This study aimed to describe the type of evidence available for and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC). The evidence was assessed with the European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS).

All data on applications submitted to PBAC between 2010 and 2020 were independently extracted in duplicate from PBAC Public Summary Documents available online. Any disagreements were resolved through discussion. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website. Substantial benefit for the ESMO-MCBS was defined as a grade A or B for (neo)adjuvant intent and four or five for palliative intent.

In the study period, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68%) for solid tumors (116 in the palliative setting) and 58 (32%) for hematological cancers. A total of 138 (76%) indications were recommended for public funding, 40 (22%) were rejected, and four (2%) were deferred. Randomized controlled trials (RCTs) were the main source of evidence in 154 indications (85%) and single-arm studies in 27 (15%) indications. RCTs were available in 113 (91%) and 41 (71%) of the solid tumor and hematological cancer indications, respectively. In submissions with RCTs, mature overall survival (OS) was reported in 81 (53%) indications. For indications with a statistically significant improvement in OS, the median gain was 3.0 months (range 0.9 to 17.0) for solid tumors and 8.2 months (range 1 to 9.1) for hematological cancers. The ESMO-MCBS score was available for 99 solid tumor indications. For indications in the palliative setting, 47 (52%) had substantial clinical benefit according to ESMO-MCBS v1.1, including 35 (51%) indications recommended by PBAC and six (35%) indications that were rejected.

These results show that only a minority of cancer medicine indications considered by PBAC are supported by a good level of evidence and provide a modest extension of patient survival.