People are dying as the drug approval process remains behind closed doors, inquiry hears.
Greater transparency is needed from the federal government around its health technology assessment process for drugs to treat rare and less common cancers, according to Rare Cancers Australia CEO Ms Christine Cockburn.
Speaking today to the Select Committee on equitable access to diagnosis and treatment for individuals with rare and less common cancers, Ms Cockburn said a lack of clarity regarding the drug approval process following a PBAC submission was hampering progress on accelerating approval pathways for new and emerging cancer therapies.
“These processes in Australia, the HTA and the PBAC processes are highly opaque,” she said.
“Once the submission is made to PBAC, the conversations between government and the sponsor are confidential and remain confidential, so it’s very difficult for us to know exactly what the challenges are [for getting new drugs approved].
“These frameworks are slow, cumbersome and not fit for purpose, and people are dying with rare diseases when there are medicines here, they are being used in other indications and the agreement is packed away in its standard of care.”
Increasing financial incentives for pharmaceutical companies seeking to undergo the assessment process, particularly larger US-based sponsors, was also crucial to improving treatment options for patients with rare and less common cancers, Ms Cockburn said.
“We cannot continue to say that without enormous randomised clinical trial evidence, you cannot bring medicine [for market approval] in Australia.”
Ms Megan Varlow, Director of Cancer Control Policy at Cancer Council Australia, agreed, saying small patient cohorts and a lack of sufficient data significantly limited investment in clinical trial research for rare and less common cancers.
“Medicines and technologies need to be financially viable in Australia for sponsors to be even interested in the first place in being able to participate in the Australian environment, and that’s a challenge,” she said.
“Then there’s the question of the process that has to be followed – it is n extremely expensive process to engage in.
“If you use the example of repurposing a medicine that’s currently only approved for one indication, but evidence shows that it is going to be helpful for something else, we can’t do that repurposing process because it’s too expensive for us to be able to run [trials].
“And because of the financial viability aspects, sponsors then often don’t choose to do that, because it just doesn’t give them the return that makes it viable in Australia.
“The question of how you can have a consistent process that everybody understands and can follow, but that makes allowances for that is really the challenge, and I don’t have a beautiful bow-wrapped solution that we could provide.”
According to Ms Cockburn and Ms Varlow, rampant disparities and underfunding of patient travel and accommodation schemes was another vital issue that state and federal governments needed to address.
“Every state has a program that financially supports people to access health care [but] they are wildly inadequate, [covering] not very much of the out-of-pocket costs at all,” Ms Cockburn said.
“I will correct myself if I’m wrong, but I think there may be a state which provides $45 a night for accommodation. I don’t think you can even stay in a backpacker hostel for that price.
“I would also say the states don’t provide that support where you’re accessing a clinical trial, as it’s not standard of care, it’s experimental.
Ms Varlow added that: “It’s not just wildly inadequate, it’s also wildly different with different states and territories providing different rates.
“Most states very firmly say that it’s a subsidy, they’re not aiming to cover the costs of care, but it means that lots of people with rare and less common cancers, and in fact, people in regional areas with common cancers, don’t end up receiving the treatment that’s been recommended because they can’t afford to travel and to stay somewhere and to receive that care.
“There is a role for the federal government in supporting the states and territories to think about how the programs could be implemented in a more consistent way, and to improve the level of support that they provide to people.”
Both witnesses also highlighted equity gaps in accessing specialist diagnostic and treatment services for rare and less common cancers.
According to Ms Varlow, current funding mechanisms segregating state and federal government funding hampered the communication and collaboration across primary and tertiary care needed to improve rare cancer diagnosis and treatment, particularly in regional and rural areas.
“The ability for clinicians in those areas to work with others across boundaries and those centres of excellence [varies] depending on where they are,” Ms Varlow said.
“Some places do it well, some places they’re not allowed to do it at all, so [clinicians] have ‘illegal’ conversations with their colleagues in other areas, rather than being able to officially consult.
“So there’s a question of, how do we better support that sort of collaboration across borders, the borders within states as well as across states? How does clinical governance cope with that, and how do ethics approvals also consider that?”
Ms Cockburn added: “Without action on equity, we cannot hope to improve the outcomes of people living with rare and less common cancers, and for them to have the same chance at life as everyone else.
“It has been 10 years since Kate and Richard Vines first launched RCA’s Just a Little More Time report in Parliament House in Canberra, 10 years on, we are still asking for the same action to be taken.
“People with rare and less common cancers simply don’t receive the same level of support or have access to the same treatment options as those with more common cancers, and they pay for that inequity with their lives.”