Review of Valuation Methods of Preference-Based Measures of Health for Economic Evaluation in Child and Adolescent Populations: Where are We Now and Where are We Going?

Methods for measuring and valuing health benefits for economic evaluation and health technology assessment in adult populations are well developed. In contrast, methods for assessing interventions for child and adolescent populations lack detailed guidelines, particularly regarding the valuation of health and quality of life in these age groups. This paper critically examines the methodological considerations involved in the valuation of child- and adolescent-specific health-related quality of life by existing preference-based measures. It also describes the methodological choices made in the valuation of existing generic preference-based measures developed with and/or applied in child and adolescent populations: AHUM, AQoL-6D, CHU9D, EQ-5D-Y, HUI2, HUI3, QWB, 16D and 17D. The approaches used to value existing child- and adolescent-specific generic preference-based measures vary considerably. While the choice of whose preferences and which perspective to use is a matter of normative debate and ultimately for decision by reimbursement agencies and policy makers, greater research around these issues would be informative and would enrich these discussions. Research can also inform the other methodological choices required in the valuation of child and adolescent health states. Gaps in research evidence are identified around the impact of the child described in health state valuation exercises undertaken by adults, including the possibility of informed preferences; the appropriateness and acceptability of valuation tasks for adolescents, in particular tasks involving the state ‘dead’; anchoring of adolescent preferences; and the generation and use of combined adult and adolescent preferences.

[1]  Wendy J. Ungar,et al.  Economic Evaluation in Child Health , 2010 .

[2]  Mandy Ryan,et al.  For better or worse? Investigating the validity of best–worst discrete choice experiments in health , 2019, Health economics.

[3]  Jason Madan,et al.  A Systematic Review and Meta-analysis of Childhood Health Utilities , 2017, Medical decision making : an international journal of the Society for Medical Decision Making.

[4]  H. Raat,et al.  Parent-reported health outcomes in infants/toddlers: measurement properties and clinical validity of the ITQOL-SF47 , 2013, Quality of Life Research.

[5]  I. Kaitila,et al.  Quality of life in early adolescence: A sixteendimensional health-related measure (16D) , 1996, Quality of Life Research.

[6]  J. Brazier,et al.  The development of a preference‐based measure of health in children with atopic dermatitis , 2005, The British journal of dermatology.

[7]  M. Cowie National Institute for Health and Care Excellence. , 2015, European heart journal.

[8]  S. Davies,et al.  Our children deserve better: prevention pays , 2013, The Lancet.

[9]  J. Richardson,et al.  Predicting time trade-off health state valuations of adolescents in four Pacific countries using the Assessment of Quality-of-Life (AQoL-6D) instrument. , 2010, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[10]  Wolfgang Greiner,et al.  Valuation of Child Health-Related Quality of Life in the United States. , 2016, Health economics.

[11]  John Brazier,et al.  Development of the multi-attribute Adolescent Health Utility Measure (AHUM) , 2012, Health and Quality of Life Outcomes.

[12]  Mark Oppe,et al.  Valuation of EuroQol Five-Dimensional Questionnaire, Youth Version (EQ-5D-Y) and EuroQol Five-Dimensional Questionnaire, Three-Level Version (EQ-5D-3L) Health States: The Impact of Wording and Perspective. , 2018, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[13]  P. Hinds,et al.  PROMIS® pediatric self-report scales distinguish subgroups of children within and across six common pediatric chronic health conditions , 2015, Quality of Life Research.

[14]  David Parkin,et al.  Is there a case for using visual analogue scale valuations in cost-utility analysis? , 2006, Health economics.

[15]  L. Prosser,et al.  Advances in Methods and Novel Applications for Measuring Family Spillover Effects of Illness , 2019, PharmacoEconomics.

[16]  J. Brazier,et al.  Using a discrete choice experiment to estimate health state utility values. , 2012, Journal of health economics.

[17]  Katherine Stevens,et al.  Assessing the performance of a new generic measure of health-related quality of life for children and refining it for use in health state valuation , 2011, Applied health economics and health policy.

[18]  J. Brazier,et al.  Valuing Child Health Utility 9D Health States with Young Adults: Insights from a Time Trade Off Study , 2015, Applied Health Economics and Health Policy.

[19]  Andrew Briggs,et al.  Estimating Health-State Utility for Economic Models in Clinical Studies: An ISPOR Good Research Practices Task Force Report. , 2016, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[20]  M Sculpher,et al.  EOS 2D/3D X-ray imaging system: a systematic review and economic evaluation. , 2012, Health technology assessment.

[21]  Julie Ratcliffe,et al.  A Review of the Development and Application of Generic Multi-Attribute Utility Instruments for Paediatric Populations , 2015, PharmacoEconomics.

[22]  J. Brazier,et al.  Valuing child health utility 9D health states with a young adolescent sample , 2011, Applied health economics and health policy.

[23]  Paul Kind,et al.  Can adult weights be used to value child health states? Testing the influence of perspective in valuing EQ-5D-Y , 2015, Quality of Life Research.

[24]  Wendy J. Ungar,et al.  Challenges in Health State Valuation in Paediatric Economic Evaluation , 2011, PharmacoEconomics.

[25]  Christopher McCabe,et al.  Health state values for the HUI 2 descriptive system: results from a UK survey. , 2005, Health economics.

[26]  W. Furlong,et al.  Differences in preferences for neonatal outcomes among health care professionals, parents, and adolescents. , 1999, JAMA.

[27]  Michael Herdman,et al.  Development of the EQ-5D-Y: a child-friendly version of the EQ-5D , 2010, Quality of Life Research.

[28]  J. Brazier,et al.  Using a discrete choice experiment to estimate societal health state utility values , 2010 .

[29]  Koonal Shah,et al.  Anchoring Latent Scale Values for the EQ-5D-Y at 0 = Dead , 2020 .

[30]  喜多 明人,et al.  「逐条解説」子どもの権利条約 = Convention on the rights of the child , 2009 .

[31]  J. Varni,et al.  The PedsQL: measurement model for the pediatric quality of life inventory. , 1999, Medical care.

[32]  N. Hays,et al.  Feasibility and Psychometric Properties of the Infant Toddler Quality of Life (ITQOL) Questionnaire in a Community-Based Sample of Healthy Infants in China , 2018, Maternal and Child Health Journal.

[33]  A Simon Pickard,et al.  Proxy Evaluation of Health-Related Quality of Life: A Conceptual Framework for Understanding Multiple Proxy Perspectives , 2005, Medical care.

[34]  Victoria A. Miller,et al.  An Emerging Field of Research , 2015, Medical decision making : an international journal of the Society for Medical Decision Making.

[35]  M. Boyle,et al.  Multiattribute and Single‐Attribute Utility Functions for the Health Utilities Index Mark 3 System , 2002, Medical care.

[36]  J. Brazier,et al.  A Comparison of Methods for Converting DCE Values onto the Full Health-Dead QALY Scale , 2015, Medical decision making : an international journal of the Society for Medical Decision Making.

[37]  J. Richardson,et al.  Predicting time trade-off health state valuations of adolescents in four Pacific countries using the AQoL-6D instrument , 2009 .

[38]  P. Veugelers,et al.  Assessment of children’s own health status using visual analogue scale and descriptive system of the EQ-5D-Y: linkage between two systems , 2014, Quality of Life Research.

[39]  M. Eckman,et al.  Health values of adolescents with cystic fibrosis. , 2003, The Journal of pediatrics.

[40]  Katherine Stevens,et al.  Valuation of the Child Health Utility 9D Index , 2012, PharmacoEconomics.

[41]  Christine C Bennett A healthier future for all Australians: an overview of the final report of the National Health and Hospitals Reform Commission , 2009, The Medical journal of Australia.

[42]  D. Feeny,et al.  Multiattribute utility function for a comprehensive health status classification system. Health Utilities Index Mark 2. , 1996, Medical care.

[43]  Dean A. Regier,et al.  Evaluating health-related quality-of-life studies in paediatric populations , 2012, PharmacoEconomics.

[44]  M. Essink‐bot,et al.  Reliability and validity of the Infant and Toddler Quality of Life Questionnaire (ITQOL) in a general population and respiratory disease sample , 2006, Quality of Life Research.

[45]  Sarah A. Edwards,et al.  Characteristics and quality of pediatric cost-utility analyses , 2012, Quality of Life Research.

[46]  I. Kaitila,et al.  Quality of life in pre-adolescence: A 17-dimensional health-related measure (17D) , 1996, Quality of Life Research.

[47]  M. Barkham,et al.  Developing and testing methods for deriving preference-based measures of health from condition-specific measures (and other patient-based measures of outcome). , 2012, Health technology assessment.

[48]  John Brazier,et al.  Identification, Review, and Use of Health State Utilities in Cost-Effectiveness Models: An ISPOR Good Practices for Outcomes Research Task Force Report. , 2019, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[49]  N. Devlin,et al.  DO ADULT AND ADOLESCENT PREFERENCES DIFFER? , 2019 .

[50]  N. Devlin,et al.  EQ-5 D and the EuroQol Group : Past , Present and Future , 2017 .

[51]  Katherine Stevens,et al.  Developing a descriptive system for a new preference-based measure of health-related quality of life for children , 2009, Quality of Life Research.

[52]  W. Brouwer The Inclusion of Spillover Effects in Economic Evaluations: Not an Optional Extra , 2018, PharmacoEconomics.

[53]  M. Bell,et al.  Development of the multi-attribute Pediatric Asthma Health Outcome Measure (PAHOM). , 2005, International journal for quality in health care : journal of the International Society for Quality in Health Care.

[54]  Juan Manuel Ramos-Goñi,et al.  Valuing EQ-5D-Y-3L Health States Using a Discrete Choice Experiment: Do Adult and Adolescent Preferences Differ? , 2021, Medical decision making : an international journal of the Society for Medical Decision Making.

[55]  K. Eames,et al.  Measuring Health Utilities in Children and Adolescents: A Systematic Review of the Literature , 2015, PloS one.

[56]  S. Petrou Methodological issues raised by preference-based approaches to measuring the health status of children. , 2003, Health economics.

[57]  W. Brouwer,et al.  Patient and general public preferences for health states: A call to reconsider current guidelines. , 2016, Social science & medicine.

[58]  Verity Watson,et al.  Is Best–Worst Scaling Suitable for Health State Valuation? A Comparison with Discrete Choice Experiments , 2017, Health economics.

[59]  R. Keren,et al.  Measuring Health Preferences for Use in Cost-Utility and Cost-Benefit Analyses of Interventions in Children , 2012, PharmacoEconomics.

[60]  K. Stevens,et al.  Working With Children to Develop Dimensions for a Preference-Based, Generic, Pediatric, Health-Related Quality-of-Life Measure , 2010, Qualitative health research.

[61]  L. Prosser,et al.  Family Spillover Effects in Pediatric Cost-Utility Analyses , 2018, Applied Health Economics and Health Policy.

[62]  Anne W. Riley,et al.  Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO good research practices for the assessment of children and adolescents task force. , 2013, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[63]  Julie Ratcliffe,et al.  Measuring and valuing health benefits for economic evaluation in adolescence: an assessment of the practicality and validity of the child health utility 9D in the Australian adolescent population. , 2012, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[64]  G. Bonsel,et al.  Feasibility, reliability, and validity of the EQ-5D-Y: results from a multinational study , 2010, Quality of Life Research.

[65]  Richard Norman,et al.  The Impact of Different DCE-Based Approaches When Anchoring Utility Scores , 2016, PharmacoEconomics.

[66]  J. Brazier,et al.  Experience-based utility and own health state valuation for a health state classification system: why and how to do it , 2017, The European Journal of Health Economics.

[67]  N. Payakachat,et al.  Progress in measuring family spillover effects for economic evaluations , 2015, Expert review of pharmacoeconomics & outcomes research.

[68]  M. T. King,et al.  Using a discrete choice experiment to value the QLU-C10D: feasibility and sensitivity to presentation format , 2016, Quality of Life Research.

[69]  N. Devlin,et al.  EQ-5D and the EuroQol Group: Past, Present and Future , 2017, Applied Health Economics and Health Policy.

[70]  Brendan Mulhern,et al.  Estimating a Dutch Value Set for the Pediatric Preference-Based CHU9D Using a Discrete Choice Experiment with Duration. , 2018, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[71]  H. McTaggart-Cowan Elicitation of informed general population health state utility values: a review of the literature. , 2011, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[72]  Stuart Peacock,et al.  Measurement of the Quality of Life for Economic Evaluation and the Assessment of Quality of Life (Aqol) Mark 2 Instrument , 2004 .

[73]  John Brazier,et al.  Developing Adolescent-Specific Health State Values for Economic Evaluation , 2012, PharmacoEconomics.

[74]  T. Trikalinos,et al.  Recommendations for Conduct, Methodological Practices, and Reporting of Cost-effectiveness Analyses: Second Panel on Cost-Effectiveness in Health and Medicine. , 2016, JAMA.

[75]  ChiMat,et al.  Annual Report of the Chief Medical Officer 2012. Our Children Deserve Better: Prevention Pays , 2008 .

[76]  J. Ratcliffe,et al.  Scoring the Child Health Utility 9D instrument: estimation of a Chinese child and adolescent-specific tariff , 2018, Quality of Life Research.

[77]  M. Herdman,et al.  Sources and Characteristics of Utility Weights for Economic Evaluation of Pediatric Vaccines: A Systematic Review. , 2016, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[78]  John Brazier,et al.  Nothing About Us Without Us? A Comparison of Adolescent and Adult Health-State Values for the Child Health Utility-9D Using Profile Case Best-Worst Scaling. , 2016, Health economics.