VorinostatZolinza
Cutaneous T-cell lymphoma (CTCL) in patients with relapsed or chemotherapy-refractory disease who are ineligible for stem cell transplantation and have received prior systemic chemotherapy.
Decisions on record
- Meeting Mar 2017 Recommended Relapsed or refractory cutaneous T-cell lymphoma
- Meeting Nov 2016 Deferred Relapsed or refractory cutaneous T-cell lymphoma
- Meeting Mar 2011 Not recommended Anti–cancer drug
Access path
- TGA registered · Zolinza
TGA label narrower than the PBS population
- Mar 2011Not recommended
unacceptably high and uncertain cost-effectiveness ratios, limited and non-comparative clinical data, small…
- Nov 2016Deferred
Comparator changed: palliative care (though PBAC considered best available…
- Mar 2017Recommended · restricted
Evidence: Single-arm → Other
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended the Authority Required listing of vorinostat for the treatment of cutaneous T-cell lymphoma (CTCL) in the General Schedule.PSD · Mar 2017
5.2 The PBAC recommendation for listing was based on, among other matters, its assessment, as described above, that the cost-effectiveness of vorinostat would be acceptable at the price proposed in the submission.PSD · Mar 2017
6.21 The resubmission provided a modelled cost-utility analysis, compared to trial-based cost per responder analysis in the previous submission. This approach was appropriate in the context of the condition, but is not substantiated by the clinical evidence presented in the resubmission.PSD · Nov 2016
Table 5: Summary of model structure and rationale Time horizon The maximum time horizon was based on the observed median survival of 88.2 months in responders in the Australian registry data. In that registry, survival was assessed using Kaplan-Meier analyses with a maximum of 10.8 years follow-up.PSD · Nov 2016
4.4 Merck Sharp & Dohme (MSD) confirmed an ex-manufacturer price for vorinostat 100 mg x 120 of $'''''''''''''''''''''. This corresponds to a Dispensed Price for Maximum Quantity (DPMQ) of $'''''''''''', which was the same as the DPMQ proposed in the November 2016 submission.PSD · Mar 2017
4.5 At the price proposed in the 2011 submission for vorinostat and with a responder rate of 49% based on ≥ 25% decrease in the modified Severity-Weighted Assessment Tool (mSWAT), the cost per responder was $''''''''''''''''.PSD · Mar 2017
6.2 The PBAC noted and welcomed the input from individuals (2) and an organisation (1) via the Consumer Comments facility on the PBS website. The comments noted support for availability of this drug and a hope for improved quality of life under treatment.PSD · Nov 2016
6.3 Representatives of the PBAC also met with Rare Cancers Australia prior to the PBAC meeting. The meeting covered the PBAC consideration of romidepsin for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) (Romidepsin PSD, November 2016 refers) and vorinostat for the treatment of patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL).PSD · Nov 2016
Cost-effectiveness
ICER values redacted throughout the document (indicated by '''''''''). Cost per responder calculated at prior pricing ($'''''''''''''''), but not in ICER format per QALY or LY.
the PBAC noted that no comparative analysis against either comparator was presented. PBAC · 2016
Decision context
PopulationPatients with cutaneous T-cell lymphoma with relapsed or chemotherapy-refractory disease, who have received prior systemic chemotherapy and are ineligible for stem cell transplantation, intended for curative treatment.
Risk sharingPatient number cap with rebate mechanism: less than 10,000 patients in Year 1 (including grandfathered MSD EAP patients only in Year 1), with a 70% rebate for expenditure above the cap to address risk of use beyond progression or in other indications.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2017 | Recommended · restricted | — | — | Other · ORR |
| Nov 2016 | Deferred | no active therapy | — | Single-arm · ORR |
| Mar 2011 | Not recommended | palliative care (though PBAC considered best available care/chemotherapy more appropriate) | $75k–105k | RCT | Single-arm · QoL | Surrogate |
Consumer voice
Two individuals and Rare Cancers Australia provided input supporting availability of vorinostat and romidepsin for treatment of relapsed/refractory T-cell lymphomas, highlighting limited treatment options, high toxicity of alternatives, profound quality-of-life impacts from physical manifestations, inequitable access due to high out-of-pocket costs, and good tolerability of these drugs.
There are currently limited treatment options for patients with relapsed or refractory PTCL and CTCL, and available treatment options have high toxicity. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to chemotherapy-refractory disease, excludes stem cell transplant candidates, and prohibits concomitant systemic therapies; TGA label has no such exclusions.