IvosidenibTibsovo
Treatment of locally advanced or metastatic cholangiocarcinoma in patients with an IDH1 R132 variant whose disease has progressed on at least one prior line of systemic therapy.
Decisions on record
- Meeting Nov 2024 Recommended Bile duct cancer (cholangiocarcinoma) with IDH1 mutation, locally advanced or metastatic
- Meeting Jul 2024 Not recommended Locally advanced or metastatic cholangiocarcinoma with IDH1 mutation
Access path
- TGA registered · Tibsovo
TGA label narrower than the PBS population
- Jul 2024Not recommended
vs palliative care / best supportive care
- ↻ resubmittedNov 2024Recommended
Comparator changed: palliative care / best supportive care → palliative care (primary)…
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended ivosidenib, for treatment of locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation, in patients who have previously progressed on chemotherapy. The PBAC considered that there was a high clinical need for treatments for patients with locally advanced or metastatic cholangiocarcinoma, who have a very poor prognosis.PSD · Nov 2024
5.2 The PBAC considered that the proposed restriction criteria were appropriate but that a combined listing including initial, continuing and grandfather treatment would be preferred for simplicity.PSD · Nov 2024
4.7 To address the PBAC’s concerns regarding the cost-effectiveness of ivosidenib the resubmission presented an economic evaluation with revised inputs for the time horizon and overall survival extrapolation functions and a reduced price for ivosidenib (% reduction, from $ to $ AEMP). The base case incremental cost-effectiveness ratio (ICER) in the resubmission was $55,000 to < $75,000 per quality-adjusted life year (QALY).PSD · Nov 2024
4.8 The resubmission did not fully revise the model as requested by the PBAC:PSD · Nov 2024
testing and untargeted treatment/palliative care in patients with locally advanced or metastatic CCA.PSD · Nov 2024
Source: Table 1-2, p31 of the July 2024 submission For more detail on PBAC’s view, see section 5 PBAC outcome.PSD · Nov 2024
4.2 The PBAC noted and welcomed the input from individual (12), and organisational (5) contributors via the Consumer Comments facility on the PBS website. The PBAC also recalled consumer comments received in relation to the July 2024 consideration of ivosidenib.PSD · Nov 2024
4.3 The PBAC noted the advice received from Pancare Foundation, Liver Foundation and Cholangiocarcinoma Foundation Australia in support of the ivosidenib submission.PSD · Nov 2024
Cost-effectiveness
ICER range stated in base case results: $55,000 to < $75,000 per QALY
The PBAC previously considered that ivosidenib would be cost-effective with an ICER of no more than $75,000 to < $95,000/QALY. PBAC · 2024
Decision context
PopulationAdult patients with locally advanced or metastatic cholangiocarcinoma with confirmed IDH1 R132 tier I variant who have disease progression following at least one line of systemic chemotherapy, with WHO performance status 2 or less.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended | palliative care (primary); chemotherapy with FOLFOX or FOLFIRI (secondary) | $55k–75k | RCT · OS |
| Jul 2024 | Not recommended | palliative care / best supportive care | — | RCT · OS |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| ClarIDHy | Ph 3 | 187 | Progression Free Survival (PFS) as Determined by the Independent Radiology Committee (IRC) | completed |
Codependent tests
| Service | Biomarker | MSAC outcome | Year |
|---|---|---|---|
| IDH1 variant testing in cholangiocarcinoma | IDH1 p.R132X tier I variant | deferred | 2024 |
Consumer voice
Consumer input from 12 individuals and 5 organisations indicated that ivosidenib is considered effective in slowing disease progression and less toxic than chemotherapy, but costs are prohibitive without PBS subsidisation. Consumers emphasised the poor prognosis of cholangiocarcinoma, limited treatment options, serious side effects of current treatments, high financial burden, and the meaningful v
Comments indicated that current treatment options have limited efficacy and ivosidenib is considered effective in slowing disease progression and is less toxic than chemotherapy in the second line (2L) setting, but costs are prohibitive for patients without PBS subsidisation. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC adds WHO performance status ≤2 restriction and specifies tier I variant, not present in TGA label.