SelpercatinibRetevmo
Advanced or metastatic medullary thyroid cancer (MTC) with RET variants in patients with unresectable disease and documented disease progression.
Decisions on record
- Meeting Mar 2026 Recommended Medullary thyroid cancer (MTC)
- Meeting Jul 2024 Recommended Advanced or metastatic RET fusion-positive non-small cell lung cancer
- Meeting Jul 2023 Not recommended Non-small cell lung cancer (NSCLC)
Access path
- TGA registered · Retevmo
TGA label narrower than the PBS population
- Jul 2023Not recommended
vs pembrolizumab in combination with pemetrexed +…
- ↻ resubmittedJul 2024Recommended · restricted
Comparator changed: pembrolizumab in combination with pemetrexed + platinum-based…
- Mar 2026Recommended · restricted
Comparator changed: pembrolizumab in combination with platinum-based doublet chemotherapy…
- PBS listing · Restricted
From the public summary
7.1 The PBAC deferred making a recommendation for selpercatinib for the treatment of patients with unresectable and progressive locally advanced or metastatic medullary thyroid cancer (MTC) and evidence of RET variants. The PBAC deferred with a mind to recommend, pending MSAC consideration of the codependent test for RET variants.PSD · Mar 2026
The PBAC considered there was a high clinical need for patients with progressive MTC not suited to local therapy, as there are no PBS-listed treatment options for this aggressive and rare disease. The PBAC accepted the significant progression free survival (PFS) benefit observed for selpercatinib compared to best supportive care but considered the magnitude of the overall survival (OS) benefit was highly uncertain.PSD · Mar 2026
6.45 The submission presented a modelled economic evaluation, based on an indirect comparison of selpercatinib and BSC in a population of patients with advanced or metastatic MTC who have a confirmed RET variant.PSD · Mar 2026
6.46 The type of economic evaluation presented was a cost utility analysis (CUA).PSD · Mar 2026
to placebo in terms of efficacy and has an inferior, but clinically manageable, safety profile.PSD · Mar 2026
1.3 The populations eligible for testing and treatment were inconsistently defined throughout the submission. The ESCs agreed with the evaluation that it was appropriate that: 1 OFFICIALPSD · Mar 2026
6.2 The PBAC noted and welcomed the input from health care professionals (3) and organisations (3) via the Consumer Comments facility on the PBS website. The comments from the health care professionals described the potential efficacy and quality of life benefits with selpercatinib. It was also stated that selpercatinib was associated with minimal adverse events.PSD · Jul 2024
6.3 Input was again received from Rare Cancers Australia and the Lung Foundation Australia. Rare Cancers Australia highlighted that patients with NSCLC with rare mutations are often faced with an extremely poor prognosis and stated that such patients need treatment options that provide significant improvements in survival.PSD · Jul 2024
Cost-effectiveness
ICER value not stated in the public summary document.
Decision context
PopulationPatients with histologically and cytologically confirmed advanced or metastatic MTC with a RET alteration (somatic or germline), WHO ECOG PS ≤2, unresectable disease, and documented disease progression.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Recommended · restricted | best supportive care | — | RCT |
| Jul 2024 | Recommended · restricted | pembrolizumab in combination with platinum-based doublet chemotherapy | $75k–95k | RCT · PFS |
| Jul 2023 | Not recommended | pembrolizumab in combination with pemetrexed + platinum-based chemotherapy (first-line); docetaxel (pre-treated setting) | — | Single-arm · ORR |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| LIBRETTO-001 | Ph 1, PHASE2 | 857 | Phase 1: Maximum Tolerated Dose (MTD) | active not recruiting |
| LIBRETTO-431 | Ph 3 | 261 | Progression Free Survival (PFS) by Blinded Independent Central Review (BICR) (With Pembrol… | active not recruiting |
Consumer voice
Health care professionals and patient advocacy organisations supported selpercatinib listing, highlighting efficacy, quality of life benefits, minimal adverse events, and accessibility advantages for patients with rare NSCLC mutations, particularly those in rural and remote areas.
patients with NSCLC with rare mutations are often faced with an extremely poor prognosis and stated that such patients need treatment options that provide significant improvements in survival Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line treatment; TGA label permits any line (locally advanced or metastatic).
Listed in law
PBS listings commencing 1 Feb 2025 were made by these 6 determinations under the National Health Act 1953. A determination covers every listing that commenced that day, so this is the legal instrument in force for the date — not a document naming this medicine. Open it and check.
| Determination | Made | Status | |
|---|---|---|---|
| PB 1 of 2025 | 30 Jan 2025 | Repealed | PDF ↗ |
| PB 1 of 2025 | 30 Jan 2025 | Repealed | PDF ↗ |
| PB 2 of 2025 | 30 Jan 2025 | Repealed | PDF ↗ |
| PB 2 of 2025 | 30 Jan 2025 | Repealed | PDF ↗ |
| PB 3 of 2025 | 30 Jan 2025 | Repealed | PDF ↗ |
| PB 3 of 2025 | 30 Jan 2025 | Repealed | PDF ↗ |