BelzutifanWelireg
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.
Decision on record
- Meeting Jul 2024 Recommended Von Hippel-Lindau disease with renal cell carcinoma, central nervous system haemangioblastomas, or pancreatic neuroendocrine tumours
Access path
- TGA registered · Welireg
TGA label narrower than the PBS population
- Jul 2024Recommended · restricted
vs Active surveillance
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the listing of belzutifan, on the basis that it should be available as a General Schedule Authority Required (telephone/online PBS Authorities system) listing for the treatment of 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).PSD · Jul 2024
7.2 The PBAC noted the qualitative evidence on the patient experience in Australia that was provided in the submission and is summarised in Table 3, above. The PBAC acknowledged the lifelong burden on patients and families of multiple surgeries undertaken for tumours with high symptom burden, or those carrying a high risk of organ dysfunction, or metastasis.PSD · Jul 2024
6.35 The submission presented a modelled economic evaluation of belzutifan plus active surveillance (belzutifan arm, BEL+AS) compared to active surveillance (AS arm) for the treatment of VHL disease associated tumours; based on results from the MAIC using data from LS-004 and VHL-NHS (see Comparative effectiveness), non-randomised data from the pre-treatment period of LS-004 and real-world observational data from the Optum Clinformatics claims database …PSD · Jul 2024
6.36 Optum CDM was a retrospective study based on a US Claims database with administrative data of VHL patients (identified via an algorithm) between 2007 and 2020 that has previously been utilised in Wang 20239 (a cost-consequence study of belzutifan).PSD · Jul 2024
6.32 The submission described belzutifan + active surveillance as superior in terms of effectiveness and inferior in terms of safety compared to active surveillance. The ESC considered that the clinical claim was supported by the evidence presented in LS-004 alone (e.g. tumour growth rates before and after belzutifan, number of surgeries before and after belzutifan, and tumour response rates).PSD · Jul 2024
6.33 The PBAC considered that the claim of superior comparative effectiveness was reasonable.PSD · Jul 2024
6.2 The PBAC noted and welcomed the input from individuals (516), health care professionals (10) and organisations (3) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with belzutifan including reducing tumour size to potentially negate/reduce the need for further surgery.PSD · Jul 2024
6.3 The PBAC noted and welcomed the input from the Medical Oncology Group of Australia (MOGA) via the Consumer Comments facility on the PBS website. The MOGA expressed strong support for belzutifan and listed it amongst its highest priority for PBS listing, to address significant unmet need in rare tumours.PSD · Jul 2024
Cost-effectiveness
ICER value is redacted/commercially sensitive; document states effective price is redacted ('$') while published price is $22,060.00 per 90-tablet pack
Decision context
PopulationAdult 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
Risk sharingSponsor 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
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2024 | Recommended · restricted | Active surveillance | — | RCT · ORR |
Consumer voice
Consumer input from 516 individuals, 10 healthcare professionals, and 3 organisations, along with advice from medical and rare cancer organisations, described significant benefits of belzutifan including reduction in tumour size to avoid/reduce surgery, improved quality of life, better psychological well-being, and preservation of renal function. Consumers highlighted the substantial physical and
The comments described a range of benefits of treatment with belzutifan including reducing tumour size to potentially negate/reduce the need for further surgery. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to non-metastatic RCC/pNET and WHO performance status 0-1; TGA label has no metastatic status or performance status requirements.