There has been “remarkable progress” in research funding and opening up access to treatments for rare cancers in the last 12 months but significant barriers still remain, a new report concludes.

One year after presenting its Rare Solutions Report to federal government, the industry-supported lobby group Rare Cancers Australia has welcomed the $248 million funding commitment for rare and less common cancers (RLC).

In a progress update launched at a Canberra meeting on 11 September, Rare Cancer Australia said it was happy to report unprecedented funding of $206 million over five years for the Rare Cancers, Rare Disease and Unmet Need Clinical trials program and a further $41 million NHMRC funding for rare cancers.

“This research, while not only saving the health system down the line with reduced acute care costs and greater productivity, is structured in a way to maximise options for funded treatment for RLC patients who have few subsidised options, providing hope for those who need it most,” the report said

There has also been significant progress on the groups calls for more flexibility in patient access to medications for rare cancers, including the TGA reforms allowing seven successful  priority reviews for RLC cancer indications and three successful provisional reviews to enable faster registration.

However there was still a need to address the issue of Orphan Drug designation, with Rare Cancers Australia recommending an 18-month fee waiver for applications to register medicines for small populations of patients.

The group also welcomed moves towards more flexible PBS subsidies for multiple cancer indications for PD-1 and PD-L1 checkpoint inhibitor immunotherapies. The Pharmaceutical Benefits Advisory Committee is currently considering a system in which PBS listing submissions may be accepted for multiple indications.

But the progress on research and medication access will also need to be backed with greater clinical collaboration so that patients can get the right diagnosis, the right specialist and the right treatment in a timely manner, according to Rare Cancer Australia

While there had been new funding to support international clinical trial collaborations there was also a need to create consistent approaches to outcomes data collection, the report said. The backlash against the MyHealth Record threatened what could be an important source of medical information for people with rare cancers, it noted.

“For instance, the adoption of managed access programs with real world evidence collection for RLC cancers (via MyHealthRecord) would improve flexibility of reimbursement and thus timeliness and affordability of treatment for patients,” it said.

The report also flags that genomics will be the future of cancer care and says it “is critical that Australia has a policy and regulatory environment that promotes patient confidence in evolving genomics research.”