Inside the tensions shaping PBAC decisions

The evolving relationship between sponsors, the Pharmaceutical Benefits Advisory Committee (PBAC) and the health technology assessment (HTA) system came under scrutiny at the Market Access Summit hosted by LifeSciences Australia.

The panel was chaired by Anne O’Neill, Chief Commercial Officer at ANDHealth, who previously held roles with the NSW Health Office for Health and Medical Research. Panellists were Professor Andrew Wilson, Co-Director of the Menzies Centre for Health Policy and Economics at The University of Sydney and former Chair of the Pharmaceutical Benefits Advisory Committee (PBAC), and A/Professor Kate Mahon, a medical oncologist and PBAC/ESC member.

Professor Wilson opened with a blunt assessment of the stalled HTA Review reforms.

“I share the frustration that the HTA Review still sits in limbo while government makes up its minds what it does or doesn’t want to do in relation to it,” said Professor Wilson.

He stressed that the HTA Review was intended to make the assessment process fit-for-purpose rather than alter the underlying policy settings governing the pricing of innovative medicines, an issue that has taken on renewed significance following the introduction of Most Favoured Nation (MFN) policies.

“That was a shortcoming,” Professor Wilson said reflecting on his time as the Chair of the HTA Review Implementation Advisory Group (IAG). “We realised that at the time when we were doing it.”

He also stressed that the HTA Review’s 50 recommendations should be considered as an interconnected package rather than a menu of isolated reforms. Incremental changes, he cautioned, could create unintended consequences elsewhere in the system if the broader architecture of the proposed reforms is overlooked.

Earlier engagement without compromising process integrity

A central question for the panel was how the PBAC could facilitate earlier engagement and greater transparency with sponsors without compromising the independence and integrity of the assessment process.

For A/Professor Mahon, the issue is partly rooted in the difference between assessing evidence at a population level and understanding what treatment means for an individual patient.

Drawing on her clinical experience, she said decision-making extends well beyond survival outcomes. Quality of life, the ability to maintain everyday activities and personal priorities can all materially influence the value of a treatment from a patient’s perspective. Earlier and more meaningful sponsor engagement, she suggested, could help ensure that clinical trial evidence better captures the outcomes that matter to patients.

Professor Wilson, meanwhile, identified the period after a PBAC recommendation as a significant weakness in the current system. Once a recommendation is made, responsibility shifts to the sponsor to determine whether and how to negotiate the next stage. He characterised the process as relatively passive and questioned whether the existing framework provides sufficient mechanisms or incentives for meaningful price negotiation.

The challenge of capturing broader societal value

The panel considered whether the PBAC’s decision-making framework should more systematically account for the wider economic and societal benefits associated with earlier access to innovative medicines.

Asked by Health Industry Hub why such considerations have yet to be incorporated systematically into PBAC decisions, Professor Wilson pointed to the limited availability of robust evidence quantifying these benefits.

“What we have is a lot of speculation about what the societal benefits will do,” he said. “There are very few situations where the benefits are actually measured, and one of the great examples is with the GLP1 inhibitors and weight reduction.”

He acknowledged that the issue warrants further examination but warned that adding additional dimensions of value could make PBAC decisions more difficult to interpret.

“We already have a complex decision-making framework. The more that we introduce some of these other things into it, the more complex we make it and the harder it is to explain the basis of why we’re making those judgements,” Professor Wilson explained. “It’s something that is still worth exploring. I’m not ruling it out.”

He nevertheless accepted that there are “circumstances” in which productivity gains and the consequences of the carer burden could reasonably form part of the assessment.

Yet these “circumstances” are already a common reality for many patients and families. Chronic diseases, rare diseases and cancers significantly affect an individual’s ability to participate in the workforce, while the demands of patient support places a substantial burden on families and carers. The question is whether these well recognised and recurring impacts should already be considered more systematically by the PBAC rather than on an ad hoc basis.

What does a stronger PBAC submission look like?

The panel was then asked what sponsors could do, in practical terms, to improve their prospects of securing a “confident and positive recommendation” from PBAC.

For Professor Wilson, greater transparency around the assumptions underpinning a submission would be a useful starting point. He argued that decision-makers should be able to understand how a company arrived at the parameters and proposed price contained in its submission. Greater visibility into the commercial and strategic considerations behind those positions, he suggested, could reduce uncertainty for the PBAC and make the basis for the sponsor’s submission clearer.

A/Professor Mahon echoed the importance of transparency, noting that uncertainty about the rationale behind a submission can consume considerable time during the assessment process. She argued that a more open relationship between sponsors and decision-makers could help reduce the need to speculate about why particular assumptions or positions have been adopted. While recognising the political and commercial complexities surrounding medicines access, she emphasised the shared objective of ensuring effective treatments reach patients.

Ultimately, the panel discussion pointed to the tensions at the heart of the current HTA: how to create greater transparency, more effective engagement and a sophisticated understanding of value while preserving the independence of the PBAC assessment process.

In reimagining healthcare across the entire patient journey, Health Industry Hub^TM is the only one-stop-hub uniting the diversity of the Pharma, MedTech, Diagnostics & Biotech sectors to inspire meaningful change.

The Health Industry Hub^TM content is copyright protected. Access is available under individual user licenses. Please click here to subscribe and visit T&Cs here.