The Clinician-reported Genetic Testing Utility Index (C-GUIDE) for Prenatal Care: Initial evidence of content and construct validity.

Purpose: To develop and assess the face and construct validity of the Clinician-reported Genetic Testing Utility Index (C-GUIDE) for genetic testing in prenatal care.

Methods: After a literature review and consultation with clinical experts, a preliminary draft of C-GUIDE Prenatal was developed. Its face and content validity were then assessed by 19 prenatal genetics' providers using interviews and surveys.

A methodological guide for implementing and interpreting results of probabilistic analysis.

Introduction: Probabilistic analysis, also referred to as probabilistic sensitivity analysis (PSA), is used extensively in cost-effectiveness evaluations of health technologies. We present methodological guidance for implementing probabilistic analysis and interpreting its results for policy and decision-making.

Methods: We review the methodological issues related to common practices in probabilistic analysis, explore aspects that are currently not widely addressed in the health economics literature, and provide an overview of recent methodological developments.

A Systematic Review of Methods and Practice for Integrating Maternal, Fetal, and Child Health Outcomes, and Family Spillover Effects into Cost-Utility Analyses.

Background: Maternal-perinatal interventions delivered during pregnancy or childbirth have unique characteristics that impact the health-related quality of life (HRQoL) of the mother, fetus, and newborn child. However, maternal-perinatal cost-utility analyses (CUAs) often only consider either maternal or child health outcomes. Challenges include, but are not limited to, measuring fetal, newborn, and infant health outcomes, and assessing their impact on maternal HRQoL.

Paving the path for implementation of clinical genomic sequencing globally: Are we ready?

Despite the emerging evidence in recent years, successful implementation of clinical genomic sequencing (CGS) remains limited and is challenged by a range of barriers. These include a lack of standardized practices, limited economic assessments for specific indications, limited meaningful patient engagement in health policy decision-making, and the associated costs and resource demand for implementation. Although CGS is gradually becoming more available and accessible worldwide, large variations and disparities remain, and reflections on the lessons learned for successful implementation are sparse.

Development of a Value Assessment Framework for Pediatric Health Technologies Using Multicriteria Decision Analysis: Expanding the Value Lens for Funding Decision Making.

Objectives: A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach.

Methods: We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework.

Recommendations for Emerging Good Practice and Future Research in Relation to Family and Caregiver Health Spillovers in Health Economic Evaluations: A Report of the SHEER Task Force.

Background: Omission of family and caregiver health spillovers from the economic evaluation of healthcare interventions remains common practice. When reported, a high degree of methodological inconsistency in incorporating spillovers has been observed.

Aim: To promote emerging good practice, this paper from the Spillovers in Health Economic Evaluation and Research (SHEER) task force aims to provide guidance on the incorporation of family and caregiver health spillovers in cost-effectiveness and cost-utility analysis.

A Systematic Review of Methods Used by Pediatric Cost-Utility Analyses to Include Family Spillover Effects.

Background: A child's health condition affects family members' health and well-being. However, pediatric cost-utility analysis (CUA) commonly ignores these family spillover effects leading to an incomplete understanding of the cost and benefits of a child's health intervention. Methodological challenges exist in assessing, valuing, and incorporating family spillover effects.

Cost-utility analysis of teduglutide compared to standard care in weaning parenteral nutrition support in children with short bowel syndrome.

Background & Aims: A growing proportion of children with short bowel syndrome (SBS) remain dependent on long-term parenteral nutrition (PN). Teduglutide offers the potential for more children to decrease PN support and achieve enteral autonomy (EA), but at a significant expense. This study aims to assess the incremental costs of teduglutide plus standard of care compared to standard of care alone in weaning PN support per quality-adjusted life year (QALY) gained in children with SBS.

Why it is so challenging to perform economic evaluations of interventions in autism and what to do about it.

Economic evaluation is used to determine the optimal provision of services and programs under budget constraints and to inform public and private payer funding decisions. To maximize value-for-money in the design and delivery of programs and services for persons with autism spectrum disorder (ASD), it's essential to generate high-quality economic evidence to inform budget allocation. There is a paucity however, of economic evaluations of interventions for ASD.

Assessing the Performance of the Clinician-reported Genetic Testing Utility InDEx (C-GUIDE): Further Evidence of Inter-rater Reliability.

Purpose: Advanced genomic and genetic testing technologies are quickly diffusing into clinical practice, but standardized approaches to assessing their clinical utility are limited. Previous work developed and generated preliminary evidence of validity for a novel outcome measure, the Clinician-reported Genetic testing Utility InDEx (C-GUIDE). C-GUIDE is a 17-item measure that captures the utility of genetic testing from the providers' perspective.

Protocol for a Prospective, Observational Cost-effectiveness Analysis of Returning Secondary Findings of Genome Sequencing for Unexplained Suspected Genetic Conditions.

Purpose: Although costly, genome-wide sequencing (GWS) detects an extensive range of variants, enhancing our ability to diagnose and assess risk for an increasing number of diseases. In addition to detecting variants related to the indication for testing, GWS can detect secondary variants in BRCA1, BRCA2, and other genes for which early intervention may improve health. As the list of secondary findings grows, there is increased demand for surveillance and management by multiple specialists, adding pressure to constrained health care budgets.

Evaluating the correlations of cost and utility parameters from summary statistics for probabilistic analysis in economic evaluations.

Objectives: The correlations between economic modeling input parameters directly impact the variance and may impact the expected values of model outputs. However, correlation coefficients are not often reported in the literature. We aim to understand the correlations between model inputs for probabilistic analysis from summary statistics.

Translating Precision Health for Pediatrics: A Scoping Review.

Precision health aims to personalize treatment and prevention strategies based on individual genetic differences. While it has significantly improved healthcare for specific patient groups, broader translation faces challenges with evidence development, evidence appraisal, and implementation. These challenges are compounded in child health as existing methods fail to incorporate the physiology and socio-biology unique to childhood.

A Comparison of Preference-Based, Generic and Disease-Specific Health-Related Quality of Life in Pediatric Inflammatory Bowel Disease.

Objective: Generic preference-based HRQOL assessments used expressly for economic evaluation have not been examined in pediatric Crohn's disease (CD) and ulcerative colitis (UC). The objective was to further assess the construct validity of preference-based HRQOL measures in pediatric IBD by comparing the Child Health Utility 9 Dimensions (CHU9D) and Health Utilities Index (HUI) to the disease-specific IMPACT-III and to the generic PedsQL in children with CD and with UC.

Methods: The CHU9D, HUI, IMPACT-III and/or PedsQL were administered to Canadian children aged 6 to 18 years with CD and UC.

A comparison of the Child Health Utility 9D and the Health Utilities Index for estimating health utilities in pediatric inflammatory bowel disease.

Purpose: Health utilities are challenging to ascertain in children and have not been studied in pediatric Crohn's disease (CD) and ulcerative colitis (UC). The objective was to assess discriminative validity by comparing utilities elicited using the Child Health Utility-9 Dimension (CHU9D) to the Health Utilities Index (HUI) across multiple disease activity scales in pediatric UC and CD.

Methods: Preference-based instruments were administered to 188 children with CD and 83 children with UC aged 6 to 18 years.

Infliximab Pricing in International Economic Evaluations in Inflammatory Bowel Disease to Inform Biologic and Biosimilar Access Policies: A Systematic Review.

Unlabelled: Policies mandating the use of lower cost biosimilars in patients with inflammatory bowel disease (IBD) have created concerns for patients who prefer their original biologic. To inform the cost-effectiveness of biosimilar infliximab treatment in IBD by systematically reviewing the effect of infliximab price variation on cost-effectiveness for jurisdictional decision making. MEDLINE, Embase, Healthstar, Allied and Complementary Medicine, Joanna Briggs Institute EBP Database, International Pharmaceutical Abstracts, Health and Psychosocial Instruments, Mental Measurements Yearbook citation databases, PEDE, CEA registry, HTA agencies.

Variable patterns of daily activity participation across settings in autistic youth: A latent profile transition analysis.

What people do or engage in in their daily lives, or daily life participation, is often linked to their state of being happy and healthy, as well as potential for living independently. To date, little research has been conducted on daily activity participation by autistic youth at home, at school or in the community. Learning more about individual differences in participation levels and what might influence them can help to create custom supports for autistic youth and their families.

Cost-effectiveness of Universal School- and Community-Based Vision Testing Strategies to Detect Amblyopia in Children in Ontario, Canada.

Importance: Screening for amblyopia in primary care visits is recommended for young children, yet screening rates are poor. Although the prevalence of amblyopia is low (3%-5%) among young children, universal screening in schools and mandatory optometric examinations may improve vision care, but the cost-effectiveness of these vision testing strategies compared with the standard in primary care is unknown.

Objective: To evaluate the relative cost-effectiveness of universal school screening and mandated optometric examinations compared with standard care vision screening in primary care visits in Toronto, Canada, with the aim of detecting and facilitating treatment of amblyopia and amblyopia risk factors from the Ontario government's perspective.

Measuring the association between behavioural services and outcomes in young children with autism spectrum disorder.

Background: Children with autism spectrum disorder (ASD) receive a wide range of services.

Aims: To examine the association between behavioural services received by children with ASD between ages 2 and 5 years and outcomes during primary school years.

Methods: A total of 414 preschool-aged children diagnosed with ASD were enrolled at five Canadian sites and were assessed within four months of diagnosis (T1), six months later (T2), 12 months later (T3), at school entry (T4), and then annually (T5-T8) to 11 years of age.

Stress, anxiety, depression, and health-related quality of life in caregivers of children with intestinal failure receiving parenteral nutrition: A cross-sectional survey study.

Background: Improved survival rates for children with intestinal failure (IF) have resulted in an increased population of children receiving long-term parenteral nutrition (PN). Our objective was to determine burden on caregivers of children with IF receiving long-term PN.

Methods: We performed a cross-sectional study of caregivers of children with IF receiving long-term PN in our intestinal rehabilitation program.