MELBOURNE, Australia Although Australia's Therapeutic Goods Administration (TGA) approved its first CAR T therapy in 2018, the country is lacking a system to reimburse those advanced therapies, and industry is calling on government to revalue gene therapies so that patients can access them.
"Global approval doesn't mean Australia approval," Medicines Australia CEO Elizabeth de Somer said during the AusBiotech conference. "Government needs a new way of valuing the benefit of therapies that are a one-off cure differently than other drugs. Part of that value discussion needs to include what happens downstream by keeping patients in the workforce, off welfare and contributing to society.
"There needs to be a clear link between market access and all the things that come before that. Market access is the goal, so companies won't put resources into research if the market access piece is missing," she warned.
"Government is aware of advancements in the pipeline, but the government is not ready to solve the problem," she said.
Othon Gervasio, medical director for Novartis Oncology, agreed there is need for a larger value proposition about rapidly evolving technology, but he said industry is also scrambling with preparing for those new cell therapies.
When Novartis' CAR T therapy, Kymriah (tisagenlecleucel), was approved in just five years, the industry was "still used to tablets and pills, and there is a need to change the mindset from a quality perspective as well."
"When we talk about price, it is important to look at value and the complexity of the manufacturing supply chain," he said, "and although most health economic systems are not ready, they are adaptable, so the partnership between industry and academia is important to change that mindset."
Cartherics Pty Ltd. CEO Alan Trounson said his investors are concerned that "we can't actually deliver products to patients, and that patients are looking for trials to enter so they don't have to pay."
Trounson said industry needs to come together to put pressure on politicians to change the system now.
Health Minister Greg Hunt has said he wants to get new technology to patients, but he doesn't have the policy levers to do that, said de Somer.
CART T only available for pediatric indication
The government announced its first funding for CAR T therapy Kymriah in Australia for pediatric patients with acute lymphoblastic leukemia (ALL), Gervasio said.
"The process wasn't easy, and we still don't have an answer for adult patients with ALL. We have gotten acknowledgment from government that patients are likely to benefit from therapy, but it's taking too long and we are collaborating with government to answer their questions."
Normally, therapies are evaluated through the Pharmaceutical Benefit Advisory Committee (PBAC) for listing on the government-subsidized Pharmaceutical Benefits Scheme (PBS). However, Novartis' CAR T therapy was evaluated by the Medical Services Advisory Committee (MSAC) and funded through the public hospital system via the National Health Reform Agreement.
Currently, patients have to travel overseas to access treatment, even though the therapy has been available in the U.S. since 2017 and was approved in the U.K. and Canada in late 2018.
The Australian government funds the medical treatment overseas program (MTOP), which allows patients to travel overseas to get treatment.
"The policy framework now is quite rigid," de Somer said. "You have the PBS access pathway and the medical services advisory committee pathway, and whether those two pathways actually deliver the kind of service to assess new treatment is not clear to us.
"We think there should be a way to adapt those pathways or develop a new, more flexible pathway to look at things that are not a service or medicine on its own but a combination such as CAR T.
"They might need a different way of looking at new therapies such as CAR T therapy, and they might need a new way of valuing them because of the benefits derived," she added. "Australia's PBS negotiates very hard on where they see the value and cost-benefit in purchasing drugs for Australian patients, and Australians get a good deal because of health technology assessments, but they often don't take into account other values."
For example, technology assessments consider health outcomes, but they don't take into account net outcomes such as lifetime value.
Pharma companies are increasingly frustrated with the reimbursement process in Australia, she said.
"Medicines Australia has long called for a fit-for-purpose system to ensure these treatments can be accommodated," de Somer said, noting that in Medicines Australia's budget submission, the association called for bipartisan support for a multistakeholder review of the National Medicines Policy to ensure breakthrough treatments and therapies like CAR T are available for Australian patients.
There is a huge need to get big pharma, biotech and academia talking to each other so they can come at government with a single voice. She said a call to action is desperately needed.
Another issue: Manufacturing
Yet another missing piece in Australia has been access to cell manufacturing facilities for gene therapies.
Novartis recently negotiated manufacturing agreements for a new cell and gene manufacturing process with Cell Therapies Pty, which operates under the Peter MacCallum Cancer Centre in Melbourne.
Industry has been pushing for Australia to become a leader in advanced manufacturing of transformative therapies and a hub for those treatments in the Asia Pacific region, so the move is long overdue.
Cell Therapies was granted a T cells GMP manufacturing license by the TGA, which allows the company to collect, manufacture, test, store and distribute commercial T-cell products, including CAR T-cell therapies.
The move makes Cell Therapies the first Australian local manufacturing hub for commercially approved products. Once approved, patients will be able to get products faster, rather than having to wait for therapies manufactured in Europe or North America, said Cell Therapies CEO Dawn Driscoll.
"Cell Therapies was way ahead of its time," said Driscoll. "We are a new model and new way of manufacturing and distributing therapies and are on the brink of a major expansion as we transition from being research based to seeing how we can serve the market."