Australian Public Healthcare System Research Articles

Combined population genomic screening for three high-risk conditions in Australia: a modelling study

No previous health-economic evaluation has assessed the impact and cost-effectiveness of offering combined adult population genomic screening for mutliple high-risk conditions in a national public healthcare system. This modeling study assessed the impact of offering combined genomic screening for hereditary breast and ovarian cancer, Lynch syndrome and familial hypercholesterolaemia to all young adults in Australia, compared with the current practice of clinical criteria-based testing for each condition separately. The intervention of genomic screening, assumed as an up-front single cost in the first annual model cycle, would detect pathogenic variants in seven high-risk genes. The simulated population was 18-40 year-olds (8,324,242 individuals), modelling per-sample test costs ranging AU$100-$1200 (base-case AU$200) from the year 2023 onwards with testing uptake of 50%. Interventions for identified high-risk variant carriers follow current Australian guidelines, modelling imperfect uptake and adherence. Outcome measures were morbidity and mortality due to cancer (breast, ovarian, colorectal and endometrial) and coronary heart disease (CHD) over a lifetime horizon, from healthcare-system and societal perspectives. Outcomes included quality-adjusted life years (QALYs) and incremental cost-effectiveness ratio (ICER), discounted 5% annually (with 3% discounting in scenario analysis). Over the population lifetime (to age 80 years), the model estimated that genomic screening per-100,000 individuals would lead to 747 QALYs gained by preventing 63 cancers, 31 CHD cases and 97 deaths. In the total model population, this would translate to 31,094 QALYs gained by preventing 2612 cancers, 542 non-fatal CHD events and 4047 total deaths. At AU$200 per-test, genomic screening would require an investment of AU$832 million for screening of 50% of the population. Our findings suggest that this intervention would be cost-effective from a healthcare-system perspective, yielding an ICER of AU$23,926 (∼£12,050/€14,110/US$15,345) per QALY gained over the status quo. In scenario analysis with 3% discounting, an ICER of AU$4758/QALY was obtained. Sensitivity analysis for the base case indicated that combined genomic screening would be cost-effective under 70% of simulations, cost-saving under 25% and not cost-effective under 5%. Threshold analysis showed that genomic screening would be cost-effective under the AU$50,000/QALY willingness-to-pay threshold at per-test costs up to AU$325 (∼£164/€192/US$208). Our findings suggest that offering combined genomic screening for high-risk conditions to young adults would be cost-effective in the Australian public healthcare system, at currently realistic testing costs. Other matters, including psychosocial impacts, ethical and societal issues, and implementation challenges, also need consideration. Australian Government, Department of Health, Medical Research Future Fund, Genomics Health Futures Mission (APP2009024). National Heart Foundation Future Leader Fellowship (102604).

Novel machine learning model for predicting multiple unplanned hospitalisations

BackgroundIn the Australian public healthcare system, hospitals are funded based on the number of inpatient discharges and types of conditions treated (casemix). Demand for services is increasing faster than public...

THE LONG ARTHROPLASTY WAITING TIMES EFFECT ON SARCOPENIA (THE LATE STUDY) IN THE SOUTH AUSTRALIA PUBLIC HEALTHCARE SYSTEM: A PILOT STUDY

The progressive painful and disabling predicament of patients with severe osteoarthritis awaiting a total hip or knee arthroplasty (THA/TKA) results in a decline in muscle mass, strength and function also known as Sarcopenia.We conducted a cross-sectional, prospective study of patients on the waiting-list for a THA/TKA in the South Australian public healthcare system and compared the findings to healthy participants and patients newly referred from their general practitioners. Participants with a history of joint replacements, pacemakers and cancers were excluded from this study. Outcomes of this study included (i) sarcopenia screening (SARC-F ≥4); (ii) sarcopenia, defined as low muscle strength (hand grip strength M<27kg; F<16kg), low muscle quality (skeletal muscle index M<27%, F<22.1%) and low physical performance (short physical performance battery ≤8). Additional outcomes include descriptions of the recruitment feasibility, randomisation and suitability of the assessment tools.29 healthy controls were recruited; following screening, 83% (24/29) met the inclusion criteria and 75% (18/24) were assessed. 42 newly referred patients were recruited; following screening, 67% (30/45) met the inclusion criteria and 63% (19/30) were assessed. 68 waiting list patients were recruited; following recruitment, 24% (16/68) met the inclusion criteria and 75% (12/16) were assessed. Preliminary data shows increasing waiting time is associated with higher SARC-F scores, lower hand grip strength and lower muscle quality.As a pilot study, preliminary data demonstrate that: (1) study subjects’ willingness to participate will enable a larger study to be conducted to establish the prevalence of sarcopenia and the diagnostic cut-off points for this patient group. (2) SARC-F is a suitable tool to screen for sarcopenia. (3) There is a positive correlation between waiting time for a THA/TKA and sarcopenia.

O74 - Support for midwives to practice with professional autonomy within their full scope of practice: Evidence from a scoping review

Midwives in Australia are educated and professionally accountable to practice in partnership with childbearing women and their families, yet most do not practice to their scope. There is an urgent need to review models of maternity care in Australia to facilitate growth in midwifery continuity of carer, relationship building, and partnership to optimise perinatal health and wellbeing, and to reduce midwifery burnout. This systematic scoping review of the literature maps the role and scope of contemporary midwifery practice in Australia to identify key issues that impact upon midwives practising to their full scope of practice. A scoping review guided by Arksey and O’Malley’s five-stage methodological framework, together with a ‘best fit’ framework synthesis using the Nursing and Midwifery Board of Australia’s Midwifery Standards for Practice. Following initial screening, 517 publications were retrieved, and with full text review evidence from 96 papers was included in this review. Key themes that emerged from the review included the central tenet of Partnership with women, facilitated by the Professional roles of the midwife, and mediated by Contextual influences . Tensions were identified between the midwifery scope of practice associated with optimal outcomes for women and babies supported by current evidence and the actual role and scope of most midwives employed in models of care in the current Australian public healthcare system. There is a mismatch between the operational parameters for midwifery practice in Australia and the evidence-based models of continuity of midwifery carer that are associated with optimal outcomes for childbearing women and babies and the midwives themselves. There is an urgent need to review the representation of the midwifery profession at leadership, executive and policy levels to support the role of the midwife to practice with professional autonomy within their full scope of practice.

A cost-effectiveness model for a decision to adopt temporary single-patient rooms to reduce risks of healthcare-associated infection in the Australian public healthcare system.

The cost-effectiveness of patient isolation as part of an infection prevention effort is poorly understood. The potential to reduce risks of transmission saving costs and improving health outcomes is strong, yet up front investments in patient isolation are required. We report a cost-effectiveness model to inform adoption of a portable single isolation rooms into Australian publicly funded acute hospitals. Information is harvested from a range of contemporary sources to reveal the expected changes to total costs and total health benefits measured by life years gain. An Incremental cost-effectiveness ratio is estimated with uncertainty in all model parameters included by probabilistic sensitivity analysis. The adoption decision was found to change total costs per 100,000 occupied bed-days by $1,429,011 and generate health benefits of 436 life years. The mean cost per life year gained is $3278. The probability an adoption decision is cost saving is 2.1%. There is some evidence that adoption of rediroom is likely to be a cost-effective solution for Australian hospitals. Important caveats and assumptions need to be considered when interpreting this conclusion.

Prescription trends and costs of diabetes medications in Australia between 2003 and 2019: an analysis and review of the literature.

Since the turn of the century, the prevalence of diabetes mellitus in Australia has increased, primarily due to rising rates of Type 2 diabetes. Simultaneously, the landscape of diabetes medications has evolved significantly. The change in prescribing trends and public spending on diabetes medications within Australia during this period are not well defined. To establish the frequency and cost of dispensed diabetes medications in the Australian public healthcare system between 2003 and 2019. We performed a longitudinal nationwide observational study using data obtained from the Pharmaceutical Benefits Scheme (PBS) and Medicare Benefits Schedule websites, which contain information on frequency and spending of diabetes medications dispensed in Australia. The total number of PBS-subsidised prescriptions dispensed for diabetes increased from 5 218 690 in 2003 to 12 188 568 in 2019, and spending increased from $117 241 031 to $598 904 983. Of the non-insulin agents, metformin was consistently the most frequently dispensed agent, with a rapid growth in metformin combination tablets. Dispensation of sulphonylureas and thiazolidinediones have declined, with a simultaneous increase in dipeptidyl peptidase-4 inhibitors, sodium-glucose co-transporter 2 inhibitors and glucagon-like peptide-1 receptor agonists. Our data show a large growth in the use of diabetes medications between 2003 and 2019. The rapid growth in dispensing of drugs with proven cardiovascular and renal benefits reflect the evolving approach of diabetes treatment, from a historical approach targeting glycaemic control alone, to a modern individualised approach targeting specific co-morbidities.

Delivering high throughput urgent exome sequencing at a diagnostic laboratory

Background: The clinical utility and feasibility of urgent exome sequencing established by the MRFF Acute Care Flagship (ACF)1 resulted in an increase in expedited referrals. Managing this demand while managing turnaround times (TAT) required implementation of a high throughput workflow and increased resourcing. Method: Retrospective audit of exome referrals at a public diagnostic genetics laboratory, NSW Health Pathology Randwick Genomics. Primary outcome was TAT for expedited exomes, measured from receipt to report issued. A referrer satisfaction survey was conducted. Results: Annual expedited and total exome referral numbers have grown from 0 expedited/56 total exomes in 2017 to 18/292 in 2018 to 48/430 in 2019 to 96/548 in 2020 to 234/1005 in 2021 (1/1/21–18/11/21). The median TAT for expedited exomes outside the ACF was 26 days in 2018, 24 days in 2019, 19 days in 2020 and 14 days in 2021. ACF TAT was 5, 6, and 9 days for 2018, 2019 and 2020 respectively. Referrers reported satisfaction with this service. Discussion: This study demonstrates that urgent/expedited exome reports can be provided in clinically relevant TAT, despite increasing referral numbers, using laboratory process redesign and modest staffing increases. Maintaining this level of service is essential for prenatal and acute inpatient/ICU testing. Reference 1. Australian Genomics Health Alliance Acute Care Flagship. Feasibility of ultra-rapid exome sequencing in critically ill infants and children with suspected monogenic conditions in the Australian public health care system. JAMA 2020; 323: 2503–11.

Cost-utility analysis of breast reduction surgery for women with symptomatic breast hypertrophy.

To assess the cost-effectiveness of breast reduction surgery for women with symptomatic breast hypertrophy in Australia. Cost-utility analysis of data from a prospective cohort study. Adult women with symptomatic breast hypertrophy assessed for bilateral breast reduction at the Flinders Medical Centre, a public tertiary hospital in Adelaide, April 2007 - February 2018. The control group included women with breast hypertrophy who had not undergone surgery. Health care costs (for the surgical admission and other related hospital costs within 12 months of surgery) and SF-6D utility scores (measure of health-related quality of life) were used to calculate incremental costs per quality-adjusted life-year (QALY) gained over 12 months, extrapolated to a 10-year time horizon. Of 251 women who underwent breast reduction, 209 completed the baseline and at least one post-operation assessment (83%; intervention group); 124 of 350 invited women waiting for breast reduction surgery completed the baseline and 12-month assessments (35%; control group). In the intervention group, the mean SF-6D utility score increased from 0.313 (SD, 0.263) at baseline to 0.626 (SD, 0.277) at 12 months; in the control group, it declined from 0.296 (SD, 0.267) to 0.270 (SD, 0.257). The mean QALY gain was consequently greater for the intervention group (adjusted difference, 1.519; 95% CI, 1.362-1.675). The mean hospital cost per patient was $11857 (SD, $4322), and the incremental cost-effectiveness ratio (ICER) for the intervention was $7808 per QALY gained. The probability of breast reduction surgery being cost-effective was 100% at a willingness-to-pay threshold of $50000 per QALY and 88% at $28033 per QALY. Breast reduction surgery for women with symptomatic breast hypertrophy is cost-effective and should be available to women through the Australian public healthcare system.

Resource utilization and disaggregated cost analysis of bariatric surgery in the Australian public healthcare system.

ObjectivesTo present a comprehensive real-world micro-costing analysis of bariatric surgery.MethodsPatients were included if they underwent primary bariatric surgery (gastric banding [GB], gastric bypass [GBP] and sleeve gastrectomy [SG]) between 2013 and 2019. Costs were disaggregated into cost items and average-per-patient costs from the Australian healthcare systems perspective were expressed in constant 2019 Australian dollars for the entire cohort and subgroup analysis. Annual population-based costs were calculated to capture longitudinal trends. A generalized linear model (GLM) predicted the overall bariatric-related costs.ResultsN = 240 publicly funded patients were included, with the waitlist times of ≤ 10.7 years. The mean direct costs were $11,269. The operating theatre constituted the largest component of bariatric-related costs, followed by medical supplies, salaries, critical care use, and labour on-costs. Average cost for SG ($12,632) and GBP ($15,041) was higher than that for GB ($10,049). Operating theatre accounted for the largest component for SG/GBP costs, whilst medical supplies were the largest for GB. We observed an increase in SG and a decrease in GB procedures over time. Correspondingly, the main cost driver changed from medical supplies in 2014–2015 for GB procedures to operating theatre for SG thereafter. GLM model estimates of bariatric average cost ranged from $7,580 to $36,633.ConclusionsWe presented the first detailed characterization of the scale, disaggregated profile and determinants of bariatric-related costs, and examined the evolution of resource utilization patterns and costs, reflecting the shift in the Australian bariatric landscape over time. Understanding these patterns and forecasting of future changes are critical for efficient resource allocation.Supplementary InformationThe online version contains supplementary material available at 10.1007/s10198-021-01405-x.

A Budget Impact Model for the use of Drug-Eluting Stents in Patients with Symptomatic Lower-Limb Peripheral Arterial Disease: An Australian Perspective

PurposeImprovement in long-term outcomes through innovative, cost-effective medical technologies is a focus for endovascular procedures aimed at treating symptomatic lower-limb peripheral arterial disease (PAD). The advent of drug-eluting stents (DES) has improved symptomatic PAD treatment via a reduction in high rates of target lesion revascularisation (TLR). The present study aimed to compare the 5-year financial impact of treatment with Eluvia, a new paclitaxel-eluting stent, versus treatment with Zilver PTX, a drug-coated stent, among patients in Australia by developing a budget impact model (BIM).MethodsA BIM was developed from an Australian public hospital payer perspective using Australian national cost weights (AUD), published literature, and public hospital audit data. Clinical outcomes, including clinically driven TLRs (CD-TLRs), adverse events, and length of stay, were based on the 2-year results of the IMPERIAL trial, which compared Eluvia DES to Zilver PTX.ResultsAssuming EVP eligibility rate of 80% and DES uses rate ranging from 10 to 28% (superficial femoral artery lesions only), the 5-year model forecasted a treatment population between 14,428 and 40,399 patients. The model estimated 1499–4198 fewer CD-TLRs and 16,515–46,243 fewer hospital days with Eluvia DES use. This translated to 5-year potential savings of $4.3–$12.1 million to the Australian public hospital payer attributable to reduced CD-TLRs for Eluvia DES and $33.1–$92.6 million to Australian public hospitals owing to reduced adverse events and hospital bed days.ConclusionEluvia DES use as treatment for symptomatic lower-limb PAD could lead to potential savings for the Australian public healthcare system based on improved patient outcomes.

PSU11 Resource Utilization and Disaggregated Cost Analysis of Bariatric Surgery in the Australian Public Healthcare System

To present a real-world micro-costing analysis of bariatric surgery in the Australian public healthcare system. In this retrospective cohort study, patients undergoing primary bariatric surgery (gastric banding [GB], gastric bypass [GBP] and sleeve gastrectomy [SG]) for the fiscal years 1st-July-2013 to 30th-June-19 were identified through the Tasmanian Department of Health’s administrative databases. Disaggregated, average-per-patient, and overall costs (2019 AU$) were presented. Subgroup analyses by surgical type were conducted. Annual population-based costs were calculated to capture longitudinal trends. A generalized linear model (GLM) was performed to predict the total costs for a base case scenario. 240 patients were included. Mean direct costs were $11,269 (US$7,799). The operating theatre cost bucket constituted the largest component of bariatric surgery-related costs, followed by costs for medical supplies, salaries, CCU use, and labour on-costs. Average costs for SG ($12,632) and GBP ($15,041) were higher than those for GB ($10,049). Operating theatre-related costs accounted for the largest component for SG and GBP, whilst medical supplies were the largest for GB. We observed an increase in SG/GBP and a decrease in GB procedures over time. Correspondingly, the main cost driver changed from medical supplies in 2014-2015 for GB procedures to operating theatre after 2014-2015 for SG/GBP procedures. In addition to surgery type, CCU use, number of comorbidities and the presence of complications were also associated with bariatric expenditures. GLM modelling of these parameters showed that the average cost for a representative cohort ranged from $7,580 to $36,633. This analysis presented the first detailed characterisation of the scale, disaggregated profile and determinants of costs of bariatric surgery and examined the evolution of resource utilization patterns and costs, reflecting the shift in Australian bariatric surgery landscape overtime. Understanding these patterns and forecasting of future changes are critical for healthcare resource allocation and sustainable budgetary planning.

Integrating a Pilot Newborn Screening for Spinal Muscular Atrophy Into the Australian Public Healthcare System

Integrating a Pilot Newborn Screening for Spinal Muscular Atrophy Into the Australian Public Healthcare System

Patient satisfaction with physiotherapists is not inferior to surgeons in an arthroplasty review clinic: non-inferiority study of an expanded scope model of care.

Objective This study evaluated whether the satisfaction of patients attending a physiotherapy-led postarthroplasty review out-patient clinic was non-inferior to that of patients attending traditional surgeon clinics. Methods Using a modified nine-item Visit-Specific Satisfaction Instrument (VSQ-9), 50 patients attending the physiotherapy clinic and 50 patients attending surgeon clinics were surveyed. Sample means (±s.d.) were calculated for each domain of the VSQ-9. Non-inferiority testing was performed using 95% confidence intervals (CIs) of the adjusted mean difference to examine whether normalised patient satisfaction scores in the physiotherapy group were no worse than those in the surgeon group. Results Both groups were satisfied (overall domain; 100% of both groups rated good-excellent). Based on mean item score, there was strong evidence that the satisfaction of the physiotherapy group was non-inferior to that of the surgeon group (adjusted mean difference (physiotherapy - surgeon) in mean score 5.1 (95% CI -0.3, 10.4). Furthermore, the mean differences in all but one of the nine domains were in favour of the physiotherapy group in this study. Conclusion The satisfaction of patients attending a physiotherapy arthroplasty review clinic is not inferior to that of patients attending traditional surgeon clinics. What is known about the topic? A substitution model of care where an advanced practice physiotherapist reviews routine postarthroplasty patients instead of surgeons originated in the UK and has been adopted in Canada and Australia to address the increasingly overloaded public out-patient health services. Evidence of clinical and cost effectiveness has been demonstrated but, to the best of the authors' knowledge, only one paper has evaluated consumer engagement, and that paper was in a Canadian population. What does this paper add? This study evaluated the satisfaction of patients in an Australian public healthcare system and was able to demonstrate that patients seen by physiotherapists were no less satisfied than those seen by surgeons in traditional clinics. The findings support those reported in the Canadian study. This is an important step in the development and acceptance of these substitution model of care innovations locally. What are the implications for practitioners? The results of this study will provide an important addition to the evidence of the clinical efficacy of this model of care: that of consumer acceptance. This will assist with planning, expansion and rollout of similar initiatives in Australia.

PP329 An Australian Cost-Effectiveness Analysis Of The EluviaTM Drug-Eluting Stent For Treatment Of Symptomatic Lower-Limb Peripheral Artery Disease

IntroductionDespite advances in endovascular interventions, including the introduction of drug-eluting stents (DES), high target lesion revascularization (TLR) rates still burden the treatment of symptomatic lower-limb peripheral arterial disease (PAD). EluviaTM, a novel, sustained-release, paclitaxel-eluting DES, was shown to further reduce TLRs when compared with the paclitaxel-coated Zilver® PTX® stent, in the IMPERIAL randomized controlled trial. This evaluation estimated the cost-effectiveness of Eluvia when compared with Zilver PTX in Australia, based on 12-month clinical outcomes from the IMPERIAL trial.MethodsA state-transition, decision-analytic model with a 12-month time horizon was developed from an Australian public healthcare system perspective. Cost parameters were obtained from the Australian National Hospital Cost Data Collection Cost Report (2016–17). All costs were captured in Australian dollars (AUD), where AUD 1 = USD 0.69 (June 2020). Complete sets of clinical parameters (primary patency loss, TLR, amputation, and death) and cost parameters from their respective distributions were bootstrapped in samples of 1,000 patients, for each intervention arm of the model. One-way and probabilistic sensitivity analyses were performed.ResultsAt 12 months, modeled TLR rates were 4.5 percent for Eluvia and 8.9 percent for Zilver PTX, and mean total direct costs were AUD 6,537 [USD 4,511] and AUD 6,908 [USD 4,767], respectively (Eluvia average per patient savings; overall cohort=AUD 371 [USD 256]; diabetic cohort=AUD 625 [USD 431]). In probabilistic sensitivity analyses, Eluvia was cost-effective relative to Zilver PTX in 92.0 percent of all simulations at a threshold of $10,000 per TLR avoided. Eluvia was more effective and less costly (dominant) than Zilver PTX in 76.0 percent of simulations.ConclusionsIn the first year after the intervention, Eluvia was more effective and less costly than Zilver PTX, making Eluvia the dominant treatment strategy for treatment of symptomatic lower-limb PAD, from an Australian public healthcare system perspective. These findings should be considered when formulating policy and practice guidelines in the context of priority setting and making evidence-based resource allocation decisions for treatment of PAD in Australia.

Feasibility of Ultra-Rapid Exome Sequencing in Critically Ill Infants and Children With Suspected Monogenic Conditions in the Australian Public Health Care System

Multiple studies have shown that genomic testing has a high diagnostic yield and an impact on clinical management for patients with suspected genetic conditions. Therefore, there has been a push worldwide to apply rapid genomic sequencing in critically ill neonatal and pediatric patients. The goal of this study was to investigate the practicality of applying ultrarapid genomic testing for critically ill neonatal and pediatric patients with suspected monogenic conditions in Australia. The study recruited a total of 108 patients prospectively from March 2018 to February 2019, and data were collected until May 2019. Of the 12 hospitals that acted as collaborating sites, 5 were women's hospitals, 3 women's and children's hospitals, and 4 children's hospitals. Eligible patients included those who were admitted to a neonatal or pediatric intensive care unit (NICU or PICU) and were referred to clinical genetics for a possible monogenic condition. Chromosomal microarray was a requirement before enrollment if there was a suspected chromosomal condition. Chromosomal microarrays were performed concurrently with ultrarapid exome sequencing when the probability of a positive result on microarray was low. Additionally, rapid mitochondrial genome sequencing was performed alongside ultrarapid exome sequencing if a mitochondrial condition was suspected. When possible, ultrarapid exome sequencing was performed in both parents as well as the child (trio). The primary outcome of this study measured the time from the last sample received to the ultrarapid exome sequencing report finalized. Other outcomes measured were the diagnostic yield, change in clinical management after the report was finalized, number of reports returned before hospital discharge, and time from admission to the report being finalized. Of the 108 patients enrolled, the median age was 28 days, with a range of 0 days to 17 years. Overall, 34% were female, 57% were NICU admissions, 33% were PICU admissions, and 9% were from other hospital wards. The mean time it took from the last sample received to the report being issued was 3.3 days (95% confidence interval [CI], 3.2-3.5 days). The mean time from hospital admission to the sequencing report being finalized was 17.5 days (95% CI, 14.6-21.1 days). The mean time from hospital admission to clinical genetics referral was 8.1 days (95% CI, 6.0-10.8 days). Overall, 94% of reports were issued within the goal of 5 calendar days, and 56 molecular diagnoses were made for 55 patients (51%). Among the 62 patients in the NICU, 35 had a clear molecular diagnosis from ultrarapid exome sequencing (56%), whereas in the 36 PICU patients, 17 had a clear molecular diagnosis (47%), and in the 10 patients from other hospital wards, 3 had a clear molecular diagnosis (30%). After the ultrarapid exome sequencing report was finalized, clinical management changed for 48 of the 108 patients enrolled (44%). Overall, the ultrarapid sequencing reports that revealed a clear molecular diagnosis were regarded as “very useful” or “useful” by referring providers in 52 of 55 cases (95%) and “neutral” in 3 of 55 cases (5%). Negative reports were regarded as “very useful” or “useful” in 31 of 53 cases (58%), “neutral” in 20 of 53 cases, and “not useful” in 2 of 53 cases (4%). Altogether, this study demonstrates the utility of ultrarapid exome sequencing in critically ill pediatric patients with a suspected monogenic condition in Australia.

Cost-effectiveness of dapagliflozin in chronic heart failure: an analysis from the Australian healthcare perspective

Abstract Background The Dapagliflozin and Prevention of Adverse Outcomes in Heart Failure (DAPA-HF) trial demonstrated that dapagliflozin reduced heart failure hospitalisations and mortality in patients with established heart failure, regardless of diabetic status. Purpose To assess the cost-effectiveness of dapagliflozin in addition to standard care versus standard care alone in patients with chronic heart failure, from the perspective of the Australian public healthcare system. Methods A Markov model populated with 1000 hypothetical individuals was constructed based on the DAPA-HF trial to assess the clinical outcomes and costs of patients with established heart failure and reduced ejection fraction over a lifetime time horizon. The model consisted of three health states: “Alive and event-free”, “Alive after non-fatal hospitalisation for heart failure” or “Dead”. Costs and utilities were estimated from published sources. Outcomes of interest were the incremental cost-effectiveness ratios (ICERs) in terms of cost per quality-adjusted life year (QALY) gained and cost per year of life saved (YoLS). All outcomes were discounted at a rate of 5% annually. Results Over a lifetime analysis, addition of dapagliflozin to standard care in patients with chronic heart failure prevented 88 acute heart failure hospitalisations (including readmission), and saved 416 (discounted) years of life and 288 (discounted) QALYs, at an additional cost of A$3,692,440 or €2,263,204 (discounted). This resulted in ICERs of A$8,875 (€5,439) per YoLS and A$12,482 (€7,650) per QALY gained, well below the Australian arbitrary willingness-to-pay threshold of A$50,000 (€30,645). Conclusion From the Australian public healthcare perspective, dapagliflozin is cost-effective when used as an adjunct therapy to standard care compared to standard care alone for the treatment of chronic heart failure with reduced ejection fraction. Funding Acknowledgement Type of funding source: None

Health economic evaluation of screening and treating children with familial hypercholesterolemia early in life: many happy returns on investment?

Abstract Background There are no studies that have specifically investigated the cost-effectiveness of cascade screening of children for heterozygous familial hypercholesterolemia (FH) and treatment of affected individuals with statins to prevent coronary heart disease (CHD). Purpose This study explores the cost-effectiveness of this strategy from the perspective of the Australian public healthcare system. Methods A lifetime Markov model with four health states (Alive without CHD, Alive with CHD, Dead from fatal CHD, and Dead from other causes) was developed to simulate the progression of ten- year-old children screened for FH and treated immediately with statins if found to have FH. The underlying prevalence of FH in this target population was assumed to be 56.8%, and the sensitivity and specificity of testing was 100%. The comparator was usual care, which assumed that subjects started statins spontaneously at a later point or when they experienced a cardiovascular event. The effect of reducing low-density lipoprotein cholesterol (LDL-C) on the risk of a first event at each age assumed that risk was proportional to total lifetime exposure and was implemented using Mendelian randomisation analysis data. Cost and other outcome data were sourced from published sources. Outcome of interests were costs in Australian dollars (AUD), life years gained (LYG) and quality-adjusted life years (QALYs) gained, as well as incremental cost-effectiveness ratios (ICERs) of costs per LYG and per QALY gained. All future costs and outcomes were discounted by 5% annually. Results Undiscounted results showed that compared with usual care, cascade screening of ten year-old children for FH and initiation of treatment of affected individuals saved 7.77 LYG and 7.53 QALYs per person over a lifetime. With 5% annual discounting, there were 0.97 LYG and 1.07 QALYs gained per person, at an additional cost of $3,244. These equated to ICERs of $3334 per LYG and $3023 per QALY gained. The equivalent ICERs in USD would be $5089 per LYG gained and $4615 per QALY gained. Sensitivity analysis showed the results to be robust. Conclusions Compared to usual care, cascade screening of ten year old children for FH and treating affected individuals is likely to be highly cost-effective. Table 1. Granular cost and benefit data Funding Acknowledgement Type of funding source: None

Feasibility of Ultra-Rapid Exome Sequencing in Critically Ill Infants and Children With Suspected Monogenic Conditions in the Australian Public Health Care System

Widespread adoption of rapid genomic testing in pediatric critical care requires robust clinical and laboratory pathways that provide equitable and consistent service across health care systems. To prospectively evaluate the performance of a multicenter network for ultra-rapid genomic diagnosis in a public health care system. Descriptive feasibility study of critically ill pediatric patients with suspected monogenic conditions treated at 12 Australian hospitals between March 2018 and February 2019, with data collected to May 2019. A formal implementation strategy emphasizing communication and feedback, standardized processes, coordination, distributed leadership, and collective learning was used to facilitate adoption. Ultra-rapid exome sequencing. The primary outcome was time from sample receipt to ultra-rapid exome sequencing report. The secondary outcomes were the molecular diagnostic yield, the change in clinical management after the ultra-rapid exome sequencing report, the time from hospital admission to the laboratory report, and the proportion of laboratory reports returned prior to death or hospital discharge. The study population included 108 patients with a median age of 28 days (range, 0 days to 17 years); 34% were female; and 57% were from neonatal intensive care units, 33% were from pediatric intensive care units, and 9% were from other hospital wards. The mean time from sample receipt to ultra-rapid exome sequencing report was 3.3 days (95% CI, 3.2-3.5 days) and the median time was 3 days (range, 2-7 days). The mean time from hospital admission to ultra-rapid exome sequencing report was 17.5 days (95% CI, 14.6-21.1 days) and 93 reports (86%) were issued prior to death or hospital discharge. A molecular diagnosis was established in 55 patients (51%). Eleven diagnoses (20%) resulted from using the following approaches to augment standard exome sequencing analysis: mitochondrial genome sequencing analysis, exome sequencing-based copy number analysis, use of international databases to identify novel gene-disease associations, and additional phenotyping and RNA analysis. In 42 of 55 patients (76%) with a molecular diagnosis and 6 of 53 patients (11%) without a molecular diagnosis, the ultra-rapid exome sequencing result was considered as having influenced clinical management. Targeted treatments were initiated in 12 patients (11%), treatment was redirected toward palliative care in 14 patients (13%), and surveillance for specific complications was initiated in 19 patients (18%). This study suggests feasibility of ultra-rapid genomic testing in critically ill pediatric patients with suspected monogenic conditions in the Australian public health care system. However, further research is needed to understand the clinical value of such testing, and the generalizability of the findings to other health care settings.

Health economic evaluation of screening and treating children with familial hypercholesterolemia early in life: Many happy returns on investment?

Background and aimsThere are no studies that have specifically investigated the cost-effectiveness of cascade screening of children for heterozygous familial hypercholesterolemia (FH) and treatment of affected individuals with statins to prevent coronary heart disease (CHD). This study explores the cost-effectiveness of this strategy from the perspective of the Australian public healthcare system. MethodsA lifetime Markov model with four health states (Alive without CHD, Alive with CHD, Dead from fatal CHD, and Dead from other causes) was developed to simulate the progression of ten-year-old children screened for FH and treated immediately with statins if found to have FH. The underlying prevalence of FH in this target population was assumed to be 56.8%, and the sensitivity and specificity of testing were 100%. The comparator was usual care, which assumed that subjects started statins spontaneously at a later point or when they experienced a cardiovascular event. The effect of reducing low-density lipoprotein cholesterol (LDL-C) on the risk of a first event at each age assumed that risk was proportional to total lifetime exposure and was implemented using Mendelian randomisation analysis data. Cost and other outcome data were sourced from published sources. Outcome of interests were costs in Australian dollars (AUD), life years gained (LYG) and quality-adjusted life years (QALYs) gained, as well as incremental cost-effectiveness ratios (ICERs) of costs per LYG and per QALY gained. All future costs and outcomes were discounted by 5% annually. ResultsUndiscounted results showed that compared with usual care, cascade screening of ten year-old children for FH and initiation of treatment of affected individuals saved 7.77 LYG and 7.53 QALYs per person over a lifetime. With 5% annual discounting, there were 0.97 LYG and 1.07 QALYs gained per person, at net reduction cost of -$1134. The cascade screening of ten-year-old children for FH and initiation of treatment compared to usual case was a cost saving approach. In 51.2% of iterations, screening and initiation with statin were cost saving and in 48.8% of iterations were cost-effective. In most of the one-way sensitivity and scenario analyses, the ICER stayed within the accepted Australian threshold. ConclusionsCompared to usual care, cascade screening of ten-year-old children for FH and treating affected individuals are likely to be cost saving.

The cost-effectiveness of icosapent ethyl in combination with statin therapy compared with statin alone for cardiovascular risk reduction.

The aim of this study was to estimate the cost-effectiveness, from the perspective of the Australian public healthcare system, of icosapent ethyl in combination with statin therapy compared with statin alone for the prevention of cardiovascular disease. A Markov model populated with data from the Reduction of Cardiovascular Events with Icosapent Ethyl-Intervention Trial was designed to predict the effectiveness and costs of icosapent ethyl in combination with statins compared with statins alone over a 20-year time horizon. Data inputs for costs and utilities were sourced from published sources. The annual costs of icosapent ethyl were assumed to be AUD1637 (USD2907) per person. All future costs and outcomes were discounted annually by 5%. The main outcome of interest was incremental cost-effectiveness ratios in terms of cost per quality adjusted life year (QALY) gained and per year of life saved (YoLS). Over a 20-year time horizon, compared with statin alone, icosapent ethyl in combination with statin was estimated to cost an additional AUD$13,022 per person, but led to 0.338 YoLS and 0.289 QALYs gained (all discounted). These equated to incremental cost-effectiveness ratios of AUD45,036 per QALY gained and AUD38,480 per YoLS. Sub-analyses for primary and secondary prevention were AUD96,136 and AUD35,935 per QALY gained, respectively. The results were sensitive to time-horizon, age related trends and the acquisition price of icosapent ethyl. Compared with statin alone, icosapent ethyl in combination with statin therapy is likely to be cost-effective in the prevention of cardiovascular disease assuming a willingness-to-pay threshold of AUD50,000 per QALY gained, especially in the secondary preventive setting.