EdaravoneRadicava
Treatment of amyotrophic lateral sclerosis (ALS) in adults who are independent in activities of daily living, have normal respiratory function, and where treatment is initiated within two years of disease onset.
Decisions on record
- Meeting Mar 2026 Recommended Amyotrophic lateral sclerosis (ALS) no PSD
- Meeting Mar 2024 Recommended Amyotrophic lateral sclerosis (ALS)
- Meeting Nov 2023 Not recommended Amyotrophic lateral sclerosis
Access path
- TGA registered · Radicava
TGA label narrower than the PBS population
- Nov 2023Not recommended
vs placebo (standard of care with or without riluzole)
- ↻ resubmittedMar 2024Recommended · restricted
Comparator changed: placebo (standard of care with or without riluzole) → current…
- Dec 2025Recommended · restricted
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended the listing of edaravone, for treatment of amyotrophic lateral sclerosis (ALS) in patients who are independent in activities of daily living and where treatment is initiated within two years of disease onset, on the basis that it should be available only under special arrangements under Section 100. The PBAC recommended Community Access on the HSD program for continuing treatment only.PSD · Mar 2024
The PBAC acknowledged the high clinical need for effective treatments for amyloid lateral sclerosis (ALS). The PBAC recalled that in November 2023 it did not recommend edaravone for this indication, noting that edaravone was not cost-effective at the price proposed in the submission and that the economic model included a number of optimistic assumptions that were likely to underestimate the incremental cost- effectiveness ratio.PSD · Mar 2024
4.13 The base case incremental cost-effectiveness ratio (ICER) for the revised model was $95,000 to < $115,000 per QALY. The PBAC previously considered that in the context of the high clinical need an ICER of up to $95,000 to < $115,000 per QALY gained would be considered acceptable (paragraph 7.15, edaravone PSD, November 2023 PBAC meeting).PSD · Mar 2024
4.14 In the base case model presented, the resubmission removed the mortality calibration as requested by the PBAC. The removal of the mortality calibration reduced the discounted incremental QALY gain from 0.76 QALYs to 0.34 QALYs. The base case economic evaluation included an overall survival benefit of 0.52 years (undiscounted) over a 10-year time horizon.PSD · Mar 2024
has similar (non-inferior) safety compared to standard of care with or without riluzole. Source: Table 1-1, p3 of the November 2023 submission.PSD · Mar 2024
ALS=amyotrophic lateral sclerosis; ALSFRS-R=ALS Functional Rating Scale – Revised For more detail on PBAC’s view, see section 5 PBAC outcome.PSD · Mar 2024
4.3 Motor Neurone Disease Australia noted that edaravone has been shown to be clinically effective and safe and has been approved for use in motor neurone disease (MND) in several other countries including the United States, Canada and Japan. The comments noted that subsequent real-world data analysis has shown a material improvement in overall survival with edaravone, as has a systematic review and meta- analysis.PSD · Mar 2024
4.4 Two health care professionals noted the current lack of effective treatment options for ALS and emphasised the importance of slowing disease progression with edaravone at an early stage of the disease. The health care professionals noted the effect of ALS on patients and families, stating that edaravone allowed patients to remain well for longer rather than living with advanced disability.PSD · Mar 2024
Cost-effectiveness
This is a minor submission for an administrative/access change to an existing HSD listing, not a new therapeutic evaluation. No new economic model or ICER calculation was performed.
Decision context
PopulationAdults with amyotrophic lateral sclerosis diagnosed by a neurologist, with symptom duration of not more than 2 years, at least 80% of predicted forced vital capacity, able to ambulate and use upper limbs without assistance, without tracheostomy or respiratory failure, and with ALSFRS-R score of at least 2 on each individual item.
Risk sharingExisting Risk Sharing Arrangement (RSA) provides for redacted percentage reimbursement for use beyond the caps based on original financial estimates; any additional expenditure would potentially be mitigated under the existing RSA.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Dec 2025 | Recommended · restricted | — | — | — |
| Mar 2024 | Recommended · restricted | current standard of care with or without riluzole | $95k–115k | RCT · ALSFRS-R |
| Nov 2023 | Not recommended | placebo (standard of care with or without riluzole) | — | RCT · ALS Functional Rating Scale (ALSFRS-R) after 6 cycles (6 months) |
Consumer voice
Motor Neurone Disease Australia, two healthcare professionals, and 54 individuals (ALS patients, family members, and carers) submitted comments supporting edaravone listing, emphasizing its clinical effectiveness, safety, real-world improvements in survival, and the urgent need for treatment options given the devastating nature of ALS and its impact on quality of life.
edaravone has been shown to be clinically effective and safe and has been approved for use in motor neurone disease (MND) in several other countries including the United States, Canada and Japan. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC adds specific functional requirements (ALSFRS-R ≥2 per item, FVC/SVC ≥80%) and excludes tracheostomy/prior respiratory failure, narrowing TGA's broader criteria.