Systematic Review of Conceptual, Age, Measurement and Valuation Considerations for Generic Multidimensional Childhood Patient-Reported Outcome Measures

Kwon, Joseph; Freijser, Louise; Huynh, Elisabeth; Howell, Martin; Chen, Gang; Khan, Kamran; Daher, Shahd; Roberts, Nia; Harrison, Conrad; Smith, Sarah C.; Devlin, Nancy; Howard, Kirsten; Lancsar, Emily; Bailey, Cate; Craig, Jonathan C.; Dalziel, Kim; Hayes, Alison; Mulhern, Brendan; Wong, Germaine; Ratcliffe, Julie; Petrou, Stavros

doi:10.1007/s40273-021-01128-0

Cited by 42 publications

(43 citation statements)

References 137 publications

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“…The valuation of child health is key to conducting economic evaluations to inform decisions on the reimbursement and pricing of health interventions for children. Previous reviews have investigated the measurement of child PROMs [ 5 , 19 ]. In the current review, we have focused on valuation approaches.…”

Section: Discussionmentioning

confidence: 99%

“…A range of PROMs has been developed for use in children and adolescents, and value sets have been produced for some of these from which utility values can be derived [ 5 ]. Patient-reported outcome measures with value sets for children include the 16D [ 6 ], the 17D [ 7 ], the Adolescent Health Utility Measure (AHUM) [ 8 ], the adolescent version of the Assessment of Quality of Life (AQoL-6D Adolescent) [ 9 ], the Child Health Utility instrument (CHU9D) [ 10 ], the youth version of the EQ-5D (EQ-5D-Y) [ 11 ], the Health Utilities Index Mark 2 (HUI2) [ 12 ] and Mark 3 (HUI3) [ 13 ], the Quality of Well Being Self-Administered (QWB-SA) scale [ 14 ] and the Infant health-related Quality of life Instrument (IQI) [ 15 ], each of which covers different age groups and domains.…”

Section: Introductionmentioning

confidence: 99%

“…Previous reviews have described the measurement characteristics of children’s generic multidimensional PROMs accompanied by preference-based value sets [ 5 , 19 , 20 ]; however, information on the elicitation methods and perspectives used to obtain these values is lacking. In addition to these value sets for childhood PROMs, other valuation research includes methodological studies, and studies aimed at valuing specific vignettes or scenarios concerning child health.…”

Section: Introductionmentioning

confidence: 99%

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Preference Elicitation Techniques Used in Valuing Children’s Health-Related Quality-of-Life: A Systematic Review

Bailey

Howell²,

Raghunandan³

et al. 2022

PharmacoEconomics

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Background and Objectives Valuing children’s health states for use in economic evaluations is globally relevant and is of particular relevance in jurisdictions where a cost-utility analysis is the preferred form of analysis for decision making. Despite this, the challenges with valuing child health mean that there are many remaining questions for debate about the approach to elicitation of values. The aim of this paper was to identify and describe the methods used to value children’s health states and the specific issues that arise in the use of these methods. Methods We conducted a systematic search of electronic databases to identify studies published in English since 1990 that used preference elicitation methods to value child and adolescent (under 18 years of age) health states. Eligibility criteria comprised valuation studies concerning both child-specific patient-reported outcome measures and child health states defined in other ways, and methodological studies of valuation approaches that may or may not have yielded a value set algorithm. Results A total of 77 eligible studies were identified from which data on country setting, aims, condition (general population or clinically specific), sample size, age of respondents, the perspective that participants were asked to adopt, source of values (respondents who completed the preference elicitation tasks) and methods questions asked were extracted. Extracted data were classified and evaluated using narrative synthesis methods. The studies were classified into three groups: (1) studies comparing elicitation methods ( n = 30); (2) studies comparing perspectives ( n = 23); and (3) studies where no comparisons were presented ( n = 26); selected studies could fall into more than one group. Overall, the studies varied considerably both in methods used and in reporting. The preference elicitation tasks included time trade-off, standard gamble, visual analogue scaling, rating/ranking, discrete choice experiments, best-worst scaling and willingness to pay elicited through a contingent valuation. Perspectives included adults’ considering the health states from their own perspective, adults taking the perspective of a child (own, other, hypothetical) and a child/adolescent taking their own or the perspective of another child. There was some evidence that children gave lower values for comparable health states than did adults that adopted their own perspective or adult/parents that adopted the perspective of children. Conclusions Differences in reporting limited the conclusions that can be formed about which methods are most suitable for eliciting preferences for children’s health and the influence of differing perspectives and values. Difficulties encountered in drawing conclusions from the data (such as lack of consensus and poor reporting making it difficult for users to choose and inter...

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Preference Elicitation Techniques Used in Valuing Children’s Health-Related Quality-of-Life: A Systematic Review

Bailey

Howell²,

Raghunandan³

et al. 2022

PharmacoEconomics

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“…Paediatric Patient-Reported Outcome Measures (PROMs) have become increasingly important in health outcomes research with an increase in use in clinical trials and evaluating health systems [1,2]. Multi-attribute PROMs aim to capture the subjective constructs of health across physical, social and psychological functioning [3,4].…”

Section: Introductionmentioning

confidence: 99%

“…There are currently over 35 published generic PROMs for children and adolescents younger than 18 years [1] of which the EQ-5D-Y and Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core scale have been frequently cited [1,2,15]. The EQ-5D-Y was adapted from the EQ-5D, an adult measure, to include youth friendly wording and examples [16].…”

Section: Introductionmentioning

confidence: 99%

Comparison of the EQ-5D-Y-5L, EQ-5D-Y-3L and PedsQL in children and adolescents

Verstraete

Scott

2022

J Patient Rep Outcomes

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Background There is an increased use of Patient-Reported Outcome Measures (PROMs) in children and adolescents. The aim of this study was to compare the feasibility, concurrent validity and known-group validity of the EQ-5D-Y-3L (Y-3L), EQ-5D-Y-5L (Y-5L) and PedsQL self-report PROMs. Methods Five hundred and fifty children and adolescents, aged 8–15-years, with acute and chronic health conditions and a general population sample were recruited from schools and hospitals in Cape Town South Africa. All respondents self-completed the Y-5L, PedsQL, Self-Rated Health Question and Y-3L. Feasibility of the measures was determined by comparing the number of missing responses. Convergent validity was assessed by Spearman’s and Intra-class correlations on the corresponding items and summary scores respectively. Known-groups validity across health conditions was assessed across the summary scores of the measures with analysis of variance (ANOVA). Results The Y-3L and Y-5L had a total of 1% and 3.5% missing responses compared to 19% on the PedsQL. Similar items on the PedsQL and Y-3L/Y-5L showed high correlations (> 0.5) and related items showed moderate correlations (0.3). PedsQL total score was moderately and significantly associated with Y-3L and Y-5L level sum and VAS scores. The Y-3L and Y-5L level sum and VAS scores showed significant differences between known health groups whereas the PedsQL only showed differences between those with acute and chronic illness. Conclusion The results of this study show that the Y-3L and Y-5L showed comparable psychometric validity to the PedsQL. When considering the choice between the PedsQL, Y-5L and Y-3L these study results indicate that the EQ-5D-Y instruments (Y-3L and Y-5L) are recommended for studies assessing known-group validity or where missing data should be minimised. The PedsQL generic measure may be preferable in future studies including the general population where a ceiling effect is anticipated. When considering the choice between the Y-5L and the Y-3L there was no systematic difference in the validity between these instruments or between the Y-3L or Y-5L and the PedsQL. Thus, the selection of EQ-5D-Y measures to include in future studies should be guided by the characteristics of the population to be tested.

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Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD)

Matza,

Stewart,

Fournier

et al. 2024

Eur J Health Econ

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Introduction Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B. Methods Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD. The health states were valued in time trade-off interviews with adult general population respondents in the UK. Results Interviews were completed with 202 participants (50.0% female; mean age = 41.3 years). The health state representing ASMD without impairment had the highest mean utility for both the adult and child health states (0.92/0.94), and severe ASMD had the lowest mean utility (0.33/0.45). Every child health state had a significantly greater utility than the corresponding adult health state. Differences between adult/child paired states ranged from 0.02 to 0.13. Subgroup analyses explored the impact of parenting status on valuation of child health states. Discussion Greater severity of ASMD was associated with lower mean utility. Results have implications for valuation of pediatric health states. The resulting utilities may be useful in cost-utility modeling estimating the value of treatment for ASMD.

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Systematic Review of Conceptual, Age, Measurement and Valuation Considerations for Generic Multidimensional Childhood Patient-Reported Outcome Measures

Cited by 42 publications

References 137 publications

Preference Elicitation Techniques Used in Valuing Children’s Health-Related Quality-of-Life: A Systematic Review

Preference Elicitation Techniques Used in Valuing Children’s Health-Related Quality-of-Life: A Systematic Review

Comparison of the EQ-5D-Y-5L, EQ-5D-Y-3L and PedsQL in children and adolescents

Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD)

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