PBAC's latest decision on Tafamidis: Recommended with restriction (2023). Considered for Treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) in patients with NYHA class I-II heart failure and an end-diastolic interventricular septal wall thickness of at least 12 mm.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
4
first 2020
Submissions
4
2020 → 2023
Eligible population
Adult patients with wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) with NYHA class I-II heart failure, end-diastolic interventricular septal wall thickness ≥12 mm, eGFR >25 mL/min/1.73 m², and confirmed evidence of transthyretin precursor protein by histological confirmation or bone scintigraphy with negative monoclonal protein tests.
Therapy area
Cardiovascular
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
All-cause mortality, cardiovascular-related hospitalisations, six-minute walk test distance, quality of life (KCCQ-OS, EQ-5D-3L), cardiovascular-related mortality, TTR stabilisation
Key trials
ATTR-ACT
Comparator
standard management of ATTR-CM (e.g. diuretics and anti-arrhythmics)
Economic model
CUA
Eligible patients/year
300
ICER note
ICER values are redacted in the document; the text indicates ranges ($135,000 to <$155,000 and $95,000 to <$115,000) but these are masked with footnotes and not explicitly stated as the actual ICER in AUD.
ICER (historical)
$155k/QALY–$255k/QALY across 2 submissions (2021) — the latest submission carried no numeric base case. No single PSD states this combined range; see source PSDs.
Risk sharing
Risk-sharing arrangement in place — Risk sharing arrangement proposed with expenditure caps at 100% of estimated expenditure with % rebate in Years 1-3 of listing and at 105% of estimated expenditure with % rebate in Years 4-6 of listing. Special pricing arrangement with effective price for Years 1-3 and Years 4-6 (values redacted).
Submission history
Jul 2020: Recommended with restriction · Authority Required
Mar 2021: Recommended with restriction · Authority Required — ICER $255k/QALY
Mar 2021: Recommended with restriction · Authority Required — ICER $255k/QALY
Jul 2023: Recommended with restriction · Authority Required