Protocol for a multisite randomised controlled trial assessing the effect of the Telehealth for Early Developmental Intervention in babies born very preterm (TEDI-Prem) programme on neurodevelopmental outcomes and parent well-being.

Introduction: Infants born very preterm (VPT, <32 weeks' gestation) are at increased risk for neurodevelopmental impairments including motor, cognitive and behavioural delay. Parents of infants born VPT also have poorer mental health outcomes compared with parents of infants born at term.We have developed an intervention programme called TEDI-Prem (Telehealth for Early Developmental Intervention in babies born very preterm) based on previous research.

Aversion to income, ethnic, and geographic related health inequality: Evidence from Australia.

This study investigated the Australian general public's views on trade-offs between reducing health inequalities and improving total health. It elicited relative equity weights, comparing inequalities in life expectancy at birth across three equity-relevant dimensions: income (comparing poorest versus richest fifth), ethnic (comparing Indigenous versus non-Indigenous), and geographic (comparing rural/remote versus major cities). A benefit trade-off exercise was administered via online survey to a sample of Australian adults (n = 3105) using quota sampling to ensure population representativeness across key demographic variables (age, gender, state of residence, household income and education level).

What are the priorities of consumers and carers regarding measurement for evaluation in mental healthcare? Results from a Q-methodology study.

Background: The purpose of this study was to identify and describe common views of people with lived experience of mental health challenges - consumers and carers, families and supporters - of what they consider the most important measures to include in health economic evaluations which assess the incremental value of competing options in mental health care.

Methods: Participants (n = 111) were people living in the state of Victoria, Australia, who identified as consumers of mental healthcare (n = 38); carers, family members and/or supporters (n = 43); or both (n = 30). Factor analysis based on Q-Methodology was used to identify clusters of people who hold similar viewpoints.

Goal-Directed Rehabilitation Versus Standard Care for Individuals with Hereditary Cerebellar Ataxia: A Multicenter, Single-Blind, Randomized Controlled Superiority Trial.

Objective: Rehabilitation is thought to reduce ataxia severity in individuals with hereditary cerebellar ataxia (HCA). This multicenter, randomized controlled superiority trial aimed to examine the efficacy of a 30-week goal-directed rehabilitation program compared with 30 weeks of standard care on function, ataxia, health-related quality of life, and balance in individuals with an HCA.

Methods: Individuals with an autosomal dominant or recessive ataxia (aged ≥15 years) were enrolled at 5 sites in Australia.

How should we fund integrated primary care for children in Australia? A resource allocation study.

Objectives: Integrated primary care provides health and social care services to intervene early and support children and families. Funding of integrated care is a barrier to care provision, but evidence is limited for which funding models are most appropriate. Our study aimed to provide expert judgement on what funding model, or mix of models, are most likely effective for integrating primary care for families with children aged 0-12 years in Australia.

Deriving the PedsUtil health state classification system to measure health utilities for pediatric populations based on the PedsQL: a confirmatory factor analysis.

Background: An important methodological challenge in conducting pediatric economic evaluations is estimating the preference-based health-related quality of life (HRQoL) of children. Current methods are highly variable and there is no single instrument available to value HRQoL consistently across multiple pediatric age groups. The Pediatric Quality of Life Inventory (PedsQL) is a non-preference-based generic HRQoL instrument validated for children 2-18 years, but it cannot be directly used in economic evaluations.

Selecting PedsQL items to derive the PedsUtil health state classification system to measure health utilities in children.

Background: There is a lack of preference-based health-related quality of life (HRQoL) measures that consistently value health across a full range of child age groups. The PedsQL is a generic HRQoL instrument validated for children 2-18 years, but it is not preference-based. The objective of this study was to derive the PedsUtil health state classification system from the PedsQL as a basis for a preference-based HRQoL measure for children.

Comparing Generic Paediatric Health-Related Quality-of-Life Instruments: A Dimensionality Assessment Using Factor Analysis.

Background: Widely used generic instruments to measure paediatric health-related quality of life (HRQoL) include the EQ-5D-Y-5L, Child Health Utility 9 Dimension (CHU-9D), Paediatric Quality of Life Inventory (PedsQL) and Health Utilities Index (HUI). There are similarities and differences in the content of these instruments, but there is little empirical evidence on how the items they contain relate to each other, and to an overarching model of HRQoL derived from their content.

Objective: This study aimed to explore the dimensionality of the instruments using exploratory factor analysis (EFA).

A Comparison of the Psychometric Properties of the EQ-5D-Y-3L and EQ-5D-Y-5L Using Paediatric Multi-Instrument Comparison (P-MIC) Study Data.

Background: The EQ-5D-Y is a generic health-related quality of life (HRQoL) measure suitable for children and adolescents. There are two versions of the instrument, which differ in response levels: the three- (Y-3L) and five-level (Y-5L) versions. The Y-5L has been developed to improve the psychometric performance of the Y-3L.

The performance of the EQ-HWB-S as a measure of quality-of-life of caregivers in families that have experienced adverse events.

Purpose: The recently developed EQ Health and Wellbeing Instrument (EQ-HWB) is a broad, generic measure of quality-of-life designed to be suitable for caregivers. The aim of this study was to investigate performance and validity of the 9-item version (EQ-HWB-S) for caregivers where families had experienced adverse-life-events.

Methods: Using survey data from caregivers of children aged 0-8 years attending a community-health centre in 2021-2022, the general performance, feasibility, convergent and known-group validity, responsiveness-to-change, and test-retest reliability of the EQ-HWB-S was assessed.

A happiness approach to valuing health states for children.

Preference weights are widely used to score generic health states into utility indexes for estimation of quality adjusted life years (QALYs) and to aid health care funding decisions. To date, health state utilities are predominantly derived using stated preference methods based on decision utility. This paper tests an alternative and generates preference weights using experienced utility for children based on the Child Health Utility 9D (CHU9D) descriptive system.

Exploring the Use of Pictorial Approaches in the Development of Paediatric Patient-Reported Outcome Instruments: A Systematic Review.

Introduction: Children may find self-reporting health-related quality of life (HRQoL) using patient-reported outcome measures (PROMs) presented in text-based formats difficult, particularly younger children and children with developmental delays or chronic illness. In such cases, pictorial PROMs (where pictorial representations are used alongside or to replace text) may offer a valid alternative.

Aim: This systematic literature review focused on identifying and describing paediatric PROMs that incorporate pictorial approaches, providing children with more effective means to express their HRQoL.

Comparing the Psychometric Performance of Generic Paediatric Health-Related Quality of Life Instruments in Children and Adolescents with ADHD, Anxiety and/or Depression.

Objective: The aim of this study was to examine the validity, reliability and responsiveness of common generic paediatric health-related quality of life (HRQoL) instruments in children and adolescents with mental health challenges.

Methods: Participants were a subset of the Australian Paediatric Multi-Instrument Comparison (P-MIC) study and comprised 1013 children aged 4-18 years with attention-deficit/hyperactivity disorder (ADHD) (n = 533), or anxiety and/or depression (n = 480). Participants completed an online survey including a range of generic paediatric HRQoL instruments (PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, CHU9D) and mental health symptom measures (SDQ, SWAN, RCADS-25).

Psychometric Properties of Child Health Utility 9D (CHU9D) Proxy Version Administered to Parents and Caregivers of Children Aged 2-4 Years Compared with Pediatric Quality of Life Inventory™ (PedsQL).

Objective: This study examines the psychometric properties of the Child Health Utility 9D (CHU9D) proxy version administered to parents/caregivers of 2-4-year-old Australian children compared with Pediatric Quality of Life Inventory™ version 4.0 (PedsQL).

Methods: Data collected in 2021/2022 from parents/caregivers of 2-4-year-olds from the Australian pediatric multi-instrument comparison study were used.

An Investigation of Inter-Rater and Intra-Proxy Agreement in Measuring Quality of Life of Children in the Community Using the EQ-5D-Y-3L.

Background: Self-reporting of health-related quality of life (HRQoL) in children is not always feasible. To date, proxy perspectives (Proxy versions 1 and 2) using the EQ-5D-Y-3L have not been explored for its impact on agreement with child self-report. Proxy version 1 requires the proxy to consider their own view of the child's HRQoL (proxy-proxy), while with Proxy version 2, the proxy is asked to respond as they believe their child would self-report their HRQoL (proxy-child).

An economic evaluation of point-of-care ultrasound for children presenting to the emergency department with suspected septic arthritis of the hip.

Objective Diagnosing septic arthritis of the hip in children is time-sensitive, with earlier diagnosis improving outcomes. Point-of-care ultrasound (POCUS) requires specialised training and resources in emergency departments (ED) to potentially lower costs through reducing patient time in ED. We aimed to compare the costs of using POCUS for suspected septic arthritis of the hip to current practice.

The Validity of the EuroQol Health and Wellbeing Short Version (EQ-HWB-S) Instrument in Parents of Children With and Without Health Conditions.

Background:  The EuroQol Health and Wellbeing Short Version (EQ-HWB-S) instrument has been developed to measure the health and wellbeing of care-recipients and their caregivers for use in economic evaluation.The EQ-HWB-S has nine items, and pilot UK preference weights have now been developed.

Objective: We aimed to investigate the validity of the instrument in parents of children with and without health conditions.

Psychometric Performance Comparison of the Adapted versus Original Versions of the EQ-5D-Y-3L and -Y-5L in Proxy Respondents for 2- to 4-Year-Olds.

Introduction: Few preference-weighted instruments are available to measure health-related quality of life in young children (2-4 years of age). The EQ-5D-Y-3L and EQ-5D-Y-5L were recently modified for this purpose.

Objective: The aim of this study was to test the psychometric properties of these adapted versions for use with parent proxies of children aged 2-4 years and to compare their performance with the original versions.

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.

The cost of proband and trio exome and genome analysis in rare disease: A micro-costing study.

Purpose: Rare disease genomic testing is a complex process involving various resources. Accurate resource estimation is required for informed prioritization and reimbursement decisions. This study aims to analyze the costs and cost drivers of clinical genomic testing.

Evaluating the cost-effectiveness of [F]FDG-PET/CT for investigation of persistent or recurrent neutropenic fever in high-risk haematology patients.

Background: A recent randomised trial demonstrated [F]fluorodeoxyglucose positron-emission tomography in combination with low-dose CT (FDG-PET/CT), compared to standard of care computed tomography (CT) imaging, positively impacted antimicrobial management and outcomes of acute leukaemia and haematopoietic stem cell transplant recipients with persistent and recurrent neutropenic fever. We conducted an economic evaluation from a healthcare perspective alongside the clinical trial.

Methods: Unit costs in Australian dollars were applied to all resources used (antimicrobials, diagnostic tests, ICU and hospital bed days).

Cost-effectiveness analysis of probiotic peanut oral immunotherapy (PPOIT) versus placebo in Australian children with peanut allergy alongside a randomised trial.

Objective: To compared the cost-effectiveness of coadministration of a probiotic adjuvant with peanut oral immunotherapy (PPOIT) with placebo (no treatment) in children with peanut allergy.

Design: Prospectively planned cost-effectiveness analysis alongside a randomised control trial.

Setting: The Royal Children's Hospital, Melbourne, Australia.

Development of a microcosting protocol to determine the economic cost of diagnostic genomic testing for rare diseases in Australia.

Introduction: Genomic testing is a relatively new, disruptive and complex health technology with multiple clinical applications in rare diseases, cancer and infection control. Genomic testing is increasingly being implemented into clinical practice, following regulatory approval, funding and adoption in models of care, particularly in the area of rare disease diagnosis. A significant barrier to the adoption and implementation of genomic testing is funding.

Comparative Psychometric Performance of Common Generic Paediatric Health-Related Quality of Life Instrument Descriptive Systems: Results from the Australian Paediatric Multi-Instrument Comparison Study.

Objective: The aim of this study was to compare the psychometric performance of common generic paediatric health-related quality-of-life instrument descriptive systems (PedsQL generic core 4.0, EQ-5D-Y-3L, EQ-5D-Y-5L, Child Health Utility 9D [CHU9D], Assessment of Quality of Life 6D [AQoL-6D], and Health Utilities Index Mark 3 [HUI3]) by child age, report type, and health status.

Methods: Data for children aged 5-18 years were from the Australian Paediatric Multi-Instrument Comparison study.