12 January 2024 - The public summary documents (first time decisions not to recommend and deferrals) from the September 2023 PBAC intracycle meeting are now available.

There is just one new/revised PBS for this round - patisiran sodium for the treatment of patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy.

Some insights:

• The PBAC noted that the resubmission had provided a new base case economic evaluation which did not follow the PBAC’s advice concerning amendment of the utility increments/decrements, and considered that the resubmission had not demonstrated cost effectiveness for patisiran sodium.
• The Committee noted that reducing these increments/decrements to 80% of base case values, as proposed in the resubmission, resulted in an incremental cost effectiveness ratio of $255,000 - $355,000/QALY gained. The PBAC considered that the previously specified requirement for an incremental cost effectiveness ratio of no more than $155,000 -$255,000/QALY gained remained appropriate for patisiran sodium, and that the concerns about treatment-related utility increments/decrements remained.
• The PBAC considered that the resubmission’s proposal for a managed access program did not sufficiently address its concern regarding the uncertain cost effectiveness of the proposed listing.

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