Benjamin Daniels

Aim. To assess the impact of the 1 September 2022 unrestricted Pharmaceutical Benefits Scheme (PBS) listing of rituximab, which removed indication-specific Authority restrictions, on Australian dispensing and government expenditure, and off-label use at a tertiary transplant centre. Methods. We performed a controlled interrupted time series analysis of monthly national PBS dispensing from January 2015 to December 2025 (interruption 1 September 2022; methotrexate as control) using segmented quasi-Poisson regression, modelling the level and trend of rituximab dispensing per 100 000 population and national PBS expenditure. In parallel, we conducted a single-centre retrospective audit of on-label and off-label rituximab dispensing and Individual Patient Use (IPU) approvals over the same period. Results. Nationally, the rituximab dispensing rate rose immediately by 12% at September 2022 (95% CI 2% to 22%; P = 0.015) and the trend increased by 1.7% per month thereafter (95% CI 1.3% to 2.1%; P < 0.001), reversing a pre-intervention decline. The post-intervention trend rose in every jurisdiction; methotrexate showed no national change. National benefits paid fell from $156.1 million (2015) to $28.1 million (2025) despite rising services. At the single centre, 466 off-label IPU approvals were recorded, led by lung transplant and haematology, and local activity remained stable after September 2022. Conclusion. The unrestricted PBS listing was followed by a sustained national increase in rituximab dispensing at lower overall cost, while single-centre off-label activity did not change. As subsidised access broadens, aggregating IPU data on off-label indications and outcomes could support quality use of medicines and inform repurposing.

Purpose: To quantify the real‐world use and describe characteristics of paediatric patients initiating biologic medicines for immune‐mediated inflammatory diseases (IMIDs). Methods: Dispensing claims for Australians aged less than 18 years were used to examine uptake and use trends of nine biologic medicines used for the treatment of IMIDs. A 15% random sample of the population was used for dispensings between 2013 and 2017, and a 30% sample for dispensings between 2017 and 2020. Incidence and prevalence rates of use per 1 000 000 population per year were calculated. Patient demographics at the time of first dispensing were determined and the number of unique medicines dispensed per person across the study period was calculated. Results: Incidence of use of biologics increased from 108 per million in 2014 to 192 per million in 2020, while prevalence increased from 352 per million to 877 per million over the same period. 1131 children were dispensed at least one biologic medicine; infliximab, adalimumab or etanercept accounted for 80% of dispensings. Mean age at first dispensing was 14 years (standard deviation: 3 years) and 51% were male. Of children initiated on biologics, 55% were first dispensed treatment for inflammatory bowel diseases and 29% for inflammatory arthropathies. While most children were dispensed only one unique biologic medicine, 15% were dispensed two, and 3% three or more over the study period. Conclusions: These results suggest that children remain on treatment for extended periods of time and nearly one in five children were dispensed multiple biologic medicines. Post‐market studies should be prioritised to monitor safety among these groups.

Background: Polypharmacy is associated with increased risks of adverse outcomes in people with cancer and medicine use evolves over the course of the disease trajectory. We estimated the prevalence of polypharmacy in people with colorectal cancer (CRC), stratified by extent of disease spread at diagnosis, during each year from 1 year prior to 5 years following diagnosis and determined factors associated with polypharmacy at diagnosis and 2- and 5-year postdiagnosis. Patients and Methods: Our retrospective cohort study used linked administrative health records from New South Wales (NSW), Australia to examine medicine use in all NSW residents ≥18 years diagnosed with CRC between 2013 and 2017. We defined polypharmacy as concomitant exposure to ≥5 medicines, excluding antineoplastics, during at least one 90-day quarter in each year from diagnosis. We used logistic regression to examine associations between polypharmacy and relevant factors. Results: Of 19 056 people diagnosed with CRC, 6797 (35%), 8482 (45%), and 3777 (20%) were diagnosed with localized, regional, and metastatic disease, respectively. Within these groups, 74%-79% experienced polypharmacy at any time during the study period; with nearly 50% experiencing polypharmacy 2 through 5 years from diagnosis. Age (≥70 years), female sex, comorbidities, colon cancer diagnosis, and socioeconomic disadvantage were associated with increased likelihood of polypharmacy at all landmarks. Conclusion: Polypharmacy is highly prevalent among Australians with CRC. For those treated with curative intent, higher polypharmacy rates are concerning, indicating excess morbidity and impaired quality of life among survivors cured of cancer but who may continue to experience poorer health than the general population.

Background Risk management plans (RMPs) are a critical element of pharmacovigilance. However, few studies have examined the quality and type of information included in RMPs, and none has examined the RMPs in the Australian medicines regulatory context. Objectives This study aims to characterise safety concerns, particularly missing information listed in the current Australian RMPs for commonly used biologic medicines, and identify additional pharmacovigilance and risk minimisation activities proposed to address identified gaps. Methods A descriptive review of RMPs included in the Australian Public Assessment Reports (2009–2024) was performed for 15 biologic medicines approved for use and universally funded in Australia for inflammatory arthropathies, inflammatory bowel diseases and inflammatory skin conditions. We extracted and quantified safety concerns (important identified risks, important potential risks and missing information) from the latest Australian Public Assessment Reports, and further categorised missing information by specific populations and conditions. We then qualitatively described the additional activities proposed. Results There were 246 safety concerns listed for the 15 medicines of interest: 85 important identified risks (34.6%), 81 important potential risks (32.9%) and 80 instances of missing information (32.5%). More than half (n = 9, 60%) of the reviewed medicines listed children and adolescents as the most common populations with missing information. Pregnant women (n = 8, 53%) and those with hepatic and renal impairment (n = 7, 47%) were also commonly listed as having missing information. Additional pharmacovigilance activities were proposed for two thirds of the medicines (n = 10, 77%) where missing information was listed. Only one third of the reviewed medicines (n = 5, 33%) had specific proposals or protocols listed in the current Australian Public Assessment Reports to address missing information. Conclusions Our study identified important gaps in RMPs for commonly used biologic medicines at the post-market phase. Despite some medicines having an extensive market history, these safety concerns remain unaddressed. Regular monitoring and critical review of RMPs are recommended to prioritise post-market studies and address outstanding safety concerns.

Background: The Medicines Intelligence (MedIntel) Data Platform is an anonymised linked data resource designed to generate real‐world evidence on prescribed medicine use, effectiveness, safety, costs and cost‐effectiveness in Australia.Results: The platform comprises Medicare‐eligible people who are ≥18 years and residing in New South Wales (NSW), Australia, any time during 2005–2020, with linked administrative data on dispensed prescription medicines (Pharmaceutical Benefits Scheme), health service use (Medicare Benefits Schedule), emergency department visits (NSW Emergency Department Data Collection), hospitalisations (NSW Admitted Patient Data Collection) plus death (National Death Index) and cancer registrations (NSW Cancer Registry). Data are currently available to 2022, with approval to update the cohort and data collections annually. The platform includes 7.4 million unique people across all years, covering 36.9% of the Australian adult population; the overall population increased from 4.8 M in 2005 to 6.0 M in 2020. As of 1 January 2019 (the last pre‐pandemic year), the cohort had a mean age of 48.7 years (51.1% female), with most people (4.4 M, 74.7%) residing in a major city. In 2019, 4.4 M people (73.3%) were dispensed a medicine, 1.2 M (20.5%) were hospitalised, 5.3 M (89.4%) had a GP or specialist appointment, and 54 003 people died. Anti‐infectives were the most prevalent medicines dispensed to the cohort in 2019 (43.1%), followed by nervous system (32.2%) and cardiovascular system medicines (30.2%).Conclusion: The MedIntel Data Platform creates opportunities for national and international research collaborations and enables us to address contemporary clinically‐ and policy‐relevant research questions about quality use of medicines and health outcomes in Australia and globally.

Objectives: Australian lockdowns in response to the initial coronavirus disease 2019 (COVID-19) outbreak in 2020 were associated with small and transient changes in the use of systemic cancer therapy. We aimed to investigate the impacts of the longer and more restrictive lockdowns in the Australian states of New South Wales (NSW) and Victoria during both the Delta subvariant lockdowns in mid-2021 and the Omicron subvariant outbreak in late 2021/early 2022. Study type: Population-based, controlled interrupted time series analysis. Methods: We conducted a national observational study using de-identified records of government-subsidised cancer medicines dispensed to a random 10% sample of Australians between July 2018 and July 2022. We used controlled interrupted time series analysis to investigate changes in the dispensing, initiation and discontinuation of all cancer medicines dispensed to residents of NSW and Victoria, using the rest of Australia as a control series. We used quasi-Poisson regression to model weekly counts and estimate incidence rate ratios (IRRs) for the effect of (each) the Delta phase lockdown and the Omicron outbreak on our systemic cancer therapy outcomes. Results: Between July 2018 and July 2022, cancer medicines were dispensed 592 141 times to 33 198 people in NSW and Victoria. Overall, there were no changes to the rates of dispensing, initiation or discontinuation of antineoplastics during the Delta phase lockdowns. In both states during the Omicron outbreak, there were significant decreases in the dispensing of antineoplastics (NSW IRR 0.89; 95% confidence interval [CI] 0.84, 0.93. Victoria IRR 0.92; 95% CI 0.88, 0.96) and in the initiation of endocrine therapy (NSW IRR 0.85; 95% CI 0.74, 0.99. Victoria IRR 0.78; 95% CI 0.65, 0.94), and no changes in the discontinuation of any systemic cancer therapy. Conclusions: The 2021 lockdowns and 2021/2022 Omicron outbreaks in NSW and Victoria had significant impacts on the dispensing, initiation and discontinuation of systemic cancer therapies, however, the overall effects were minimal. The impacts of lockdowns were less significant than the Omicron outbreaks, suggesting COVID-19 infection, health system capacity, and patient and community concerns were important factors for treatment changes.

The Medicines Intelligence (MedIntel) Data Platform is an anonymised linked data resource designed to generate real-world evidence on prescribed medicine use, safety, costs and cost-effectiveness in Australia. The platform comprises Medicare-eligible people who are ≥18 years and residing in New South Wales (NSW), Australia, any time during 2005-2020, with linked data on dispensed prescription medicines (Pharmaceutical Benefits Scheme), health service use (Medicare Benefits Schedule), emergency department visits (NSW Emergency Department Data Collection), hospitalisations (NSW Admitted Patient Data Collection), cancer notifications (NSW Cancer Registry), fact and cause of death (National Death Index). Data are currently available to 2022, with approval to update the cohort and data collections annually. The platform includes 7.4 million unique people across all years, covering 36.9% of the Australian adult population; the overall population increased from 4.8M in 2005 to 6.0M in 2020. As of 1 January 2019 (the last pre-pandemic year), the cohort had a mean age of 48.7 years (51.1% female), with most people (4.4M, 74.7%) residing in a major city. In 2019, 4.4M people (73.3%) were dispensed a medicine, 1.2M (20.5%) were hospitalised, 5.3M (89.4%) had a GP or specialist appointment, and 54 003 people died. Anti-infectives were the most prevalent medicines dispensed to the cohort in 2019 (43.1%), followed by nervous system (32.2%) and cardiovascular system medicines (30.2%). The MedIntel Data Platform creates opportunities for national and international research collaborations and enables us to address contemporary clinically- and policy-relevant research questions about quality use of medicines and health outcomes in Australia and globally. Five Key Points: The Medicines Intelligence (MedIntel) Data Platform is a purpose-built, real-world data resource designed to generate evidence on prescribed medicine use, safety, costs and cost-effectiveness in Australia This is a whole of population anonymised linkage of medicine and health records of Medicare-eligible adults (≥18 years) residing in Australia’s most populous state, New South Wales, from 2005 onwards The platform comprised 7.4M unique people across all years, representing 36.9% of the Australian adult population; the population increased from 4.8M in 2005 to 6.0M in 2020 As of 1 January 2019 (the last pre-pandemic year), the cohort had a mean age of 48.7 years (51.1% female), with most people (4.4M, 74.7%) residing in a major city The MedIntel Data Platform creates unique research opportunities for national and international research collaborations exploring population-level medicine use and outcomes Plain Language Summary: The Medicines Intelligence (MedIntel) Data Platform is a new linked data resource established to generate evidence on prescribed medicine use, safety, costs, and cost-effectiveness in Australia. It adheres to best practice privacy principles, with no identifying information available to researchers. The platform comprises Medicare-eligible people who are ≥18 years and residing in New South Wales (NSW), Australia, any time during 2005-2020, with linked data on dispensed prescription medicines, Medicare services, emergency department visits, hospitalisations, cancer notifications, and deaths. In total, the platform includes 7.4 million unique people across all years, covering 36.9% of the Australian adult population. As of 1 January 2019 (the last pre-pandemic year), the cohort had a mean age of 48.7 years (51.1% female), with most people (4.4M, 74.7%) residing in a major city. In 2019, 4.4M people (73.3%) were dispensed a medicine (most commonly anti-infective, nervous system, and cardiovascular medicines), 1.2M (20.5%) were hospitalised, 5.3M (89.4%) had a GP or specialist appointment, and 54 003 people died. Data are available until 2022 with approval for annual updates. This platform creates opportunities for national and international research collaborations and enables us to address important questions about quality use of medicines and health outcomes.