PBAC blocks tumour-agnostic funding after nearly a decade of deliberations

After eight years of debate with industry, clinicians and patient advocacy groups, the Pharmaceutical Benefits Advisory Committee (PBAC) has rejected a long-awaited tumour-agnostic funding proposal for MSD’s Keytruda (pembrolizumab) and deferred a decision on BMS’ Opdivo (nivolumab) and Yervoy (ipilimumab).

The rejection is a blow to patients diagnosed with cancer, who have been calling for equitable and timely access to tumour-agnostic immune checkpoint inhibitors, medicines that target genetic biomarkers rather than the site of cancer.

“It’s simply not good enough that after eight years of churn, the committee’s answer to the pleas of the Australian cancer community for more affordable and equitable access to cancer care is an outright ‘no’,” said Chifumi Umeda, Managing Director, MSD Australia & New Zealand.

“For almost a decade the PBAC has continually shifted the goal posts on multi-cancer funding, making it impossible to find an equitable solution for Australian cancer patients.”

The MSD proposal sought to introduce a tumour-agnostic (also called multi-indication or pan-tumour) funding model under which all approved uses of Keytruda for unresectable advanced or metastatic cancers listed on the Australian Register of Therapeutic Goods (ARTG) would be reimbursed through the PBS. Under this plan, around 700 Australians could have gained funded access to Keytruda immediately, with an additional 5,200 patients expected to benefit over the next four years. This would have covered patients with 11 rare cancer subtypes where no other immunotherapy is available on the PBS.

Yet, the PBAC concluded the prices proposed were too high, saying further discounts would be needed to demonstrate cost-effectiveness. It also argued the proposal excluded some groups with significant unmet clinical needs, such as patients with rare cancers, one of the original motivations for broad PD-L(1) inhibitor PBS listings.

This reasoning has baffled MSD, which pointed to extensive consultation with Rare Cancers Australia, leading oncologists, and government agencies throughout 2024-25, indicating that the inclusion of medicines not registered on the ARTG was considered inappropriate for a funding model.

Umeda responded bluntly, saying “MSD is deeply uneasy that the committee appears to be using Australians with rare cancers as a justification for the rejection, at a time when the rare cancer community has been the most vocal advocate for multi-cancer funding.”

The decision comes almost a decade after the government first sought PBAC advice on tumour-agnostic reimbursement of immunotherapies, following a direct request from then Federal Health Minister, Greg Hunt.

Yet progress has stalled. At present, each new Keytruda indication requires separate PBAC review, with an average delay of 447 days from TGA approval to PBS listing. Of the 35 registered indications for the drug, only 15 have made it to full or partial reimbursement, despite 40 separate submissions.

BMS, meanwhile, expressed disappointment at the PBAC’s deferral of its own tumour-agnostic listing proposal for Opdivo and Yervoy. The committee cited concerns around financial estimates and the risk-sharing arrangement (RSA) structure.

The company said in a statement that it is working with the PBAC to “navigate a prompt pathway to approval” and “is proud of the leadership role it has played in developing this innovative approach to the reimbursement of immunotherapies in Australia”. Owen Smith, Vice President and General Manager of BMS ANZ, has delved into the significant potential of the tumour-agnostic funding model in a recent Health Industry Hub interview.

The science behind these funding models is rapidly evolving. Genomic advances now allow cancers to be categorised by genetic alteration rather than origin, enabling targeted treatments for small patient groups across multiple tumour types. Yet, these therapies are often backed by single-arm trials without a standard-of-care comparator, creating uncertainty around safety, efficacy, and cost-effectiveness – complications that make health technology assessment (HTA) deliberations more complex.

Other countries, however, have found ways forward. Tumour-agnostic funding models were first introduced in Europe in 2017 and are now used by several nations to improve access while providing budget predictability.

Recent research supports adopting a “life-cycle” cost-effectiveness framework for tumour-agnostic medicines, in which economic models are continuously updated with real-world evidence to guide decisions on appraisal, re-appraisal, and de-adoption.

Australia, critics argue, remains stuck.

“Without a clear change in approach by the Federal Government, there does not appear to be a path forward for multi-cancer funding in Australia,” Umeda said. “Sadly, Australian cancer patients remain at the mercy of a painfully slow, inequitable and inefficient medicines funding system. It’s a sad day when innovation and equity are dismissed in favour of the status quo.”

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