To describe the type of evidence and the clinical benefit of cancer medicines assessed for funding in Australia by the Pharmaceutical Benefits Advisory Committee (PBAC) and to assess it 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 extracted from PBAC Public Summary Documents available online. ESMO-MCBS ratings were retrieved from the ESMO-MCBS website.

Then, 182 cancer indications for 100 cancer medicines were examined by PBAC, including 124 (68.1 percent) for solid tumors and 58 (31.9 percent) for hematological cancers. A total of 137 (75.3 percent) indications were recommended for PBS funding and 40 (21.9 percent) were rejected. Randomized clinical trials (RCTs) were the main source of evidence in 154 indications (84.6 percent), single-arm studies in 28 (15.4 percent) indications. Statistically significant improvement in overall survival (OS) was reported in 80 (44 percent) of the indications, with a median OS gain of 3.0 months (range 0.9–17.0) for solid tumors and 8.2 months (range 1–49.1) for hematological cancers when mature OS data were available. The ESMO-MCBS score was available for 99 solid tumor indications, of which 51 (51.5 percent) showed substantial clinical benefit according to ESMO-MCBS, including 40 (54.1 percent) of PBAC-recommended indications and 9 (42.9 percent) of PBAC-rejected indications. There was no association between the ESMO scoring and PBAC decision.

Most cancer medicines indications considered by PBAC were supported by RCTs. A minority showed a substantial improvement in OS.

This study aimed to assess whether there have been changes in the quality of clinical evidence submitted for government subsidy decisions on cancer medicines over the past 15 years.

We reviewed public summary documents (PSDs) reporting on subsidy decisions made by the Pharmaceutical Benefits Advisory Committee (PBAC) from July 2005 to July 2020. Information was extracted on the study design, directness of comparison, sample size, and risk of bias (RoB). Changes in the quality of evidence were assessed using regression analysis.

Overall, 214 PSDs were included in the analysis. Thirty-seven percent lacked direct comparative evidence. Thirteen percent presented observational or single-arm studies as the basis for decisions. Among PSDs presenting indirect comparisons, 78 percent reported transitivity issues. Nearly half (41 percent) of PSDs reporting on medicines supported by head-to-head studies noted there was a moderate/high/unclear RoB. PSDs reporting concerns with RoB increased by a third over the past 7 years, even after adjusting for disease rarity and trial data maturity (OR 1.30, 95% CI: 0.99, 1.70). No time trends were observed regarding the directness of clinical evidence, study design, transitivity issues, or sample size during any of the analyzed periods.

Our findings indicate that the clinical evidence supplied to inform funding decisions for cancer medicines is often of poor quality and has been deteriorating over time. This is concerning as it introduces greater uncertainty in decision making. This is particularly important as the evidence supplied to the PBAC is often the same as that supplied to other global decision-making bodies.