PBAC's latest decision on Tolvaptan: Recommended with restriction (2019). Considered for Treatment of autosomal-dominant polycystic kidney disease (ADPKD) in patients with estimated glomerular filtration rate (eGFR) between 30 and 89 mL/min/1.73 m² and rapidly progressing disease.
PBAC outcome
Recommended with restriction
Restricted
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
4
first 2017
Submissions
4
2017 → 2019
Eligible population
Adults with autosomal-dominant polycystic kidney disease (ADPKD) with eGFR between 30 and 89 mL/min/1.73 m² and rapidly progressing disease (defined as a decline in eGFR of ≥5 mL/min/1.73 m² within one year or average decline of ≥2.5 mL/min/1.73 m² per year over five years), requiring treatment by a nephrologist.
Therapy area
Rare disease
Line of therapy
Not applicable
Evidence base
Other
Primary endpoint
Cost-minimisation
Economic model
Cost-minimisation
ICER note
Cost-minimisation submission; no ICER calculated. PBAC noted recommendation was on a cost-minimisation basis.
Risk sharing
Risk-sharing arrangement in place — Risk Sharing Arrangement (RSA) in place; new single-dose packs included within existing caps of the RSA.
Submission history
Mar 2017: Deferred · Authority Required
Mar 2018: Recommended with restriction · Authority Required
Jul 2018: Recommended with restriction · Restricted
Nov 2019: Recommended with restriction · Restricted