DeferasiroxAKM DEFERASIROX
Chronic iron overload due to disorders of haemopoiesis, including transfusion-dependent malignant and non-malignant disorders of erythropoiesis, and non-transfusion-dependent thalassaemia.
Decisions on record
- Meeting Nov 2020 Recommended Chronic iron overload
- Meeting Jul 2018 Recommended Chronic iron overload
- Meeting Jul 2015 Recommended Chronic iron overload
- Meeting Nov 2014 Recommended Exjade® Novartis Pharmaceuticals Australia Pty Ltd
Access path
- TGA registered · AKM DEFERASIROX
TGA label narrower than the PBS population
- Jul 2006Recommended
vs desferrioxamine mesylate powder for injection
- Jul 2006Recommended · restricted
- Nov 2014Recommended · restricted
Comparator changed: desferrioxamine mesylate powder for injection → desferrioxamine
- Jul 2015Deferred
Comparator changed: desferrioxamine → best supportive care without iron chelation…
- Jul 2018Recommended · restricted
Comparator changed: best supportive care without iron chelation therapy; placebo (for…
- Nov 2020Recommended
Comparator changed: deferasirox dispersible tablets → Jadenu (deferasirox 90 mg, 180 mg…
- PBS listing · Restricted
From the public summary
6.1 The PBAC recommended the listing of DFXJ on the S100 HSD Program for patients with chronic iron overload due to disorders of haemopoiesis on a cost-minimisation basis to Jadenu.PSD · Nov 2020
6.3 The PBAC noted that costs to the PBS would be incurred because DFXJ has 28 tablets per pack compared to Jadenu which has 30 tablets, and therefore prescription numbers for DFXJ would be higher. The PBAC considered that, given this recommendation is on a cost-minimisation basis, the listing should not result in any additional cost to government, as it is expected to share the market with Jadenu.PSD · Nov 2020
5.9 As a minor submission, the economic analysis has not been independently evaluated.PSD · Nov 2020
5.10 The minor submission proposed the same price per tablet between the equi-effective doses of Jadenu and DFXJ (see paragraph 4.4). The proposed pricing is shown in Table 2.PSD · Nov 2020
5.7 The submission claimed non-inferior comparative effectiveness and non-inferior comparative safety of DFXJ compared with Jadenu. 3PSD · Nov 2020
5.8 The PBAC considered that the claim of non-inferior comparative effectiveness and safety was reasonably supported by the data.PSD · Nov 2020
5.2 The PBAC noted and welcomed the input from individuals (6) and health care professionals (2) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with deferasirox film coated tablets including improved compliance, fewer side effects and increased treatment options. Pharmacokinetic dataPSD · Jul 2018
5.3 Pharmacokinetic data was presented from three Phase I studies, for which references were not provided. They are described in the table below. 3PSD · Jul 2018
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated as the submission was on a cost-minimisation basis with Jadenu as comparator.
The PBAC noted that its recommendation was on a cost-minimisation basis and advised that, because DFXJ is not expected to provide a substantial and clinically relevant improvement in efficacy, or reduction of toxicity, over Jadenu, or not expected to address a high and urgent unmet clinical need given the presence of an alternative therapy, the criteria prescribed by the National Health (Pharmaceuticals and Vaccines – Cost Recovery) Regulations 2009 for Pricing Pathway A were not met. PBAC · 2020
Decision context
PopulationPatients with chronic iron overload due to disorders of haemopoiesis: transfusion-dependent malignant disorders of haemopoiesis with median life expectancy exceeding five years; transfusion-dependent non-malignant disorders of erythropoiesis; and non-transfusion-dependent thalassaemia.
Risk sharingSpecial Pricing Arrangements (SPA) apply to the listing, as noted in the administrative advice sections for each restriction.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2020 | Recommended | Jadenu (deferasirox 90 mg, 180 mg, 360 mg film coated tablets) | — | Single-arm · Bioequivalence |
| Jul 2018 | Recommended · restricted | deferasirox dispersible tablets | — | RCT · Safety |
| Jul 2015 | Deferred | best supportive care without iron chelation therapy; placebo (for NTDT) | — | Single-arm · OS |
| Nov 2014 | Recommended · restricted | desferrioxamine | $15k–45k | RCT · QoL |
| Jul 2006 | Recommended | desferrioxamine mesylate powder for injection | $15k–45k | RCT · OS | PFS | DFS | ORR | QoL | Surrogate |
| Jul 2006 | Recommended · restricted | desferrioxamine mesylate powder for injection | $15k–45k | RCT |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| THALASSA | Ph 2 | 166 | Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52 | completed |
Consumer voice
No consumer comments were received for this item.
The PBAC noted that no consumer comments were received for this item. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to transfusion-dependent malignant disorders with >5-year life expectancy, transfusion-dependent non-malignant disorders, and non-transfusion-dependent thalassaemia only.