Compassionate Access Drug Use for Solid Organ Malignancies in a Metropolitan Cancer Centre in Australia Between 2017...

The Pharmaceutical Benefits Scheme (PBS) provides affordable access to cancer treatments in Australia. Not all cancer treatments are available on the PBS. Access to non-PBS-listed cancer treatments can occur through Compassionate Access Programs (CAPs). This retrospective review describes cancer treatments used via CAPs in a Metropolitan Cancer Centre in Australia.

Patients with solid organ malignancies who had received drugs via compassionate access between 2017 and 2024 were identified via pharmacy and hospital medical records. Patients with hematological malignancies or who accessed drugs through out-of-pocket funding were excluded. CAPs were grouped into cost-share/co-pay and cost-free programs.

Of 228 cases, 148 (64.9%) received the drug cost-free, and 80 (35.1%) were on a cost-share/co-pay program. A total of 110 (48.2%) patients received the CAP drug as their first therapy. The median number of previous therapies before receiving CAP drug was 1 (range: 0-4). The median duration between diagnosis and use of CAP drug was 5.1 months (range: 0.2-96.0). The median and mean duration of CAP drug use were 5.2 and 10.3 months, respectively (range: 0.03-96.0). A total of 53 programs provided 40 drugs from 18 companies. Six drugs had both cost-share/co-pay and cost-free programs. The drug with the most patients was pertuzumab (n = 43), followed by nivolumab (n = 27) and durvalumab (n = 21). Fourteen patients accessing non-oral drugs via cost-share/co-pay programs ultimately did not receive any free drugs.

There is an increasing prevalence of novel drug use via CAPs across all types of cancers with promising impacts on patient outcomes.