TEPROTUMUMAB

The PBAC recommended the listing of teprotumumab on the PBS for the treatment of active, moderate-to-severe thyroid eye disease (TED). The PBAC reaffirmed its previous view that there is a high clinical need for patients with this condition. 

The PBAC welcomed comments from health care professionals, consumers seeking access to teprotumumab and the Orbital Plastics and Lacrimal (OPAL) and Ocular Rheumatology (EORC) units from the Royal Victorian Eye and Ear Hospital (RVEEH). Their comments emphasised the high clinical need for effective treatment options for patients living with active  moderate-to-severe TED. The comments described the substantial burden of TED and highlighted the limited effectiveness and safety concerns associated with current therapies. The PBAC noted comments also described teprotumumab as a targeted therapy with clinical benefits observed in trials, although hearing impairment was identified as a key adverse effect requiring careful patient selection and monitoring.

The PBAC noted the PBS listing for this population, recommended at its May 2025 meeting, could not be progressed, as the previously proposed price was no longer considered feasible by the sponsor. The PBAC considered that the revised average vial use per treatment course was highly uncertain and likely underestimated utilisation in practice. The PBAC considered that a price reduction would be required to achieve a cost-effective listing, together with changes to the risk sharing arrangement (RSA) to manage uncertainty related to the estimated vial usage per patient.