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INTRODUCTION Home-based treatment of febrile neutropenia (FN) in children with cancer with oral or intravenous antibiotics is safe and effective. There are limited data on the economic impact of this model of care. We evaluated the cost-effectiveness of implementing an FN programme, incorporating home-based intravenous antibiotics for carefully selected patients, in a tertiary paediatric hospital. METHODS A decision analytic model was constructed to compare costs and outcomes of the home-based FN programme, with usual in-hospital treatment with intravenous antibiotics. The programme included a clinical decision rule to stratify patients by risk for severe infection and home-based eligibility criteria using disease, chemotherapy and patient-level factors. Health outcomes (quality of life) and probabilities of FN risk classification and home-based eligibility were based on prospectively collected data between 2017 and 2019. Patient-level costs were extracted from hospital administrative records. Cost-effectiveness was expressed as the incremental cost per quality-adjusted life year (QALY). FINDINGS The mean health care cost of home-based FN treatment in low-risk patients was Australian dollars (A$) 7765 per patient compared to A$20,396 for in-hospital treatment (mean difference A$12,632 [95% CI: 12,496-12,767]). Overall, the home-based FN programme was the dominant strategy, being more effective (0.0011 QALY [95% CI: 0.0011-0.0012]) and less costly. Results of the model were most sensitive to proportion of children eligible for home-based care programme. CONCLUSION Compared to in-hospital FN care, the home-based FN programme is cost-effective, with savings arising from cheaper cost of caring for children at home. These savings could increase as more patients eligible for home-based care are included in the programme.

INTRODUCTION: Home-based treatment of febrile neutropenia (FN) in children with cancer with oral or intravenous antibiotics is safe and effective. There are limited data on the economic impact of this model of care. We evaluated the cost-effectiveness of implementing an FN programme, incorporating home-based intravenous antibiotics for carefully selected patients, in a tertiary paediatric hospital. METHODS: A decision analytic model was constructed to compare costs and outcomes of the home-based FN programme, with usual in-hospital treatment with intravenous antibiotics. The programme included a clinical decision rule to stratify patients by risk for severe infection and home-based eligibility criteria using disease, chemotherapy and patient-level factors. Health outcomes (quality of life) and probabilities of FN risk classification and home-based eligibility were based on prospectively collected data between 2017 and 2019. Patient-level costs were extracted from hospital administrative records. Cost-effectiveness was expressed as the incremental cost per quality-adjusted life year (QALY). FINDINGS: The mean health care cost of home-based FN treatment in low-risk patients was Australian dollars (A$) 7765 per patient compared to A$20,396 for in-hospital treatment (mean difference A$12,632 [95% CI: 12,496-12,767]). Overall, the home-based FN programme was the dominant strategy, being more effective (0.0011 QALY [95% CI: 0.0011-0.0012]) and less costly. Results of the model were most sensitive to proportion of children eligible for home-based care programme. CONCLUSION: Compared to in-hospital FN care, the home-based FN programme is cost-effective, with savings arising from cheaper cost of caring for children at home. These savings could increase as more patients eligible for home-based care are included in the programme.

IntroductionAustralia’s current healthcare system for children is neither sustainable nor equitable. As children (0–4 years) comprise the largest proportion of all primary care-type emergency department presentations, general practitioners (GPs) report feeling undervalued as an integral member of a child’s care, and lacking in opportunities for support and training in paediatric conditions. This Strengthening Care for Children (SC4C) randomised trial aims to evaluate a novel, integrated GP-paediatrician model of care, that, if effective, will improve GP quality of care, reduce burden to hospital services and ensure children receive the right care, at the right time, closer to home.Methods and analysisSC4C is a stepped wedge cluster randomised controlled trial (RCT) of 22 general practice clinics in Victoria and New South Wales, Australia. General practice clinics will provide control period data before being exposed to the 12-month intervention which will be rolled out sequentially each month (one clinic per state) until all 22 clinics receive the intervention. The intervention comprises weekly GP-paediatrician co-consultation sessions; monthly case discussions; and phone and email paediatrician support, focusing on common paediatric conditions. The primary outcome of the trial is to assess the impact of the intervention as measured by the proportion of children’s (0–Ethics and disseminationHuman research ethics committee (HREC) approval was granted by The Royal Children’s Hospital Ethics Committee in August 2020 (Project ID: 65955) and site-specific HRECs. The investigators (including Primary Health Network partners) will communicate trial results to stakeholders and participating GPs and general practice clinics via presentations and publications.Trial registration numberAustralia New Zealand Clinical Trials Registry 12620001299998.

In the original version of this article, the author’s name Emily Lancsar was incorrectly written as Emily Lanscar. The original article has been corrected.

Outpatient parenteral antibiotic therapy after hospital admission is increasingly popular, but its use to avoid admission to hospital altogether by treating patients wholly as outpatients remains uncommon in children. One reason for the low use of treatment at home is the scarcity of evidence of its cost-effectiveness. In this planned follow-up analysis of the Cellulitis at Home or Inpatient in Children from the Emergency Department (CHOICE) trial, we aimed to assess the cost-effectiveness of an admission avoidance pathway, in which children were treated at home, compared with standard hospital care for the intravenous treatment of moderate or severe cellulitis.We did a cost-effectiveness analysis to compare home treatment with intravenous ceftriaxone versus hospital treatment with intravenous flucloxacillin in children aged 6 months to 18 years who had presented to the emergency department at The Royal Children's Hospital, Melbourne, VIC, Australia, with moderate or severe uncomplicated cellulitis. We included costs from two sources: institutional costs at a patient level and expenses incurred by families. We measured effectiveness with quality-adjusted life years (QALYs), which we derived from the Child Health Utility 9D questionnaire, and a clinical outcome of treatment failure, which was the primary outcome of the CHOICE trial. We planned to calculate the incremental cost-effectiveness ratio, defined as the difference between groups in total cost divided by the difference between groups in effectiveness. The CHOICE trial is registered at ClinicalTrials.gov, number NCT02334124.We included 180 children who comprised the per-protocol population in the CHOICE trial: 89 children in the home group and 91 children in the hospital group. The institutional cost per patient per episode was significantly lower in the home group than in the hospital group (AUS$1965 vs $3775; p0·0001). The mean cost incurred per family was $182 for the home group and $593 for the hospital group (p0·0001). Both measures of effectiveness were significantly better in the home group than in the hospital group: QALYs were 0·005 for the home group versus 0·004 for the hospital group (p0·0001), and treatment failure occurred in one (1%) patient in the home group versus seven (8%) patients in the hospital group (risk difference -6·5%, 95% CI -12·4 to -0·7; p=0·029). Calculating the incremental cost-effectiveness ratio was thus deemed redundant.Treatment at home was less costly and more effective than standard hospital care for children with moderate or severe cellulitis. These findings support development of this admission avoidance pathway in hospitals.The Royal Children's Hospital Foundation, Murdoch Children's Research Institute.

BackgroundUrinary tract infection (UTI) is a common childhood infection. Many febrile children require a urine sample to diagnose or exclude UTI. Collecting urine from young children can be time-consuming, unsuccessful or contaminated. Cost-effectiveness of each collection method in the emergency department is unknown.ObjectiveTo determine the cost-effectiveness of urine collection methods for precontinent children.MethodsA cost-effectiveness analysis was conducted comparing non-invasive (urine bag, clean catch and 5 min voiding stimulation for clean catch) and invasive (catheterisation and suprapubic aspirate (SPA)) collection methods, for children aged 0–24 months in the emergency department. Costs included equipment, staff time and hospital bed occupancy. If initial collection attempts were unsuccessful subsequent collection using catheterisation was assumed. The final outcome was a definitive sample incorporating progressive dipstick, culture and contamination results. Average costs and outcomes were calculated for initial collection attempts and obtaining a definitive sample. One-way and probabilistic sensitivity analyses were performed.ResultsFor initial collection attempts, catheterisation had the lowest cost per successful collection (GBP£25.98) compared with SPA (£37.80), voiding stimulation (£41.32), clean catch (£52.84) and urine bag (£92.60). For definitive collection, catheterisation had the lowest cost per definitive sample (£49.39) compared with SPA (£51.84), voiding stimulation (£52.25), clean catch (£64.82) and urine bag (£112.28). Time occupying a hospital bed was the most significant determinant of cost.ConclusionCatheterisation is the most cost-effective urine collection method, and voiding stimulation is the most cost-effective non-invasive method. Urine bags are the most expensive method. Although clinical factors influence choice of method, considering cost-effectiveness for this common procedure has potential for significant aggregate savings.

© 2018 American College of Emergency Physicians Study objective: To determine the cost-effectiveness of 3 clinical decision rules in comparison to Australian and New Zealand usual care: the Children's Head Injury Algorithm for the Prediction of Important Clinical Events (CHALICE), the Pediatric Emergency Care Applied Research Network (PECARN), and the Canadian Assessment of Tomography for Childhood Head Injury (CATCH). Methods: A decision analytic model was constructed from the Australian health care system perspective to compare costs and outcomes of the 3 clinical decision rules compared with Australian and New Zealand usual care. The study involved multicenter recruitment from 10 Australian and New Zealand hospitals; recruitment was based on the Australian Pediatric Head Injury Rules Study involving 18,913 children younger than 18 years and with a head injury, and with Glasgow Coma Scale score 13 to 15 on presentation to emergency departments (EDs). We determined the cost-effectiveness of the 3 clinical decision rules compared with usual care. Results: Usual care, CHALICE, PECARN, and CATCH strategies cost on average AUD $6,390, $6,423, $6,433, and $6,457 per patient, respectively. Usual care was more effective and less costly than all other strategies and is therefore the dominant strategy. Probabilistic sensitivity analyses showed that when simulated 1,000 times, usual care dominated all clinical decision rules in 61%, 62%, and 60% of simulations (CHALICE, PECARN, and CATCH, respectively). The difference in cost between all rules was less than $36 (95% confidence interval –$7 to $77) and the difference in quality-adjusted life-years was less than 0.00097 (95% confidence interval 0.0015 to 0.00044). Results remained robust under sensitivity analyses. Conclusion: This evaluation demonstrated that the 3 published international pediatric head injury clinical decision rules were not more cost-effective than usual care in Australian and New Zealand tertiary EDs. Understanding the usual care context and the likely cost-effectiveness is useful before investing in implementation of clinical decision rules or incorporation into a guideline.

The inclusion of future medical costs in cost-effectiveness analyses remains a controversial issue. The impact of capturing future medical costs is likely to be particularly important in patients with cancer where costly lifelong medical care is necessary. The lack of clear, definitive pharmacoeconomic guidelines can limit comparability and has implications for decision making. The aim of this study was to demonstrate the impact of incorporating future medical costs through an applied example using original data from a clinical study evaluating the cost effectiveness of a sepsis intervention in cancer patients. A decision analytic model was used to capture quality-adjusted life-years (QALYs) and lifetime costs of cancer patients from an Australian healthcare system perspective over a lifetime horizon. The evaluation considered three scenarios: (1) intervention-related costs (no future medical cost), (2) lifetime cancer costs and (3) all future healthcare costs. Inputs to the model included patient-level data from the clinical study, relative risk of death due to sepsis, cancer mortality and future medical costs sourced from published literature. All costs are expressed in 2017 Australian dollars and discounted at 5%. To further assess the impact of future costs on cancer heterogeneity, variation in survival and lifetime costs between cancer types and the implications for cost-effectiveness analysis were explored. The inclusion of future medical costs increased incremental cost-effectiveness ratios (ICERs) resulting in a shift from the intervention being a dominant strategy (cheaper and more effective) to an ICER of $7526/QALY. Across different cancer types, longer life expectancies did not necessarily result in greater lifetime healthcare costs. Incremental costs differed across cancers depending on the respective costs of managing cancer and survivorship, thus resulting in variations in ICERs. There is scope for including costs beyond intervention costs in economic evaluations. The inclusion of future medical costs can result in markedly different cost-effectiveness results, leading to higher ICERs in a cancer population, with possible implications for funding decisions.

AIM: Paediatric head injuries (PHI) are the most common cause of trauma-related emergency department (ED) presentations. This study sought to report the incidence of PHI in Australia, examine the temporal trends from 2014 to 2018 and estimate the patient and population-level acute care costs. METHODS: Taking a public-sector health-care perspective, we applied direct and indirect hospital costs for PHI-related ED visits and acute admissions. All costs were inflated to 2018 Australian dollars ($). The patient-level analysis was performed with data from 17 841 children

BACKGROUND Treating respiratory distress in newborns is expensive. We compared the cost-effectiveness of 2 common noninvasive therapies, nasal continuous positive airway pressure (CPAP) and nasal high-flow (nHF), for newborn infants cared for in nontertiary special care nurseries. METHODS The economic evaluation was planned alongside a randomized control trial conducted in 9 Australian special care nurseries. Costs were considered from a hospital perspective until infants were 12 months of age. A total of 754 infants with respiratory distress, born ≥31 weeks’ gestation and with birth weight ≥1200 g, 1 hour were recruited during 2015–2017. Inpatient costing records were obtained for 753 infants, of whom 676 were included in the per-protocol analysis. Two scenarios were considered: (1) CPAP versus nHF, with infants in the nHF group having “rescue” CPAP backup available (trial scenario); and (2) CPAP versus nHF, as sole primary support (hypothetical scenario). Effectiveness outcomes were rate of endotracheal intubation and transfer to a tertiary-level NICU. RESULTS As sole primary support, CPAP is more effective and on average cheaper, and thus is superior. However, nHF with back-up CPAP produced equivalent cost and effectiveness results, and there is no reason to make a decision between the 2 treatments on the basis of the cost or effectiveness outcomes. CONCLUSIONS Nontertiary special care nurseries choosing to use only 1 of the modes should choose CPAP. In units with both modes available, using nHF as first-line therapy may be acceptable if there is back-up CPAP.

1. Introduction In Victoria, the healthcare system for children is overburdened by increasing general practitioner (GP) referrals to hospital emergency departments (EDs) and outpatient clinics. GPs could be supported to manage children closer to home. 2. Practice change implemented We co-designed and implemented a GP-paediatrician integrated care model comprising: weekly paediatrician-GP co-consultation sessions at the GP practice; monthly case discussions; and phone and email support for GPs. 3. Aim and theory of change The model was designed to support GPs to deliver higher quality of care, with greater confidence for a broader range of childhood health concerns. We measured: model feasibility and acceptability; GP confidence, referrals and care quality; family experience and preference for care; and costs. 4. Targeted population and stakeholders Population: 49 participating GPs from 5 GP practices (2 metropolitan and 3 regional); and their patients aged 0 – 17 years, and families; and 2 Paediatricians. Stakeholders: The Royal Children’s Hospital; Sunshine and Werribee Mercy Hospitals; North Western Melbourne Primary Health Network (PHN); and the Victorian Department of Health and Human Services. 5. Timeline 12-month intervention. 6. Highlights (innovation, Impact and outcomes) The model was feasible and acceptable to GPs, families and paediatricians with 624 children seen in the co-consultations and 50 case discussions conducted. In pre-post testing, there was a 7% absolute reduction in ED referrals, 20% reduction in GP low value care; increased GP confidence in paediatric care (88% to 100%); and family confidence in GP care (78% to 94%). Families reported ease and comfort of receiving paediatric care closer to home. Model costs as implemented were $172 over and above usual care, per child seen in the co-consults. However, in an idealised implementation scenario, the model has the potential to be cost saving for families, state/federal governments and hospitals. 7. Comments on sustainability Sustainability planning was completed in partnership with the PHN and the practices; two have hired paediatricians, with others exploring options for access to paediatric support and learning (e.g. webinar program). The research team plan to complete a larger, multi-site multi-state trial to rigorously evaluate effectiveness and cost-effectiveness of the model in high referral areas. 8. Comments on transferability This integrated care model could be replicated for chronic complex care that burdens the hospital system, or implemented in service hubs with co-located paediatricians, or extended to include telehealth for rural/regional hubs. 9. Conclusions (key findings) Developing and embedding a GP-paediatrician integrated model of care in Australia’s primary health care system is feasible and acceptable; improves GP confidence and quality of paediatric care; benefits families and children; and may reduce referrals to hospital services. 10. Discussions Truly integrated care, delivered as a collaboration between hospitals, health networks, GPs and their practices has the potential to shape a health system in which children can receive higher quality, timelier care, closer to home. 11. Lessons learned Co-design and initial onsite-support were crucial e.g. billing; data collection; co-consult structure and purpose. The drive and desire to provide better care for children is the strongest motivator for change.

Objective This study determined caregivers’ evaluations of telehealth consultations for their child, preference over in-person consultations and potential cost savings by child condition, family socioeconomic status and location. Methods A survey was conducted of 2436 caregivers attending a telehealth consultation with their child for a broad group of conditions between 3 June and 25 August 2020 at a tertiary paediatric hospital in Melbourne, Australia. Results Most caregivers found telehealth consultations convenient, acceptable, safe and private, and capable of answering their questions and concerns. However, caregivers who spoke a language other than English and patients attending for behavioural and mental health, developmental or other (e.g. allied health) concerns were more likely to prefer in-person consultations over telehealth. Mean (±s.d.) reported cost savings on caregiver time were A$144.98 ± 99.04 per family per consultation, whereas mean (±s.d.) transport cost savings were A$84.90 ± 100.74 per family per consultation. Cost savings were greatest for families living in low and middle socioeconomic areas and regional or rural areas. Conclusions Paediatric telehealth video consultations were largely viewed favourably by caregivers, except for those attending for behavioural and mental health or developmental concerns. What is known about the topic? Adult consumers of telehealth consultations view them as useful, convenient and cost saving, but less is known about caregivers’ evaluations of telehealth consultations and potential cost savings for paediatric patients, and whether these differ by family location, socioeconomic status or child condition. What does this paper add? This is the first Australian paper to report on caregivers’ evaluations across a range of paediatric conditions and locations. Most caregivers found telehealth consultations convenient, safe, acceptable, able to answer their questions and concerns about their child’s health and cost saving. What are the implications for practitioners? Caregivers of children with behavioural, mental health or developmental problems were less likely to prefer telehealth over in-person consultations, so practitioners should consider providing such consultations in person.

Background Cancer patients are at significant risk of developing sepsis due to underlying malignancy and necessary treatments. Little is known about the economic burden of sepsis in this high-risk population. We estimate the short- and long-term healthcare costs of care of cancer patients with and without sepsis using individual-level linked-administrative data. Methods We conducted a population-based matched cohort study of cancer patients aged ≥18, diagnosed between 2010 and 2017. Cases were identified if diagnosed with sepsis during the study period, and were matched 1:1 by age, sex, cancer type and other variables to controls without sepsis. Mean costs (2018 Canadian dollars) for patients with and without sepsis up to 5 years were estimated adjusted using survival probabilities at partitioned intervals. We estimated excess cost associated with sepsis presented as a cost difference between the two cohorts. Haematological and solid cancers were analysed separately. Results 77,483 cancer patients with sepsis were identified and matched. 64.3% of the cohort were aged ≥65, 46.3% female and 17.8% with haematological malignancies. Among solid tumour patients, the excess cost of care among patients who developed sepsis was $29,081 (95%CI, $28,404-$29,757) in the first year, rising to $60,714 (95%CI, $59,729-$61,698) over 5 years. This was higher for haematology patients; $46,154 (95%CI, $45,505-$46,804) in year 1, increasing to $75,931 (95%CI, $74,895-$76,968). Conclusions Sepsis imposes substantial economic burden and can result in a doubling of cancer care costs, particularly during the first year of cancer diagnosis. These estimates are helpful in improving our understanding of burden of sepsis along the cancer pathway and to deploy targeted strategies to alleviate this burden.

ObjectiveThis study determined caregivers’ evaluations of telehealth consultations for their child, preference over in-person consultations and potential cost savings by child condition, family socioeconomic status and location. MethodsA survey was conducted of 2436 caregivers attending a telehealth consultation with their child for a broad group of conditions between 3 June and 25 August 2020 at a tertiary paediatric hospital in Melbourne, Australia. ResultsMost caregivers found telehealth consultations convenient, acceptable, safe and private, and capable of answering their questions and concerns. However, caregivers who spoke a language other than English and patients attending for behavioural and mental health, developmental or other (e.g. allied health) concerns were more likely to prefer in-person consultations over telehealth. Mean (±s.d.) reported cost savings on caregiver time were A$144.98 ± 99.04 per family per consultation, whereas mean (±s.d.) transport cost savings were A$84.90 ± 100.74 per family per consultation. Cost savings were greatest for families living in low and middle socioeconomic areas and regional or rural areas. ConclusionsPaediatric telehealth video consultations were largely viewed favourably by caregivers, except for those attending for behavioural and mental health or developmental concerns. What is known about the topic?Adult consumers of telehealth consultations view them as useful, convenient and cost saving, but less is known about caregivers’ evaluations of telehealth consultations and potential cost savings for paediatric patients, and whether these differ by family location, socioeconomic status or child condition. What does this paper add?This is the first Australian paper to report on caregivers’ evaluations across a range of paediatric conditions and locations. Most caregivers found telehealth consultations convenient, safe, acceptable, able to answer their questions and concerns about their child’s health and cost saving. What are the implications for practitioners?Caregivers of children with behavioural, mental health or developmental problems were less likely to prefer telehealth over in-person consultations, so practitioners should consider providing such consultations in person.

ObjectivesTo investigate associations between maternal body mass index (BMI) at delivery (using pregnancy-specific BMI cut-off values 5 kg/m2higher in each of the WHO groups) and clinical, theatre utilisation and health economic outcomes for women undergoing caesarean section (CS). DesignA prospective multicentre observational study. SettingSeven secondary or tertiary referral obstetric hospitals. ParticipantsOne thousand and four hundred and fifty-seven women undergoing all categories of CS. Data collectionHeight and weight were recorded at the initial antenatal visit and at delivery. We analysed the associations between delivery BMI (continuous and pregnancy-specific cut-off values) and total theatre time, surgical time, anaesthesia time, maternal and neonatal adverse outcomes, total hospital admission and theatre costs. ResultsMean participant characteristics were: age 32 years, gestation at delivery 38.4 weeks and delivery BMI 32.2 kg/m2. Fifty-five per cent of participants were overweight, obese or super-obese using delivery pregnancy-specific BMI cut-off values. As BMI increased, total theatre time, surgical time and anaesthesia time increased. Super-obese participants had approximately 27% (17 min, p ConclusionsIncreased maternal BMI was associated with increased total theatre time, surgical and anaesthesia time, increased total hospital admission costs and theatre costs. Clinicians and health administrators should consider these clinical risks, time implications and financial costs when managing pregnant women.

BACKGROUND:The measurement and valuation of health-related quality of life for and by young people are increasingly important, yet research on the impact of study perspective and validity of preferences obtained from young populations remains limited. OBJECTIVE:The objective of this study was to evaluate the feasibility and validity of collecting EQ-5D Youth version (EQ-5D-Y) preferences from adolescents, adults, and adults from a child perspective. METHODS:A profile case best-worst scaling (BWS) online survey was administered to representative Australian and Spanish adult (age ≥ 18 years) and child (age 11-17 years) samples. Adults were told to either answer from their own perspective or for a hypothetical 10-year-old child. Marginal best- and worst-choice frequencies, analysis of dominant choices, self-reported difficulty completing the tasks, and time to complete tasks were used to determine the validity of responses. RESULTS:In Australia, 2134 adults and 1010 adolescents completed the survey. In Spain, 2007 adults and 1000 adolescents completed it. Analysis of marginal choice frequencies and dominant choices indicated that the pattern of responses between adolescents and adults was similar. For Australian respondents, having no mobility problems was rated as best by adolescents, while adults rated having no pain and discomfort as 'best'. In Spain, both adults and adolescents rated no pain or discomfort as 'best'. Australian adolescents rated very worried, sad or unhappy as 'worst', while Spanish adolescents, Spanish adults and Australian adults rated a lot of pain and discomfort as 'worst'. CONCLUSIONS:Results suggest preferences from adolescents using direct BWS are valid. Our descriptive analysis also suggest that there are age-related and country-specific differences in elicitation values for the EQ-5D-Y.

In health economics, the use of patient recall of health care utilisation information is common, including in national health surveys. However, the types and magnitude of measurement error that relate to different recall periods are not well understood. This study assessed the accuracy of recalled doctor visits over 2-week, 3-month, and 12-month periods by comparing self-report with routine administrative Australian Medicare data. Approximately 5,000 patients enrolled in an Australian study were pseudo-randomised using birth dates to report visits to a doctor over three separate recall periods. When comparing patient recall with visits recorded in administrative information from Medicare Australia, both bias and variance were minimised for the 12-month recall period. This may reflect telescoping that occurs with shorter recall periods (participants pulling in important events that fall outside the period). Using shorter recall periods scaled to represent longer periods is likely to bias results. There were associations between recall error and patient characteristics. The impact of recall error is demonstrated with a cost-effectiveness analysis using costs of doctor visits and a regression example predicting number of doctor visits. The findings have important implications for surveying health service utilisation for use in economic evaluation, econometric analyses, and routine national health surveys.

OBJECTIVE: The severe epilepsies of infancy (SEI) are a devastating group of disorders that pose a major care and economic burden on society; early diagnosis is critical for optimal management. This study sought to determine the incidence and etiologies of SEI, and model the yield and cost-effectiveness of early genetic testing. METHODS: A population-based study was undertaken of the incidence, etiologies, and cost-effectiveness of a whole exome sequencing-based gene panel (targeted WES) in infants with SEI born during 2011-2013, identified through electroencephalography (EEG) and neonatal databases. SEI was defined as seizure onset before age 18 months, frequent seizures, epileptiform EEG, and failure of ≥2 antiepileptic drugs. Medical records, investigations, MRIs, and EEGs were analyzed, and genetic testing was performed if no etiology was identified. Economic modeling was performed to determine yield and cost-effectiveness of investigation of infants with unknown etiology at epilepsy onset, incorporating targeted WES at different stages of the diagnostic pathway. RESULTS: Of 114 infants with SEI (incidence = 54/100 000 live births/y), the etiology was determined in 76 (67%): acquired brain injuries (n = 14), focal cortical dysplasias (n = 14), other brain malformations (n = 17), channelopathies (n = 11), chromosomal (n = 9), metabolic (n = 6), and other genetic (n = 5) disorders. Modeling showed that incorporating targeted WES increased diagnostic yield compared to investigation without targeted WES (48/86 vs 39/86). Early targeted WES had lower total cost ($677 081 U.S. dollars [USD] vs $738 136 USD) than late targeted WES. A pathway with early targeted WES and limited metabolic testing yielded 7 additional diagnoses compared to investigation without targeted WES (46/86 vs 39/86), with lower total cost ($455 597 USD vs $661 103 USD), lower cost per diagnosis ($9904 USD vs $16 951 USD), and a dominant cost-effectiveness ratio. SIGNIFICANCE: Severe epilepsies occur in 1 in 2000 infants, with the etiology identified in two-thirds, most commonly malformative. Early use of targeted WES yields more diagnoses at lower cost. Early genetic diagnosis will enable timely administration of precision medicines, once developed, with the potential to improve long-term outcome.

IntroductionMeasures of health-related quality of life (HRQoL), accompanied by the values (or utilities) required to estimate quality-adjusted life-years, are crucial for determining health benefits within economic evaluation and health technology assessment. Several generic and condition-specific measures or instruments of HRQoL, accompanied by values, currently exist for application with child populations. However, there is a lack of a structured summary of guidelines and recommendations for applying these measures in practice. This protocol describes a systematic review of guidelines and recommendations for child and proxy completion of child-specific measures of HRQoL. The aims of the review are to (1) identify and summarise published guidelines and recommendations for existing child-specific measures of HRQoL, (2) determine whether the identified guidelines and recommendations differ by instrument and child characteristics, (3) identify current gaps in these guidelines and recommendations and (4) identify best practices for child self and proxy assessment in paediatric HRQoL measurement for economic evaluation and health technology assessment.Methods and analysisThe review will identify, collate and synthesise published guidelines and recommendations for existing child-specific utility measures of HRQoL. Electronic databases to be searched include the Cochrane Library, Medline, Scopus, Web of Science, EconLit, PsycINFO, CINAHL, Embase and Informit. The search will be extended to websites of (1) international organisations for health technology assessment, (2) regulation, health economics and HRQoL outcomes research and (3) instrument developers. Three reviewers will independently screen titles and abstracts against the inclusion criteria. A narrative synthesis will describe the key features of the guidelines identified.Ethics and disseminationEthical approval is not required as the proposed systematic review will not use primary data. A paper of the systematic review will be submitted to a peer-reviewed journal for publication.PROSPERO registration numberCRD42020207160.

We estimated the inpatient resource use for a Fontan patient from birth to adulthood and explored factors that might induce cost differences (2014 US dollar). Inpatient costing records from 4 hospitals with greatest numbers of Fontan patients in Australia and New Zealand were linked with the Fontan registry database. Inpatient records between July 1995 and September 2014 for 420 Fontan patients were linked, and the most frequent primary diagnoses were hypoplastic left heart syndrome (20.7%), tricuspid atresia (19.7%), and double inlet left ventricle (17.1%). The mean hospital cost for a Fontan patient from birth to 18 years of age was estimated to be $390,601 (95% confidence interval [CI] $264,703 to $516,499), corresponding to 164 (95% CI 98 to 231) inpatient days. The cost incurred from birth through to Fontan completion (the staged procedures period) was $219,482 (95% CI $202,410 to $236,553) and the cost thereafter over 15 years was $146,820 (95% CI $44,409 to $249,231), corresponding to 82 (95% CI 72 to 92) and 65 (95% CI 18 to 112) inpatient days, respectively. Costs were higher in male and hypoplastic left heart syndrome patients in the staged procedures period (p

Introduction: Reviewing rCGM patterns may prompt lifestyle or therapeutic changes to achieve glycaemic targets but evidence for rCGM use in primary care management of T2D is limited. Methods: Two-arm RCT. Participants: Adults with T2D, HbA1c ≥0.5% above target, prescribed ≥2 non-insulin glycemia medications or insulin. Intervention: 1-hour diabetes education and (at 0/3/6/9/12 months): HbA1c assessment + wearing FreeStyle Libre Pro (Abbott) for up to 14 days prior to being discussed at a clinic visit. Physicians were trained in interpreting ambulatory glucose profiles. Control: 3-monthly ‘usual care’ clinic visits + r-CGM device worn (blinded, research data) at 0 and 12 months. Primary outcome: difference in mean HbA1c at 12 months. Secondary outcomes: mean differences in time in range (TIR: 4-10 mmol/L) and diabetes-specific distress (PAID) at 12 months, and HbA1c at 6 months. ITT analysis. Results: In 25 primary care practices, participants were: 299 adults with T2D, aged (mean(SD)) 60(10) years, HbA1c: 8.9(1.2)%, diabetes duration (median(IQR)) of 12(8,20) years. At 12 months, the between-group difference in mean HbA1c was 0.2% (p=0.112). The estimated mean percentage TIR was 8.4% higher in the intervention than the control arm (p=0.004). Diabetes-specific distress did not differ between arms (0.5; p=0.71). At 6 months, HbA1c was significantly lower in the intervention arm (0.5%; p Discussion: Use of 3-monthly rCGM in adults with T2D in primary care does not improve HbA1c at 12 months. We showed a statistically and clinically significant reduction in HbA1c at 6 months in the intervention group, as well as significant improvements in TIR at 12 months, with no change in diabetes distress. Our findings suggest the primary impact of r-CGM use on HbA1c in this setting is short term, and not associated with increase in the number of diabetes medications. Disclosure J. Furler: Research Support; Self; Abbott, Sanofi. D.N. O’Neal: None. J. Speight: Research Support; Self; AstraZeneca, Medtronic, Sanofi. Speaker’s Bureau; Self; Novo Nordisk A/S, Roche Diabetes Care. J.E. Manski-Nankervis: Research Support; Self; Australian National Health and Medical Research Council, Boehringer Ingelheim International GmbH, Diabetes Australia, Eli Lilly and Company. Speaker’s Bureau; Self; RACGP. S. Thuraisingam: None. E. Holmes-Truscott: Research Support; Self; Sanofi. Speaker’s Bureau; Self; Novo Nordisk Inc. K.R. De La Rue: None. L.E. Ginnivan: None. R.C. Doyle: None. K. Khunti: Advisory Panel; Self; Amgen Inc., AstraZeneca, Eli Lilly and Company, Merck Sharp & Dohme Corp., Novo Nordisk A/S, Sanofi. Consultant; Self; AstraZeneca, Boehringer Ingelheim International GmbH, Eli Lilly and Company, Janssen Pharmaceuticals, Inc., Merck Sharp & Dohme Corp., Novartis AG, Novo Nordisk A/S, Pfizer Inc., Sanofi-Aventis, Servier, Takeda Pharmaceutical Company Limited. Research Support; Self; AstraZeneca, Boehringer Ingelheim International GmbH, Eli Lilly and Company, Merck Sharp & Dohme Corp., Novartis AG, Novo Nordisk A/S, Pfizer Inc., Sanofi-Aventis. Speaker’s Bureau; Self; AstraZeneca, Berlin-Chemie AG, Boehringer Ingelheim International GmbH, Eli Lilly and Company, Janssen Pharmaceuticals, Inc., Menarini Group, Merck Sharp & Dohme Corp., Novartis AG, Novo Nordisk A/S, Roche Pharma, Sanofi, Servier, Takeda Pharmaceutical Company Limited. M. Catchpool: None. K. Dalziel: None. J.I. Chiang: None. I. Blackberry: None. R. Audehm: Advisory Panel; Self; AstraZeneca, Novartis Pharmaceuticals Corporation. M. Kennedy: Advisory Panel; Self; AstraZeneca, Boehringer Ingelheim Pharmaceuticals, Inc., Merck Sharp & Dohme Corp., Novo Nordisk Inc., Sanofi. M.J. Clark: None. A.J. Jenkins: Advisory Panel; Self; Abbott, Australian Diabetes Society, Medtronic. Research Support; Self; Abbott, GlySens Incorporated, Medtronic, Mylan. Speaker’s Bureau; Self; Eli Lilly and Company, Novo Nordisk Inc. A.S. Januszewski: None. D. Liew: Advisory Panel; Self; AstraZeneca, Bayer AG. Research Support; Self; AbbVie Inc., AstraZeneca, Bristol-Myers Squibb Company, CSL Behring, Pfizer Inc. P.M. Clarke: None. J.D. Best: Consultant; Self; Abbott. Funding National Health and Medical Research Council of Australia (APP1104241); Sanofi Australia; Abbott Diabetes Care

To assess use and needs for general practitioner, specialist, emergency department, hospital inpatient, and hospital outpatient services for children with special health care needs (CSHCN). To assess the level of CSHCN's needs compared with other children.Health service use and needs for children aged 4 to 15 years were assessed using the nationally representative Longitudinal Study of Australian Children survey with 25,011 observations. Odds ratios of service use and unmet needs for CSHCN compared to other children were estimated. CSHCN were further grouped as those 1) needing only additional medication, 2) needing only additional medical care, or 3) needing both as defined by the CSHCN screener questions.The prevalence of those meeting criteria for CSHCN was 16.1%. In the past 12 months, 77.7% of CSHCN used any general practitioner care and 46.4%, 21.7%, 12.4%, and 15.0%, respectively, used specialty care, emergency department, hospital inpatient, and outpatient services. Approximately half of CSHCN were identified as having special needs due to needing additional medication. Their service use and unmet needs are only slightly elevated. Another group of CSHCN who need both more medication and medical care comprises less than one third of all CSHCN. These children have the highest odds of using specialty care (odds ratio 12.3, P.001), and of having unmet specialty care need (odds ratio 7.4, P.001) compared to children without special needs.Our findings highlight the importance of ensuring specialty care for CSHCN, especially the one third with most elevated needs. This estimate might guide future care planning.

Objective The management of low-risk febrile neutropenia (FN) patients through ambulatory programs has demonstrated comparative safety and effectiveness to in-patient strategies. However, there is limited evidence of benefits of changing practice, particularly on a national scale. The aim of this study was to estimate costs and benefits of the program over a 10-year time horizon. Methods A comparative cost analysis from a health system perspective was performed, comparing costs and length of stay (LOS) of patients enrolled in an ambulatory program to a historical cohort who did not receive the program. Generalised linear models were used for analysis and bootstrapped to account for uncertainty. National data of identified FN admissions were used to inform future projections, with varying proportions of low-risk patients and eligibility for the ambulatory program. Results The overall LOS for patients in ambulatory cohort was 1.9 days shorter (95% confidence interval (CI) 1.0–2.8 days), a 50% reduction in in-patient bed-days. Although patients in the ambulatory cohort incurred additional costs due to care received outside hospital (mean (± s.d.) A$828.03 ± 124.30), the mean total cost incurred remained substantially lower than that of the historical cohort (A$2979 lower; 95% CI A$772–5391). On a national scale, this could translate into A$62.7 million in costs averted and 41 347 bed-days saved over 10 years if the low-risk prediction rate and eligibility for ambulatory programs remained at currently observed rates. Conclusions The wider implementation of a safe and effective ambulatory program to manage low-risk FN patients can result in significant return-on-investment for the healthcare system by eliminating avoidable costs due to unnecessary lengthy hospital admissions. What is known about the topic? There is strong evidence demonstrating out-patient treatment of low-risk FN patients to be an effective and cost-effective strategy compared with continued in-patient hospitalisation. What does this paper add? This study demonstrates the sustainability of the ambulatory program in ensuring cost benefits and in-patient beds through real-life implementation data. It also provides evidence of the substantial cost and bed-days potentially averted when the cost savings and difference in LOS are estimated on a national scale over a 10-year time horizon. What are the implications for practitioners? The management of low-risk FN patients through ambulatory or out-patient programs is a safe and effective approach. There is strong evidence demonstrating the likely cost savings and considerable bed-days saved, which can be reallocated to meet other medical demands.

Background Epilepsy surgery is an alternative to continued antiepileptic drugs (AEDs) in children with drug-resistant epilepsy (DRE). Objective The objective of the study was to measure, model, and compare the medical costs and impacts on health-related quality of life (HRQL) of epilepsy surgery versus continued medical treatment with AEDs in children with DRE. Methods A decision analytic model was created to estimate the cost-effectiveness of epilepsy surgery relative to continued medical treatment with AEDs. The model was based on costing and effectiveness data collected from 105 children with DRE who were operated on at the Royal Children's Hospital, Melbourne, Australia. The mean cost of conducting epilepsy surgery was AU$ 61,417 per person. Effectiveness of continued medical treatment was sourced from best available literature. In the absence of published utility values for pediatric patients with epilepsy and ethical approval to contact patients directly, HRQL was estimated by four clinicians using the Child Health Utility 9 Dimension (CHU9D). Outcome measures were seizure freedom and quality-adjusted life years (QALYs). Results The costs over 7.6 years of follow-up were AU$ 219,297 for the surgical treatment group compared with AU$ 170,583 for the medical treatment group. The incremental cost-effectiveness ratio (ICER) for surgically vs medical treatment was AU$ 76,538 per additional patient attaining seizure freedom and AU$ 75,541 per additional QALY gained. Conclusion Epilepsy surgery resulted in a greater reduction of seizures and improvement in HRQL but was more expensive than continued medical treatment with AEDs. Including benefits outside of a healthcare perspective would likely lead to a more compelling cost-effective argument.

IntroductionThe majority of children who sustain a concussion will recover quickly, but a significant minority will experience ongoing postconcussive symptoms, known as postconcussion syndrome (PCS). These symptoms include emotional, behavioural, cognitive and physical symptoms and can lead to considerable disability. The neurobiological underpinnings of PCS are poorly understood, limiting potential clinical interventions. As such, patients and families frequently re-present to clinical services, who are often ill equipped to address the multifactorial nature of PCS. This contributes to the high cost of concussion management and the disability of children experiencing PCS. The aims of the present study are: (1) to plot and contrast recovery pathways for children with concussion from time of injury to 3 months postinjury, (ii) evaluate the contribution of acute biomarkers (ie, blood, MRI) to delayed recovery postconcussion and (3) estimate financial costs of child concussion to patients attending the emergency department (ED) of a tertiary children’s hospital and factors predicting high cost.Methods and analysisTake C.A.Re is a prospective, longitudinal study at a tertiary children’s hospital, recruiting and assessing 525 patients aged 5–Ethics and disseminationEthical approval has been obtained through the Royal Children’s Hospital Melbourne Human Research Ethics Committee (33122). We aim to disseminate the findings through international conferences, international peer-reviewed journals and social media.Trial registration numberACTRN12615000316505; Results.

Background General Practice Optimising Structured Monitoring to Improve Clinical Outcomes in Type 2 Diabetes (GP-OSMOTIC) is a multicentre, individually randomised controlled trial aiming to compare the use of intermittent retrospective continuous glucose monitoring (r-CGM) to usual care in patients with type 2 diabetes attending general practice. The study protocol was published in the British Medical Journal Open and described the principal features of the statistical methods that will be used to analyse the trial data. This paper provides greater detail on the statistical analysis plan, including background and justification for the statistical methods chosen, in accordance with SPIRIT guidelines. Objective To describe in detail the data management process and statistical methods that will be used to analyse the trial data. Methods An overview of the trial design and primary and secondary research questions are provided. Sample size assumptions and calculations are explained, and randomisation and data management processes are described in detail. The planned statistical analyses for primary and secondary outcomes and sub-group analyses are specified along with the intended table layouts for presentation of the results. Conclusion In accordance with best practice, all analyses outlined in the document are based on the aims of the study and have been pre-specified prior to the completion of data collection and outcome analyses. Trial registration Australian New Zealand Clinical Trials Registry, ACTRN12616001372471. Registered on 3 August 2016.

IntroductionWhile most children recover from a concussion shortly after injury, approximately 30% experience persistent postconcussive symptoms (pPCS) beyond 1-month postinjury. Existing research into the treatment of pPCS have evaluated unimodal approaches, despite evidence suggesting that pPCS likely represent an interaction across various symptom clusters. The primary aim of this study is to evaluate the effectiveness of a multimodal, symptom-tailored intervention to accelerate symptom recovery and increase the proportion of children with resolved symptoms at 3 months postconcussion.Methods and analysisIn this open-label, assessor-blinded, randomised clinical trial, children with concussion aged 8–18 years will be recruited from The Royal Children’s Hospital (The RCH) emergency department, or referred by a clinician, within 17 days of initial injury. Based on parent ratings of their child’s PCS at ~10 days postinjury, symptomatic children (≥2 symptoms at least 1-point above those endorsed preinjury) will undergo a baseline assessment at 3 weeks postinjury and randomised into either Concussion Essentials (CE, n=108), a multimodal, interdisciplinary delivered, symptom-tailored treatment involving physiotherapy, psychology and education, or usual care (UC, n=108) study arms. CE participants will receive 1 hour of intervention each week, for up to 8 weeks or until pPCS resolve. A postprogramme assessment will be conducted at 3 months postinjury for all participants. Effectiveness of the CE intervention will be determined by the proportion of participants for whom pPCS have resolved at the postprogramme assessment (primary outcome) relative to the UC group. Secondary outcome analyses will examine whether children receiving CE are more likely to demonstrate resolution of pPCS, earlier return to normal activity, higher quality of life and a lower rate of utilisation of health services, compared with the UC group.Ethics and disseminationEthics were approved by The RCH Human Research Ethics Committee (HREC: 37100). Parent, and for mature minors, participant consent, will be obtained prior to commencement of the trial. Study results will be disseminated at international conferences and international peer-reviewed journals.Trial registration numberACTRN12617000418370; pre-results.

IntroductionEmerging evidence indicates that rehabilitation can improve ataxia, mobility and independence in everyday activities in individuals with hereditary cerebellar ataxia. However, with the rarity of the genetic ataxias and known recruitment challenges in rehabilitation trials, most studies have been underpowered, non-randomised or non-controlled. This study will be the first, appropriately powered randomised controlled trial to examine the efficacy of an outpatient and home-based rehabilitation programme on improving motor function for individuals with hereditary cerebellar ataxia.Methods and analysisThis randomised, single-blind, parallel group trial will compare a 30-week rehabilitation programme to standard care in individuals with hereditary cerebellar ataxia. Eighty individuals with a hereditary cerebellar ataxia, aged 15 years and above, will be recruited. The rehabilitation programme will include 6 weeks of outpatient land and aquatic physiotherapy followed immediately by a 24- week home exercise programme supported with fortnightly physiotherapy sessions. Participants in the standard care group will be asked to continue their usual physical activity. The primary outcome will be the motor domain of the Functional Independence Measure. Secondary outcomes will measure the motor impairment related to ataxia, balance, quality of life and cost-effectiveness. Outcomes will be administered at baseline, 7 weeks, 18 weeks and 30 weeks by a physiotherapist blinded to group allocation. A repeated measures mixed-effects linear regression model will be used to analyse the effect of the treatment group for each of the dependent continuous variables. The primary efficacy analysis will follow the intention-to-treat principle.Ethics and disseminationThe study has been approved by the Monash Health Human Research Ethics Committee (HREC/18/MonH/418) and the Human Research Ethics Committee of the Northern Territory Department of Health and Menzies School of Health Research (2019/3503). Results will be published in peer-reviewed journals, presented at national and/or international conferences and disseminated to Australian ataxia support groups.Trial registration numberACTRN12618000908235.

Background The number and age demographic of the future Fontan population is unknown. Methods Population projections were calculated probabilistically using microsimulation. Mortality hazard rates for each Fontan recipient were calculated from survivorship of 1353 Fontan recipients in the Australia and New Zealand Fontan Registry, based on Fontan type, age at Fontan, gender and morphology. Projected rates of new Fontan procedures were generated from historical rates of Fontan procedures per population births. Results At the end of 2014, the living Fontan population of Australia and New Zealand was 1265 people from an Australian and New Zealand regional population of 28 million (4.5 per 100,000 population). Of those, 165 (13%) received an atrio-pulmonary (AP) procedure, 262 (21%) a lateral tunnel (LT) procedure and 838 (66%) an extra-cardiac conduit (ECC) procedure. This population is expected to grow to 1917 (95% CI: 1846: 1986) by 2025 (5.8 per 100,000 population), with 149 (8%) AP procedures, 254 (13%) LT procedures, and 1514 (79%) ECC procedures. By 2045, the living Fontan population is expected to reach 2986 (95% CI: 2877: 3085; 7.2 per 100,000 population). The average age of the Fontan population is expected to increase from 18years in 2014 to 23years (95% CI: 22–23) by 2025, and 31years (95% CI: 30–31) by 2045. Conclusion The Australian and New Zealand population of patients alive after a Fontan procedure will double over the next 20years increasing the demand for heart-failure services and cardiac transplantation. Greater consideration for the needs of this mostly adult Fontan population will be necessary.

Despite a lack of evidence supporting the use of angiotensin-converting enzyme (ACE) inhibitors in patients with a Fontan circulation, their use is frequent. We decided to identify the rationale for ACE inhibitor therapy in patients within the Australia and New Zealand Fontan Registry.All patients in the Registry taking an ACE inhibitor at last follow up were identified, and a review of medical records was undertaken to determine the rationale for treatment initiation and reasons for treatment continuation or dose increase.In 2015, 36% of the surviving patients in the Registry (462/1268) were taking an ACE inhibitor. Indications for initiation of therapy were ventricular systolic or diastolic dysfunction (29%), atrioventricular valve regurgitation (19%), preservation of normal ventricular function (7%), prolonged effusions at Fontan (6%), hypertension (6%), other (6%) and unknown (2%). No indication was stated in the remaining patients (25%). Those with hypoplastic left heart syndrome were more likely to be on an ACE inhibitor than those with an alternative primary morphology (70% vs 32%; p0.001). Only 36% of the patients treated with an ACE inhibitor at last follow up (166/462) had an indication that would generally justify treatment in a two-ventricle circulation.It is likely that the use of ACE inhibitors in patients with a Fontan circulation is excessive within our region. The coordination of prospective, multicentre studies and initiatives such as the Australia and New Zealand Fontan Registry will facilitate further investigations to guide treatment decisions in the growing Fontan population.

Objective: Pentoxifylline has previously been shown to increase haemoglobin levels in patients with chronic kidney disease (CKD) and erythropoietin-stimulating agent (ESA)-hyporesponsive anaemia in the HERO multi-centre double-blind, randomized controlled trial. The present study evaluated the effects of pentoxifylline on oxidative stress in ESA-hyporesponsive CKD patients. Methods: This sub-study of the HERO trial compared 15 patients in the pentoxifylline arm (400 mg daily) and 17 in the matched placebo arm on oxidative stress markers: plasma total F2-isoprostanes, protein carbonyls, glutathione peroxidase (GPX), and superoxide dismutase (SOD) activities. Results: Pentoxifylline did not significantly alter total F2-isoprostanes (adjusted mean difference (MD) 35.01 pg/ml, P = 0.11), SOD activity (MD 0.82 U/ml, P = 0.07), GPX activity (MD −6.06 U/l, P = 0.09), or protein carbonyls (MD −0.04 nmol/mg, P = 0.52). Replicating results from the main study, pentoxifylline significantly increased haemoglobin concentration compared with controls (MD 7.2 g/l, P = 0.04). Conclusions: Pentoxifylline did not alter oxidative stress biomarkers, suggesting that alternative mechanisms may be responsible for the agent's ability to augment haemoglobin levels in CKD patients with ESA-hyporesponsive anaemia.

© 2020 AHHA Open Access. Objective: This study reports on the characteristics of individuals conducting health service research (HSR) in Australia and New Zealand, the perceived accessibility of resources for HSR, the self-reported impact of HSR projects and perceived barriers to conducting HSR. Methods: A sampling frame was compiled from funding announcements, trial registers and HSR organisation membership. Listed researchers were invited to complete online surveys. Close-ended survey items were analysed using basic descriptive statistics. Goodness of fit tests determined potential associations between researcher affiliation and access to resources for HSR. Open-ended survey items were analysed using thematic analysis. Results: In all, 424 researchers participated in the study (22% response rate). Respondents held roles as health service researchers (76%), educators (34%) and health professionals (19%). Most were employed by a university (64%), and 57% held a permanent contract. Although 63% reported network support for HSR, smaller proportions reported executive (48%) or financial (26%) support. The least accessible resources were economists (52%), consumers (49%) and practice change experts (34%) researchers affiliated with health services were less likely to report access to statisticians (P < 0.001), economists (P < 0.001), librarians (P = 0.02) and practice change experts (P = 0.02) than university-affiliated researchers. Common impacts included conference presentations (94%), publication of peer-reviewed articles (87%) and health professional benefits (77%). Qualitative data emphasised barriers such as embedding research culture within services and engaging with policy makers. Conclusions: The data highlight opportunities to sustain the HSR community through dedicated funding, improved access to methodological expertise and greater engagement with end-users. What is known about the topic?: HSR faces several challenges, such as inequitable funding allocation and difficulties in quantifying the effects of HSR on changing health policy or practice. What does this paper add?: Despite a vibrant and experienced HSR community, this study highlights some key barriers to realising a greater effect on the health and well-being of Australian and New Zealand communities through HSR. These barriers include limited financial resources, methodological expertise, organisational support and opportunities to engage with potential collaborators. What are the implications for practitioners?: Funding is required to develop HSR infrastructure, support collaboration between health services and universities and combine knowledge of the system with research experience and expertise. Formal training programs for health service staff and researchers, from short courses to PhD programs, will support broader interest and involvement in HSR.

The monetary value of a quality-adjusted life-year (QALY) is frequently used to assess the benefits of health interventions and inform funding decisions. However, there is little consensus on methods for the estimation of this monetary value. In this study, we use life satisfaction as an indicator of 'experienced utility', and estimate the dollar equivalent value of a QALY using a fixed effect model with instrumental variable estimators. Using a nationally-representative longitudinal survey including 28,347 individuals followed during 2002-2015 in Australia, we estimate that individual's willingness to pay for one QALY is approximately A$42,000-A$67,000, and the willingness to pay for not having a long-term condition approximately A$2000 per year. As the estimates are derived using population-level data and a wellbeing measurement of life satisfaction, the approach has the advantage of being socially inclusive and recognizes the significant meaning of people's subjective valuations of health. The method could be particularly useful for nations where QALY thresholds are not yet validated or established.

BackgroundIt is a public heath priority to understand why many children with mental health problems fail to access mental health services. This study aims to quantify under-recognition of children’s mental health problems by parents across income quintiles.MethodsWe estimated under-recognition with parent-reported mental health problems and the Strengths and Difficulties Questionnaire (SDQ) using a nationally representative Australian data set for children aged 4–15 years with 24 269 person-wave observations.ResultsUnder-recognition was the highest in the lowest income quintile, with 11.5% of children from the lowest income quintile families who scored in the clinical range on the SDQ perceived by parents as having no mental health problems. For the highest income quintile this was 2.4%. In terms of gender and age, under-recognition was greater for boys and younger children.ConclusionsParent’s mental health literacy, especially for low-income families, warrants prioritised attention from researchers, clinicians and policymakers.

Background Cost-effectiveness analysis (CEA) is frequently used as an input for guiding priority setting in health. However, CEA seldom incorporates information about trade-offs between total health gains and equity impacts of interventions. This study investigates to what extent equity considerations have been taken into account in CEA in low- and middle-income countries (LMICs), using rotavirus vaccination as a case study. Methods Specific equity-related indicators for vaccination were first mapped to the Guidance on Priority Setting in Health Care (GPS-Health) checklist criteria. Economic evaluations of rotavirus vaccine in LMICs identified via a systematic review of the literature were assessed to explore the extent to which equity was considered in the research objectives and analysis, and whether it was reflected in the evaluation results. Results The mapping process resulted in 18 unique indicators. Under the ‘disease and intervention’ criteria, severity of illness was incorporated in 75% of the articles, age distribution of the disease in 70%, and presence of comorbidities in 5%. For the ‘social groups’ criteria, relative coverage reflecting wealth-based coverage inequality was taken into account in 30% of the articles, geographic location in 27%, household income level in 8%, and sex at birth in 5%. For the criteria of ‘protection against the financial and social effects of ill health’, age weighting was incorporated in 43% of the articles, societal perspective in 58%, caregiver’s loss of productivity in 45%, and financial risk protection in 5%. Overall, some articles incorporated the indicators in their model inputs (20%) while the majority (80%) presented results (costs, health outcomes, or incremental cost-effectiveness ratios) differentiated according to the indicators. Critically, less than a fifth (17%) of articles incorporating indicators did so due to an explicit study objective related to capturing equity considerations. Most indicators were increasingly incorporated over time, with a notable exception of age-weighting of DALYs. Conclusion Integrating equity criteria in CEA can help policy-makers better understand the distributional impact of health interventions. This study illustrates how equity considerations are currently being incorporated within CEA of rotavirus vaccination and highlights the components of equity that have been used in studies in LMICs. Areas for further improvement are identified. Electronic supplementary material The online version of this article (10.1186/s12962-018-0102-2) contains supplementary material, which is available to authorized users.

Objective: The purpose of this paper is to provide some learnings for the NDIS from the referral pattern and cost of implementing the Partners in Recovery initiative of Gippsland. Method: Information on referral areas made for each consumer was collated from support facilitators. Cost estimates were determined using budget estimates, administrative costs and a literature review and are reported from a government perspective. Results: Sixty-three per cent of all referrals were made to organisations that provided multiple types of services. Thirty-one per cent were to Mental Health Community Support Services. Eighteen per cent of referrals were made to clinical mental health services. The total cost of providing the service for a consumer per year (set-up and ongoing) was estimated to be AUD$15,755 and the ongoing cost per year was estimated to be AUD$13,434. The cost of doing nothing is likely to cost more in the longer term, with poor mental health outcomes such as hospital admission, unemployment benefits, prison, homelessness and psychiatric residential care. Conclusions: Supporting recovery in persons with Severe and Persistent Mental Illness is likely to be economically more beneficial than not doing so. Recovery can be better supported when frequently utilised services are co-located. These might be some learnings for the NDIS.

Infection and sepsis are common problems in cancer management affecting up to 45% of patients and are associated with significant morbidity, mortality and healthcare utilisation.ObjectiveTo develop and implement a whole of hospital clinical pathway for the management of sepsis (SP) in a specialised cancer hospital and to measure the impact on patient outcomes and healthcare utilisation.MethodsA multidisciplinary sepsis working party was established. Process mapping of practices for recognition and management of sepsis was undertaken across all clinical areas. A clinical pathway document that supported nurse-initiated sepsis care, prompt antibiotic and fluid resuscitation was implemented. Process and outcome measures for patients with sepsis were collected preimplementation (April–December 2012), postimplementation cohorts (April–December 2013), and from January to December 2014.Results323 patients were evaluated (111 preimplementation, 212 postimplementation). More patients with sepsis had lactate measured (75.0% vs 17.2%) and appropriate first dose antibiotic (90.1% vs 76.1%) (all pConclusionsThe SP was associated with significant improvement in patient outcomes and reduced costs. The SP has been sustained since 2013, and has been successfully implemented in another hospital with further implementations underway in Victoria.

OBJECTIVES: We sought to determine the quality of life after the arterial switch operation (ASO) using the Short Form 36 questionnaire in adult survivors. METHODS: All patients (n = 107) who underwent the ASO and were 18 years of age or older living in the Australian state of Victoria with a contact telephone number were identified from the hospital database. Fifty-one (48%) patients were 18-24 years old and 56 (52%) patients were 25-34 years old. Patients completed the Short Form 36 quality of life questionnaire via telephone. The results of the 8 domains of the Short Form 36 questionnaire and the derived health state summary score (Short Form 6-Dimension) were compared against mean scores from age-matched Australian population data. RESULTS: Compared with the Australian population age-matched data, 18- to 24-year-old ASO patients ranked their health higher in 3 of the 8 domains (P

Children living in families with parental substance abuse are at high risk of child maltreatment and associated adverse outcomes. A trial of methadone-maintained parents randomised to the Parents under Pressure (PuP) parenting programme reported significant improvements in family functioning relative to standard care or a brief intervention, as indicated by a reduction in scores on the Child Abuse Potential Inventory. We sought to determine the cost-effectiveness of the PuP programme to inform policy and programme implementation. The cost per expected case of child maltreatment prevented was calculated and compared with the estimated lifetime costs of maltreatment and sensitivity analyses were conducted. Compared to usual care, the PuP programme costs an additional AU$8777 (£4880) per family to deliver. Assuming the most conservative estimate of one in five cases of maltreatment prevented, a cost-effectiveness estimate of AU$43 975 (£24 451) per case of potential maltreatment prevented for the PuP group was obtained. This is significantly less than the estimated mean lifetime cost of a case of child maltreatment of AU$200 000 (2013 present value) (£110 000). For 100 families in this population treated with PuP, there would be a net present value saving of an estimated AU$3.1 million (£1.7 million).

Introduction Nasal high-flow (nHF) therapy is a popular mode of respiratory support for newborn infants. Evidence for nHF use is predominantly from neonatal intensive care units (NICUs). There are no randomised trials of nHF use in non-tertiary special care nurseries (SCNs). We hypothesise that nHF is non-inferior to nasal continuous positive airway pressure (CPAP) as primary support for newborn infants with respiratory distress, in the population cared for in non-tertiary SCNs. Methods and analysis The HUNTER trial is an unblinded Australian multicentre, randomised, non-inferiority trial. Infants are eligible if born at a gestational age ≥31 weeks with birth weight ≥1200 g and admitted to a participating non-tertiary SCN, are 1 hour. Infants are randomised to treatment with either nHF or CPAP. The primary outcome is treatment failure within 72 hours of randomisation, as determined by objective oxygenation, apnoea or blood gas criteria or by a clinical decision that urgent intubation and mechanical ventilation, or transfer to a tertiary NICU, is required. Secondary outcomes include incidence of pneumothorax requiring drainage, duration of respiratory support, supplemental oxygen and hospitalisation, costs associated with hospital care, cost-effectiveness, parental stress and satisfaction and nursing workload. Ethics and dissemination Multisite ethical approval for the study has been granted by The Royal Children’s Hospital, Melbourne, Australia (Trial Reference No. 34222), and by each participating site. The trial is currently recruiting in eight centres in Victoria and New South Wales, Australia, with one previous site no longer recruiting. The trial results will be published in a peer-reviewed journal and will be presented at national and international conferences. Trial registration number Australian and New Zealand Clinical Trials Registry (ANZCTR): ACTRN12614001203640; pre-results.

Providing equitable care is an objective of many national healthcare systems. Using the birth cohort of the nationally representative Longitudinal Study of Australian Children linked with the Medicare Benefits Scheme billing data who were recruited in 2004 at ages 0–1 years and assessed biennially for six waves, we assessed the distribution of out-of-hospital government Medicare spending by household income. 4853 children followed over 11 years were included in the study. Distributions of major spending components including general practitioner and specialist care were assessed using concentration indices. Trends in the inequalities as children grow were investigated. The results showed that after controlling for health care needs, total government Medicare spending over 0–11 years of age favoured the rich (concentration index 0.041). The Medicare spending for general practitioner care was equal (concentration index 0.005) while for specialist care and diagnostics and imaging were ‘pro-rich’ (concentration index 0.108 and 0.088 respectively). Children from poorer families were most disadvantaged when aged 0–1 years in specialist spending, and the disparity lessened as children approached adolescence. Our findings suggest that income-related inequalities exist in government Medicare spending particularly in the first few years of life. As early years of life are a critical window in childhood development and building block for future health, the results warrant further investigation and attention from policy makers.