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OBJECTIVE: This research assesses the impact of an outcome-based payment arrangement (OBA) linking complete remission (CR) to survival as a means of maintaining cost-effectiveness for a chimeric antigen receptor T cell (CAR-T) therapy in young patients with acute lymphoblastic leukemia (ALL). METHODS: A partitioned survival model (PSM) was used to model the cost-effectiveness of tisagenlecleucel versus blinatumomab in ALL from the Australian healthcare system perspective. A decision tree modeled different OBAs by funneling patients into a series of PSMs based on response. Outcomes were informed by individual patient data, while costs followed Australian treatment practices. Costs and quality-adjusted life years (QALYs) were combined to calculate a single incremental cost-effectiveness ratio (ICER), reported in US dollars (2022) at a discount rate of 5% on costs and outcomes. RESULTS: For the base case, incremental costs and benefit were $379,595 and 4.27 QALYs, giving an ICER of $88,979. The ICER was most sensitive to discount rate ($57,660-$75,081), "cure point" ($62,718-$116,206) and extrapolation method ($76,018-$94,049). OBAs had a modest effect on the ICER when response rates varied. A responder-only payment was the most effective arrangement for maintaining the ICER ($88,249-$89,434), although this option was associated with the greatest financial uncertainty. A split payment arrangement (payment on infusion followed by payment on response) reduced variability in the ICER ($82,650-$99,154) compared with a single, upfront payment ($77,599-$107,273). CONCLUSION: OBAs had a modest impact on reducing cost-effectiveness uncertainty. The value of OBAs should be weighed against the additional resources needed to administer such arrangements, and importantly overall cost to government.

AbsractThis paper examines the potential effect of Direct-to-Consumer (DTC) advertising on consumers' behavioral intentions in relation to a medical issue. Using an online experiment, 1295 people were randomized to two information conditions. One group watched an advertisement for a hypothetical cold sore medicine, while a second (control) group did not view the advertisement, before both groups answered questions on symptoms. The responses were analyzed based on group allocation and the respondents' experience with cold sores. Results indicate that those who viewed the advertisement were more likely to choose the product, and the advertisement had larger effects based on consumer experience.

Lost productivity is one of the largest costs associated with foodborne illness (FBI); however, the methods used to estimate lost productivity are often criticised for overestimating the actual burden of illness. A discrete choice experiment (DCE) was undertaken to elicit preferences to avoid six possible FBIs and estimate whether ability to work, availability of paid sick leave and health-related quality of life affect willingness-to-pay (WTP) to avoid FBI. Respondents (N = 1918) each completed 20 DCE tasks covering two different FBIs [gastrointestinal illness, flu-like illness, irritable bowel syndrome (IBS), Guillain–Barre syndrome (GBS), reactive arthritis (ReA), or haemolytic uraemic syndrome (HUS)]. Attributes included: ability to work, availability of sick leave, treatment costs and illness duration. Choices were modelled using mixed logit regression and WTP was estimated. The WTP to avoid a severe illness was higher than a mild illness. For chronic conditions, the marginal WTP to avoid a chronic illness for one year, ranged from $531 for mild ReA ($1412 for severe ReA) to $1025 for mild HUS ($2195 for severe HUS). There was a substantial increase in the marginal WTP to avoid all the chronic conditions when the ability to work was reduced and paid sick leave was not available, ranging from $6289 for mild IBS to $11,352 for severe ReA. Including factors that reflect productivity and compensation to workers influenced the WTP to avoid a range of FBIs for both acute and chronic conditions. These results have implications for estimating the burden and cost of FBI.

The study characterised differences in costs associated with raising a child between four rare disorders and examined the associations between these costs with clinical severity. Caregivers of 108 individuals with Prader-Willi, Angelman (AS), Chromosome 15q Duplication and fragile X (FXS) syndromes completed a modified Client Services Receipt Inventory and participants completed intellectual/developmental functioning and autism assessments. AS incurred the highest yearly costs per individual ($AUD96,994), while FXS had the lowest costs ($AUD33,221). Intellectual functioning negatively predicted total costs, after controlling for diagnosis. The effect of intellectual functioning on total costs for those with AS was significantly different to the other syndromes. The study highlights the significant costs associated with these syndromes, particularly AS, linked with severity of intellectual functioning.

Objective The aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia. Methods The survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics. Results There were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training. Conclusions Private sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.

372 Background: Incorporating surrogate outcomes for survival into economic evaluations for localized prostate cancer (LPC) has the potential to reduce the time needed to subsidise new therapies but may increase uncertainty regarding true benefit. The ICECaP study demonstrated that metastasis-free survival (MFS) is a strong surrogate of overall survival (OS) in LPC. We sought to model the benefits to society of earlier funding decisions, and to understand the context of societal trade-offs between aspects of drug value and time to reimbursement decision-making in LPC. Methods: Societal benefits were modelled using a 4-health state (biochemical recurrence (BCR), BCR-free, metastasis and death) partitioned survival model to evaluate the cost-effectiveness of abiraterone plus prednisolone (AAP) plus standard of care (SoC) compared to SoC alone in LPC. Survival outcomes were dependent on the ICECaP surrogacy relationship. Treatment pathways and costs reflected Australian practice and quality of life was informed by the literature. The model evaluated the difference between deciding to reimburse AAP now, based on MFS, versus wait for OS 3 years. Societal trade-offs were captured using a population based discrete choice experiment exploring the influence on preference for drug reimbursement of cost-effectiveness, cost to Government, adverse events, proportions expected to benefit, nature of benefits, the completeness of data and the time of follow-up. Preferences were analysed using mixed logit analyses. Results: The analysis of societal benefits showed that using MFS as a surrogate for OS could prevent 117 patients from recurrence, 30 from metastases and save 63 lives in the short-term when AAP is compared with SoC for LPC patients in Australia. Delaying the reimbursement decision for 3 years results in a loss of 4.48 years, 0.47 quality adjusted life years, and increase the cost to $11,558 per patient over the lifetime. The analysis of societal trade-offs reflected results from 1,003 community members and showed an inclination to favour reimbursing interventions that benefit a high proportion of patients (OR=2.10 CI 95%=1.91, 2.32), have a lower cost-effectiveness ratio (OR=0.81 CI 95%= 0.78, 0.88) and an aversion for evidence from ongoing studies (OR= 0.83 CI 95%= 0.78, 0.88). Conclusions: Using MFS as surrogate for funding decision in LPC is likely to produce benefits to society in terms of improved short and long-term outcomes. This contrasts with societal preferences to delay reimbursement decisions to increase certainty. This suggests, improved cost-effectiveness could be used to compensate for the uncertainty arising from the use of surrogate outcomes in reimbursement decisions. The use of evidence-based surrogate outcomes in economic evaluations to support reimbursement decisions can reduce the time it takes new interventions to become available improving the benefits to society.

Health related quality of life (HRQoL) is rarely routinely measured in the clinical setting. In the absence of patient reported data, clinicians rely on proxy and informal estimates to support clinical decisions. This study compares clinician estimates (proxy) with patient reported HRQoL in patients with advanced heart failure and examines factors influencing discrepancies. Seventy-five patients with heart failure, (22 females, 53 males) completed the EQ-5D-5L questionnaire. Thirty-nine clinicians (11 medical, 23 nursing, 5 allied health) completed the proxy version (V1) producing 194 dyads. Correlation was assessed using Spearman’s rank tests, systematic bias was examined with Bland–Altman analyses. Inter-rater agreement at the domain level, was investigated using linear weighted Kappa statistics while factors influencing the IRG were explored using independent student t-tests, analysis of variance and regression. There was a moderate positive correlation between clinician HRQoL estimates and patient reported utility (r = 0.38; p

IntroductionThe evaluation of the Victorian Healthy Homes Program (VHHP) will generate evidence about the efficacy and cost-effectiveness of home upgrades to improve thermal comfort, reduce energy use and produce health and economic benefits to vulnerable households in Victoria, Australia.Methods and analysisThe VHHP evaluation will use a staggered, parallel group clustered randomised controlled trial to test the home energy intervention in 1000 households. All households will receive the intervention either before (intervention group) or after (control group) winter (defined as 22 June to 21 September). The trial spans three winters with differing numbers of households in each cohort. The primary outcome is the mean difference in indoor average daily temperature between intervention and control households during the winter period. Secondary outcomes include household energy consumption and residential energy efficiency, self-reported respiratory symptoms, health-related quality of life, healthcare utilisation, absences from school/work and self-reported conditions within the home. Linear and logistic regression will be used to analyse the primary and secondary outcomes, controlling for clustering of households by area and the possible confounders of year and timing of intervention, to compare the treatment and control groups over the winter period. Economic evaluation will include a cost-effectiveness and cost-benefit analysis.Ethics and disseminationEthical approval was received from Victorian Department of Human Services Human Research Ethics Committee (reference number: 04/17), University of Technology Sydney Human Research Ethics Committee (reference number: ETH18-2273) and Australian Government Department of Veterans Affairs. Study results will be disseminated in a final report and peer-reviewed journals.Trial registration numberACTRN12618000160235.

Background Prognostic uncertainty and the need for proxy decision-making owing to cognitive impairment in advanced dementia, adds complexity to end-of-life care planning within the long-term care setting. Case conferences provide a structure to facilitate difficult conversations and an opportunity for family and clinicians to engage in prospective planning, and reach agreement on goals of end-of-life care. Objective To explore interactions between multidisciplinary healthcare clinicians and families during facilitated case conferences on end-of-life care for residents with advanced dementia. Methods A qualitative approach was used. Transcripts of audio-recorded case conferences facilitated by a trained registered nurse were coded by two independent researchers and analysed inductively. Transcripts were selected from an available pool until thematic saturation was reached. Emerging themes were confirmed with the wider research group. Results Thematic saturation was reached after 25 transcripts. An overarching theme concerned the ways in which clinicians and families bridged medical and person-centred perspectives. Subthemes included: details of day-to-day care versus establishing overall goals of care; expression of emotion versus retreat from emotion; and missed opportunities versus expressed cues. Successful facilitation served to ‘bridge the gap’ between family and clinicians. Conclusion Facilitation of case conferences for residents with advanced dementia should focus on ensuring that: clinicians do not miss opportunities to discuss end-of-life care; discussions on the minutiae of care regularly return to the resident’s broader goals of care; and information on dementia and treatments provided by clinicians is integrated with advice by family members regarding the resident’s premorbid values and likely preferences.

Background: Nasal disinfection with 0.5% povidone-iodine (PVP-I) may be a useful adjunct in the management of COVID-19. The purpose of this article is to confirm the in vitro activity of the PVP-I nasal spray against SARS-CoV-2 and whether that may translate into reduced nasal shedding in vivo. Methods: Two SARS-CoV-2 virus isolates were exposed to 0.5% PVP-nasal spray (Nasodine®) for different times in vitro, with PCR and cell culture used to assess impact on viral infectivity and RNA copies. An open label in vivo single arm pilot study of 14 subjects with positive COVID-19 PCR diagnosis was undertaken. Baseline nasal swabs were collected to quantify SARS-CoV-2 pre-treatment, followed by a single 0.5% PVP nasal spray application (1.12 mL). Nasal swabs were collected at 5, 15, and 60 minutes post-dose to assess immediate and residual impact of treatment. Results: In vitro, the nasal spray reduced infectivity by 3.5 log10 TCID50/mL (99.97%) after 15 seconds exposure and eliminated detectable viral infectivity after 60 seconds; there was no effect on viral RNA detection by PCR. In vivo, culturable virus (VOC beta/B.1.351 variant) was obtained from 6 of 14 PCRconfirmed positive subjects; in these subjects, 5 minutes after the single PVP-I dose, the mean viral titre was reduced by 65% versus baseline and by 79% versus baseline at 60 minutes post-dose. 5 of the 6 subjects (83%), had reduction or cessation of viral shedding at 5 minutes in all 6 subjects, virus titers 60 minutes post-dose were below baseline value. 0.5% PVP-I treatment didn’t interfere with the laboratory diagnosis of COVID-19 via PCR-detection of viral RNA in humans. Conclusions: 0.5% PVP-I nasal spray is rapidly virucidal to SARS-CoV-2 in vitro using exposure times consistent with nasal residence; single in vivo nasal administration reduced infectious viral titers in COVID-19 subjects with culturable virus. A single application of 0.5% PVP-I nasal spray does not interfere with PCR-mediated laboratory diagnosis of COVID-19. We are undertaking a large double blinded randomized controlled trial to confirm if repeated application of 0.5% PVP-I nasal spray over a longer period could be useful in suppressing viral shedding and transmission risk in COVID-positive patients.

ObjectivesGhana is in the process of formally introducing health technology assessment (HTA) for health decision making. Similar to other low- and middle-income countries, evidence suggests that the lack of data and human capacity is a major barrier to the conduct and use of HTA. This study assessed the current human and data capacity available in Ghana to undertake HTA.MethodsAs economic evaluation (EE) forms an integral part of HTA, a systematic review of EE studies undertaken in Ghana was conducted to identify the quality and number of studies available, methods and source of data used, and local persons involved. The literature search was undertaken in EMBASE (including MEDLINE), PUBMED, and Google Scholar. The quality of studies was evaluated using the Consolidated Health Economics Evaluation Reporting Standards. The number of local Ghanaians who contributed to authorship were used as a proxy for assessing human capacity for HTA.ResultsThirty-one studies were included in the final review. Overall, studies were of good quality. Studies derived their effectiveness, resource utilization and cost data mainly from Ghana. The most common source of cost data was from the National Health Insurance Scheme pricing list for medicines and tariffs. Effectiveness data were mostly derived from either single study or intervention programs. Sixty out of 199 authors were Ghanaians (30 percent); these authors were mostly involved in data collection and study conceptualization.ConclusionsHuman capacity for HTA in Ghana is limited. To introduce HTA successfully in Ghana, policy makers would need to develop more local capacity to undertake Ghanaian-specific HTA.

Background Childhood speech, language and communication needs (SLCN) impose a significant burden on individuals, families and society. There are explicit costs related to increased health utilization and expenditure. Additionally, there may be indirect costs associated with a child's employment prospects in the long term because of the child's low literacy and numeracy, which in turn affects adult labour force participation (LFP). Several reviews have identified that there is paucity of published evidence on the costs of SLCN. Motivated by broad policy implications, and the lack of prior research in this area, this paper calculates the indirect costs and workplace productivity losses of children with SLCN. Aims To estimate the indirect costs of SLCN associated with a child's reduced long-term productivity. Methods & procedures Using 12 years of data from a longitudinal study of Australian children, we employed a panel fixed-effects model to estimate academic achievement at 14-15 years of age. Using these estimates, we employed a human capital approach (HCA) to estimate the projected LFP for children with SLCN, measured by workforce participation and foregone wages. LFP is estimated by extrapolating a child's academic achievement at 14-15 years of age to adulthood outcomes. Outcomes & results The results showed that a 1 SD (standard deviation) decrease in SLCN is equivalent to 0.19 (95% confidence interval (CI) = 0.09, 0.30) SD decrease in academic achievement at 14-15 years, 0.79% (95% CI = 0.37, 1.21) decrease in work participation and A$453 (95% CI = A$207, A$674) per annum in lost wages. The average work participation penalty across all level of SLCN (-3, -2, -1) is A$628 (95% CI = A$236, A$894) per person per year. Based on the prevalence of 8.3% from our sample, this equates to lifetime costs of A$21.677 billion (US$14.28 billion, €13.08 billion, £11.66 billion) for children with SLCN in Australia. Speech pathology treatment appears to have a positive impact on work participation and wages. On average A$355 (95% CI = A$346, A$355) per person per year could be saved through treatment or identification (the difference in lost wages for children with and without speech pathology treatment at each SLCN level (-1, -2 ,-3) calculated as a weighted average). This equates to lifetime savings of A$5.22 billion (US$3.44 billion, €3.15 billion, £2.81 billion) for children with SLCN in Australia. Conclusions & implications Overall, the findings showed that SLCN are associated with increased indirect costs through reduced workforce participation. The evidence from this study can be used to inform policies on the societal costs of SLCN. What this paper adds What is already known on this subject Childhood SLCN impose significant burden on individuals, families and society. There are explicit costs related to increased health utilization and expenditure. Additionally, there may be indirect costs associated with a child's employment prospects in the long term because of the child's low literacy and numeracy, which in turn affects adult LFP. Several reviews have identified that there is paucity of published evidence on the costs of SLCN. Motivated by broad policy implications, and the lack of prior research in this area, this paper calculates the indirect costs and workplace productivity losses of children with SLCN. What this paper adds to existing knowledge This study estimates the academic achievement and indirect costs of SLCN associated with a child's reduced long-term productivity. Using 12 years of data from a longitudinal study of Australian children, we employed a panel fixed-effects model to estimate academic achievement at 14-15 years of age. Using these estimates, we used a human capital approach to estimate the projected LFP for children with SLCN, measured by workforce participation and foregone wages. LFP is estimated by extrapolating a child's academic achievement at 14-15 years of age to adulthood outcomes. What are the potential or actual clinical implications of this work? SLCN are associated with increased indirect costs through reduced workforce participation. The evidence from this study provides one of the first indirect cost estimates of how SLCN impacts LFP through educational achievement. Early identification, intervention and screening for SLCN may be useful offsets to reduce the economic effects identified here.

The reimbursement of immune checkpoint inhibitors is challenging. Funding these technologies involves the careful balance between awarding innovation and ensuring affordability as increases in drug spending compete directly with other health care and social expenditure. This narrative review examines the recommendations of 2 health technology assessment agencies—the Australian Pharmaceutical Benefits Advisory Committee and the British National Institute of Clinical Excellence—to determine the factors that contribute to the approval and rejection of immune checkpoint inhibitors as well as the use of manage entry schemes and risk management strategies to control expenditure. Reimbursement decisions from 6 immune checkpoint inhibitor drugs (ipilimumab, pembrolizumab, nivolumab, durvalumab, atezolizumab, avelumab) covering 10 different cancers were examined. The extrapolation of survival beyond the clinical trial and lack of head‐to‐head evidence are some of the main issues relating to cost effectiveness. Payers managed financial risks using different mechanisms such as risk share agreements and financial caps. This review of the reimbursement decisions and subsequent financial impact in Australia and the UK suggests budgets for immune checkpoint inhibitor therapy have been well managed so far. Through risk agreements and managed entry programmes, the example of immune checkpoint inhibitor therapies illustrates that industry and payers can effectively collaborate to ensure that innovative, but expensive, drugs can be made readily available to patients.

OBJECTIVES: The Sunbeam trial significantly reduced falls in long-term aged care (LTC) residents. The current study's primary objective was to undertake subgroup analysis of the Sunbeam trial, to determine whether the intervention was effective for reducing falls in LTC residents with mild-moderate cognitive impairment/dementia. Secondary objectives were to determine intervention effects on cognitive and physical function. DESIGN: Subgroup analysis of a cluster randomized controlled trial (RCT). SETTING AND PARTICIPANTS: Permanent residents of LTC in Australia who participated in the Sunbeam trial with Addenbrooke's Cognitive Examination-Revised (ACE-R) scores 14 = main trial inclusion criteria). METHODS: Of 221 participants, 148 had an ACE-R

Refereed/Peer-reviewed Importance: The effects of altered neural processing, defined as altering neural networks responsible for perceptions of pain and function, on chronic pain remains unclear. Objective: To estimate the effect of a graded sensorimotor retraining intervention (RESOLVE) on pain intensity in people with chronic low back pain. Design, Setting, and Participants: This parallel, 2-group, randomized clinical trial recruited participants with chronic (>3 months) nonspecific low back pain from primary care and community settings. A total of 276 adults were randomized (in a 1:1 ratio) to the intervention or sham procedure and attention control groups delivered by clinicians at a medical research institute in Sydney, Australia. The first participant was randomized on December 10, 2015, and the last was randomized on July 25, 2019. Follow-up was completed on February 3, 2020. Interventions: Participants randomized to the intervention group (n = 138) were asked to participate in 12 weekly clinical sessions and home training designed to educate them about and assist them with movement and physical activity while experiencing lower back pain. Participants randomized to the control group (n = 138) were asked to participate in 12 weekly clinical sessions and home training that required similar time as the intervention but did not focus on education, movement, and physical activity. The control group included sham laser and shortwave diathermy applied to the back and sham noninvasive brain stimulation. Main Outcomes and Measures: The primary outcome was pain intensity at 18 weeks, measured on an 11-point numerical rating scale (range, 0 [no pain] to 10 [worst pain imaginable]) for which the between-group minimum clinically important difference is 1.0 point. Results: Among 276 randomized patients (mean [SD] age, 46 [14.3] years; 138 [50%] women), 261 (95%) completed follow-up at 18 weeks. The mean pain intensity was 5.6 at baseline and 3.1 at 18 weeks in the intervention group and 5.8 at baseline and 4.0 at 18 weeks in the control group, with an estimated between-group mean difference at 18 weeks of -1.0 point ([95% CI, -1.5 to -0.4]; P =.001), favoring the intervention group. Conclusions and Relevance: In this randomized clinical trial conducted at a single center among patients with chronic low back pain, graded sensorimotor retraining, compared with a sham procedure and attention control, significantly improved pain intensity at 18 weeks. The improvements in pain intensity were small, and further research is needed to understand the generalizability of the findings. Trial Registration: ANZCTR Identifier: ACTRN12615000610538.

Background Children with intellectual disability (ID) frequently have significant educational, social and health care needs, resulting in caregivers often experiencing a wide range of negative effects. This paper aims to determine the impact of childhood ID on caregivers' health-related quality of life (HRQoL) across co-morbid diagnostic groups. The second aim of this study is to determine the risk factors associated with lower HRQoL in this population. Methods Caregivers of a child with ID aged between 2 and 12 years old completed an online survey to determine their HRQoL using the EQ-5D-5L measure. They were also asked demographic questions and about their dependent child's level of behavioural and emotional difficulties. Results Of the total sample of 634 caregivers, 604 caregivers completed all five questions of the EQ-5D-5L. The mean age of caregivers was 39.1 years and 91% were women. Caregivers spent on average 66.6 h per week caring for their child related to their child's disability. The mean EQ-5D-5L score of caregivers was 0.80 (95% confidence interval: 0.79, 0.82), which is below the estimated Australian population norms (mean utility score of 0.92) for the age-equivalent population. Caregivers of children with autism spectrum disorders reported the lowest HRQoL (0.77, 95% confidence interval: 0.74, 0.79) of the five included co-morbid diagnostic groups. Caregivers with a lower income, a perceived low level of social support and children with higher degree of behavioural and emotional problems were likely to have a statistically lower HRQoL. Conclusions This is the first study to produce utility values for caregivers of children with ID. The utility values can be used to compare health states and can be used to inform comparative cost-effectiveness analyses. Demonstrating that caregivers of children with ID have reduced HRQoL and that this is associated with the degree of behavioural and emotional problems has important policy implications, highlighting the potential for policy interventions that target behavioural and emotional problems to improve outcomes for caregivers.

Aims: The aim of this study was to estimate the cost-effectiveness of nivolumab versus everolimus for second-line treatment of renal cell carcinoma (RCC) based on mature data, updated safety and decreased everolimus price. Materials and methods: A 3-state (pre-progression/progression-free disease, progressive disease and death) Markov model was developed from the perspective of the Australian health care system. Two scenarios were tested. Scenario 1 used 30-months clinical data and scenario 2 used updated 80-months clinical data with updated everolimus price. Inputs for quality-of-life and costs were informed by the literature and government sources. Incremental cost-effectiveness ratio (ICER) per quality adjusted life years (QALY) gained was reported and an ICER threshold of AU$75,000 was assumed. Threshold analysis was performed, and uncertainty was explored using one-way and probabilistic sensitivity analyses. Results: In scenario 1, the model estimated 1.73 QALYs at a cost of AU$105,000 for nivolumab and 1.48 QALYs at AU$38,000 for everolimus with an ICER = AU$266,871/QALY gained. A rebate of 54.4% was needed for nivolumab to reach the ICER threshold. For scenario 2, 1.93 QALYs at AU$111,418 was estimated for nivolumab and 1.60 QALYs at AU$31,942 for everolimus with an ICER of AU$213,320/QALY gained. The rebate needed to reach the ICER threshold was 54.9%. One-way sensitivity analyses for both scenarios showed that the cost of nivolumab, time horizon and utilities were main drivers. The cost-effectiveness acceptability curves highlighted the differences in cost-effectiveness of the two scenarios, as well as significant uncertainty in the results. Conclusions: A 54% rebate of the published price is needed for nivolumab to be cost-effective in Australia for the treatment of RCC. At that rebate, nivolumab remains cost-effective despite severe price erosion of everolimus because of improved longer term follow-up data. We recommend that generic price erosion should be accounted for when performing cost-effectiveness analysis.

The study characterised differences in costs associated with raising a child between four rare disorders and examined the associations between these costs with clinical severity. Caregivers of 108 individuals with Prader-Willi, Angelman (AS), Chromosome 15q Duplication and fragile X (FXS) syndromes completed a modified Client Services Receipt Inventory and participants completed intellectual/developmental functioning and autism assessments. AS incurred the highest yearly costs per individual ($AUD96,994), while FXS had the lowest costs ($AUD33,221). Intellectual functioning negatively predicted total costs, after controlling for diagnosis. The effect of intellectual functioning on total costs for those with AS was significantly different to the other syndromes. The study highlights the significant costs associated with these syndromes, particularly AS, linked with severity of intellectual functioning.

BackgroundOne model of care that has not been tested for chronic low back pain (LBP) is patient-led goal setting. We aimed to compare the clinical effectiveness and healthcare use of a patient-led goal setting approach (intervention) with simple advice to exercise (control) over 12 months.MethodsAn assessor-blinded randomised controlled trial. Intervention was education combined with patient-led goal setting compared with a control group receiving a standardised exercise programme. The primary outcomes were back pain disability and pain intensity. Secondary outcomes were quality of life, kinesiophobia, self-efficacy, depression, anxiety and stress. Outcomes and healthcare use were assessed immediately post-treatment (2 months) and after 4 and 12 months. Analysis was by intention to treat.ResultsSeventy-five patients were randomly assigned to either the intervention (n=37) or the control (n=38) group. Using linear mixed model analyses, adjusted mean changes in primary outcomes of disability and pain intensity were greater in the intervention group than in the control group (disability post-treatment: pConclusionA patient-led goal setting intervention was significantly more effective than advice to exercise for improving outcomes in disability, pain intensity, quality of life, self-efficacy and kinesiophobia in chronic LBP. These improvements were maintained at 12 months. Smaller effects were seen in measures of depression, anxiety and stress.Trial registration numberACTRN12614000830695.

BackgroundInternational research has focused on screening and mass media campaigns to promote earlier patient presentation and detect lung cancer earlier. This trial tested the effect of a behavioural intervention in people at increased risk of lung cancer on help-seeking for respiratory symptoms.MethodsParallel, individually randomised controlled trial. Eligible participants were long-term smokers with at least 20 pack-years, aged 55 and above. The CHEST intervention entailed a consultation to discuss and implement a self-help manual, followed by self-monitoring reminders to encourage help-seeking for respiratory symptoms. The control group received a brief discussion about lung health. Both groups had baseline spirometry. Telephone randomisation was conducted, 1:1, stratified Medical Research Council (MRC) dyspnoea score and general practice. Participants could not be blinded; data extraction and statistical analyses were performed blinded to group assignment. The primary outcome was respiratory consultation rates.ResultsWe randomised 551 participants (274 intervention, 277 control) from whom the primary outcome was determined for 542 (269 intervention, 273 control). There was a 40% relative increase in respiratory consultations in the intervention group: (adjusted rates (95% CI) intervention 0.57 (0.47 to 0.70), control 0.41 (0.32 to 0.52), relative rate 1.40 (1.08 to 1.82); p=0.0123). There were no significant differences in time to first respiratory consultation, total consultation rates or measures of psychological harm. The incremental cost-effectiveness ratio was $A1289 per additional respiratory consultation.ConclusionsA behavioural intervention can significantly increase consulting for respiratory symptoms in patients at increased risk of lung cancer. This intervention could have an important role in primary care as part of a broader approach to improve respiratory health in patients at higher risk.Trial registration numberAustralian New Zealand Clinical Trial Registry (1261300039 3752). This was registered pre-results.

© 2019, Springer Nature Switzerland AG. Background: In 2009, mandatory folic acid fortification of bread-making flour was introduced in Australia to reduce the birth prevalence of preventable neural tube defects (NTDs) such as spina bifida. Before the introduction of the policy, modelling predicted a reduction of 14–49 NTDs each year. Objective: Using real-world data, this study provides the first ex-post evaluation of the cost effectiveness of mandatory folic acid fortification of bread-making flour in Australia. Methods: We developed a decision tree model to compare different fortification strategies and used registry data to quantify the change in NTD rates due to the policy. We adopted a societal perspective that included costs to industry and government as well as healthcare and broader societal costs. Results: We found 32 fewer NTDs per year in the post-mandatory folic acid fortification period. Mandatory folic acid fortification improved health outcomes and was highly cost effective because of the low intervention cost. The policy demonstrated improved equity in outcomes, particularly in birth prevalence of NTDs in births from teenage and indigenous mothers. Conclusions: This study calculated the value of mandatory folic acid fortification using real-world registry data and demonstrated that the attained benefit was comparable to the modelled expected benefits. Mandatory folic acid fortification (in addition to policies including advice on supplementation and education) improved equity in certain populations and was effective and highly cost effective for the Australian population.

© 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research Background: Risk-sharing arrangements (RSAs) can be used to mitigate uncertainty about the value of a drug by sharing the financial risk between payer and pharmaceutical company. We evaluated the projected impact of alternative RSAs for non–small cell lung cancer (NSCLC) therapies based on real-world data. Methods: Data on treatment patterns of Dutch NSCLC patients from four different hospitals were used to perform “what-if” analyses, evaluating the costs and benefits likely associated with various RSAs. In the scenarios, drug costs or refunds were based on response evaluation criteria in solid tumors (RECIST) response, survival compared to the pivotal trial, treatment duration, or a fixed cost per patient. Analyses were done for erlotinib, gemcitabine/cisplatin, and pemetrexed/platinum for metastatic NSCLC, and gemcitabine/cisplatin, pemetrexed/cisplatin, and vinorelbine/cisplatin for nonmetastatic NSCLC. Results: Money-back guarantees led to moderate cost reductions to the payer. For conditional treatment continuation schemes, costs and outcomes associated with the different treatments were dispersed. When price was linked to the outcome, the payer's drug costs reduced by 2.5% to 26.7%. Discounted treatment initiation schemes yielded large cost reductions. Utilization caps mainly reduced the costs of erlotinib treatment (by 16%). Given a fixed cost per patient based on projected average use of the drug, risk sharing was unfavorable to the payer because of the lower than projected use. The impact of RSAs on a national scale was dispersed. Conclusions: For erlotinib and pemetrexed/platinum, large cost reductions were observed with risk sharing. RSAs can mitigate uncertainty around the incremental cost-effectiveness or budget impact of drugs, but only when the type of arrangement matches the setting and type of uncertainty.

BackgroundThe National Institute for Health and Care Excellence (NICE) prefers the use of the generic EQ-5D instrument to estimate quality-adjusted life years (QALYs), and recommends that condition-specific instruments only be used when EQ-5D data are not available or not appropriate.ObjectiveThis study aimed to compare the utility gain and cost-effectiveness results of using the generic EQ-5D-3L instrument to the condition-specific Quality-of-Life Utility Measure-Core 10 dimensions (QLU-C10D) by applying both sets of values in a published cost-utility analysis (CUA) of immunotherapy for metastatic melanoma.MethodsQuality-of-life data were drawn from a clinical study in which both QLQ-C30 and EQ-5D-3L tools were used. The potential influence of the two instruments on cost-effectiveness was assessed using a three-state Markov model. Descriptive statistics and standard health economic outputs were compared between analyses that applied the two different utility measures.ResultsMean baseline utility values as measured by the QLU-C10D (mean = 0.744, SD = 0.219) were not statistically different (p > 0.05) compared to values derived from EQ-5D-3L (mean = 0.735, SD = 0.239). The two instruments were correlated (Pearson's correlation = 0.74); however, concordance was low (Lin's concordance correlation coefficient < 0.90) at baseline. The model predicted slightly higher QALYs gained when using EQ-5D-3L over QLU-C10D-derived utilities (1.87 vs 1.74, respectively). This resulted in an incremental cost-effectiveness ratio of US$30.5K when using EQ-5D-3L utilities, compared to US$32.7K when using QLU-C10D utilities. Cost-effectiveness acceptability curves based on the two sets of utilities were almost indistinguishable.ConclusionThis study supports the use of the generic EQ-5D instrument in immunotherapy treated metastatic melanoma, and found no additional benefit for using the disease-specific QLU-C10D when using Australian weights.

Speech and language acquisition is one of the key development indicators of optimal literacy development in infancy and early childhood. Over the last decade there has been increasing interest in the development of theoretical frameworks which underpin the underlying complexity of a child’s language developmental landscapes. This longitudinal study aims to measure the impact of genetic and environmental risk and protective factors on speech, language, and communication development (SLCN) among 5000 infants in Australia. Using robust panel fixed-effects models, the results demonstrate that there are clear and consistent effects of protective factors and SLCN associated with the infant’s family [coefficient (SD) = 0.153, 95% standard error (SE) = 8.76], the in utero environment [coefficient (SD) = 0.055, standard error (SE) = 3.29] and early infant health [coefficient (SD) = 0.074, standard error (SE) = 5.28]. The impact of family and in utero health is dominant at aged 2 to 3 years (relative to 0 to 1 years) across the domains of language and communication and more dominant from birth to 1 years for speech acquisition. In contrast, the evidence for the impact of genetics on SLCN acquisition in infancy, is less clear. The evidence from this study can be used to inform intervention policies.

PURPOSE: Up to 40% of cancer patients treated with neurotoxic chemotherapies experience chemotherapy-induced peripheral neuropathy (CIPN). Currently, there is no gold standard assessment tool for CIPN and there is little information in the literature on patient preferences for such assessments. This study aims to address this gap by identifying the features of a CIPN assessment tool that cancer patients value. METHODS: An online discrete choice experiment (DCE) survey of neurotoxic chemotherapy-treated patients was implemented. Respondents completed 8 choice questions each. In each choice question, they chose between two hypothetical CIPN assessment tools, each described by six attributes: impact on quality of life; level of nerve damage detected; questionnaire length; physical tests involved; impact on clinic time; impact on care. RESULTS: The survey was completed by 117 respondents who had a range of cancers of which breast cancer was the most common. Respondents favoured an assessment tool that includes a physical test and that asks about impact on quality of life. Respondents were strongly opposed to clinicians, alone, deciding how the results of a CIPN assessment might influence their care especially their chemotherapy treatment. They were concerned about small changes in their CIPN, independent of clinical relevance. Respondents were willing to add half an hour to the usual clinic time to accommodate the CIPN assessment. CONCLUSION: The findings of this DCE will assist clinicians in choosing an assessment tool for CIPN that is satisfactory to both clinician and patient.

Objectives The objective of this review was to identify sources of variability in cost-effectiveness analyses of chimeric antigen receptor T-cell (CAR-T) therapies, tisagenlecleucel and axicabtagene ciloleucel, evaluated by health technology assessment (HTA) agencies, focusing on young compared with older patients. Methods HTA evaluations in pediatric acute lymphoblastic leukemia (ALL) and adult diffuse large B-cell lymphoma (DLBCL) were included from Australia, Canada, England, Norway, and the United States. Key clinical evidence, economic approach, and outcomes (costs, quality-adjusted life-years [QALYs] and incremental cost-effectiveness ratios) were summarized. Results Fourteen HTA evaluations were identified (5 ALL, 9 DLBCL [4 tisagenlecleucel, 5 axicabtagene]). Analyses were naive comparisons of prospective single-arm studies for the CAR-Ts with retrospective cohort studies for the comparators. Key clinical evidence and economic model approaches were generally consistent by CAR-T and indication, although outcomes varied. Notably, incremental QALYs varied substantially in ALL (3.67-10.6 QALYs gained), whereas variation in DLBCL was less (1.21-1.97 [tisagenlecleucel], 1.97-3.40 [axicabtagene]). Discounting of costs and outcomes varied, with the highest QALYs generated for tisagenlecleucel in ALL (10.95) associated with the lowest discount rate (1.5%) and vice versa (4.97 QALYs; 5% discount rate). The approach to extrapolation of overall survival data varied, even where the same empirical data were used. Conclusion Modeled, long-term treatment benefit in young patients may be associated with greater uncertainty compared with adults because of potential life-long benefits with cell and gene therapies. This reflects the methodological challenges identified by HTA agencies associated with single-arm, short-term studies.

ObjectivesThe friction cost approach (FCA) is one way to estimate lost productivity, which considers the time taken to replace an employee, known as the friction period. The friction period may be influenced by local labour market conditions, limiting the relevance of international FCA estimates. The objective was to estimate the time and costs of replacing an employee in Australia.MethodsStaff responsible for recruitment in businesses across Australia were surveyed about the last management and non-management employee hired, workforce composition, friction period time and costs, and team dynamic effects. Primary analyses were conducted on respondents that recruited in the past 12 months. The friction period was decomposed into three periods: recruitment decision, recruitment period, and training period. Descriptive statistics of the friction period time and costs, and team dynamic effects were calculated.ResultsThe sample consisted of Australian businesses (N = 274), primarily micro-organisations (2-4 employees, 44%) in urban locations (75%). The time (12.3 weeks; SD 15.1) and costs ($6230; SD $17,502) to replace a manager were higher than those to replace non-managers (10.0 weeks, SD 13.01; $2666, sd $7849). The training period represented the longest time component in replacing an employee (38-40% of the total friction period). There was an increasing impact on other employees' productivity, particularly for absent managers as time off work increased.ConclusionsThe friction period in Australia was similar to international estimates. Interestingly, the friction period mainly consisted of time outside the recruitment period; the decision to recruit and the training period.

Background Statistical analysis plans describe the planned data management and analysis for clinical trials. This supports transparent reporting and interpretation of clinical trial results. This paper reports the statistical analysis plan for the RESOLVE clinical trial. The RESOLVE trial assigned participants with chronic low back pain to graded sensory-motor precision training or sham-control. Results We report the planned data management and analysis for the primary and secondary outcomes. The primary outcome is pain intensity at 18-weeks post randomization. We will use mixed-effects models to analyze the primary and secondary outcomes by intention-to-treat. We will report adverse effects in full. We also describe analyses if there is non-adherence to the interventions, data management procedures, and our planned reporting of results. Conclusion This statistical analysis plan will minimize the potential for bias in the analysis and reporting of results from the RESOLVE trial. Trial registration ACTRN12615000610538 ( https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=368619 ).

Although health technology assessment (HTA) is intended to provide policymakers with objective information, the likelihood that a health decision-maker (HDM) will use this information is associated with their knowledge, role and perception of the HTA process. In Ghana, policymakers are working towards formalising the use of HTA, but HDM knowledge of and attitude towards HTA are not known. Between March and May 2016, we conducted in-depth interviews and used inductive thematic analysis to explore Ghanaian HDMs (n = 23) and researchers' (n = 4) perceptions of and barriers to HTA and identify ways to promote HTA. We compare our findings with those reported in previous studies conducted in low-and-middle-income countries. Common themes were that resources, political and cultural factors act as barriers to HTA use. Recommendations made in previous studies which were also identified in this study included the need for the development of both human and data capacity, allocating funds to HTA and stakeholder involvement in HTA processes. Specific recommendations made by Ghanaian HDMs and researchers in this study focused on the establishment of an HTA body: its location, the constitution of the appraisal team, the type of evidence to appraise and who makes the final decision. The findings provide important information in the context of current planning to institutionalise HTA in Ghana. Addressing the identified barriers will enable policymakers to maximise the chances of realising the expected benefits of HTA, as participants who are potential producers and end-users are likely to use what they have contributed to.

Grutters et al recently investigated the role of early health economic modelling of health technologies by undertaking a secondary analysis of health economic modelling assessments performed by their group. Our commentary offers a broad perspective on the potential utility of early health economic modelling to inform health technology assessment (HTA) and decision-making around reimbursement of new health technologies. Further we provide several examples to compliment Grutters and colleagues’ observations.

Health technology assessment (HTA) was introduced in Australia for the reimbursement of pharmaceuticals in 1992 and in the following years for procedures, diagnostic tests, and devices. The Australian health system is largely funded by the government. The Pharmaceutical Benefits Scheme is a national list of prescription pharmaceuticals for which the patient pays a small copayment. HTA submissions to the Pharmaceutical Benefits Scheme are assessed by the Pharmaceutical Benefits Advisory Committee. The Medical Benefits Scheme provides ambulatory medical services and HTA submissions are assessed by the Medical Services Advisory Committee. This article describes the processes of reimbursement in Australia as well as the special case of codependent technologies (eg, diagnostic test and a therapeutic drug) where a combined Medical Services Advisory Committee and Pharmaceutical Benefits Advisory Committee application is required. There are many future challenges for HTA in Australia, with growing pressure to provide early access to promising treatments and high cost personalized medicines looming on the horizon. However, Australia is well placed to deal with these issues as the early adoption of HTA and coexistence between industry, academia and the payer has proven to be a fertile environment for developing capacity to undertake and evaluate HTA.

Background There is limited research quantifying the direct and indirect economic costs associated with intellectual disability (ID) in Australia. Costs incurred by families, governments and broader society include time spent providing care, absenteeism and increased healthcare utilisation. The purpose of this research is to quantify the costs associated with ID in childhood using a range of methods to collect cost data. Methods Costs included healthcare service utilisation, pharmaceutical use, caregiver productivity losses and time spent providing care because of the child's disability. The sample comprised caregivers with a child with ID aged between 2 and 10 years old recruited in Australia. Healthcare service utilisation and pharmaceutical use were obtained from routinely collected administrative claims data. Healthcare utilisation not captured in the routinely collected administrative data and absenteeism data were obtained from a retrospective recall-based questionnaire. Time spent providing care because of the child's disability was obtained using a time-use diary. Results The total cost of ID in Australia was estimated to be AUD 72 027 per year per child, and the total cost of ID in childhood was estimated to be AUD 12.5 billion per year. The cost to governments of ID in childhood was estimated to be AUD 6385 per child per year, resulting in a total cost to government of AUD 1.1 billion per year. Conclusions This is the first study to estimate the direct and indirect costs associated with ID in childhood. The results of this research demonstrate the considerable economic impact of ID in childhood on families, governments and broader society in terms of both direct and indirect costs. An understanding of the cost implications of any intervention are critical in assisting policymakers in planning and prioritising of health services.

Objective: To estimate the cost-effectiveness of a strength and balance exercise programme (SUNBEAM) which has been shown to be clinically effective in reducing the rate of falls in residents of aged care facilities. Design: An economic evaluation was conducted alongside a pragmatic cluster randomized controlled trial that included 16 residential care facilities and 221 participants. Mean participant age was 86 years, 65% were female and 78% relied on a mobility aide. A cost-effectiveness analysis examined the costs of providing the exercise programme and costs of health service use arising from falls in each arm (intervention and usual care) over 12 months. Main measures: Incremental cost-effectiveness ratios were calculated for the cost per fall avoided. Costs were bootstrapped to obtain adjusted confidence intervals for the incremental cost-effectiveness ratios. Results: Of 63 facilities contacted, 16 met the eligibility criteria and were randomized to the intervention or usual care (1:1). There were 142 falls in the intervention group and 277 in the usual care group. 72 injurious falls occurred in the intervention group versus 157 with usual care. Delivery of the SUNBEAM programme cost $463 per participant. The mean total cost of each fall (regardless of group) was $400.09 and the mean cost of each injurious fall was $708.27. The incremental cost-effectiveness ratio was $22 per fall per person avoided with the mean bootstrapped incremental cost-effectiveness ratio $18 per fall avoided (95% CI: −$380.34 to $417.85). Conclusion: The SUNBEAM programme can be considered cost-effective, relative to other fall-prevention interventions in older adults.

Prescribing medications for nursing home residents with advanced dementia should focus on optimizing function and comfort, reducing unnecessary harms and aligning care goals with a palliative approach.The aim of the study was to estimate the proportion of Australian nursing home residents with advanced dementia receiving potentially inappropriate medications, and identify those most commonly prescribed and factors associated with their use.Data were collected through retrospective audit of medication charts.Two hundred eighteen nursing home residents with advanced dementia from 20 nursing homes participated in a cluster-randomized controlled trial of case conferencing (the IDEAL Study) from June 2013 to December 2014.Inappropriate drug use was defined as medications classified as "never appropriate" by the Palliative Excellence in Alzheimer Care Efforts (PEACE) program criteria. Generalized linear mixed models were used to identify variables predicting use of "never" appropriate medications.Over a quarter (n = 65, 30%) of residents received at least one medication classed as "never" appropriate, the most common being lipid-lowering agents (n = 38, 17.4%), antiplatelet agents (n = 18, 8.3%), and acetylcholinesterase inhibitors (n = 16, 7.3%). Residents who had been at the nursing home for ≤10 months (odds ratio [OR] 5.60, 95% confidence interval [CI] 1.74-18.06) and 11-21 months (OR 5.41, 95% CI 1.67-17.75) had significantly greater odds of receiving a never appropriate medication compared with residents who had been at the nursing home for5 years.Use of potentially inappropriate medications in Australian nursing home residents with advanced dementia is common. A greater understanding of the rationale that underpins prescribing of medications is required.

Background: Assessing health-related quality of life (HRQOL) in people with advanced dementia is challenging but important for informed decision-making. Proxy measurement of this construct is difficult and is often rated lower than self-report. Accurate proxy rating of quality of life in dementia is related to identification of concepts important to the person themselves, as well as the sensitivity of the measures used. The main aim of this study was to compare the performance of two instruments—QUALID and EQ-5D-5L—on measuring HRQOL in people with advanced dementia. Methods: In a sub-study nested within a cluster-RCT we collected proxy(nurse)-completed EQ-5D-5L and QUALID measures at baseline, 3, 6, 9 and 12 months’ follow-up for people with advanced dementia, residing in 20 nursing homes across Australia. Spearman’s rank correlations, partial correlations and linear regressions were used to assess the relationship between the HRQOL instrument scores and their changes over time. Results: The mean weight from 284 people for the EQ-5D-5L and QUALID at baseline were 0.004 (95% CI − 0.026, 0.033) and 24.98 (95% CI 24.13, 25.82), respectively. At 12 months’ follow-up, 115 participants remained alive. EQ-5D-5L weights and QUALID scores at baseline and at follow-up were moderately correlated (r = − 0.437; p < 0.001 at 12 months). Changes within QUALID and EQ-5D-5L across the same follow-up periods were also correlated (r = − 0.266; p = 0.005). The regression analyses support these findings. Conclusion: Whilst these quality of life instruments demonstrated moderate correlation, the EQ-5D-5L does not appear to capture all aspects of quality of life that are relevant to people with advanced dementia and we cannot recommend the use of this instrument for use within this population. The QUALID appears to be a more suitable instrument for measuring HRQOL in people with severe dementia, but is not preference-based, which limits its application in economic evaluations of dementia care.

This study aimed to describe Australian long-term care (LTC) personnel’s knowledge and attitudes concerning palliative care for residents with advanced dementia, and explore relationships with LTC facility/personnel characteristics. An analysis was undertaken of baseline data from a cluster randomised controlled trial of facilitated family case conferencing for improving palliative care of LTC residents with advanced dementia (the ‘IDEAL Study’). Participants included any LTC personnel directly involved in residents’ care. Knowledge and attitudes concerning palliative care for people with advanced dementia were measured using the questionnaire on Palliative Care for Advanced Dementia. Univariate and multivariate analyses explored relationships between personnel knowledge/attitudes and facility/personnel characteristics. Of 307 personnel in the IDEAL Study, 290 (94.5%) from 19/20 LTCFs provided sufficient data for inclusion. Participants included 9 (2.8%) nurse managers, 59 (20.5%) registered nurses, 25 (8.7%) enrolled nurses, 187 (64.9%) assistants in nursing/personal care assistants and 9 (3.1%) care service employees. In multivariate analyses, a facility policy not to rotate personnel through dementia units was the only variable associated with more favourable overall personnel knowledge and attitudes. Other variables associated with favourable knowledge were a designation of nursing manager or registered or enrolled nurse, and having a preferred language of English. Other variables associated with favourable attitudes were tertiary level of education and greater experience in dementia care. Like previous international research, this study found Australian LTC personnel knowledge and attitudes regarding palliative care for people with advanced dementia to be associated with both facility and personnel characteristics. Future longitudinal research is needed to better understand the relationships between knowledge and attitudes, as well as between these attributes and quality of care.

© 2018, Springer International Publishing AG, part of Springer Nature. Objectives: This research produces a preference-based monetary valuation of informal care provided to children with intellectual disability (ID) that can be directly applied in economic evaluations. Methods: A discrete-choice experiment (DCE) was designed to elicit an individual’s willingness to accept compensation for different care tasks. Respondents were presented choice sets that included a care package comprising different amounts and types of care and asked to choose between the care package provided free of charge or providing that care themselves and receiving cash compensation. The care package included personal care, social support, household errands and housework, with the value of compensation, number of care hours provided and types of care varied across the choice sets. Choices were analysed using a generalised multinomial logit model and latent class model. Results: A representative sample of 198 caregivers completed the survey (response rate 52%). Participants were recruited in Australia. Overall, caregivers would accept a minimum of Australian dollars ($A)20.61 to provide 1 h of care. The preferences for assistance varied significantly with different types of care tasks. Individuals placed the highest value on receiving assistance with social support ($A35.96) and the least value on receiving assistance with household errands ($A-0.92) Conclusions: This study produces a value of informal care provided to children with ID that can be directly applied in economic evaluations. The study shows that informal care tasks are not valued equally. Caregivers placed the most value on receiving assistance with social support, which may reflect the time spent by caregivers on these tasks.

Background This study aimed to determine the cost-effectiveness of contrast-enhanced magnetic resonance imaging (CE-MRI) compared with multiphase CE computed tomography (CE-CT) scan to characterize suspected liver lesions in patients with known colorectal carcinoma. Methods A decision analytic model linking diagnostic accuracy to health outcomes in patients with colorectal carcinoma was constructed. The model assumed that CE-MRI has superior sensitivity and equivalent specificity to CE-CT, and patients with a colorectal liver metastasis could be eligible for curative surgery or chemotherapy and palliation. Delayed diagnosis or misdiagnosis was associated with worse health outcomes (disutility). Cost-effectiveness was calculated as the incremental cost relative to the incremental benefit, the benefit was estimated using quality-adjusted life years. Sensitivity analyses were conducted to test the robustness of the results. Results The clinical evidence supports increased sensitivity of CE-MRI compared with CE-CT (0.943 versus 0.768). CE-MRI was more effective and more costly than CE-CT. The incremental cost-effectiveness ratio was estimated to be $40 548 per quality-adjusted life year gained. The model is most sensitive to the cost of MRI, cost of palliative treatment and the disutility associated with delayed palliative care. The results were also sensitive to the assumptions made about the clinical algorithm. Conclusion The results provide evidence of the potential cost-effectiveness associated with CE-MRI for the diagnosis of liver metastases in patients with identified colorectal carcinoma. CE-MRI can be recommended as cost-effective provided it replaces CE-CT and that improved diagnostic accuracy results in earlier, curative, disease management.

Background:Palliative care for nursing home residents with advanced dementia is often sub-optimal due to poor communication and limited care planning. In a cluster randomized controlled trial, registered nurses (RNs) from 10 nursing homes were trained and funded to work as Palliative Care Planning Coordinators (PCPCs) to organize family case conferences and mentor staff. This qualitative sub-study aimed to explore PCPC and health professional perceptions of the benefits of facilitated case conferencing and identify factors influencing implementation.Method:Semi-structured interviews were conducted with the RNs in the PCPC role, other members of nursing home staff, and physicians who participated in case conferences. Analysis was conducted by two researchers using a thematic framework approach.Results:Interviews were conducted with 11 PCPCs, 18 other nurses, eight allied health workers, and three physicians. Perceived benefits of facilitated case conferencing included better communication between staff and families, greater multi-disciplinary involvement in case conferences and care planning, and improved staff attitudes and capabilities for dementia palliative care. Key factors influencing implementation included: staffing levels and time; support from management, staff and physicians; and positive family feedback.Conclusion:The facilitated approach explored in this study addressed known barriers to case conferencing. However, current business models in the sector make it difficult for case conferencing to receive the required levels of nursing qualification, training, and time. A collaborative nursing home culture and ongoing relationships with health professionals are also prerequisites for success. Further studies should document resident and family perceptions to harness consumer advocacy.

Study design: Randomised double-blind factorial-design placebo-controlled trial. Objective: Urinary tract infections (UTIs) are common in people with spinal cord injury (SCI). UTIs are increasingly difficult to treat due to emergence of multi-resistant organisms. Probiotics are efficacious in preventing UTIs in post-menopausal women. We aimed to determine whether probiotic therapy with Lactobacillus reuteri RC-14+Lactobacillus GR-1 (RC14-GR1) and/or Lactobacillus rhamnosus GG+Bifidobacterium BB-12 (LGG-BB12) are effective in preventing UTI in people with SCI. Setting: Spinal units in New South Wales, Australia with their rural affiliations. Methods: We recruited 207 eligible participants with SCI and stable neurogenic bladder management. They were randomised to one of four arms: RC14-GR1+LGG-BB12, RC14-GR1+placebo, LGG-BB12+ placebo or double placebos for 6 months. Randomisation was stratified by bladder management type and inpatient or outpatient status. The primary outcome was time to occurrence of symptomatic UTI. Results: Analysis was based on intention to treat. Participants randomised to RC14-GR1 had a similar risk of UTI as those not on RC14-GR1 (HR 0.67; 95% CI: 0.39–1.18; P = 0.17) after allowing for pre-specified covariates. Participants randomised to LGG-BB12 also had a similar risk of UTI as those not on LGG-BB12 (HR 1.29; 95% CI: 0.74–2.25; P = 0.37). Multivariable post hoc survival analysis for RC14-GR1 only vs. the other three groups showed a potential protective effect (HR 0.46; 95% CI: 0.21–0.99; P = 0.03), but this result would need to be confirmed before clinical application. Conclusion: In this RCT, there was no effect of RC14-GR1 or LGG-BB12 in preventing UTI in people with SCI. Published version National Health and Medical Research Council (NHMRC) is the organisation responsible for funding the supply of probiotics and matching placebo selected by the researchers for this study and budgeted within the NHMRC grant. CHR Hansen, Horsholm, Denmark has been paid commercial rates for providing the intervention product and placebo. The company had no input into the design of the trial.

© 2016 The Speech Pathology Association of Australia Limited Published by Taylor & Francis. Purpose: This study investigated the relationship between children’s language difficulties and health care costs using the 2004–2012 Longitudinal Study of Australian Children (LSAC). Method: Language difficulties were defined as scores ≤1.25SD below the standardised mean on measures of directly assessed receptive vocabulary (4–9 years) and teacher-reported language and literacy (10–13 years). Participant data were individually linked to administrative data, which were sourced from Australia’s universal subsidised healthcare scheme (Medicare). Result: It was found that healthcare costs over each 2-year age band were higher for children with language difficulties than without in the 4–5-year-age bracket (mean difference = AU$357, 95%CI $59, $659), in the 6–7-year-age bracket (mean difference = AU$602, 95%CI $136, $1068) and in the 10–11-year-age bracket (mean difference = AU$504, 95%CI $153, $854). Out-of-pocket costs, that is the portion of healthcare costs paid for by the family, were also higher for children with than without language difficulties in the 4–5-year-age bracket (mean difference = AU$123, 95%CI $46, $199), in the 6–7-year-age bracket (mean difference = AU$176, 95%CI $74,278) and in the 10–11-year-age bracket (mean difference = AU$79, 95%CI $6, $152). Medical services accounted for 97% of total healthcare cost differences. Conclusion: Overall the findings from this study suggest that language difficulties are associated with increased healthcare costs at key developmental milestones, notably early childhood and as a child approaches the teenage years.

STUDY DESIGN A prospective, single-arm, pre-postintervention study. OBJECTIVE The aim of this study was to test the preliminary effectiveness of a patient-led goal-setting intervention on improving disability and pain in chronic low back pain. SUMMARY OF BACKGROUND DATA An effective intervention for the treatment of chronic low back pain remains elusive despite extensive research into the area.An intervention using patient-centered goal setting to drive intervention strategies and encourage self-management for patients suffering chronic low back was developed. METHODS A single group longitudinal cohort pilot study was conducted. Twenty participants (male = nine) experiencing chronic low back pain were involved in a patient-led goal-setting intervention, facilitated by a physiotherapist over a 2-month period with two monthly follow-up sessions after treatment conclusion. Participants, guided by the therapist, identified problem areas of personal importance, defined goals, and developed evidence-based strategies to achieve the goals. Participants implemented the strategies independently between sessions. Primary outcome measures of disability and pain intensity were measured at baseline, 2, and 4 months. Secondary measures of quality of life, stress and anxiety, self-efficacy, and fear of movement were also taken. RESULTS Significant improvements (repeated analysis of variance P