The latest Pharmaceutical Benefits Advisory Committee (PBAC) Meeting was held in July and it recommended the Public Benefit Scheme (PBS) listing of Vyndamax (tafamidis) 61 mg for patients with transthyretin cardiac amyloid cardiomyopathy with New York Heart Association Class I-II heart failure. pbac-web-outcomes-07-2023.pdf (pbs.gov.au) see pg 32. Tafamidis has been submitted several times before for consideration of PBS listing (Medicare funding) but not been approved based on financial concerns. There have been similar concerns in the USA and other countries. https://www.axios.com/2021/07/08/pfizer-drug-prices-medicare-oig-kickbacks-tafamidis. Negotiations between the Australian Government and the pharmaceutical company, Pfizer will now proceed. It will take time for the financial arrangements and PBS program development to occur before tafamidis can be accessed on Medicare.
In the same PBAC meeting, patisiran was submitted for the first time for consideration of PBS listing for treatment of patients with hereditary ATTR amyloidosis with polyneuropathy. (see pg 23 of the meeting outcome document). The PBAC considered there was a high unmet need in the requested patient population, and that efficacy and safety of patisiran compared with best supportive care had been demonstrated by the clinical evidence. However, PBAC considered that the submission had not demonstrated that patisiran was cost – effective. The PBAC nominated the early resolution re-submission pathway for this item. PBAC submissions for high cost therapies for rare diseases are rarely accepted on first submission and this recommendation for the early resolution pathway is considered a good outcome”.