Challenges in Measuring and Valuing Children’s Health-Related Quality of Life
Nancy Devlin, PhD, Centre for Health Policy, University of Melbourne, Australia; Rosie Lovett, PhD, National Institute for Health and Care Excellence, Manchester, UK; Donna Rowen, PhD, School of Health and Related Research (ScHARR), University of Sheffield, UK
Children hold a special place in any society. They are among its most vulnerable members and children’s health and well-being affects both their lifelong opportunities and the future for us all. One of the basic responsibilities of any society is to care for children in the best and most compassionate manner possible.1
But even where children are concerned, there are choices to be made about healthcare and opportunity costs of those choices. It is important to ensure that preventive and treatment options for children are both effective and cost-effective. Making good decisions requires evidence about both the costs and outcomes of technologies where children are the intended beneficiaries.
Methods widely used to measure and value health-related quality of life (HRQoL) and generate quality-adjusted life years (QALYs) in adults are problematic when applied to children.2 There are 3 key challenges.
First, the aspects of HRQoL that matter to children may be different than for adults, and there are challenges with asking very young and very sick children to self-report their health. Such issues can be overcome to some extent by the use of child-and adolescent-specific HRQoL measures (such as CHU9D and EQ-5D-Y), administered via self-report or parental proxy. There are ISPOR guidelines on the use of patient-reported outcomes (PROs) in children3 although challenges remain, especially in measuring HRQoL in very young children.4
Second, summarizing these measures of HRQoL by converting to utilities (as needed for estimating QALYs) raises further issues: should these be based on the stated preferences of adult members of the general public? How do we ask them to imagine and value children’s health states? Should we ask children for their preferences? Is it feasible and ethical to ask children to complete tasks involving trade-offs between life and death? Methods and issues relating to valuation of child HRQoL are summarized in Rowen et al.5
Third, how do we interpret the estimates of child QALYs that are produced? Are they measuring the same thing? Does a child QALY have the same value to society as an adult QALY, or should child QALY gains be given more weight by decision makers?
We wanted to explore the extent to which these issues are being encountered by health technology assessment (HTA) agencies in evidence submitted to them and their views about how to improve evidence and decision making concerning children.
The ISPOR and NICE International HTA Roundtable on Child HRQoL
An HTA Roundtable on these issues was held as a joint initiative of ISPOR and the National Institute for Health and Care Excellence (NICE) International in October 2020. Invitations were sent to agencies that had well-established HTA processes and were likely to have considered the issues outlined above. There were 22 attendees representing 11 HTA agencies: NICE (England); Scottish Medicines Consortium (Scotland); Canadian Agency for Drugs and Technologies in Health (Canada); The Dental and Pharmaceutical Benefits Agency (TLV) (Sweden); Ministry of Health (Singapore); Swedish Agency for Health Technology Assessment and Assessment of Social Services (Sweden); Instance Nationale de l’Evaluation & de l’Accréditation en Santé (Tunisia); Danish Medicines Council (Denmark); National Institute of Pharmacy and Nutrition (Hungary); Norwegian Medicines Agency; Technology Assessment at SickKids (TASK) (Canada); and the Agency for Health Technology Assessment and Tariff System (Poland).
Box 1. Key findings from the HTA Roundtable on child HRQoL
The aim was to share experiences and views on measuring and valuing children’s HRQoL for use in cost-effectiveness analyses and the use of these results in HTA decision making. Prior to the Roundtable, participants watched an introductory video, which can be accessed here, and answered a survey. The Roundtable was held online and entailed a structured discussion, facilitated by the authors.
The principal findings from the HTA Roundtable are reported in Box 1.
Despite the availability of HRQoL instruments designed and validated for children, and the growing availability of utilities to accompany them, HTA Roundtable participants noted that very little good-quality HRQoL evidence is currently being submitted to support HTA of technologies for children. There are gaps in evidence, frequent use of measures and utilities intended for adults, and inconsistent practices. This reinforces the conclusions drawn from reviews of submissions on pediatric technologies made to NICE6 and PBAC.7 There is a considerable gap in evidence on children’s HRQoL, and urgent action is required to address this.
Figure. Are children's QALY gains given extra weight over adults' QALY gains in HTA? Responses from attendees at the ISPOR/NICE International Roundtable on pediatric HRQoL.
While HTA bodies are aware of the challenges in measuring and valuing HRQoL in children, no HTA body currently provides explicit methods guidance. Roundtable participants agreed that clearer methods guidance on child HRQoL and QALY estimation would provide clearer signals to industry about the need to collect evidence on pediatric HRQoL, as well as about what instruments and utilities to use.
Roundtable participants suggested that an effective way to make progress would be to develop consensus statements collaboratively by HTA bodies and academia. International consensus on those aspects of methods that can be common across borders could increase the use of pediatric HRQoL measures in clinical trials and improve the quality of evidence available for HTA. An ISPOR emerging good practices taskforce on pediatric utilities is planned for 2022 and should provide guidance on methods for valuing child HRQoL. Ultimately, however, some aspects of methods—such as whose preferences should be reflected in HRQoL utilities and whether child and adult QALYs should be treated equally in decision making—require value judgements that must be made by local HTA agencies.
Acknowledgment: ISPOR and NICE International are grateful to all those who participated in this HTA Roundtable. The views here are those of the authors and do not necessarily reflect the views of NICE.
References
1. Mandela N. (2003) Address by Nelson Mandela at opening of Walter Sisulu Paediatric Cardiac Centre for Africa, Johannesburg. Published November 7, 2003. Accessed 19th November 202. www.mandela.gov.za/mandela_speeches/2003/031107_cardiac.htm
2. Devlin N, Norman R, Ratcliffe J, et al for the QUality Of life in Kids: key evidence for decision makers in Australia (QUOKKA) Project Team. London Office of Health Economics. Do child QALYs = adult QALYs? Five reasons why they might not. Published February 4, 2020. Accessed 19th November 2021. www.ohe.org/news/do-child-qalysadult-qalys-five-reasons-why-they-might-not.
3. Matza S, Patrick D, Riley A, 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. Value Health. 2013;16 (4):460-479. https://doi.org/10.1016/j.jval.2013.04.004
4. Lamb A, Murray A, Lovett R.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
5. Rowen D, Rivero-Arias O, Devlin N. et al. 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? PharmacoEconomics. 2020;38:325-340. https://doi.org/10.1007/s40273-019-00873-7
6. Hill H, Rowen D, Pennington R, Wong R, Wailoo A. A review of the methods used to generate utility values in NICE technology assessments for children and adolescents. Value Health. 2020;23(7):907-917. https://doi.org/10.1016/j.jval.2020.02.011
7. Bailey C, Dalziel K, Cronin P, Devlin N, Viney R for the QUality Of life in Kids: Key evidence for decision makers in Australia (QUOKKA) Project Team. How are child-specific utility instruments used in decision making in Australia? a review of Pharmaceutical Benefits Advisory Committee Public Summary Documents. Pharmacoeconomics. Published online November 5, 2021. https://doi.org/10.1007/s40273-021-01107-5
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