Aims: People with rare cancers face limited access to subsidised therapies due to restrictive Health Technology Assessment methods that are challenging for small populations. This causes significant financial toxicity. Rare Cancers Australia (RCA) used pan-tumour evidence, advocacy and lived experience data to influence reimbursement policy within Australia’s Pharmaceutical Benefits Scheme (PBS).
Methods: Across eight years (2018-2026) RCA worked with patient advocates to reframe equity issues, expose real‑world data on financial toxicity, and collaborate with patient organisations, clinicians and pharmaceutical companies to develop solutions to one of the most significant causes of financial toxicity for people with rare cancers: out-of-pocket costs for targeted therapies. Through surveys, reports, roundtables, media, and sustained engagement with Parliamentarians, government and sponsors, RCA helped increase public and political awareness and encouraged sponsors to make pan-tumour submissions to the Pharmaceutical Benefits Advisory Committee (PBAC).
Results: The convergence of evidence, advocacy and lived experience data contributed to the PBAC’s landmark recommendation for a broad multi‑cancer PBS listing for an immunotherapy in September 2025, with patient access from 1 March 2026. PBAC made a second multi-cancer recommendation in December 2025, which is awaiting PBS listing. These decisions recognise that tumour‑agnostic access can reduce financial toxicity for patients who previously had to self‑fund recommended treatments. It is expected that over 10,000 patients could benefit from the current listing, reducing the cost per script from $,15,400 to the co-payment.
Conclusions: Welcomed as a long‑awaited step toward equitable access, this first broad, multi‑cancer immunotherapy listing acknowledges the people with rare cancers who face inequity and high out-of-pocket costs due to policy barriers. RCA continues to champion the collective influence of patients, clinicians and pharmaceutical developers on medicines access policy, including the evolution of multi-cancer PBS listings.