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Background Regulatory pathways adopted by the United States Food Drug and Administration (FDA) and Australian Therapeutic Goods Administration (TGA) enable expedited approval of medicines that are thought to offer significant clinical advantage over existing options for severe diseases.Objectives To review Australian accessibility to medicines approved through the FDA breakthrough therapy designation (BTD) process including timelines and approvals by the TGA and Pharmaceutical Benefits Advisory Committee (PBAC) for listing on the Pharmaceutical Benefits Scheme (PBS).Methods Retrospective review of published reports from the FDA, TGA, PBAC and PBS for BTDs from 1 January 2013–31 August 2023. Uniform data about BTD and milestone dates were collected. Analysis included all BTDs approved by FDA until 31-August-2022. Main outcome measures: Rates of approval by TGA and PBAC, and PBS-listing; and median (interquartile range, IQR) time from FDA submission to FDA approval, and FDA approval to TGA approval, PBAC approval and PBS listing for cancer and non-cancer medicines.Results Of 237 BTDs across 156 medicines, 68% were approved by the TGA, and 37% were listed on the PBS. Median (IQR) time from FDA submission to FDA approval was shorter for cancer compared to non-cancer; 179 days (140–210) vs 232 days (181–245), p

Abstract The clinical application of Pharmacogenomics (PGx) has improved patient safety. However, comprehensive PGx testing has not been widely adopted in clinical practice, and significant opportunities exist to further optimize PGx in cancer care. This systematic review and meta‐analysis aim to evaluate the safety outcomes of reported PGx‐guided strategies (Analysis 1) and identify well‐studied emerging pharmacogenomic variants that predict severe toxicity and symptom burden (Analysis 2) in patients with cancer. We searched MEDLINE, EMBASE, CENTRAL, clinicaltrials.gov, and International Clinical Trials Registry Platform from inception to January 2023 for clinical trials or comparative studies evaluating PGx strategies or unconfirmed pharmacogenomic variants. The primary outcomes were severe adverse events (SAE; ≥ grade 3) or symptom burden with pain and vomiting as defined by trial protocols and assessed by trial investigators. We calculated pooled overall relative risk (RR) and 95% confidence interval (95%CI) using random effects models. PROSPERO, registration number CRD42023421277. Of 6811 records screened, six studies were included for Analysis 1, 55 studies for Analysis 2. Meta‐analysis 1 (five trials, 1892 participants) showed a lower absolute incidence of SAEs with PGx‐guided strategies compared to usual therapy, 16.1% versus 34.0% (RR = 0.72, 95%CI 0.57–0.91, p = 0.006, I2 = 34%). Meta‐analyses 2 identified nine medicine(class)‐variant pairs of interest across the TYMS, ABCB1, UGT1A1, HLA‐DRB1, and OPRM1 genes. Application of PGx significantly reduced rates of SAEs in patients with cancer. Emergent medicine‐variant pairs herald further research into the expansion and optimization of PGx to improve systemic anti‐cancer and supportive care medicine safety and efficacy.

Abstract This study explored the acceptability of a novel pharmacist‐led pharmacogenetics (PGx) screening program among patients with cancer and healthcare professionals (HCPs) taking part in a multicenter clinical trial of PGx testing (PACIFIC‐PGx ANZCTR:12621000251820). Medical oncologists, oncology pharmacists, and patients with cancer from across four sites (metropolitan/regional), took part in an observational, cross‐sectional survey. Participants were recruited from the multicenter trial. Two study‐specific surveys were developed to inform implementation strategies for scaled and sustainable translation into routine clinical care: one consisting of 21 questions targeting HCPs and one consisting of 17 questions targeting patients. Responses were collected from 24 HCPs and 288 patients. The 5‐to‐7‐day PGx results turnaround time was acceptable to HCP (100%) and patients (69%). Most HCPs (92%) indicated that it was appropriate for the PGx clinical pharmacist to provide results to patients. Patients reported equal preference for receiving PGx results from a doctor/pharmacist. Patients and HCPs highly rated the pharmacist‐led PGx service. HCPs were overall accepting of the program, with the majority (96%) willing to offer PGx testing to their patients beyond the trial. HCPs identified that lack of financial reimbursements (62%) and lack of infrastructure (38%) were the main reasons likely to prevent/slow the implementation of PGx screening program into routine clinical care. Survey data have shown overall acceptability from patients and HCPs participating in the PGx Program. Barriers to implementation of PGx testing in routine care have been identified, providing opportunity to develop targeted implementation strategies for scaled translation into routine practice.