Pharmaceutical Benefits Scheme Research Articles

OP27 Impact Of Generic Entry Of Pharmaceuticals On Drug Prices In Australia

IntroductionPolicy makers are keen to introduce cost containment measures in medicine spending due to aging populations and fiscal pressure. A major price reform was applied to the Australian Pharmaceutical Benefits Scheme (PBS) in 2014. This aimed to stimulate price reductions by increasing competition among generics, amidst growing evidence at the time of unnecessarily high generic medicine prices in Australia. The aim of this study was to estimate the effect of patent expiration and generic competition on drug prices, while controlling for other determinants of drug prices in Australia.MethodsA dataset from publicly available sources was constructed using monthly data on the price of drugs listed on the PBS from October 2014 to July 2022. The information included the generic drug name, item code, date, approved ex-manufacturer price, dispensed price per maximum quantity, and brand names. This was supplemented with monthly government spending and number of prescriptions filled per item code. A fixed effects regression model was used to estimate the effect of patent expiration and generic competition on dispensed drug prices.ResultsThe model estimated that introducing generics in Australia led to an 18 percent decrease in prices, excluding further decreases resulting from other controlled variables. The price elasticity of total prescriptions filled was estimated to be -0.6, suggesting that a one percent increase in prescriptions filled resulted in drug prices being lowered by 0.6 percent. This reflects the fact that, on average, firms reacted by reducing prices to increase market share when faced with an increase in quantity demanded. Each extra competitor was estimated to result in a reduction in prices of roughly 1.8 percent.ConclusionsThese results show that entry of generics into the Australian pharmaceutical market resulted in a significant reduction in drug prices. However, this alone does not provide empirical support for the effectiveness of these price reforms in generating savings by inducing generic competition, especially over other forms of pharmaceutical regulation.

PP87 Glecaprevir/pibrentasvir (Maviret®) Remains A Cost-effective Treatment For Chronic Hepatitis C Virus Infection After Changes To The Treated Population

IntroductionThe first direct-acting antiviral (DAA) therapies for chronic Hepatitis C virus (HCV) infection were reimbursed via Australia’s Pharmaceutical Benefits Scheme (PBS) in March 2016. This was based on the recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) that the regimens would be acceptably cost-effective at an incremental cost-effectiveness ratio (ICER) of AUD15,000/quality-adjusted life-year (QALY). Broad access to DAA therapies has been a key strategy in driving a national health goal to eliminate viral hepatitis as a major health threat by 2030. Since the initial PBS listings for DAA therapies and subsequent listings of newer DAA treatments such as Maviret, the demographics and disease characteristics of currently treated patients have markedly changed. The aim of our analysis was to reassess the cost-effectiveness of Maviret, accounting for the changes of the treated population characteristics and retreatment in first-line failures and reinfected individuals.MethodsTo assess the cost-effectiveness six years after initial listing of Maviret, an update was made to the Markov model used to achieve PBS reimbursement for Viekira-Pak® in May 2016. Amendments to the Viekira-Pak model include: changes to baseline age and fibrosis distribution of treated patients, and incorporation of retreatment of first-line failures (those not achieving a sustained virologic response (SVR)) and reinfected individuals. Treatment-related inputs including SVR response rates, adverse events, treatment-related disutility and discontinuations were sourced from pivotal glecaprevir/pibrentasvir clinical trials.ResultsUsing the published price of Maviret, the ICER is above AUD15,000/QALY but well below the commonly used ICER threshold in other chronic diseases (AUD45,000/QALY). When the confidential effective price is used, the ICER is under the AUD15,000/QALY cost-effectiveness threshold set by the PBAC for DAA therapies.ConclusionsDespite substantial changes to the population seeking treatment in Australia since reimbursement in 2016, Maviret remains a cost-effective treatment for chronic HCV infection.

OP14 Cost-Utility Analysis Of Regorafenib For Patients With Hepatocellular Carcinoma Who Progressed On Sorafenib Treatment

IntroductionIn the RESORCE trial, regorafenib was shown to provide overall survival (OS) benefit for patients with hepatocellular carcinoma (HCC) that has progressed on sorafenib treatment. Subsequently, it was approved by the Therapeutic Goods Administration for the treatment of patients with HCC who were previously treated with sorafenib; however, regorafenib is still not recommended by the Pharmaceutical Benefits Advisory Committee in Australia. We aimed to assess the cost effectiveness of regorafenib as a second-line therapy for patients with HCC who progressed on sorafenib from an Australian healthcare perspective.MethodsWe developed a Markov model to compare the cost effectiveness of regorafenib with best supportive care (BSC) as a second-line therapy for HCC after treatment with sorafenib. The health outcomes of life-years and quality-adjusted life-years (QALYs) were derived from the RESORCE trial. Survival benefits sourced from the RESORCE trial were fitted with the parametric model to estimate survival beyond the follow-up period. Drug costs and costs associated with adverse events (AEs) were sourced from published literature and the Independent Health and Aged Care Pricing Authority cost report. Model validity was verified using probabilistic sensitivity analyses.ResultsThe incremental monthly cost of treatment with regorafenib was AUD19,273 (USD13,374), with an incremental life-year gain of 0.38, compared with BSC. The incremental QALYs gained with regorafenib were 0.24, resulting in a base-case incremental cost-effectiveness ratio (ICER) of AUD80,511 (USD55,872) per QALY. In the probabilistic sensitivity analyses across scenarios, the ICER remained above the conventional threshold of AUD50,000 (USD34,698) per QALY, with a zero probability of being cost effective at this willingness-to-pay threshold.ConclusionsAt the current price, second-line treatment with regorafenib in patients with HCC that has progressed on sorafenib was not cost effective at the conventional willingness-to-pay threshold from an Australian health-system perspective.

PP93 Health Technology Assessments For Rare Diseases In Australia: A Case Study On Cystic Fibrosis

IntroductionCurrently, no cure exists for the 1 in 2,500 Australian babies born with potentially fatal cystic fibrosis (CF). The authors conducted a health technology assessment (HTA) case study analysis of all regulatory approved CF treatments in Australia from January 1994 to July 2022. Submissions were also made under the Therapeutics Goods Administration and Pharmaceutical Benefits Advisory Committee (TGA-PBAC) parallel process.MethodsPublic summary and source materials were researched to understand relevant clinical and health economic evidence requirements, and access decisions from Australia’s lead HTA body, PBAC.ResultsThe review found that there are more than seven approved products in Australia. Of those, all four novel CF transmembrane conductance regulator (CFTR) modulating medications, which treat the underlying disease, received an orphan drug designation and were eventually listed. However, initial HTA decisions were mixed, with one recommended (25%), one not recommended (25%), and two deferred (50%). Clinical efficacy, cost-effectiveness, clinical need, as well as patient/carer-centric perspectives were most influential in HTA recommendations. Like other rare disease treatments, price, high incremental cost-effectiveness ratios (ICERs), uncertainty around cost-effectiveness and/or efficacy were key barriers to positive decisions. Notably, Australian stakeholders did not recommend CF medicines when their ICERs significantly exceeded a threshold of AUD200,000 (USD134,700) per quality-adjusted life year (QALY) gained. Administratively, Australia addresses risks associated with poor cost-effectiveness and high costs through managed access programs, risk-sharing agreements (RSA) and special pricing arrangements.Recently approved elexacaftor-tezacaftor-ivacaftor would be inaccessible to many Australian patients without inclusion in the Pharmaceutical Benefits Scheme (PBS); this placement increases access by limiting patients’ payments to AUD42.50 (USD28.62) maximum per prescription. Alternatively, manufacturers of therapies for other chronic or rare life-threatening conditions can participate in Australia’s Highly Specialised Drugs Program and/or Life Saving Drugs Program to facilitate access.ConclusionsCompanies can accelerate and optimize market access by using the TGA-PBAC parallel process. Other Asia-Pacific countries can model components of Australia’s approach to advancing access to innovative, live-saving therapies.

HTA23 Reasons for a Negative Pharmaceutical Benefits Advisory Committee (PBAC) Review in Rare Diseases in Australia: A Comprehensive Analysis

HTA23 Reasons for a Negative Pharmaceutical Benefits Advisory Committee (PBAC) Review in Rare Diseases in Australia: A Comprehensive Analysis

Denosumab and Mortality in a Real-World Setting: A Comparative Study.

Denosumab (Dmab) is increasingly prescribed worldwide. Unlike bisphosphonates (BPs), its effect on mortality has yet to be well explored. This study examined the association between Dmab and all-cause mortality compared with no treatment in subjects with a fracture and BPs in subjects without a fracture. The study population was from the Sax Institute's 45 and Up Study (n = 267,357), a prospective population-based cohort with questionnaire data linked to hospital admissions (Admitted Patients Data Collection [APDC] data were linked by the Centre for Health Record Linkage), medication records (Pharmaceutical Benefits Scheme [PBS] provided by Services Australia), and stored securely (secure data access was provided through the Sax Institute's Secure Unified Research Environment [SURE]). The new-user cohort design with propensity-score (PS) matching was implemented. In the fracture cohort, Dmab and oral BP users were matched 1:2 to no treatment (Dmab: 617 women, 154 men; oral BPs: 615 women, 266 men). In the no-fracture cohort, Dmab users were matched 1:1 with oral BPs and zoledronic acid (Zol) users (Dmab:oral BPs: 479 men, 1534 women; Dmab:Zol: 280 men, 625 women). Mortality risk was measured using sex-specific pairwise multivariable Cox models. In the fracture cohort, compared with no treatment, Dmab was associated with 48% lower mortality in women (hazard ratio [HR] = 0.52, 95% confidence interval [CI] 0.36-0.72) but not in men. Oral BPs were associated with 44% lower mortality in both sexes (women HR = 0.56, 95% CI 0.42-0.77; men HR = 0.56, 95% CI 0.40-0.78). In the no-fracture cohort, compared with BPs, Dmab was associated with 1.5- to 2.5-fold higher mortality than oral BPs (women HR = 1.49, 95% CI 1.13-1.98; men HR = 2.74; 95% CI 1.82-4.11) but similar mortality to Zol. Dmab in women and oral BPs were associated with lower post-fracture mortality than no treatment. However, Dmab users had generally higher mortality than oral BP users in those without fractures. © 2023 American Society for Bone and Mineral Research (ASBMR).

Psychosocial safety climate (PSC) and working conditions, predictors of mental health and antidepressant and opioid use in Australia: a study protocol for longitudinal data linkage

IntroductionWork-related stress is a social determinant of global health that represents a huge cost to workers’ health and reduces work performance. In Australia, mental well-being is a pressing national issue—with...

PP132 Health Technology Assessment Agencies’ Expectations Regarding Patient Experience Data in Australia, China, And Japan

IntroductionHealth technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.MethodsPublished HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.ResultsAustralia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.ConclusionsThere are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.

PP147 What Does Real World Evidence (RWE) Offer Health Technology Assessment (HTA) Procedures In Australia?

IntroductionMedical device health technology assessment (HTA) in Australia is largely coordinated by the Medical Services Advisory Committee (MSAC). Its remit to improve the public’s health by deciding where to allocate public healthcare funding, can be enhanced by considering real world evidence (RWE). Existing data sources have limitations that can be addressed through RWE, including coverage of Australian patient populations who may not meet trial eligibility criteria, and long-term follow-up through data linkage and datasets. We partnered with a university to explore what information could be gained from an analysis of linked administrative patient data, with a view to addressing current evidence gaps and/or limitations. The findings can be used as a source of local data to define patient populations, estimate actual costs of care, and enable more comprehensive economic modeling to inform medical device HTA.MethodsThe University-developed New South Wales Cardiovascular Cohort dataset, comprising person-level longitudinal NSW administrative data for all patients admitted to hospital with a cardiovascular diagnosis from 2001 onwards, linked to national Medicare Benefits Schedule and Pharmaceutical Benefits Scheme claims data, was interrogated.ResultsWorking with RWE is resource intensive in terms of time and costs. The potential of these data was revealed as the research progressed. It was possible to continually refine the data analyzed and reported,as well as expand the data requested. Varied expertise is required to accurately analyze the administrative datasets, particularly clinical classification skills and expertise in methods for causal inference using observational data. Findings from this study will enable the refinement of information for MSAC submissions, including identifying the most relevant patient population and reporting comprehensive costs, beyond an admitted hospital setting. The data will enhance engagement with clinicians and refine messaging, for example regarding patient risk factors.ConclusionsRWE enhances Australian HTA applications. Local data, extended periods of time and insights not apparent from a focus on admitted hospital episodes can be revealed. Data can be refined during the process for specificity and applicability.

The disease-modifying therapy utilisation and cost trend for multiple sclerosis in Australia between 2013 and 2022

Background: The MS disease-modifying therapies (DMTs) prescribing landscape in Australia have changed over time. Objectives: This study evaluated the utilisation and cost trends of MS-related DMTs in Australia over 10 years and investigated differences between States/Territories. Methods: The prescription and costs of 16 DMTs were extracted from the Pharmaceutical Benefits Scheme for 2013–2022. Descriptive approaches analysed the total number of people prescribed DMTs and total DMT costs per 10,000 population, proportions of prescriptions/costs by DMT groups and the number of people prescribed each individual DMT and costs of each DMT over the 10-year period. All estimates were for Australia and each State/Territory individually. Results: The number of people prescribed DMT and costs per 10,000 population had substantial growth between 2013 and 2022: 125%/164% for Australia, and 94%–251%/129%–373% for individual States/Territories. Higher efficacy group accounted for 54% of total people prescribed DMTs in 2013 and 75% in 2022. Fingolimod was the most popular DMT until 2020, then was dominated by ocrelizumab. The trends of individual DMT prescriptions and costs differed between states particularly in Western Australia (WA), Tasmania and Northern Territory (NT). Conclusion: DMT prescriptions and costs continuously increased over the last decade, particularly for higher efficacy DMTs, and their trends differed between States/Territories.

Chronic Obstructive Pulmonary Disease Adverse Event Signals Associated with Potential Inhibitors of Glutathione Peroxidase 1: A Sequence Symmetry Analysis.

Prior molecular modelling analysis identified several medicines as potential inhibitors of glutathione peroxidase 1 (GPx1) which may contribute to development or progression of chronic obstructive pulmonary disease (COPD). This study investigates 40 medicines (index medicines) for signals of COPD development or progression in a real-world dataset. Sequence symmetry analysis (SSA) was conducted using a 10% extract of Australian Pharmaceutical Benefits Scheme (PBS) claims data between January 2013 and September 2019. Patients must have been initiated on an index medicine and a medicine for COPD development or progression within 12 months of each other. Sequence ratios were calculated as the number of patients who initiated an index medicine followed by a medicine for COPD development or progression divided by the number who initiated the index medicine second. An adjusted sequence ratio (aSR) was calculated which accounted for changes in prescribing trends. Adverse drug event signals (ADEs) were identified where the aSR lower 95% confidence interval (CI) was greater than 1. Twenty-one of 40 (53%) index medicines had at least one ADE signal of COPD development or progression. Signals of COPD development, as identified using initiation of tiotropium, were observed for atenolol (aSR 1.32, 95% CI 1.23-1.42) and naproxen (aSR 1.14, 95% CI 1.06-1.23). Several signals of COPD progression were observed, including initiation of fluticasone propionate/salmeterol following initiation of atenolol (aSR 1.44, 95% CI 1.30-1.60) and initiation of aclidinium/formoterol following initiation of naproxen (aSR 2.21, 95% CI 1.34-3.65). ADE signals were generated for several potential GPx1 inhibitors; however, further validation of signals is required in large well-controlled observational studies.

Identifying Longer-Term Health Events and Outcomes and Health Service Use of Low Birthweight CALD Infants in Australia

IntroductionApproximately one-third of all births in Australia each year are by culturally and linguistically diverse (CALD) women. CALD women are at an increased risk of adverse pregnancy and birth outcomes including prematurity and low birthweight. Infants born weighing less than 2500 g are susceptible to increased risk of ill health and morbidities such as cognitive defects including cerebral palsy, and neuro-motor functioning.MethodsAn existing linked administrative dataset, Maternity 1000 was utilized for this study which has identified all children born in Queensland (QLD), Australia, between 1st July 2012 to 30th June 2018 from the QLD Perinatal Data Collection. This has then been linked to the QLD Hospital Admitted Patient Data Collection, QLD Hospital Non-Admitted Patient Data Collection, QLD Emergency Department Data Collection, and Medicare Benefits Schedule and Pharmaceutical Benefits Scheme Claims Records between 1 and 2012 to 30th June 2019.ResultsCulturally and linguistically diverse infants born with low birthweight had higher mean and standard deviation of all health events and outcomes; potentially preventable hospitalisations, hospital re-admissions, ED presentations without admissions, and development of chronic diseases compared to non-CALD infants born with low birthweight.DiscussionResults from this study highlight the disparities in health service use and health events and outcomes associated with low birthweight infants, between both CALD and Australian born women. This study has responded to the knowledge gap of low birthweight on the Australian economy by identifying that there are significant inequalities in access to health services for CALD women in Australia, as well as increased health events and poor birth outcomes for these infants when compared to those of mothers born in Australia.

Exploring the Evolution of Statin Pricing in Australia: Observations of Price Disclosure Effects on Pharmaceutical Benefits Scheme Expenditure

ObjectivesThe high cardiovascular disease burden globally and in Australia necessitates attention on statin expenditure, the primary pharmacological intervention for cardiovascular disease risk factors. The Pharmaceutical Benefits Scheme (PBS) subsidies approved statins for Australians. Managing PBS government expenditure occurs through price control strategies of statutory price decreases upon first generic entry and price disclosure. This study investigates the impact price control measures had on statin price evolution and government expenditure between 2010 and 2022. MethodsPrescription and pricing data were obtained from Services Australia Medicare Statistics, and price reduction strategies from the PBS. Summary statistics compared and described statin price, prescription, number of brands, market share, and government expenditure to atorvastatin, fluvastatin, pravastatin, rosuvastatin, and simvastatin price control timelines. ResultsStatin prices exposed to price control measures decreased irrespective of dosage and correlated with reductions in government expenditure, with a comparison of 2010 and 2022 showing annual statin expenditure declined by AU$833.5 million (83.25%) whereas prescriptions reduced by 3.0 million (15.7%). Effects of price disclosure on atorvastatin and rosuvastatin market share suggest industry-prompted price reductions may arise from market share loss, whereas reasons external to pricing prompted rosuvastatin to gain market share. ConclusionsLimited publications on contemporary effects of statin price control measures exist. This investigation found these measures reduced government expenditure for statins by AU$949.1 million, with the price reduction correlating with price control measures. In addition to affirming price control mechanisms remain effective in contemporary times, this investigation provides data for key insights into the Australian statin industry.

On- and off-label utilisation of antipsychotics in Australia (2000-2021): Retrospective analysis of two medication datasets.

To determine antipsychotic utilisation patterns in Australian adults from 2005 to 2021, with a focus on on-label and off-label prescriptions. We examined antipsychotic dispensing trends in adults from 2005 to 2021 using a 10% sample of the Pharmaceutical Benefits Scheme (PBS) dataset, which contains patient-level information on medicines dispensed throughout Australia. The lack of diagnostic information in PBS was substituted by analysing BEACH (Bettering the Evaluation And Care of Health) dataset, a cross-sectional national survey from 2000 to 2016, consisting of data from general practitioner-patient encounters. There were 5.6 million dispensings for 164,993 patients in PBS throughout this period; 69% patients had >1 dispensing, with a median of 6 per patient. Calculating the estimated period of exposure gave a total of 693,562 treatment episodes, with a median duration of 80 days. There were steady increases in both the incidence and prevalence of antipsychotic dispensings, mainly due to oral second-generation antipsychotics. The most commonly prescribed antipsychotics were quetiapine, olanzapine and risperidone, with a significant portion of patients receiving low-dose quetiapine without dose titration. Analysis of diagnostic indications from BEACH indicated that 27% of antipsychotic prescriptions were off-label for indications such as depression, dementia, anxiety and insomnia, at much lower prescribed daily dosages. The increasing prescribing and off-label use highlights concerns about chronic adverse effects caused by antipsychotics. The combined analysis of medication dispensings and the diagnostic indications for which they are prescribed is a novel approach and throws a spotlight on the need for additional monitoring of antipsychotics.

Abstract 14262: Cancer - A Driver of Heart Failure in People With Diabetes

Introduction: Despite the risk of heart failure (HF) in the survivorship phase of some cancers, the risk is not high enough to justify HF screening. However, people with diabetes mellitus (DM) have an increased risk of both HF and cancer. Examining this at-risk population may allow for better understanding of the effect cancer has on the development of HF. Hypothesis: Among people with DM, HF is more common in those with than without prior cancer. Methods: Data from the Australian National Diabetes Services Scheme (NDSS; 1,114,356 DM, types 1 and 2) from 2010 to 2019, was linked with public hospital data, the National Death Index and national Pharmaceutical Benefits Scheme (PBS) database. People with cancer were identified by the prescription of chemotherapy agents on the PBS dataset or a hospital admission with cancer. Incident HF was detected via first hospitalisation with HF as the primary diagnosis or death due to HF. The model was adjusted for age and sex. Results: Of the total cohort (1,114,356 people with DM), 48,014 experienced HF during 6,663,046 person-years of follow up from 2010-2019. Specifically, of 175,656 people with DM and cancer, 8,464 experienced HF during 644,620 person-years of follow up. The HF incidence rate ratio (IRR) of people with DM and cancer was 1.63 (95%CI: 1.59-1.67), compared to people with DM but no cancer. Cancer admission alone had an IRR of 1.97 (1.90-2.05), with an IRR of 1.50 (1.46-1.54) in DM treated with chemotherapy with or without admission. Conclusion: Cancer diagnosis is associated with an increased incidence of HF among people with DM. The higher IRR of people with cancer admission alone supports the association of cancer with HF irrespective of chemotherapy. Further studies controlling for confounders of cardiovascular risk and refinements by age are required to justify grounds for HF screening.

Impact of the COVID-19 pandemic on delivery of prostate cancer care in Australia: An interrupted time series analysis.

The COVID-19 pandemic led to a major disruption to health services across the world. The aim of this population-based study was to assess the downstream effects of the pandemic on diagnostic tests and treatment activities related to prostate cancer (PC). The Australian Government Department of Health Medicare Benefits Schedule and the Pharmaceutical Benefits Scheme databases were queried from January 2010 to June 2022. Two interrupted time series were performed Pre-COVID (January 2010 to February 2020) and peri-COVID (March 2020 to June 2022). Temporal modeling was performed to account for seasonal variation. Pre-COVID-19, monthly prostate-specific antigen (PSA) testing showed a declining trend and testing decreased by 81 tests per 100 000 annually. A single-month 38% drop in PSA testing was observed in April 2020; this corresponded to Australia's first wave. No change was observed in the rate of prostate biopsies. Peri-COVID-19 outbreaks, there was a slight shift toward the use of long-acting androgen deprivation therapy (ADT) at 4% with a predilection still for short-acting agents. with no registered change in the overall volume of radiotherapy or surgery. There were no deficits in the number of diagnostic and treatment activities for men with PC. Aside from a slight shift toward long-acting ADT use during the pandemic, no other patterns were observed. The longer-term impact such as missed diagnosis or late presentation affecting chances of survival due to COVID-19 is yet to be ascertained.

Sodium-Glucose Cotransporter 2 Inhibitors in South Australia: The Magic Before the Fame

Recent clinical trials have demonstrated that sodium-glucose cotransporter 2 inhibitors (SGLT2i), which were previously only indicated in treatment of type 2 diabetes mellitus (T2DM), can markedly reduce heart failure hospitalisation (HFH), with less striking potential reductions in acute coronary syndromes and cardiac arrhythmias. To evaluate the impact of SGLT2i on cardiovascular outcomes in real-world practice, we performed a retrospective cohort analysis on South Australian (SA) data. A total of 842 individuals with T2DM receiving SGLT2i were identified from SA public hospitals between 2011 and 2019. Episodes of care were temporally matched with those of 3,128 individuals with T2DM not receiving SGLT2i (control). Baseline characteristics were adjusted using inverse probability treatment weighting. The incidence of cardiovascular events at 12 and 24 months was evaluated using coded (International Classification of Diseases, Tenth Revision, Australian Modification [ICD-10-AM]) data. The primary outcome of HFH was lower with SGLT2i use at 12 months (adjusted hazard ratio [HRadj] 0.44; 95% confidence interval [CI] 0.29-0.68; p<0.001) and 24 months. There were also lower hospitalisations due to acute myocardial infarction (HRadj 0.42; 95% CI 0.21-0.85; p=0.015) and atrial or ventricular arrhythmias (HRadj 0.29; 95% CI 0.14-0.59; p=0.001), with no difference observed in hospitalisation due to ischaemic cerebrovascular events. There was no difference in all-cause mortality at 12 months but interestingly a higher rate at 24 months (HRadj 2.08; 95% CI 1.59-2.72; p<0.001). Despite this, similar reductions in cardiovascular outcomes were observed at 24 months. Use of SGLT2i in patients with T2DM in SA was associated with reductions in cardiovascular events even before their recent Pharmaceutical Benefits Scheme (PBS) listing for heart failure. Furthermore, this analysis supports that SGLT2i play a role not only in HFH reduction but also in reducing coronary and tachyarrhythmic events. This real-world evidence supports the use of SGLT2i as broadly protective cardiovascular drugs.

Medication use in people with cystic fibrosis before and after modulator therapy

BackgroundLong-term changes in medication dispensings post cystic fibrosis transmembrane conductance regulator (CFTR) modulator initiation have not been described. Our study aimed to investigate changes in medication use following the initiation of modulator therapy in people with cystic fibrosis (PwCF) in Australia. MethodsUsing a 10% sample of the Australian Pharmaceutical Benefits Scheme (PBS) data between 2013 and 2022, linear regression was used to analyse dispensings in PwCF who initiated any modulator (cases) and matched PwCF controls not dispensed a modulator. The difference in mean number of total monthly dispensings pre- and post-modulator initiation was analysed, with separate analyses by medication class. ResultsA total of 247 cases were matched 1:1 to controls (case and control median age 21 years (IQR: 13–32), 55.1% male). Immediately after modulator initiation, the mean number of dispensings was 0.9 higher in the modulator group, but then decreased to the level of controls after approximately 5 years. After 7.5 years, cases had decreased opioids compared to the pre-modulator period (β-coefficient: −0.00131, 95% CI: −0.00164, −0.00097) whilst controls did not (β: −0.00014, 95% CI: −0.00042, 0.00014). Over the same time period controls had an increase in psychotropics (β: 0.00389, 95% CI:0.00295, 0.00484) whilst cases remained stable (β: −0.00014, 95% CI: −0.0006, 0.00031). Women's health medications increased in cases (β:0.00026, 95% CI:0.0001, 0.00042) but decreased in controls (β: 0.00044, 95% CI: 0.00063, −0.00025). ConclusionsModulator initiation in PwCF was associated with decreased dispensings of opioids and psychotropics, and increased dispensings of women's health medications, suggesting improved patient outcomes across multiple clinical domains.

O024 Sodium oxybate for refractory narcolepsy and idiopathic hypersomnia: clinical and employment outcomes

Abstract Background Sodium oxybate (SOX) is an evidenced based treatment for narcolepsy. SOX is not available in Australia through the pharmaceutical benefits scheme. In 2018, Western Australia (WA) listed sodium oxybate on the state formulary for the treatment of refractory narcolepsy. We examine the demographics, clinical and employment outcomes of patients treated with SOX. Methods We undertook a retrospective study of patients treated with sodium oxybate in WA for refractory narcolepsy or idiopathic hypersomnia (IH). A database of SOX patients was cross checked with pharmacy prescribing data from across WA public hospitals. Electronic medical records were used to collect demographic, polysomnographic, prescribing and outcome data. Clinical outcomes were based on the Epworth sleepiness scale, clinical assessment and employment status. Patients that responded to SOX are to be compared to non-responders using appropriate statistical methodology. Progress to date The study has been registered as a hospital clinical audit (number 49852). A total of 50 patients prescribed SOX for the treatment of a central hypersomnia have been identified. A request for sodium oxybate dispensing data across WA has been made. Review of 10 patient’s records has been completed. Correspondence, sleep studies, MSLT, HLA testing and prescriptions are being reviewed for the remaining 40 patients. Intended outcome and impact Describe the characteristics of patients treated with SOX in WA. Explore whether clinical or sleep study characteristics predict response to SOX. Assess patient reported outcomes from treatment with SOX based on symptoms and changes in employment status.

Determining the Impact of Opioid Policy on Substance Use and Mental Health-Related Harms: Protocol for a Data Linkage Study.

Increasing harms related to prescription opioids over the past decade have led to the introduction of a range of key national and state policy initiatives across Australia. These include introducing a mandatory real-time prescription drug-monitoring program in the state of Victoria from April 2020 and a series of changes to subsidies for opioids on the Pharmaceutical Benefit Scheme from June 2020. Together, these changes aim to influence opioid supply and reduce harms related to prescription opioids, yet few studies have specifically explored how these policies have influenced opioid prescribing and related harms in Australia. The aim of this study is to examine the impact of a range of opioid-related policies on hospital admissions and emergency department (ED) presentations in Victoria, Australia. In particular, the study aims to understand the effect of various opioid policies and opioid-prescribing changes on (1) the number and rates of ED presentations and hospital admissions attributed to substance use (ie, opioid and nonopioid related) or mental ill-health (eg, suicide, self-harm, anxiety, and depression), (2) the association between differing opioid dose trajectories and the likelihood of ED presentations and hospital admissions related to substance use and mental ill-health, and (3) whether changes in an individual's opioid prescribing change the risk related to ED presentations and hospital admissions related to substance use and mental ill-health. We will conduct a population-level linked data study. General practice health records obtained from the Population Level Analysis and Reporting platform are linked with person-level data from 3 large hospital networks in Victoria, Australia. Interrupted time series analysis will be used to examine the impact of opioid policies on a range of harms, including the rates of presentations related to substance use (opioid and nonopioid) and mental ill-health among the primary care cohort. Group-based trajectory modeling and a case-crossover design will be used to further explore the impact of changes in opioid dosage and other covariates on opioid and nonopioid poisonings and mental ill-health-related presentations at the patient level. Given that this paper serves as a protocol, there are currently no results available. The deidentified primary health data were sourced from electronic medical records of approximately 4,717,000 patients from 542 consenting general practices over a 6-year period (2017-2022). The submission of results for publication is planned for early 2024. This study will add to the limited evidence base to help understand the impact of opioid policies in Australia, including whether intended or unintended outcomes are occurring as a result. EU PAS Register EUPAS104005; https://www.encepp.eu/encepp/viewResource.htm?id=104006. DERR1-10.2196/51825.