Special Issue "Assessing Value in Child Health"

A special issue of Children (ISSN 2227-9067). This special issue belongs to the section "Global and Public Health".

Deadline for manuscript submissions: closed (31 March 2021).

Special Issue Editor

Prof. Wendy J. Ungar
E-Mail Website
Guest Editor
1. Technology Assessment at Sick Kids (TASK), Child Health Evaluative Sciences, The Hospital for Sick Children (SickKids) Research Institute, Toronto, M5G 1X8 ON, Canada;
2. Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, M5T 3M6 ON, Canada
Interests: health technology assessment; child health; cost-effectiveness analysis; health-related quality of life; genomics; neurodevelopmental disorders

Special Issue Information

Dear Colleagues,

Assessing value in child health is increasingly important as health care systems face difficult choices with regard to what services and programs to offer. Researchers, practitioners, and funding and policy decision makers appreciate that value extends far beyond assessments of efficacy and safety of new treatments and interventions. Measuring cost-effectiveness, evaluating effects on quality of life for patients and their caregivers, determining preferences for treatments and services from patients, families and members of the public, and consideration of the ethical implications of new complex technologies all contribute to an understanding of value. Conducting research to measure elements of value often necessitates novel methods and approaches. Conducting such research in child health is particularly challenging given the paucity of valid pediatric patient-reported outcome (PRO) instruments, the lack of clinical trial data sources, reliance on proxies, and many other methodologic and data-related constraints. This creates exciting opportunities for methodologic research to develop and validate tools, instruments, statistical approaches, and models to measure elements of value in child health. 

Alongside research to measure value and expand the methodologic toolbox for such assessments in child health, there is growing interest in the development of valid value frameworks to assist funding and policy decision makers called upon to make evidence-informed decisions based on the highest quality evidence.

This Special Issue invites submissions that pertain to the topic of assessing value in child health. Manuscripts that address the following topics are welcome:

  • Health technology assessment (HTA) in child health;
  • Pediatric cost-effectiveness and willingness-to-pay analysis;
  • Measuring preferences (utility) of patients, family members or members of the public for pediatric health states;
  • Health-related quality of life and cost spillover effects in caregivers of children with a health condition;
  • Valuation of preferences for pediatric interventions and technologies by patients, family members, clinicians, and members of the public using quantitative or qualitative methods;
  • Ethical analysis for valuation of complex emerging technologies in child health;
  • The development of tools, instruments, statistical approaches or models to measure elements of value in child health;
  • Value frameworks for funding and policy decision making for pediatric interventions and services.

Prof. Wendy J. Ungar
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Children is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 1800 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • Health technology assessment 
  • Cost-effectiveness analysis 
  • Willingness-to-pay 
  • Health state preferences 
  • Spillover effects 
  • Preference ascertainment 
  • Ethical analysis 
  • Value frameworks

Published Papers (9 papers)

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Editorial

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Editorial
Assessing Value in Child Health
Children 2021, 8(11), 972; https://doi.org/10.3390/children8110972 - 27 Oct 2021
Viewed by 362
Abstract
Assessing value in child health is increasingly important as health care systems face difficult choices with regard to what services and programs for children to fund and deliver [...] Full article
(This article belongs to the Special Issue Assessing Value in Child Health)

Research

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Article
The Challenges of Measuring and Valuing Quality of Life in Preschool Children: A Retrospective Review of NICE Appraisals
Children 2021, 8(9), 765; https://doi.org/10.3390/children8090765 - 31 Aug 2021
Cited by 2 | Viewed by 572
Abstract
Health technology assessment agencies evaluate interventions across the lifespan. However, there is no consensus about best-practice methods to measure health-related quality of life (HRQoL) in preschool children (<5 years) and data are often scarce. We reviewed methods used to capture the HRQoL of [...] Read more.
Health technology assessment agencies evaluate interventions across the lifespan. However, there is no consensus about best-practice methods to measure health-related quality of life (HRQoL) in preschool children (<5 years) and data are often scarce. We reviewed methods used to capture the HRQoL of preschool children in past National Institute for Health and Care Excellence (NICE) appraisals to establish whether there is a need for better methods in this area and if so, to identify priority research areas. We identified past NICE appraisals that included preschool children, examining the methods used to generate utility values and whether committees believed these captured HRQoL adequately. Of the 12 appraisals, most used generic HRQoL measures designed for adults. Measures were usually completed by adult patients or clinical experts. Committees frequently commented on limitations in the HRQoL data. While acknowledging that data collection may be challenging, committees would value evidence based on HRQoL data from parents or guardians collected as part of a clinical trial. We identified several research priorities including the psychometric properties of existing measures; the feasibility and validity of valuation studies; and mapping. Progress in these areas will help ensure that the aspects of HRQoL which matter to children and their families are captured in NICE evaluations. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
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Article
Psychometric Performance of HRQoL Measures: An Australian Paediatric Multi-Instrument Comparison Study Protocol (P-MIC)
Children 2021, 8(8), 714; https://doi.org/10.3390/children8080714 - 20 Aug 2021
Cited by 1 | Viewed by 721
Abstract
Background: There is a lack of psychometric evidence about pediatric health-related quality of life (HRQoL) instruments. Evidence on cost effectiveness, involving the measurement of HRQoL, is used in many countries to make decisions about pharmaceuticals, technologies, and health services for children. Additionally, valid [...] Read more.
Background: There is a lack of psychometric evidence about pediatric health-related quality of life (HRQoL) instruments. Evidence on cost effectiveness, involving the measurement of HRQoL, is used in many countries to make decisions about pharmaceuticals, technologies, and health services for children. Additionally, valid instruments are required to facilitate accurate outcome measurement and clinical decision making. A pediatric multi instrument comparison (P-MIC) study is planned to compare the psychometric performance and measurement characteristics of pediatric HRQoL instruments. Methods: The planned P-MIC study will collect data on approximately 6100 Australian children and adolescents aged 2–18 years via The Royal Children’s Hospital Melbourne and online survey panels. Participants will complete an initial survey, involving the concurrent collection of a range of pediatric HRQoL instruments, followed by a shorter survey 2–8 weeks later, involving the collection of a subset of instruments from the initial survey. Children aged ≥7 years will be asked to self-report HRQoL. Psychometric performance will be assessed at the instrument, domain, and item level. Conclusions: This paper describes the methodology of the planned P-MIC study, including benefits, limitations, and likely challenges. Evidence from this study will guide the choice of HRQoL measures used in clinical trials, economic evaluation, and other applications. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
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Article
Bilateral Permanent Childhood Hearing Loss and Health-Related Quality of Life in Adolescence
Children 2021, 8(6), 484; https://doi.org/10.3390/children8060484 - 07 Jun 2021
Cited by 1 | Viewed by 687
Abstract
Little is known about the impact of bilateral permanent childhood hearing loss (PCHL) on health-related quality of life (HRQoL). The objective of this study was to describe preference-based and non-preference based HRQoL outcomes in adolescence, from both self and proxy perspectives, amongst participants [...] Read more.
Little is known about the impact of bilateral permanent childhood hearing loss (PCHL) on health-related quality of life (HRQoL). The objective of this study was to describe preference-based and non-preference based HRQoL outcomes in adolescence, from both self and proxy perspectives, amongst participants of the Hearing Outcomes Project. The Health Utilities Index Marks II (HUI2) and III (HUI3) and the PedsQLTM Version 4.0 Generic Core Scales were used to measure HRQoL based on self and parent proxy reports in 114 adolescents aged 13–19 years, 76 with bilateral PCHL and 38 with normal hearing, recruited from a population sample that was followed up from birth to adolescence. Descriptive statistics and multivariable analyses were used to estimate the relationship between severity of PCHL and HRQoL outcomes. PCHL was associated with decrements in mean multi-attribute utility score that varied between 0.078 and 0.148 for the HUI2 (p = 0.001) and between 0.205 and 0.315 for the HUI3 (p < 0.001), dependent upon the national tariff set applied and respondent group. Multivariable analyses revealed that, after controlling for clinical and sociodemographic covariates, mean HUI3 multi-attribute utility scores were significantly lower in adolescents with moderately severe, severe and profound hearing loss than in adolescents with normal hearing. Significant differences in physical functioning, social functioning, psychosocial functioning and total PedsQLTM scores were only observed when assessments by parents were relied upon, but these dissipated in the multivariable analyses. Bilateral PCHL is associated with poorer HRQoL outcomes in adolescence. Further studies conducted are needed to understand the trajectory and underpinning mechanisms of HRQoL outcomes following PCHL. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
Article
Newborn Screening and Treatment of Phenylketonuria: Projected Health Outcomes and Cost-Effectiveness
Children 2021, 8(5), 381; https://doi.org/10.3390/children8050381 - 12 May 2021
Cited by 1 | Viewed by 620
Abstract
The objective of this study was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to recommended diet treatment and a newly available drug treatment (sapropterin dihydrochloride). A computer simulation model was [...] Read more.
The objective of this study was to evaluate the cost-effectiveness of newborn screening and treatment for phenylketonuria (PKU) in the context of new data on adherence to recommended diet treatment and a newly available drug treatment (sapropterin dihydrochloride). A computer simulation model was developed to project outcomes for a hypothetical cohort of newborns with PKU. Four strategies were compared: (1) clinical identification (CI) with diet treatment; (2) newborn screening (NBS) with diet treatment; (3) CI with diet and medication (sapropterin dihydrochloride); and (4) NBS with diet and medication. Data sources included published literature, primary data, and expert opinion. From a societal perspective, newborn screening with diet treatment had an incremental cost-effectiveness ratio of $6400/QALY compared to clinical identification with diet treatment. Adding medication to NBS with diet treatment resulted in an incremental cost-effectiveness ratio of more than $16,000,000/QALY. Uncertainty analyses did not substantially alter the cost-effectiveness results. Newborn screening for PKU with diet treatment yields a cost-effectiveness ratio lower than many other recommended childhood prevention programs even if adherence is lower than previously assumed. Adding medication yields cost-effectiveness results unlikely to be considered favorable. Future research should consider conditions under which sapropterin dihydrochloride would be more economically attractive. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
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Article
An Assessment of the Validity and Reliability of the Pediatric Child Health Utility 9D in Children with Inflammatory Bowel Disease
Children 2021, 8(5), 343; https://doi.org/10.3390/children8050343 - 27 Apr 2021
Cited by 1 | Viewed by 475
Abstract
Health utilities relevant to children are lacking, compromising health funding and policy decisions for children. The Child Health Utility 9D (CHU9D) is a recently developed preference-based health utility instrument designed for use in children. The objective was to examine the validity of the [...] Read more.
Health utilities relevant to children are lacking, compromising health funding and policy decisions for children. The Child Health Utility 9D (CHU9D) is a recently developed preference-based health utility instrument designed for use in children. The objective was to examine the validity of the CHU9D in a cohort of 285 Canadian children aged 6.5 to 18 years of age with Crohn’s disease (CD) and ulcerative colitis (UC), (collectively inflammatory bowel disease (IBD)). The correlation and agreement between paired CHU9D and Health Utility Index (HUI) assessments were determined with Spearman coefficients and Bland–Altman levels of agreement. Total and domain utilities were calculated for the CHU9D using Australian adult and youth tariffs. Algorithms for HUI2 and HUI3 were used. Domain correlations were determined between domains with expected overlap between instruments. In CD and in UC, correlations between CHU9D, HUI2, and HUI3 utilities ranged between 0.62 to 0.67 and 0.67 to 0.69, respectively (p < 0.05). CHU9D utilities were lower using youth tariffs compared to adult tariffs. A large range in health utilities suggested a heterogeneous quality of life. The CHU9D is a good option for preference-based utility measurement in pediatric IBD. Additional research is required to derive pediatric tariffs to conduct economic evaluation in children. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
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Review

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Review
Monetary Valuation of Children’s Cognitive Outcomes in Economic Evaluations from a Societal Perspective: A Review
Children 2021, 8(5), 352; https://doi.org/10.3390/children8050352 - 29 Apr 2021
Cited by 2 | Viewed by 598
Abstract
Cognitive ability in childhood is positively associated with economic productivity in adulthood. Expected gains in economic output from interventions that protect cognitive function can be incorporated in benefit–cost and cost-effectiveness analyses conducted from a societal perspective. This review summarizes estimates from high-income countries [...] Read more.
Cognitive ability in childhood is positively associated with economic productivity in adulthood. Expected gains in economic output from interventions that protect cognitive function can be incorporated in benefit–cost and cost-effectiveness analyses conducted from a societal perspective. This review summarizes estimates from high-income countries of the association of general cognitive ability, standardized as intelligence quotient (IQ), with annual and lifetime earnings among adults. Estimates of the association of adult earnings with cognitive ability assessed in childhood or adolescence vary from 0.5% to 2.5% per IQ point. That range reflects differences in data sources and analytic methods. We take a conservative published estimate of a 1.4% difference in market productivity per IQ point in the United States from a recent study that controlled for confounding by family background and behavioral attributes. Using that estimate and the present value of lifetime earnings calculated using a 3% discount rate, the implied lifetime monetary valuation of an IQ point in the United States is USD 10,600–13,100. Despite uncertainty and the exclusion of non-market productivity, incorporation of such estimates could lead to a fuller assessment of the benefits of public health and clinical interventions that protect the developing brains of fetuses, infants, and young children. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
Review
Incorporating Cascade Effects of Genetic Testing in Economic Evaluation: A Scoping Review of Methodological Challenges
Children 2021, 8(5), 346; https://doi.org/10.3390/children8050346 - 27 Apr 2021
Cited by 2 | Viewed by 861
Abstract
Cascade genetic testing is indicated for family members of individuals testing positive on a genetic test, and is particularly relevant for child health because of their vulnerability and the long-term health and economic implications. Cascade testing has patient- and health system-level implications; however [...] Read more.
Cascade genetic testing is indicated for family members of individuals testing positive on a genetic test, and is particularly relevant for child health because of their vulnerability and the long-term health and economic implications. Cascade testing has patient- and health system-level implications; however cascade costs and health effects are not routinely considered in economic evaluation. The methodological challenges associated with incorporating cascade effects in economic evaluation require examination. The purpose of this scoping review was to identify published economic evaluations that considered cascade genetic testing. Citation databases were searched for English-language economic evaluations reporting on cascade genetic testing. Nineteen publications were included. In four, genetic testing was used to identify new index patients—cascade effects were also considered; thirteen assessed cascade genetic testing strategies for the identification of at-risk relatives; and two calculated the costs of cascade genetic testing as a secondary objective. Methodological challenges associated with incorporating cascade effects in economic evaluation are related to study design, costing, measurement and valuation of health outcomes, and modeling. As health economic studies may currently be underestimating both the cost and health benefits attributable to genetic technologies through omission of cascade effects, development of methods to address these difficulties is required. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
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Review
Utility of Genetic Testing from the Perspective of Parents/Caregivers: A Scoping Review
Children 2021, 8(4), 259; https://doi.org/10.3390/children8040259 - 27 Mar 2021
Cited by 5 | Viewed by 942
Abstract
In genomics, perceived and personal utility have been proposed as constructs of value that include the subjective meanings and uses of genetic testing. Precisely what constitutes these constructs of utility and how they vary by stakeholder perspective remains unresolved. To advance methods for [...] Read more.
In genomics, perceived and personal utility have been proposed as constructs of value that include the subjective meanings and uses of genetic testing. Precisely what constitutes these constructs of utility and how they vary by stakeholder perspective remains unresolved. To advance methods for measuring the value of genetic testing in child health, we conducted a scoping review of the literature to characterize utility from the perspective of parents/caregivers. Peer reviewed literature that included empiric findings from parents/caregivers who received genetic test results for an index child and was written in English from 2016–2020 was included. Identified concepts of utility were coded according to Kohler’s construct of personal utility. Of 2142 abstracts screened, 33 met inclusion criteria. Studies reflected a range of genetic test types; the majority of testing was pursued for children with developmental or neurodevelopmental concerns. Coding resulted in 15 elements of utility that mapped to Kohler’s four domains of personal utility (affective, cognitive, behavioural and social) and one additional medical management domain. An adapted construct of utility for parents/caregivers may enable specific and standardized strategies for researchers to use to generate evidence of the post-test value of genetic testing. In turn, this will contribute to emerging methods for health technology assessment and policy decision making for genomics in child health. Full article
(This article belongs to the Special Issue Assessing Value in Child Health)
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