Poster Presentation Clinical Oncology Society of Australia Annual Scientific Meeting 2026

From Breakthrough to Bedside: Evidence Characteristics Associated with Timely Australian Access to Precision Cancer Therapies (146353)

Sherrie SS Sadri 1 , Abhishek AD Debnath 2 , Angela Elizabeth AD Djaja 2 , Yi Jaye YL Lim 2 , Aishwarya Shalom AR Raj 2 , Anthony AT Tascone 2 , Senthil SL Lingaratnam 1
  1. Pharmacy, peter maccallum cancer centre, Melbourne, VIC, Australia
  2. Pharmacy, Monash University, Melbourne, VIC, Australia

Abstract

Aim:

To examine the association between clinical evidence characteristics and the time from initial U.S. Food and Drug Administration (FDA) approval to Therapeutic Goods Administration (TGA) registration and positive Pharmaceutical Benefits Advisory Committee (PBAC) recommendation for cancer medicines granted Breakthrough Therapy Designation (BTD). Three common tumour streams - lung cancer, breast cancer, and lymphoma – were selected to enable cross-tumour comparison while limiting clinical heterogeneity.

Methods:

A retrospective review was conducted of FDA-approved BTD indications between 2012 and 2022. Times from FDA approval to TGA registration and PBAC positive recommendation were summarised using median days and interquartile ranges (IQR). Variables included pivotal study design (RCT vs. Non-RCT), trial phase (Phase III/IV vs. Phase I/II), submission type (new medicine vs. new indication), and primary endpoint (OS/PFS vs. others). Comparisons were between independent groups performed using Mann–Whitney U test.

Results:

Thirty-eight BTD indications were identified across lymphoma (n=15), lung cancer (n=13) and breast cancer (n=10). RCT-supported submissions were associated with shorter TGA registration times than non-RCTs submissions (median 224vs491 days; p =0.011). Similar trend for time to PBAC recommendation was not statistically significant (509vs836 days; p=0.128). Phase III/IV trials were similarly associated with faster TGA registration than Phase I/II studies (median 244vs692 days; p=0.035). Submissions supported by OS/PFS endpoints were associated with shorter TGA registration times than those using other endpoints (median 249vs.551 days; p=0.038) but not for PBAC recommendation (median 678vs.705; p=0.43). There was no difference for median TGA registration times (249vs.382 days; p=0.157) and PBAC recommendation times (678vs.623 days; p=0.509) between new indications and new medicines

Conclusion:

Evidence maturity, characterised by randomised study designs, later-phase trials and clinically meaningful efficacy endpoints, appears to facilitate faster Australian regulatory registration for BTD indications, but does not necessarily translate into faster reimbursement recommendations.