CarfilzomibKyprolis
Treatment of relapsed or refractory multiple myeloma (RRMM) after at least one prior therapy, in combination with lenalidomide and dexamethasone.
Decisions on record
- Meeting Mar 2022 Recommended Multiple myeloma
- Meeting Jul 2020 Recommended Multiple myeloma (MM)
- Meeting Nov 2018 Not recommended Multiple myeloma
- Meeting Mar 2018 Recommended Multiple myeloma
- Meeting Jul 2017 Recommended Multiple myeloma
- Meeting Nov 2016 Not recommended Multiple myeloma
Access path
- TGA registered · Kyprolis
TGA label narrower than the PBS population
- Nov 2016Not recommended
vs Bortezomib with dexamethasone (Bd) for Cd regimen; placebo…
- ↻ resubmittedJul 2017Recommended · restricted
Comparator changed: Bortezomib with dexamethasone (Bd) for Cd regimen; placebo plus…
- Mar 2018Recommended · restricted
Evidence: RCT → Other
- Nov 2018Not recommended
deficiencies in stakeholder consultation, evidence evaluation and cost assessment, insufficient evidence from ENDEAVOR…
- ↻ resubmittedJul 2020Recommended · restricted
Listing: Restricted → Authority Required
- Mar 2022Recommended
Comparator changed: Cd56 BIW (carfilzomib 56 mg/m² twice weekly with dexamethasone) →…
- PBS listing · Authority Required
From the public summary
The PBAC recommended the listing of carfilzomib in combination with lenalidomide and dexamethasone (CLd) for the treatment of relapsed or refractory multiple myeloma (RRMM), on the basis that it should be available only under special arrangements under Section 100 (Efficient Funding of Chemotherapy). Listing was recommended on the basis of a cost-minimisation approach (CMA) versus carfilzomib 41PSD · Mar 2022
The PBAC noted and welcomed the input from consumers and organistions via the Consumer Comments facility which supported the CLd resubmission and described the desire for alternate RRMM therapies.PSD · Mar 2022
ICER was in excess of $ 1/QALY The resubmission requested a revision to the carfilzomib RSA expenditure caps to allow for other regimens which have been cost-minimised to Cd to share these caps based on a precedent set with PBd.PSD · Mar 2022
Submission presented combined estimates for The resubmission stated that the proposed both Cd and CLd. The submission assumed expenditure caps will not result in an increase 20% of patients would use Cd and 80% would use CLd overall PBS/RPBS cost to the RRMM market.PSD · Mar 2022
Indirect treatment comparison - CLd vs Cd On the basis of the ITC, the resubmission claimed that CLd was non-inferior in terms of effectiveness compared to Cd.PSD · Mar 2022
The ESC considered that the clinical claim in terms of comparative effectiveness was likely supported, noting that there were uncertainties relating to:PSD · Mar 2022
The PBAC noted and welcomed the input from individuals (12) and organisations (3) via the Consumer Comments facility on the PBS website. The comments described the impact of multiple myeloma on patients and their families, and a desire for access to additional treatments for patients with RRMM.PSD · Mar 2022
The PBAC noted the comments from Myeloma Australia’s Medical and Scientific Advisory Group (MSAG), which supported the proposed listing of CLd on the basis of the safety and efficacy results from the ASPIRE trial. The PBAC noted the comments from Myeloma Australia, which strongly supported the proposed listing and 11PSD · Mar 2022
Cost-effectiveness
Cost-minimisation approach used; no ICER calculated by design.
The PBAC noted the proposed change to the pomalidomide restriction was not supported by trial evidence, and there is a risk of loss of cost-effectiveness for pomalidomide use. PBAC · 2018
Decision context
PopulationAdults with relapsed or refractory multiple myeloma who have received at least one prior therapy, receiving carfilzomib in combination with lenalidomide and dexamethasone.
Risk sharingSpecial Pricing Arrangements apply; Section 100 (Efficient Funding of Chemotherapy) listing with streamlined Authority Required restrictions.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2022 | Recommended | carfilzomib in combination with dexamethasone (Cd) | — | RCT · PFS |
| Jul 2020 | Recommended · restricted | Cd56 BIW (carfilzomib 56 mg/m² twice weekly with dexamethasone) | — | RCT · ORR |
| Nov 2018 | Not recommended | — | — | RCT · ORR |
| Mar 2018 | Recommended · restricted | — | — | Other |
| Jul 2017 | Recommended · restricted | bortezomib + dexamethasone | — | RCT · OS |
| Nov 2016 | Not recommended | Bortezomib with dexamethasone (Bd) for Cd regimen; placebo plus lenalidomide and dexamethasone for CLd regimen | — | RCT · Other |
Clinical evidence
Consumer voice
Consumers and patient organisations (12 individuals, 3 organisations) supported listing of carfilzomib (CLd) for relapsed/refractory multiple myeloma, emphasising the importance of treatment access and remission outcomes. Some consumers reported cardiac toxicity cases with carfilzomib.
The comments described the impact of multiple myeloma on patients and their families, and a desire for access to additional treatments for patients with RRMM. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to relapsed/refractory disease and mandates specific combination therapy (lenalidomide + dexamethasone), whereas TGA label permits any patient with ≥1 prior therapy.