TrabectedinYONDELIS
Advanced (unresectable and/or metastatic) leiomyosarcoma in patients who have received prior chemotherapy treatment including an anthracycline.
Decisions on record
- Meeting Jul 2023 Withdrawn Leiomyosarcoma no PSD
- Meeting Jul 2021 Recommended Advanced unresectable/metastatic leiomyosarcoma and liposarcoma
Access path
- TGA registered · YONDELIS
TGA label narrower than the PBS population
- Jul 2021Recommended · restricted
vs pazopanib
- PBS listing · Authority Required
From the public summary
The PBAC recommended the Section 100 (Efficient Funding of Chemotherapy), Authority Required (STREAMLINED) listing of trabectedin for the treatment of leiomyosarcoma (LMS). Noting the evidence presented for trabectedin included a population with liposarcoma (LPS) and that the treatment effect was similar across both sarcomas, together with the high clinical need for additional treatment options for LPS that is a rare and aggressive cancer, the PBAC also …PSD · Jul 2021
The PBAC’s recommendation for listing in LMS was based on, among other matters, its assessment, that the cost-effectiveness of trabectedin would be acceptable if it were cost-minimised against pazopanib. The PBAC’s recommendation for listing in LPS was based on, among other matters, its assessment, that the cost-effectiveness of trabectedin would be acceptable if it were priced the same as for the LMS population.PSD · Jul 2021
The submission presented a cost-minimisation analysis of trabectedin compared with pazopanib. The key components and assumptions of the cost-minimisation analysis are summarised below.PSD · Jul 2021
Table 7: Key components and assumptions of the cost-minimisation analysis Component Claim or assumption Therapeutic claim: effectiveness Based on evidence presented, efficacy is assumed to be non-inferior to pazopanib.PSD · Jul 2021
containing regimen, trabectedin is as effective as pazopanib at improving progression-free survival. Source: Table 1 of the submission.PSD · Jul 2021
The submission described trabectedin as non-inferior in terms of effectiveness and safety compared to pazopanib in the treatment of LMS. The presented evidence did not adequately support the therapeutic conclusion presented in the submission.PSD · Jul 2021
The PBAC noted and welcomed the input from health care professionals (7) and organisations (2) via the Consumer Comments facility on the PBS website. The comments described the need for additional treatment options for LMS (and also LPS, for which PBS subsidy was not specifically sought) and highlighted that trabectedin is currently being used in clinical practice through the TGA Special Access Scheme at a great financial cost to patients.PSD · Jul 2021
The PBAC noted the advice received from Rare Cancers Australia, which highlighted the often poor prognosis associated with LMS and the need for additional therapeutic options for treating this rare condition.PSD · Jul 2021
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated by design.
The submission presented a cost-minimisation analysis of trabectedin compared with pazopanib. PSD · 2021
Decision context
PopulationAdults aged 18 years or older with advanced (unresectable and/or metastatic) leiomyosarcoma who have received prior chemotherapy treatment including an anthracycline, with ECOG performance status of 2 or less.
Risk sharingSpecial Pricing Arrangement (SPA) discussed; sponsor indicated willingness to negotiate an SPA for trabectedin based on the SPA for pazopanib.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2021 | Recommended · restricted | pazopanib | — | RCT · PFS |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| TSAR | Ph 3 | 103 | Progression Free Survival | completed |
Consumer voice
Healthcare professionals and organisations highlighted the need for additional treatment options for leiomyosarcoma, noting that trabectedin is currently accessed through the TGA Special Access Scheme at significant financial cost to patients. Rare Cancers Australia emphasised the poor prognosis of the condition and the need for new therapeutic options.
trabectedin is currently being used in clinical practice through the TGA Special Access Scheme at a great financial cost to patients Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to leiomyosarcoma only (excludes liposarcoma) and adds ECOG performance status ≤2 requirement.