Rare disease report exposes the need for broader HTA reform buy-in

The patient community is urging the Federal Health Minister Mark Butler to accelerate the implementation of the Health Technology Assessment (HTA) Review reforms, following the release of a new report showing Australia continues to lag comparable nations in funding rare disease medicines.

The All Too Rare report, developed by IQVIA and funded by Alexion, AstraZeneca Rare Disease, found Australia ranked 14 out of 15 comparator countries for the number of rare disease medicines funded over the past decade.

Speaking to Health Industry Hub, Patient Voice Initiative (PVI) CEO Ann Single said the findings reinforce longstanding concerns about delays in access to innovative medicines and the need to make Australia a more attractive market for rare disease therapies.

“The first thing that’s really important is that most of the rare disease medicines that are listed on the Pharmaceutical Benefits Scheme (PBS) are not getting through on the first HTA submission,” Single said, reflecting on the data from the report. “For our patient communities, a day can be a long time living with a rare condition. Having it take more than one submission most [78%] of the time is concerning.”

Another striking finding was Australia’s comparative position internationally.

“Something that surprised me was looking at other jurisdictions and the difference in how many medicines they had,” she said. “While there can be multiple reasons for that, we really want to encourage companies to bring innovative medicines here. Being lower down the list than most countries is concerning.”

Only 39% of medicines granted orphan drug designation over the analysis period have progressed to reimbursement, with patients waiting an average of 22 months between registration and PBS listing. Among the examples cited is BioMarin’s Palynziq (pegvaliase), the first enzyme replacement therapy developed for people with phenylketonuria (PKU), which has been TGA approved since 2021 but not yet PBS listed due to failed pricing negotiations.

The Alexion report adds to a growing body of recent reports examining medicines access in Australia, particularly in the push to implement the recommendations of the HTA Review. Rather than creating ‘report fatigue’, Single believes the accumulating evidence is strengthening the case for reform.

“What we want to be careful to do is not get jaded by the number of reports, but to think critically about what they mean and what they’re telling us,” she explained. “Every report is giving us more detail to support the HTA Review recommendations. They come from particular perspectives and sometimes people might see different reasons for what’s happening. But what they’re doing is giving us content that we can discuss with each other.”

While acknowledging the government’s intention to progress the HTA reforms, Single expressed frustration that the implementation roadmap remains unpublished despite repeated calls from stakeholders.

Asked what she believed was behind the delay, Single suggested the urgency of HTA reforms may not yet be fully appreciated beyond the health sector.

“It’s easy to think that HTA reform is just an issue for the Department of Health or the health sector,” she reflected. “I’m just not sure if the delay is that people outside the Department of Health [including Treasury and broader consumers] don’t see this as the burning issue that those of us in the health system do.”

The need for broader HTA reforms buy-in has been voiced by several MPs and revealed by the lack of action from the Expenditure Review Committee (ERC).

Releasing the HTA Review implementation roadmap would also enable patient organisations to contribute more effectively.

“It’s critical for understanding how we effectively partner and how patient communities decide where we put our energies,” she emphasised. “There’s a lot of stuff we can still do, but we could work so much more efficiently if we could see what that roadmap looked like. We don’t understand why we haven’t been shown it yet.”

Nicole Gaupset, General Manager, Alexion ANZ, said “There are two million Australians living with one of the 7,000 known rare diseases and, while we are seeing great strides in our understanding of these diseases, only 5% have an effective treatment available. Many of these medicines will be life changing and provide new treatment options for patients and their families for the first time.”

There are also growing concern among patient groups about the prospect of medicines being withdrawn from the PBS, in light of the recent debates relating to breast cancer and multiple sclerosis (MS) drugs.

“When there’s a treatment that a patient can’t access, that’s a really tough position to be in,” Single told Health Industry Hub. “But to have medicines removed from the PBS is the only thing worse than not being able to access new treatments. Patient communities express that to me often.”

Drawing on her role as President of the Health Technology Assessment International (HTAi), Single said discussions at the recent annual meeting reinforced that while countries can learn from one another, healthcare decision-making must ultimately reflect local priorities and values.

“We had some of the greatest diversity we’ve ever had at that HTAi meeting – more than 65 countries,” she said. “What it really highlighted was the importance of the local setting and of community values in driving your process.”

“While I’ve always been keen that we learn from other countries, borrow and adapt where appropriate, it really brought home to me just how important it is in HTA to think about what we believe as a society about health, where we want to incentivise innovation and how we want to shape universal health coverage. Being explicit about that and knowing that we agree about it as a society, is really critical.”

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