. 2022 Apr;40(4):379-431.

doi: 10.1007/s40273-021-01128-0. Epub 2022 Jan 24.

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

Joseph Kwon^#¹, Louise Freijser^#², Elisabeth Huynh³, Martin Howell⁴, Gang Chen⁵, Kamran Khan⁶, Shahd Daher⁷, Nia Roberts⁸, Conrad Harrison⁹, Sarah Smith¹⁰, Nancy Devlin², Kirsten Howard⁴, Emily Lancsar³, Cate Bailey², Jonathan Craig¹¹, Kim Dalziel¹², Alison Hayes⁴, Brendan Mulhern¹³, Germaine Wong⁴, Julie Ratcliffe^#¹⁴, Stavros Petrou^#¹⁵

Affiliations

¹ School of Health and Related Research, University of Sheffield, Sheffield, England, UK.
² Centre for Health Policy, University of Melbourne, Melbourne, Australia.
³ Department of Health Services Research and Policy, Australian National University, Canberra, Australia.
⁴ School of Public Health, University of Sydney, Sydney, Australia.
⁵ Centre for Health Economics, Monash University, Melbourne, Australia.
⁶ Centre for Health Economics at Warwick, University of Warwick, Coventry, England, UK.
⁷ Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, England, UK.
⁸ Bodleian Health Care Libraries, University of Oxford, Oxford, England, UK.
⁹ Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, England, UK.
¹⁰ Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, England, UK.
¹¹ College of Medicine and Public Health, Flinders University, Adelaide, Australia.
¹² Health Economics Unit, University of Melbourne, Melbourne, Australia.
¹³ Centre for Health Economics Research and Evaluation, University of Technology Sydney, Sydney, Australia.
¹⁴ Caring, Futures Institute, College of Nursing and Health Sciences, Flinders University, Adelaide, Australia.
¹⁵ Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, England, UK. stavros.petrou@phc.ox.ac.uk.

^# Contributed equally.

PMID: 35072935
PMCID: PMC9007803
DOI: 10.1007/s40273-021-01128-0

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

Joseph Kwon et al. Pharmacoeconomics. 2022 Apr.

. 2022 Apr;40(4):379-431.

doi: 10.1007/s40273-021-01128-0. Epub 2022 Jan 24.

Authors

Affiliations

¹ School of Health and Related Research, University of Sheffield, Sheffield, England, UK.
² Centre for Health Policy, University of Melbourne, Melbourne, Australia.
³ Department of Health Services Research and Policy, Australian National University, Canberra, Australia.
⁴ School of Public Health, University of Sydney, Sydney, Australia.
⁵ Centre for Health Economics, Monash University, Melbourne, Australia.
⁶ Centre for Health Economics at Warwick, University of Warwick, Coventry, England, UK.
⁷ Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, England, UK.
⁸ Bodleian Health Care Libraries, University of Oxford, Oxford, England, UK.
⁹ Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, England, UK.
¹⁰ Department of Health Services Research and Policy, London School of Hygiene and Tropical Medicine, London, England, UK.
¹¹ College of Medicine and Public Health, Flinders University, Adelaide, Australia.
¹² Health Economics Unit, University of Melbourne, Melbourne, Australia.
¹³ Centre for Health Economics Research and Evaluation, University of Technology Sydney, Sydney, Australia.
¹⁴ Caring, Futures Institute, College of Nursing and Health Sciences, Flinders University, Adelaide, Australia.
¹⁵ Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, England, UK. stavros.petrou@phc.ox.ac.uk.

^# Contributed equally.

PMID: 35072935
PMCID: PMC9007803
DOI: 10.1007/s40273-021-01128-0

Erratum in

Correction to: Systematic Review of Conceptual, Age, Measurement and Valuation Considerations for Generic Multidimensional Childhood Patient-Reported Outcome Measures.
Kwon J, Freijser L, Huynh E, Howell M, Chen G, Khan K, Daher S, Roberts N, Harrison C, Smith S, Devlin N, Howard K, Lancsar E, Bailey C, Craig J, Dalziel K, Hayes A, Mulhern B, Wong G, Ratcliffe J, Petrou S. Kwon J, et al. Pharmacoeconomics. 2022 Apr;40(4):477-478. doi: 10.1007/s40273-022-01135-9. Pharmacoeconomics. 2022. PMID: 35156178 Free PMC article. No abstract available.

Abstract

Background and aims: Patient-reported outcome measures (PROMs) for children (aged ≤ 18 years) present methodological challenges. PROMs can be categorised by their diverse underlying conceptual bases, including functional, disability and health (FDH) status; quality of life (QoL); and health-related quality of life (HRQoL). Some PROMs are designed to be accompanied by preference weights. PROMs should account for childhood developmental differences by incorporating age-appropriate health/QoL domains, guidance on respondent type(s) and design. This systematic review aims to identify generic multidimensional childhood PROMs and synthesise their characteristics by conceptual basis, target age, measurement considerations, and the preference-based value sets that accompany them.

Methods: The study protocol was registered in the Prospective Register of Systematic Reviews (CRD42021230833), and reporting followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. We conducted systematic database searches for generic multidimensional childhood PROMs covering the period 2012-2020, which we combined with published PROMs identified by an earlier systematic review that covered the period 1992-2011. A second systematic database search identified preference-based value sets for generic multidimensional PROMs. The PROMs were categorised by conceptual basis (FDH status, QoL and HRQoL) and by target age (namely infants and pre-schoolers aged < 5 years, pre-adolescents aged 5-11, adolescents aged 12-18 and multi-age group coverage). Descriptive statistics assessed how PROM characteristics (domain coverage, respondent type and design) varied by conceptual basis and age categories. Involvement of children in PROM development and testing was assessed to understand content validity. Characteristics of value sets available for the childhood generic multidimensional PROMs were identified and compared.

Results: We identified 89 PROMs, including 110 versions: 52 FDH, 29 QoL, 12 HRQoL, nine QoL-FDH and eight HRQoL-FDH measures; 20 targeted infants and pre-schoolers, 29 pre-adolescents, 24 adolescents and 37 for multiple age groups. Domain coverage demonstrated development trajectories from observable FDH aspects in infancy through to personal independence and relationships during adolescence. PROMs targeting younger children relied more on informant report, were shorter and had fewer ordinal scale points. One-third of PROMs were developed following qualitative research or surveys with children or parents for concept elicitation. There were 21 preference-based value sets developed by 19 studies of ten generic multidimensional childhood PROMs: seven were based on adolescents' stated preferences, seven were from adults from the perspective of or on behalf of the child, and seven were from adults adopting an adult's perspective. Diverse preference elicitation methods were used to elicit values. Practices with respect to anchoring values on the utility scale also varied considerably. The range and distribution of values reflect these differences, resulting in value sets with notably different properties.

Conclusion: Identification and categorisation of generic multidimensional childhood PROMs and value sets by this review can aid the development, selection and interpretation of appropriate measures for clinical and population research and cost-effectiveness-based decision-making.

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Conflict of interest statement

ND and BM are members of the EuroQol Group, which developed the EQ-5D-Y, one of the measures included in review. Other authors have no conflict of interest to declare.

Figures

**Fig. 1**
The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) flow diagram for database searches for studies developing generic multidimensional childhood patient-reported outcome measures published between 1 January 2012 and 16 October 2020

**Fig. 2**
The Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) flow diagram for database searches for valuation studies for generic multidimensional childhood PROMs identified by search strategies. *PROM* patient-reported outcome measure, *QALY* quality-adjusted life year

**Fig. 3**
Number of identified generic multidimensional childhood PROMs by conceptual basis and age category. *FDH* functioning, disability and health, *HRQoL* health-related quality of life, *PROM* patient-reported outcome measure, *QoL* quality of life

**Fig. 4**
Preference-based index value range by perspective

See this image and copyright information in PMC

References

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

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

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