Impact of Structural Differences on the Modeled Cost-Effectiveness of Noninvasive Prenatal Testing.

BACKGROUND: Noninvasive prenatal testing (NIPT) was developed to improve the accuracy of prenatal screening to detect chromosomal abnormalities. Published economic analyses have yielded different incremental cost-effective ratios (ICERs), leading to conclusions of NIPT being dominant, cost-effective, and cost-ineffective. These analyses have used different model structures, and the extent to which these structural variations have contributed to differences in ICERs is unclear. AIM: To assess the impact of different model structures on the cost-effectiveness of NIPT for the detection of trisomy 21 (T21; Down syndrome). METHODS: A systematic review identified economic models comparing NIPT to conventional screening. The key variations in identified model structures were the number of health states and modeling approach. New models with different structures were developed in TreeAge and populated with consistent parameters to enable a comparison of the impact of selected structural variations on results. RESULTS: The review identified 34 economic models. Based on these findings, demonstration models were developed: 1) a decision tree with 3 health states, 2) a decision tree with 5 health states, 3) a microsimulation with 3 health states, and 4) a microsimulation with 5 health states. The base-case ICER from each model was 1) USD$34,474 (2023)/quality-adjusted life-year (QALY), 2) USD$14,990 (2023)/QALY, (3) USD$54,983 (2023)/QALY, and (4) NIPT was dominated. CONCLUSION: Model-structuring choices can have a large impact on the ICER and conclusions regarding cost-effectiveness, which may inadvertently affect policy decisions to support or not support funding for NIPT. The use of reference models could improve international consistency in health policy decision making for prenatal screening. HIGHLIGHTS: NIPT is a clinical area in which a variety of modeling approaches have been published, with wide variation in reported cost-effectiveness.This study shows that when broader contextual factors are held constant, varying the model structure yields results that range from NIPT being less effective and more expensive than conventional screening (i.e., NIPT was dominated) through to NIPT being more effective and more expensive than conventional screening with an ICER of USD$54,983 (2023)/QALY.Model-structuring choices may inadvertently affect policy decisions to support or not support funding of NIPT. Reference models could improve international consistency in health policy decision making for prenatal screening.

What attributes of digital devices are important to clinicians in rehabilitation? A cross-cultural best-worst scaling study.

BACKGROUND: Digital interventions are becoming increasingly popular in rehabilitation. Understanding of device features which impact clinician adoption and satisfaction is limited. Research in the field should be conducted across diverse settings to ensure digital interventions do not exacerbate healthcare inequities. OBJECTIVE: This study aimed to understand rehabilitation clinicians' preferences regarding device attributes and included a cross-cultural comparison. MATERIALS AND METHODS: Choice experiment methodology (best-worst scaling) was used to survey rehabilitation clinicians across Australia and Brazil. Participants completed 10 best-worst questions, choosing the most and least important device attributes from subsets of 31 attributes in a partially balanced block design. Results were analysed using multinomial models by country and latent class. Attribute preference scores (PS) were scaled to 0-100 (least to most important). RESULTS: A total of 122 clinicians from Brazil and 104 clinicians from Australia completed the survey. Most respondents were physiotherapists (83%) working with neurological populations (51%) in the private/self-employed sector (51%) who had experience using rehabilitation devices (87%). Despite preference heterogeneity across country and work sector (public/not-for-profit versus private/self-employed/other), clinicians consistently prioritised patient outcomes (PS 100.0, 95%CI: 86.2-100.0), patient engagement (PS 93.9, 95%CI: 80.6-94.2), usability (PS 81.3, 95%CI: 68.8-82.5), research evidence (PS 80.4, 95%CI: 68.1-81.7) and risk (PS 75.7, 95%CI: 63.8-77.3). In Australia, clinicians favoured device attributes which facilitate increased therapy dosage (PS 79.2, 95%CI: 62.6-81.1) and encourage patient independent practice (PS 66.8, 95%CI: 52.0-69.2). In Brazil, clinicians preferred attributes enabling device use for providing clinical data (PS 67.6, 95%CI: 51.8-70.9) and conducting clinical assessments (PS 65.6, 95%CI: 50.2-68.8). CONCLUSION: Clinicians prioritise patients' needs and practical application over technical aspects of digital rehabilitation devices. Contextual factors shape clinician preferences rather than individual clinician characteristics. Future device design and research should consider preferences and influences, involving diverse stakeholders to account for context-driven variations across cultures and healthcare settings.

Protocol for the Australian Type 1 Diabetes National Screening Pilot: Assessing the feasibility and acceptability of three general population screening models in children.

AIM: One third of Australian children diagnosed with type 1 diabetes present with life-threatening diabetic ketoacidosis (DKA) at diagnosis. Screening for early-stage, presymptomatic type 1 diabetes, with ongoing follow-up, can substantially reduce this risk (<5% risk). Several screening models are being trialled internationally, without consensus on the optimal approach. This pilot study aims to assess three models for a routine, population-wide screening programme in Australia. METHODS: An implementation science-guided pilot study to evaluate the feasibility, acceptability and costs of three screening models in children will be conducted between July 2022 and June 2024. These models are as follows: (1) Genetic risk-stratified screening using newborn heel prick dried bloodspots, followed by autoantibody testing from 11 months of age; (2) genetic risk-stratified screening of infant (6-12 months) saliva followed by autoantibody testing from 10 months of age; and (3) autoantibody screening using capillary dried bloodspots collected from children aged 2, 6 or 10 years. Cohorts for each model will be recruited from targeted geographic areas across Australia involving ≥2 states per cohort, with a recruitment target of up to 3000 children per cohort (total up to 9000 children). The primary outcome is screening uptake for each cohort. Secondary outcomes include programme feasibility, costs, parental anxiety, risk perception, satisfaction, well-being and quality of life, and health professional attitudes and satisfaction. CONCLUSIONS: This pilot is the first direct comparison of three screening implementation models for general population screening. Findings will provide evidence to inform a potential national screening programme for Australian children. TRIAL REGISTRATION: ACTRN12622000381785.

Bringing optimised COVID-19 vaccine schedules to immunocompromised populations (BOOST-IC): study protocol for an adaptive randomised controlled clinical trial.

BACKGROUND: Immunocompromised hosts (ICH) experience more breakthrough infections and worse clinical outcomes following infection with COVID-19 than immunocompetent people. Prophylactic monoclonal antibody therapies can be challenging to access, and escape variants emerge rapidly. Immunity conferred through vaccination remains a central prevention strategy for COVID-19. COVID-19 vaccines do not elicit optimal immunity in ICH but boosting, through additional doses of vaccine improves humoral and cellular immune responses. This trial aims to assess the immunogenicity and safety of different COVID-19 vaccine booster strategies against SARS-CoV-2 for ICH in Australia. METHODS: Bringing optimised COVID-19 vaccine schedules to immunocompromised populations (BOOST-IC) is an adaptive randomised trial of one or two additional doses of COVID-19 vaccines 3 months apart in people living with HIV, solid organ transplant (SOT) recipients, or those who have haematological malignancies (chronic lymphocytic leukaemia, non-Hodgkin lymphoma or multiple myeloma). Key eligibility criteria include having received 3 to 7 doses of Australian Therapeutic Goods Administration (TGA)-approved COVID-19 vaccines at least 3 months earlier, and having not received SARS-CoV-2-specific monoclonal antibodies in the 3 months prior to receiving the study vaccine. The primary outcome is the geometric mean concentration of anti-spike SARS-CoV-2 immunoglobulin G (IgG) 28 days after the final dose of the study vaccine. Key secondary outcomes include anti-spike SARS-CoV-2 IgG titres and the proportion of people seroconverting 6 and 12 months after study vaccines, local and systemic reactions in the 7 days after vaccination, adverse events of special interest, COVID-19 infection, mortality and quality of life. DISCUSSION: This study will enhance the understanding of COVID-19 vaccine responses in ICH, and enable the development of safe, and optimised vaccine schedules in people with HIV, SOT, or haematological malignancy. TRIAL REGISTRATION: ClinicalTrials.gov NCT05556720. Registered on 23rd August 2022.

Costs and healthcare use of patients with chronic kidney disease in the Northern Territory, Australia.

BACKGROUND: The burden of chronic kidney disease (CKD) is high in the Northern Territory (NT), Australia. This study aims to describe the healthcare use and associated costs of people at risk of CKD (e.g. acute kidney injury, diabetes, hypertension, and cardiovascular disease) or living with CKD in the NT, from a healthcare funder perspective. METHODS: We included a retrospective cohort of patients at risk of, or living with CKD, on 1 January 2017. Patients on kidney replacement therapy were excluded from the study. Data from the Territory Kidney Care database, encompassing patients from public hospitals and primary health care services across the NT was used to conduct costing. Annual healthcare costs, including hospital, primary health care, medication, and investigation costs were described over a one-year follow-up period. Factors associated with high total annual healthcare costs were identified with a cost prediction model. RESULTS: Among 37,398 patients included in this study, 23,419 had a risk factor for CKD while 13,979 had CKD (stages 1 to 5, not on kidney replacement therapy). The overall mean (± SD) age was 45 years (± 17), and a large proportion of the study cohort were First Nations people (68%). Common comorbidities in the overall cohort included diabetes (36%), hypertension (32%), and coronary artery disease (11%). Annual healthcare cost was lowest in those at risk of CKD (AUD$7,958 per person) and highest in those with CKD stage 5 (AUD$67,117 per person). Inpatient care contributed to the majority (76%) of all healthcare costs. Predictors of increased total annual healthcare cost included more advanced stages of CKD, and the presence of comorbidities. In CKD stage 5, the additional cost per person per year was + $53,634 (95%CI 32,769 to 89,482, p < 0.001) compared to people in the at risk group without CKD. CONCLUSION: The total healthcare costs in advanced stages of CKD is high, even when patients are not on dialysis. There remains a need for effective primary prevention and early intervention strategies targeting CKD and related chronic conditions.

The randomized controlled trial (NAVKIDS2) of a patient navigator program created for children with chronic kidney disease.

Patient navigators enable adult patients to circumnavigate complex health systems, improving access to health care and outcomes. Here, we aimed to evaluate the effects of a patient navigation program in children with chronic kidney disease (CKD). In this multi-center, randomized controlled trial, we randomly assigned children (aged 0-16 years) with CKD stages 1-5 (including children on dialysis or with kidney transplants), from low socioeconomic status backgrounds, and/or residing in remote areas, to receive patient navigation at randomization (immediate) or at six months (waitlist). The primary outcome was self-rated health (SRH) of participating children at six months, using intention to treat analysis. Secondary outcomes included caregivers' SRH and satisfaction with health care, children's quality of life, hospitalizations, and missed school days. Repeated measures of the primary outcome from baseline to six months were analyzed using cumulative logit mixed effects models. Semi-structured interviews were thematically evaluated. Of 398 screened children, 162 were randomized (80 immediate and 82 waitlist); mean age (standard deviation) of 8.8 (4.8) years with 64.8% male. SRH was not significantly different between the immediate and wait-listed groups at six months. There were also no differences across all secondary outcomes between the two groups. Caregivers' perspectives were reflected in seven themes: easing mental strain, facilitating care coordination, strengthening capacity to provide care, reinforcing care collaborations, alleviating family tensions, inability to build rapport and unnecessary support. Thus, in children with CKD, self-rated health may not improve in response to a navigator program, but caregivers gained skills related to providing and accessing care.

Financial incentives to motivate treatment for hepatitis C with direct acting antivirals among Australian adults (The Methodical evaluation and Optimisation of Targeted IncentiVes for Accessing Treatment of Early-stage hepatitis C: MOTIVATE-C): protocol for a dose-response randomised controlled study.

BACKGROUND: Untreated hepatitis C virus (HCV) infection can result in cirrhosis and hepatocellular cancer. Direct-acting antiviral (DAA) therapies are highly effective and have few side effects compared to older interferon-based therapy. Despite the Australian government providing subsidised and unrestricted access to DAA therapy for chronic HCV infection, uptake has not been sufficient to meet the global target of eliminating HCV as a public health threat by 2030. This study will offer people with HCV financial incentives of varying values in order to evaluate its effect on initiation of DAA therapy in primary care. METHODS: Australian adults (18 years or older) who self-report as having current untreated HCV infection can register to participate via an automated SMS-based system. Following self-screening for eligibility, registrants are offered a financial incentive of randomised value (AUD 0 to 1000) to initiate DAA therapy. Study treatment navigators contact registrants who have consented to be contacted, to complete eligibility assessment, outline the study procedures (including the requirement for participants to consult a primary care provider), obtain consent, and finalise enrolment. Enrolled participants receive their offered incentive on provision of evidence of DAA therapy initiation within 12 weeks of registration (primary endpoint). Balanced randomisation is used across the incentive range until the first analysis, after which response-adaptive randomisation will be used to update the assignment probabilities. For the primary analysis, a Bayesian 4-parameter EMAX model will be used to estimate the dose-response curve and contrast treatment initiation at each incentive value against the control arm (AUD 0). Specified secondary statistical and economic analyses will evaluate the effect of incentives on adherence to DAA therapy, virological response, and cost-effectiveness. DISCUSSION: This project seeks to gain an understanding of the dose-response relationship between incentive value and DAA treatment initiation, while maximising the number of people treated for HCV within fixed budget and time constraints. In doing so, we hope to offer policy-relevant recommendation(s) for the use of financial incentives as a pragmatic, efficient, and cost-effective approach to achieving elimination of HCV from Australia. TRIAL REGISTRATION: ANZCTR (anzctr.org.au), Identifier ACTRN12623000024640, Registered 11 January 2023 ( https://anzctr.org.au/Trial/Registration/TrialReview.aspx?id=384923&isReview=true ).

Putting the Choice in Choice Tasks: Incorporating Preference Elicitation Tasks in Health Preference Research.

Choice-based preference elicitation methods such as the discrete choice experiment (DCE) present hypothetical choices to respondents, with an expectation that these hypothetical choices accurately reflect a 'real world' health-related decision context and that consequently the choice data can be held to be a true representation of the respondent's health or treatment preferences. For this to be the case, careful consideration needs to be given to the format of the choice task in a choice experiment. The overarching aim of this paper is to highlight important aspects to consider when designing and 'setting up' the choice tasks to be presented to respondents in a DCE. This includes the importance of considering the potential impact of format (e.g. choice context, choice set presentation and size) as well as choice set content (e.g. labelled and unlabelled choice sets and inclusion of reference alternatives) and choice questions (stated choice versus additional questions designed to explore complete preference orders) on the preference estimates that are elicited from studies. We endeavoure to instil a holistic approach to choice task design that considers format alongside content, experimental design and analysis.

Improving Diverse and Equitable Involvement of Patients and Caregivers in Research in CKD: Report of a Better Evidence and Translation-Chronic Kidney Disease (BEAT-CKD) Workshop.

Patient and caregiver involvement can enhance the uptake and impact of research, but the involvement of patients and caregivers who are underserved and marginalized is often limited. A better understanding of how to make involvement in research more broadly accessible, supportive, and inclusive for patients with chronic kidney disease (CKD) and caregivers is needed. We conducted a national workshop involving patients, caregivers, clinicians, and researchers from across Australia to identify strategies to increase the diversity of patients and caregivers involved in CKD research. Six themes were identified. Building trust and a sense of safety was considered pivotal to establishing meaningful relationships to support knowledge exchange. Establishing community and connectedness was expected to generate a sense of belonging to motivate involvement. Balancing stakeholder goals, expectations, and responsibilities involved demonstrating commitment and transparency by researchers. Providing adequate resources and support included strategies to minimize the burden of involvement for patients and caregivers. Making research accessible and relatable was about nurturing patient and caregiver interest by appealing to intrinsic motivators. Adapting to patient and caregiver needs and preferences required tailoring the approach for individuals and the target community. Strategies and actions to support these themes may support more diverse and equitable involvement of patients and caregivers in research in CKD.

An Economic Evaluation of a Web-Based Management Support System for Children With Urinary Incontinence: The eADVICE Trial.

PURPOSE: Children who require specialist outpatient care typically wait substantial periods during which their condition may progress, making treatment more difficult and costly. Timely and effective therapy during this period may reduce the need for lengthy specialist care. This study evaluated the cost-effectiveness of an individualized, evidence-informed, web-based program for children with urinary incontinence awaiting a specialist appointment (Electronic Advice and Diagnosis Via the Internet following Computerized Evaluation [eADVICE]) compared to usual care. eADVICE was supervised by a primary physician and delivered by an embodied conversational agent. MATERIALS AND METHODS: A trial-based cost-effectiveness analysis was performed from the perspective of the health care funder as a substudy of eADVICE, a multicenter, waitlist-controlled, randomized trial. Outcomes measures were incremental cost per incremental change in continence status and quality of life on an intention-to-treat basis. Uncertainty was examined using cost-effectiveness planes, scenarios, and 1-way sensitivity analyses. Costs were valued in 2021 Australian dollars. RESULTS: The use of eADVICE was found to be cost saving and beneficial (dominant) over usual care, with a higher proportion of children dry over 14 days at 6 months (risk difference 0.13; 95%CI 0.02-0.23, P = .03) and mean health care costs reduced by $188 (95%CI $61-$315) per participant. CONCLUSIONS: An individualized, evidence-informed, web-based program delivered by an embodied conversational agent is likely cost saving for children with urinary incontinence awaiting a specialist appointment. The potential economic impact of such a program is favorable and substantial, and may be transferable to outpatient clinic settings for other chronic health conditions.

Behavioural 'nudging' interventions to reduce low-value care for low back pain in the emergency department (NUDG-ED): protocol for a 2×2 factorial, before-after, cluster randomised trial.

INTRODUCTION: Opioids and imaging are considered low-value care for most people with low back pain. Yet around one in three people presenting to the emergency department (ED) will receive imaging, and two in three will receive an opioid. NUDG-ED aims to determine the effectiveness of two different behavioural 'nudge' interventions on low-value care for ED patients with low back pain. METHODS AND ANALYSIS: NUDG-ED is a 2×2 factorial, open-label, before-after, cluster randomised controlled trial. The trial includes 8 ED sites in Sydney, Australia. Participants will be ED clinicians who manage back pain, and patients who are 18 years or over presenting to ED with musculoskeletal back pain. EDs will be randomly assigned to receive (i) patient nudges, (ii) clinician nudges, (iii) both interventions or (iv) no nudge control. The primary outcome will be the proportion of encounters in ED for musculoskeletal back pain where a person received a non-indicated lumbar imaging test, an opioid at discharge or both. We will require 2416 encounters over a 9-month study period (3-month before period and 6-month after period) to detect an absolute difference of 10% in use of low-value care due to either nudge, with 80% power, alpha set at 0.05 and assuming an intra-class correlation coefficient of 0.10, and an intraperiod correlation of 0.09. Patient-reported outcome measures will be collected in a subsample of patients (n≥456) 1 week after their initial ED visit. To estimate effects, we will use a multilevel regression model, with a random effect for cluster and patient, a fixed effect indicating the group assignment of each cluster and a fixed effect of time. ETHICS AND DISSEMINATION: This study has ethical approval from Southwestern Sydney Local Health District Human Research Ethics Committee (2023/ETH00472). We will disseminate the results of this trial via media, presenting at conferences and scientific publications. TRIAL REGISTRATION NUMBER: ACTRN12623001000695.

What Matters to Aboriginal and Torres Strait Islander Youth (WM2Y): a study protocol to develop a national youth well-being measure.

INTRODUCTION: Adolescents face challenges associated with unprecedented environmental, social and technological changes. The impacts of colonisation, intergenerational trauma, racism and socioeconomic disadvantage intensify these challenges for many Aboriginal and Torres Strait Islander adolescents. However, Aboriginal and Torres Strait Islander adolescents also have cultural, spiritual, family and community capital that fosters their well-being.To date, little research has focused on understanding and appropriately measuring the well-being of Aboriginal and Torres Strait Islander adolescents, a pivotal factor in informing and guiding programmes and interventions that support them. This study will identify the domains of well-being and develop a new preference-based well-being measure based on the values and preferences of Aboriginal and Torres Strait Islander youth (aged 12-17 years). METHODS AND ANALYSIS: This project will be conducted across three research phases: (1) qualitative exploration of well-being using PhotoYarning and yarns with adult mentors to develop candidate items; (2) Think Aloud study, quantitative survey, psychometric analysis, validity testing of candidate items and finalisation of the descriptive system; and (3) scoring development using a quantitative preference-based approach. A multinomial (conditional) logit framework will be used to analyse responses and generate a scoring algorithm for the new preference-based well-being measure. ETHICS AND DISSEMINATION: Ethics approvals have been obtained from: the Human Research Ethics Committees for each state and territory where data are being collected, the institutions where the research is being conducted and from the relevant Departments of Education. The new well-being measure will have wide applicability and can be used in assessing the effectiveness of programmes and services. This new national measure will ensure benefit and positive impact through the ability to identify and measure the aspects of well-being important to and valued by Aboriginal and Torres Strait Islander youth. Results will be published in international peer-reviewed journals and presented at conferences, and summaries will be provided to the study partner organisations and other relevant organisations.

Pathways between foodways and wellbeing for first nations Australians.

BACKGROUND: Supporting the health and wellbeing of Aboriginal and Torres Strait Islander peoples (hereafter respectfully referred to as First Nations peoples) is a national priority for Australia. Despite immense losses of land, language, and governance caused by the continuing impact of colonisation, First Nations peoples have maintained strong connections with traditional food culture, while also creating new beliefs, preferences, and traditions around food, which together are termed foodways. While foodways are known to support holistic health and wellbeing for First Nations peoples, the pathways via which this occurs have received limited attention. METHODS: Secondary data analysis was conducted on two national qualitative datasets exploring wellbeing, which together included the views of 531 First Nations peoples (aged 12-92). Thematic analysis, guided by an Indigenist research methodology, was conducted to identify the pathways through which foodways impact on and support wellbeing for First Nations peoples. RESULTS AND CONCLUSIONS: Five pathways through which wellbeing is supported via foodways for First Nations peoples were identified as: connecting with others through food; accessing traditional foods; experiencing joy in making and sharing food; sharing information about food and nutrition; and strategies for improving food security. These findings offer constructive, nationally relevant evidence to guide and inform health and nutrition programs and services to harness the strengths and preferences of First Nations peoples to support the health and wellbeing of First Nations peoples more effectively.

Differences in weight status among Australian children and adolescents from priority populations: a longitudinal study.

BACKGROUND AND SIGNIFICANCE: Australia has a high level of cultural and linguistic diversity, including Aboriginal and Torres Strait Islander peoples. Children from specific cultural and ethnic groups may be at greater risk of overweight and obesity and may bear the additional risk of socioeconomic disadvantage. Our aim was to identify differences in body-mass index z-score (zBMI) by: (1) Cultural and ethnic groups and; (2) Socioeconomic position (SEP), during childhood and adolescence. SUBJECTS/METHODS: We used data from the Longitudinal Study of Australian children (n = 9417) aged 2-19 years with 50870 longitudinal measurements of zBMI. Children were classified into 9 cultural and ethnic groups, based on parent and child's country of birth and language spoken at home. These were: (1) English-speaking countries; (2) Middle East & North Africa; (3) East & South-East Asia; (4) South & Central Asia; (5) Europe; (6) Sub-Saharan Africa; (7) Americas; (8) Oceania. A further group (9) was defined as Aboriginal and Torres Strait Islander from self-reported demographic information. Longitudinal cohort analyses in which exposures were cultural and ethnic group and family socioeconomic position, and the outcome was zBMI estimated using multilevel mixed linear regression models. We stratified our analyses over three periods of child development: early childhood (2-5 years); middle childhood (6-11 years); and adolescence (12-19 years). RESULTS: Across all three periods of child development, children from the Middle East and North Africa, the Americas and Oceania were associated with higher zBMI and children from the two Asian groups were associated with lower zBMI, when compared to the referent group (English). zBMI was socioeconomically patterned, with increasingly higher zBMI associated with more socioeconomic disadvantage. CONCLUSIONS: Our findings identified key population groups at higher risk of overweight and obesity in childhood and adolescence. Prevention efforts should prioritize these groups to avoid exacerbating inequalities in healthy weight in childhood.

The RETRIEVE Checklist for Studies Reporting the Elicitation of Stated Preferences for Child Health-Related Quality of Life.

BACKGROUND: Recent systematic reviews show varying methods for eliciting, modelling, and reporting preference-based values for child health-related quality-of-life (HRQoL) outcomes, thus producing value sets with different characteristics. Reporting in many of the reviewed studies was found to be incomplete and inconsistent, making them difficult to assess. Checklists can help to improve standards of reporting; however, existing checklists do not address methodological issues for valuing child HRQoL. Existing checklists also focus on reporting methods and processes used in developing HRQoL values, with less focus on reporting of the values' key characteristics and properties. We aimed to develop a checklist for studies generating values for child HRQoL, including for disease-specific states and value sets for generic child HRQoL instruments. DEVELOPMENT: A conceptual model provided a structure for grouping items into five modules. Potential items were sourced from an adult HRQoL checklist review, with additional items specific to children developed using recent reviews. Checklist items were reduced by eliminating duplication and overlap, then refined for relevance and clarity via an iterative process. Long and short checklist versions were produced for different user needs. The resulting long RETRIEVE contains 83 items, with modules for reporting methods (A-D) and characteristics of values (E), for researchers planning and reporting child health valuation studies. The short RETRIEVE contains 14 items for decision makers or researchers choosing value sets. CONCLUSION: Applying the RETRIEVE checklists to relevant studies suggests feasibility. RETRIEVE has the potential to improve completeness in the reporting of preference-based values for child HRQOL outcomes and to improve assessment of preference-based value sets.

Effect of a coaching intervention to enhance physical activity and prevent falls in community-dwelling people aged 60+ years: a cluster randomised controlled trial.

OBJECTIVES: To evaluate the effect of a coaching intervention compared with control on physical activity and falls rate at 12 months in community-dwelling people aged 60+ years. DESIGN: Cluster randomised controlled trial. SETTING: Community-dwelling older people. PARTICIPANTS: 72 clusters (605 participants): 37 clusters (290 participants) randomised to the intervention and 35 (315 participants) to control. INTERVENTION: Intervention group received written information, fall risk assessment and prevention advice by a physiotherapist, activity tracker and telephone-based coaching from a physiotherapist focused on safe physical activity. Control group received written information and telephone-based dietary coaching. Both groups received up to 19 sessions of telephone coaching over 12 months. OUTCOMES: The co-primary outcomes were device-measured physical activity expressed in counts per minute at 12 months and falls rate over 12 months. Secondary outcomes included the proportion of fallers, device-measured daily steps and moderate-to-vigorous physical activity (MVPA), self-reported hours per week of physical activity, body mass index, eating habits, goal attainment, mobility-related confidence, quality of life, fear of falling, risk-taking behaviour, mood, well-being and disability. RESULTS: The mean age of participants was 74 (SD 8) years, and 70% (n=425) were women. There was no significant effect of the intervention on device-measured physical activity counts per minute (mean difference 5 counts/min/day, 95% CI -21 to 31), or falls at 12 months (0.71 falls/person/year in intervention group and 0.87 falls/person/year in control group; incidence rate ratio 0.86, 95% CI 0.65 to 1.14). The intervention had a positive significant effect on device-measured daily steps and MVPA, and self-reported hours per week of walking, well-being, quality of life, and disability. No significant between-group differences were identified in other secondary outcomes. CONCLUSION: A physical activity and fall prevention programme including fall risk assessment and prevention advice, plus telephone-based health coaching, did not lead to significant differences in physical activity counts per minute or falls rate at 12 months. However, this programme improved other physical activity measures (ie, daily steps, MVPA, hours per week of walking), overall well-being, quality of life and disability. TRIAL REGISTRATION NUMBER: ACTRN12615001190594.

Establishing cost-effectiveness threshold in China: a community survey of willingness to pay for a healthylife year.

INTRODUCTION: The willingness to pay per quality-adjusted life year gained (WTP/Q) is commonly used to determine whether an intervention is cost-effective in health technology assessment. This study aimed to evaluate the WTP/Q for different disease scenarios in a Chinese population. METHODS: The study employed a quadruple-bounded dichotomous choice contingent valuation method to estimate the WTP/Q in the general public. The estimation was conducted across chronic, terminal and rare disease scenarios. Face-to-face interviews were conducted in a Chinese general population recruited from Jiangsu province using a convenience sampling method. Interval regression analysis was performed to determine the relationship between respondents' demographic and socioeconomic conditions and WTP/Q. Sensitivity analyses of removing protest responses and open question analyses were conducted. RESULTS: A total of 896 individuals participated in the study. The WTP/Q thresholds were 128 000 Chinese renminbi (RMB) ($36 364) for chronic diseases, 149 500 RMB ($42 472) for rare diseases and 140 800 RMB ($40 000) for terminal diseases, equivalent to 1.76, 2.06 and 1.94 times the gross domestic product per capita in China, respectively. The starting bid value had a positive influence on participants' WTP/Q. Additionally, residing in an urban area (p<0.01), and higher household expenditure (p<0.01), educational attainment (p<0.02) and quality of life (p<0.02) were significantly associated with higher WTP/Q. Sensitivity analyses demonstrated the robustness of the results. CONCLUSION: This study implies that tailored or varied rather than a single cost-effectiveness threshold could better reflect community preferences for the value of a healthy year. Our estimates hold significance in informing reimbursement decision-making in health technology assessment in China.

Making recommendations to subsidize new health technologies in Australia: A qualitative study of decision-makers' perspectives on committee processes.

OBJECTIVES: To explore experiences of, and perspectives on, health technology assessment (HTA) processes used to produce recommendations about subsidizing new medicines, and medical technologies in Australia, from the perspectives of those experienced in these processes. METHODS: Semi-structured interviews with a diverse group of 18 informants currently or previously members of the Pharmaceutical Benefits Advisory Committee (PBAC) or the Medical Services Advisory Committee (MSAC). Participants were interviewed September 2021-February 2022. Transcripts were analyzed using reflexive thematic analysis. RESULTS: 3 major themes were identified: contrasting technical and decision-making stages, resisting reductionist approaches, and navigating decision-making trade-offs. Participants discussed the complexities of the evaluative HTA process, especially when considering uncertainty in the evidence. As part of the current process, a deliberative decision-making stage was considered essential, allowing a flexible approach to decision making to consider factors beyond strength and quality of quantifiable data in the technical evaluation. Participants acknowledged these less-quantifiable factors were sometimes considered implicitly or were difficult to describe and this, paired with commercial in confidence requirements, presented challenges with respect to the desire to increase transparency. CONCLUSION (S): As HTA processes for new medicines and medical technologies in Australia continue to be reviewed, the balance between retaining flexibility during deliberation, confidentiality for sponsors and the public's desire for greater transparency may be a fruitful area for continuing research.

Women's Preferences for Hypofractionated Radiation Therapy for Treatment of Early-Stage Breast Cancer: A Discrete Choice Experiment.

PURPOSE: The primary treatment recommended for early-stage breast cancer is breast conserving surgery followed by external beam radiation therapy of the whole breast. Previously, radiation therapy for early-stage breast cancer was given using more fractions over longer durations. Guidelines support treatments with fewer fractions over a shorter time (hypofractionated radiation therapy). This study aimed to understand women's preferences for different features of treatments for early-stage breast cancer. METHODS AND MATERIALS: A discrete choice experiment with 12 choice tasks was conducted, describing the treatments by extent of surgery, duration of radiation treatment, need to relocate for treatment, local side effects, changes in breast appearance, costs, and difficulty with daily activities during and after treatment. Participants were women with breast cancer and from the general population. Mixed logit analyses were conducted and trade-offs between attributes estimated. RESULTS: Four hundred twenty respondents completed the discrete choice experiment. The relative importance of attributes varied by respondent characteristics; the most influential attribute for younger women was type of surgery (breast conserving surgery). Type of surgery did not influence older women's preferences. Shorter treatment duration, avoiding relocation, fewer local side effects, and less difficulty with daily activities all positively influenced treatment preference. Younger women were willing to accept 32 to 40 days of radiation treatment before a treatment that included mastectomy was potentially acceptable. CONCLUSIONS: Attributes of treatment such as duration, need for relocation, side effects, and effects on normal daily activities during and after treatment significantly influenced women's preference for treatment, including surgery. Our findings have the potential for real impact for patients and services including supporting one-on-one clinical discussions, supporting program and patient resource development, and informing service funding, organization, and delivery.