The changing face of Australian data reforms: Impact on pharmacoepidemiology research

Journal article

Robust evidence is needed to support the development, registration, subsidy and use of cancer therapies. Randomised controlled trials form a cornerstone of cancer medicines research by generating evidence about the efficacy of therapies in selected patient populations. However, their implementation is resource- and cost-intensive, and they often have limited generalisability to patients treated in routine care. Real-world evidence leverages data collected about patients receiving routine care, outside of clinical trial settings. This thesis comprises four studies that use Australian real-world data to explore the potential of real-world evidence to address evidence gaps that are not met by conventional clinical trials, using lung and breast cancer as illustrative examples. These evidence gaps relate to differences in patients, management and outcomes between clinical trials and real-world practice. First, using linked health administrative data for clients of the Australian Department of Veterans’ Affairs, we determined that most adults aged ≥65 years with advanced lung cancer would not be suitable for clinical trial participation and nearly 90% did not receive systemic cancer treatment. This demonstrates the limited generalisability of clinical trial evidence and resultant evidence gap for older adults with lung cancer. Second, we assessed how real-world patients compare with participants in immunotherapy trials, using data from a prospective, multicentre database of lung cancer patients. Only 30% of real-world lung cancer patients would be eligible for landmark immunotherapy trials, and this would be almost doubled by broadening clinical trial eligibility criteria. Third, we used national, population-wide medicines dispensing data to study real-world trastuzumab use and outcomes among patients with HER2-positive early breast cancer. Although only two-thirds of patients completed the recommended 12-month course of treatment, survival outcomes were similar to those reported in clinical trials. Finally, we studied cardiac monitoring practices among patients receiving trastuzumab for HER2-positive early breast cancer in a nation-wide cohort. Adherence to guidelines varied by year and geography, suggesting areas for improvements in cancer care at a population level. This thesis illustrates the ways in which observational research using Australian real-world data provides opportunities to complement and enhance cancer research centred on clinical trials. Maximising the potential of real-world evidence research is key to developing a robust evidence base for optimising cancer medicines use and outcomes in Australia

Authors: de Oliveira Costa, J; Bruno, C; Schaffer, AL; Raichand, S; Karanges, EA

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: Swansea University
• Journal: International Journal of Population Data Science
• Volume: 6
• Issue: 1
• Pages: 1418-1418
• Publication date: 2021-04-15
• DOI: 10.23889/ijpds.v6i1.1418
• EISSN: 2399-4908
• ISSN: 2399-4908
• Language: English
• Keywords: Population; Environmental health; Health care; Family medicine; Medicine; Pharmacoepidemiology; Pharmaceutical Benefits Scheme; Geography; Pharmacology; Medical prescription; Political science; Context (archaeology); Legislation; Government (linguistics)