School of Health and Related Research (ScHARR), University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK.
National Perinatal Epidemiology Unit, Nuffield Department of Population Health, University of Oxford, Oxford, UK.
Pharmacoeconomics. 2020 Apr;38(4):325-340. doi: 10.1007/s40273-019-00873-7.
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.
用于成人人群经济评估和卫生技术评估的健康效益衡量和估值方法已经非常成熟。相比之下,针对儿童和青少年人群的干预措施评估方法缺乏详细的指南,特别是在这些年龄段的健康和生活质量估值方面。本文批判性地审查了现有基于偏好的测量方法评估儿童和青少年特定健康相关生活质量时所涉及的方法学考虑因素。它还描述了在儿童和青少年人群中开发和/或应用的现有通用基于偏好的测量方法的估值中所做出的方法学选择:AHUM、AQoL-6D、CHU9D、EQ-5D-Y、HUI2、HUI3、QWB、16D 和 17D。用于评估现有儿童和青少年特定通用基于偏好的测量方法的方法差异很大。虽然选择使用谁的偏好和视角是规范性辩论的问题,最终由报销机构和决策者决定,但围绕这些问题进行更多的研究将具有启发性,并丰富这些讨论。研究还可以为儿童和青少年健康状况估值所需的其他方法学选择提供信息。在由成年人进行的健康状况估值练习中,描述儿童的影响方面存在研究证据的差距,包括知情偏好的可能性;对于青少年来说,估值任务的适当性和可接受性,特别是涉及“死亡”状态的任务;青少年偏好的锚定;以及成人和青少年偏好的综合生成和使用。