Aims: Genomics reveals links to tumour-agnostic drug therapies targeting actionable alterations not accessible through histotype-based treatment. However, real-world access to genomically-matched therapy in Australia remains unknown.
Methods: This cross-sectional study analysed therapy access after comprehensive genomic profiling (CGP) via the Molecular Screening and Therapeutics (MoST) program, a nationwide precision oncology platform. The study focused on rare and less common cancers (RLCC) and treatment-resistant solid tumours and reports the patient proportion with therapeutically significant genomic alterations, including high tumour mutational burden (TMB-H), microsatellite instability (MSI-H), BRAF V600E and oncogenic alterations in ERBB2, NTRK1-3 and RET, and access to molecular tumour board (MTB)-based therapy recommendations. Follow-up data cut-off was June 2024.
Results: From September 2016-December 2023, 7013 patients had valid CGP results through MoST. Of these, 4978 (71%) received an MTB recommendation. Among the 3704 patients (53%) who received systemic therapy after MTB issue, 1040 (28%) received a matched therapy. Notably, 640 of these patients (62%) accessed matched therapy(s) through clinical trials (351/640 MoST-affiliated, 55%). Similarly, 705 of 2644 RLCC patients received matched treatment, with 457 (65%) through clinical trials (260/457 MoST-affiliated, 57%). Trial participation for pan-cancer biomarkers ranged from 19-38%, with the most common drug classes being immunotherapy and inhibitors of BRAF V600E and RET (Table 1).
Conclusion: Matched therapy access linked to pan-cancer biomarkers is not universal following molecular profiling. Clinical trial participation is essential to address this access gap for Australian patients.
Table 1. Access pattern of therapies associated with pan-cancer biomarkers.
Biomarker |
Frequency |
Received matched |
Trial participation (%)* |
TMB-H |
828 |
424 |
206 (25) |
MSI-H/instable |
75 |
23 |
14 (19) |
BRAF V600E |
184 |
70 |
40 (22) |
ERBB2 alteration |
372 |
109 |
73 (20) |
NTRK fusion |
14 |
10 |
3 (21) |
RET fusion |
24 |
16 |
9 (38) |
*Relative to biomarker frequency
Supported by MSD, Merck Sharp & Dohme (Australia) Pty Limited