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Dietary education is a core component of cardiac rehabilitation (CR). It is unknown how or what dietary education is delivered across the UK. We aimed to characterise practitioners who deliver dietary education in UK CR and determine the format and content of the education sessions. A fifty-four-item survey was approved by the British Association for Cardiovascular Prevention and Rehabilitation (BACPR) committee and circulated between July and October 2021 via two emails to the BACPR mailing list and on social media. Practitioners providing dietary education within CR programmes were eligible to respond. Survey questions encompassed: practitioner job title and qualifications, resources, and the format, content and individual tailoring of diet education. Forty-nine different centres responded. Nurses (65·1 %) and dietitians (55·3 %) frequently provided dietary education. Practitioners had no nutrition-related qualifications in 46·9 % of services. Most services used credible resources to support their education, and 24·5 % used BACPR core competencies. CR programmes were mostly community based (40·8 %), lasting 8 weeks (range: 2-25) and included two (range: 1-7) diet sessions. Dietary history was assessed at the start (79·6 %) and followed up (83·7 %) by most centres; barriers to completing assessment were insufficient time, staffing or other priorities. Services mainly focused on the Mediterranean diet while topics such as malnutrition and protein intake were lower priority topics. Service improvement should focus on increasing qualifications of practitioners, standardisation of dietary assessment and improvement in protein and malnutrition screening and assessment.

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: To summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value. METHOD: Economic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations. RESULTS: Eight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations. CONCLUSION: The methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.

Background: Advanced dementia is a life-limiting illness that requires a palliative approach to care. Decline in eating/drinking represents a milestone in progression that warrants decision-making and planning of care. In long-term care (LTC), this is best conducted via family case conferences. Aim: To explore decision-making and planning regarding eating/drinking-related care in case conferences for persons with advanced dementia in LTC to inform future practice. Methods: A qualitative approach was taken, using observation of audio-recorded case conferences. Case conferences were conducted in 6 LTC facilities within the intervention arm of a cluster randomized controlled trial. Participants were LTC personnel, doctors, and families of persons with advanced dementia. Content was analysed for eating/drinking-related goals of care and the degree to which decision-making was shared. Findings: Thirty-two case conferences considered eating/drinking-related care. The goals included nutrition, hydration, weight gain and maintenance, food enjoyment, social interaction, and independence. Key considerations included secondary health issues impacting comfort, and tensions between food enjoyment versus nutrition and risk of aspiration. While families contributed essential information about eating/drinking history, sometimes decision-making was dominated by professionals and overlooked overall goals of care. Discussion: Shared decision-making regarding eating/drinking-related care for persons with advanced dementia in LTC should start with consensus on overall goals of care and include contributions to quality of life as well as risks. Family involvement should be supported beyond information-giving. Conclusion: Future studies are needed to identify the most sensitive and understandable ways for families of discussing eating/drinking-related decline in dementia.

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

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 <83 (Mini-Mental State Examination >14 = main trial inclusion criteria). METHODS: Of 221 participants, 148 had an ACE-R <83 and were included in this study. Sixteen LTC residences (clusters) were randomized to receive either the Sunbeam program or usual care. The Sunbeam program involved two 1-hour sessions/week of tailored and progressive resistance and balance training for 25 weeks followed by a maintenance program (two 30-min sessions/week of nonprogressive exercise for 6 months). Assessments were conducted at baseline, 6 months, and 12 months. Falls were recorded using routinely collected data from the LTC incident management systems. RESULTS: Rate of falls (50%) and risk of falls (31%), multiple falls (40%), and injurious falls (44%) were reduced in the intervention group. The intervention group had significantly better balance (static and dynamic) and sit-to-stand ability when compared with the control group at 6 months and significantly better dynamic balance at 12 months. There were no serious adverse events. CONCLUSIONS AND IMPLICATIONS: The Sunbeam Program significantly reduced falls and improved physical performance in cognitively impaired LTC residents. This is a novel and important finding, as many previous studies have excluded people with cognitive impairment/dementia and inconsistent findings h

This 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.

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 di

INTRODUCTION: The 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 ANALYSIS: The 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 DISSEMINATION: Ethical 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 NUMBER: ACTRN12618000160235.

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.

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.

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.

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.

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 a

BACKGROUND: The 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. OBJECTIVE: This 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. METHODS: Quality-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. RESULTS: Mean 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. CONCLUSION: This 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.

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.

OBJECTIVES: Most breast cancer cases in Ghana occur in premenopausal and perimenopausal (PPM) women. This study evaluated the cost-effectiveness of tamoxifen compared with no tamoxifen for the adjuvant treatment of hormone receptor-positive (HR+) early breast cancer (EBC) among PPM Ghanaian women. METHODS: A Markov model was constructed to synthesize data on the effectiveness, costs, and health benefits of tamoxifen. Effectiveness and utility data were sourced from a literature review. Resource use and healthcare costs were estimated from Ghanaian sources. The evaluation was conducted in 2017 from the perspective of the health system over a 15-year time horizon. The financial impact of funding tamoxifen on Ghana's National Health Insurance Scheme (NHIS) was also estimated. RESULTS: Adjuvant tamoxifen treatment for women with HR+ EBC was more effective and more costly than no-tamoxifen therapy. The incremental benefit and costs were estimated to be 1.38 quality-adjusted life-years gained and Ghana cedis (GHC) 2338 ($520), respectively. The incremental cost-effectiveness ratio was estimated to be GHC 1694 ($376). The model was sensitive to the cost of tamoxifen and utility values. The cost of tamoxifen for the treatment of HR+ EBC represents less than 0.01% GHC 96 960 ($21 547) of the current NHIS total claims expenditure. CONCLUSIONS: Tamoxifen provides additional benefits to PPM Ghanaian women with HR+ EBC and is cost-effective compared with no tamoxifen. These results support the public funding of tamoxifen under the NHIS and provide Ghanaian policy makers with vital information for future budgetary planning.

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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).

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.

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.

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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.

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

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

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.