Clinicians warn PBS bureaucracy is blocking innovative oncology treatments

The Department of Health has released an outcome statement from the Multiple Myeloma Stakeholder Meeting held back in July 2025, revealing stark tensions between clinical guidelines and access to innovative therapies.

The meeting followed the March 2025 PBAC consideration of submissions for J&J Innovative Medicine’s Darzalex (daratumumab) and Pfizer’s Elrexfio (elranatamab), exposing a landscape where regulatory bureaucracy is slowing access to the most effective oncology treatments.

Stakeholders painted a picture of dramatic evolution in MM and RRMM treatment, moving away from cytotoxic chemotherapy toward novel therapies such as immunomodulatory drugs (IMiDs), proteasome inhibitors (PIs), anti-CD38 monoclonal antibodies, and BCMA-targeted agents. PBS expenditure on multiple myeloma in 2024–25 hit $341 million – 11% of total antineoplastic spending – highlighting both the scale of disease burden and the cost pressures on government.

While government policy has curbed overall expenditure through statutory price reductions and first-new-brand reductions, clinicians warned that cost containment is coming at the expense of patient-centred flexibility.

The PBS restrictions, acting as quasi-clinical guidelines, fail to reflect contemporary treatment realities. Clinicians emphasised that effective myeloma care must be personalised. The rigid 1st/2nd/3rd line PBS nomenclature is no longer fit for purpose, and treatment sequencing should hinge on prior drug exposure rather than arbitrary lines of therapy. Limiting PBS prescribing to haematologists was proposed as a safeguard against unsafe or suboptimal prescribing, particularly as immune therapies become more toxic.

Stakeholders decried restrictions on anti-CD38 monoclonal antibodies. The March 2025 PBAC recommendation for first-line daratumumab with lenalidomide and dexamethasone for transplant-ineligible patients, still unimplemented, may see patients bypass transplantation altogether.

Clinicians also noted troubling treatment gaps for patients relapsing after long remissions. Daratumumab cannot be accessed third line, and compassionate access for fourth-line therapy often requires prior use of all PBS-listed medicines – a system misaligned with patient frailty or clinical need. Relapsed patients may even be excluded from trials if prior anti-CD38 therapy is a prerequisite.

Stakeholders advocated for first-line access to the most effective therapies, arguing that upfront use of higher-cost treatments may reduce overall healthcare spending. Clinicians stressed the impact of early treatment decisions. Clinicians called for revised PBS restrictions and unrestricted access to combinations like daratumumab + bortezomib + lenalidomide + dexamethasone (Dara-VRd) for all patients, regardless of transplant eligibility.

Other contentious points included: the need for third-line daratumumab access, proactive horizon scanning for emerging therapies, and structured clinical advice to PBAC on submissions. Clinicians questioned cross-sponsor collaboration feasibility, noting practical difficulties for non-generic combinations.

Stakeholders emphasised that HTA bodies must maintain a full view of the treatment landscape. Tools like the EpiMap Myeloma Model Project can project patient numbers and costs, helping inform PBS planning.

The PBAC Chair requested consensus advice on these issues. The Medical and Scientific Group (MSAG) of Myeloma Australia will provide a view on appropriate PBS restriction criteria for multiple myeloma medicines to the PBAC.

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