TafamidisVyndamax

7.1 The PBAC recommended the listing of tafamidis for patients with transthyretin amyloid cardiomyopathy, with NHYA class I-II heart failure. The PBAC considered that there was a clear clinical benefit for tafamidis, including a reduction in mortality. The PBAC considered that the ICER was high, but would be acceptable at the lower price proposed in the pre-PBAC response, in the context of the high unmet need for this patient population.PSD · Jul 2023

7.2 The PBAC acknowledged the consumer comments in continued support of listing tafamidis, and recognised the ongoing impact that a lack of PBS therapies for this condition was having on patients’ prognosis and quality of life.PSD · Jul 2023

In March 2021 the PBAC considered the outstanding economic The economic analysis presented in the current issues may be addressed in a simple resubmission if the resubmission is largely unchanged from the September following changes were made, without any additional 2021 model.PSD · Jul 2023

In particular, the PBAC considered that the ICER ICER to $ 1 per QALY gained (compared to the stated in the resubmission ($ 1/QALY gained) was resubmission’s base case of $ 2 per QALY gained). significantly higher than the ICER requested ($80,000/QALY The resubmission stated that the sponsor is unable to gained) and noted that it relied on tafamidis being discontinued in meet the $80,000 ICER, but reductions in the published patients with persistent class …PSD · Jul 2023

6.33 The resubmission described tafamidis 61 mg (equivalent to tafamidis meglumine 80 mg), as superior in terms of effectiveness and inferior in terms of safety, compared to standard management of ATTR-CM heart failure.PSD · Jul 2023

6.34 The PBAC previously accepted the clinical claim that tafamidis is superior in terms of efficacy and inferior in terms of safety compared with standard management (paras 7.7-7.9, Tafamidis PSD, March 2021 PBAC meeting).PSD · Jul 2023

6.2 The PBAC noted and welcomed the input from individuals (26), and organisations (2) via the Consumer Comments facility on the PBS website. The comments from patients treated with tafamidis described the benefits of treatment in terms of stabilisation of amyloid deposits in the heart.PSD · Jul 2023

6.3 The PBAC noted advice received from Amyloidosis Research Consortium (ARC), which provided results of a global survey of 390 ATTR patients (41% with CM only and 40% with both CM and PN). The most common symptoms reported for ATTR-CM patients were fatigue (87%), shortness of breath (77%), and muscle weakness or loss of strength (71%).PSD · Jul 2023