, especially with different diagnostic criteria and the use of SGLT2 inhibitors in heart failure with preserved ejection fraction (HFpEF). The PBAC advised that a submission was required that provides evidence supporting equivalence between diagnosis of ATTR-CM using the clinical trial (ATTRACT) criteria and bone scanning, and that the magnitude of benefit is the same across the whole population.