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 recommended Authority Required (Streamlined) listing for selpercatinib for the treatment of advanced or metastatic rearranged during transfection (RET) fusion- positive non-small cell lung cancer (NSCLC).PSD · Jul 2024
7.2 The PBAC noted the input from health care professionals, the Lung Foundation 43PSD · Jul 2024
6.51 The resubmission presented a stepped economic evaluation of selpercatinib compared with pembrolizumab + PC as first-line therapy for the treatment of RET fusion-positive, locally advanced or metastatic NSCLC based on an indirect comparison 25PSD · Jul 2024
6.52 The type of economic evaluation presented was a cost-effectiveness analysis and a cost-utility analysis. The resubmission presented a partitioned survival model, which was the same basic model structure as the July 2023 submission.PSD · Jul 2024
6.44 The resubmission described selpercatinib as superior in terms of effectiveness compared with pembrolizumab + PC in patients with advanced RET fusion-positive NSCLC based on the new head-to-head LIBRETTO-431 trial data. In July 2023, the PBAC could not support the claim of superior efficacy as the ITCs presented were associated with a high degree of uncertainty (paragraph 7.1, selpercatinib PSD, July 2023 PBAC meeting).PSD · Jul 2024
6.45 Patients from the LIBRETTO-431 trial treated with selpercatinib demonstrated a statistically significant (p<0.05) improvement in PFS when compared to patients in the pembrolizumab + PC arm in the ITT-pembrolizumab population and ITT-population (incremental PFS = 13.67 months).PSD · Jul 2024
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 | — | 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).