Rare but not invisible: Push to shift the HTA compass

A Rare Conversations panel convened by Patient Voice Initiative (PVI) and Alexion has intensified calls for reform, coinciding with the release of a whitepaper examining barriers to Pharmaceutical Benefits Scheme (PBS)-listed treatments for rare diseases.

Tim Fulton’s complex diagnostic and treatment journey became the catalyst for both personal determination and broader advocacy. What began as a short run to regain mobility has evolved into a lifelong mission of running for each of the known rare diseases.

“There are 2 million Australians whose story is not too different to my own,” said Fulton, Inaugural Ambassador of Rare Voices Australia and Founder of Running Rare. “Those who don’t have equitable pathways, who have an uncertain future, and who aren’t represented.”

Ann Single, CEO of Patient Voice Initiative (PVI) and President of Health Technology Assessment International (HTAi), highlighted the critical importance of embedding meaningful patient involvement within decision-making frameworks, arguing that rare disease communities must have a stronger voice in shaping policy and access pathways.

She pointed to the Czech Republic as a model of proactive reform, where government partnered with the rare community to build dedicated legislation, embed HTA processes and create a formal framework for rare disease priorities within the health system.

“We need to think about what access means for our rare community and how we ensure that, regardless of what you’re diagnosed with, rare isn’t a second-rate condition in our system,” Single emphasised.

The discussion also turned to the ongoing HTA Review reforms, with Professor Andrew Wilson, Former Chair of the Pharmaceutical Benefits Advisory Committee (PBAC), expressing dissatisfaction with Federal Health Minister Mark Butler’s response to the implementation plan.

“I have frustrations with what’s happened with the HTA Review in terms of the speed of implementation, or even for that matter, confirmation of what government has or hasn’t accepted from that Review,” he stated. “The Review has recommendations which impact more than the rare diseases community.”

When asked by Health Industry Hub which stakeholders beyond the health sector, Minister Butler and the Department of Health need to be persuaded to support funding for the implementation of the HTA Review recommendations, Professor Wilson avoided a direct response.

The role of political advocacy and community mobilisation was reinforced by Dr Gordon Reid MP, Federal Member for Robertson and Member of the House of Representatives Standing Committee on Health, who highlighted the influence of constituent engagement in shaping policy priorities.

Referring to “power of the letter” as a key advocacy tool, Dr Reid reflected on recent community efforts surrounding access to two multiple sclerosis (MS) treatments and concerns over their potential removal from the PBS.

“In my electorate, the amount of incoming correspondence on that was nothing short of extraordinary, and all of them went straight to Mark Butler, followed by phone calls,” he said. “If you’re convincing us of a particular issue and you show us how it’s going to affect our communities, then we can build the case to lobby Mark, the Prime Minister, the Cabinet for structural change.”

Sarah McGoram OAM, patient advocate and President of Patient Voice Initiative (PVI), said rare diseases often challenge the structures of traditional healthcare systems.

“By definition, we have small patient populations. There’s limited data. There’s smaller clinical trials. But that doesn’t make our lives any less important,” stated McGoram. “This whitepaper gives us evidence and numbers around that. Patient organisations give those numbers a voice. And behind that are real, individual families desperate for access to treatments that will change their lives.”

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