EDARAVONE

The PBAC recommended adding a Section 100 (Highly Specialised Drugs (HSD) Community Access (CA)) listing of edaravone for initial treatment of amyotrophic lateral sclerosis, enabling community-based neurologists to initiate therapy. The PBAC considered this change to improve flexibility and equitable access for patients through community and hospital settings, particularly in rural and remote areas. The PBAC advised that the existing Section 100 HSD Public and Private Hospital listings should be removed to avoid multiple PBS item codes, as the CA listing will also continue to allow access in hospital settings.
The PBAC recommended including a caution regarding rare but clinically significant hypersensitivity risks, such as anaphylaxis and severe asthmatic episodes in susceptible individuals based on input received from clinical representative group for this treatment area. 
The PBAC considered that patients who commenced edaravone privately and met eligibility criteria at initiation (i.e., grandfathered patients) were intended to be able to transition to PBS-subsidised therapy under the existing restrictions. The PBAC noted the sponsor had indicated the wording of the existing restrictions may require further clarification to ensure the intention of the listing is clear for prescribers of these patients. To support this, the PBAC advised amending the initial treatment criteria for clarity, enabling these patients to receive their first PBS supply under the initial treatment restriction and continue therapy under the continuing treatment restriction. The PBAC advised that prescribers should request quantities sufficient for one month per dispensing, aligned with the treatment cycle. 
The PBAC considered the financial impact of these changes to be minor as the patient groups were included in the original estimates, with only a small additional cost to the PBS due to differences in fees and mark-ups between the Section 100 HSD Public hospital and CA programs which it considered would be mitigated by the existing Risk Sharing Arrangement.