Evidence of clinical benefit of cancer medicines considered for funding in Australia

Abstract

Objectives: 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).

Methods: 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.

Results: 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.

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

Keywords: Australia; assessment; cancer medicines; extent of benefit; funding; strength of evidence.