PBAC's latest decision on Belzutifan: Recommended with restriction (2024). Considered for Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system haemangioblastomas (CNS Hb), or pancreatic neuroendocrine tumours (pNET), not requiring immediate surgery.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2024
Submissions
1
2024 → 2024
Eligible population
Adult patients with VHL disease who require therapy for associated non-metastatic RCC, CNS Hb, or non-metastatic pNET, not requiring immediate surgery, with WHO performance status 0 or 1
Therapy area
Oncology
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
ORR
Key trials
LS-004
Comparator
Active surveillance
Economic model
CUA
ICER note
ICER value is redacted/commercially sensitive; document states effective price is redacted ('$') while published price is $22,060.00 per 90-tablet pack
Risk sharing
Risk-sharing arrangement in place — Sponsor stated willingness to discuss a risk sharing arrangement (RSA) should it be needed; pre-PBAC response proposed RSA to address uncertainty related to applicability broader than clinical evidence base