Eflornithine
Post-maintenance treatment to prevent relapse in patients with high-risk neuroblastoma (HRNB) who are in remission after prior multiagent, multimodality therapy.
Decision on record
- Meeting Mar 2025 Recommended High-risk neuroblastoma, post-maintenance treatment to prevent relapse
Access path
- Mar 2025Recommended · restricted
vs standard of care (SOC)
- PBS listing · Authority Required
From the public summary
The PBAC recommended the Section 100 (Highly Specialised Drugs Program) listing of eflornithine for post-maintenance treatment to prevent relapse in patients with high- risk neuroblastoma (HRNB) who are in remission after receiving multiagent, multimodality therapy. The PBAC acknowledged the high clinical need for a post- maintenance treatment to prevent relapse of this condition.PSD · Mar 2025
The PBAC is satisfied that eflornithine provides, for some patients, a significant improvement in efficacy over the main comparator, SOC, in the post-maintenance treatment of HRNB, albeit with inferior safety.PSD · Mar 2025
The submission presented a cost-utility analysis (CUA) using a partitioned survival analysis model with four health states: event free (EF) disease, relapse, long-term remission (LT-remission), and death. The submission modelled the Stratum 1 and Stratum 2 populations separately using the same model structure but different parameters. The model diagram is presented in Figure 3. 21PSD · Mar 2025
Table 7: Summary of model structure, key inputs and rationale Component Summary Treatments Eflornithine vs SOC (no active treatment) 100 years in the model base case vs. median OS follow up 7.21 years in Study 3b Stratum 1 (Stratum 2 not reported) and 5.85 years in Study ANBL0032. The evaluation noted that while a lifetime model Time horizon may be reasonable, the extrapolation to 100 years was uncertain compared to the length of the study data.PSD · Mar 2025
Source: Table1-1, p18 of the submission. a Defined in Study NMTRC003b as: chemotherapy (5-7 cycles), surgery as indicated, consolidation therapy as indicated, radiation therapy as indicated, or anti-ganglioside 2 (GD2) antibody therapy with retinoic acid up to 6 cycles. b Defined as any patient who received additional therapy due to a suboptimal response to standard therapy.PSD · Mar 2025
The submission described eflornithine as superior in terms of effectiveness compared with SOC and inferior but manageable in terms of safety compared to SOC for both Stratum 1 and Stratum 2 patients.PSD · Mar 2025
The Australian and New Zealand Children’s Haematology/ Oncology Group (ANZCHOG) Solid Tumour Group described neuroblastoma as one of the most challenging childhood cancers that accounts for a significant proportion of paediatric cancer mortality in Australia. It supported the listing of eflornithine on the PBS, describing it as a critical therapy that requires equitable and timely access.PSD · Mar 2025
The National Paediatric Medicines Forum (NPMF) stated that the PBS listing of eflornithine will be immensely beneficial to all relevant paediatric patients diagnosed with HRNB as well as improving equitable access and outcomes for all stakeholders.PSD · Mar 2025
Cost-effectiveness
ICER value not stated in the public text; document indicates cost-effectiveness analysis was performed but specific ICER range not disclosed.
Decision context
PopulationPatients aged 1 year and older with high-risk neuroblastoma (HRNB) who have responded to prior multiagent, multimodality therapy and are in remission either at the end of upfront therapy or after any previous relapse or refractory therapy.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended · restricted | standard of care (SOC) | — | RCT · EFS, OS |
Consumer voice
Consumer input from 262 individuals, 3 healthcare professionals, and 5 organisations supported PBS listing of eflornithine for high-risk neuroblastoma. Key organisations including ANZCHOG and NPMF emphasised neuroblastoma as a serious childhood cancer requiring equitable access to this critical therapy.
neuroblastoma as one of the most challenging childhood cancers that accounts for a significant proportion of paediatric cancer mortality in Australia Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients in remission; TGA label includes all who have responded, not necessarily in remission.