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儿科经济评价中健康状态估值的挑战:QALYs 是否被禁止?

Challenges in health state valuation in paediatric economic evaluation: are QALYs contraindicated?

机构信息

Child Health Evaluative Sciences, The Hospital for Sick Children, Toronto, Ontario, Canada.

出版信息

Pharmacoeconomics. 2011 Aug;29(8):641-52. doi: 10.2165/11591570-000000000-00000.

Abstract

With the growth in the use of health economic evaluation to inform healthcare resource allocation decisions, the challenges in applying standard methods to child health have become apparent. A unique limitation is the paucity of child-specific preference-based measures. A single, valid, preference-based measure of utility that can be used in children of all ages does not exist. Thus, the ability to derive a QALY for use in cost-utility analysis (CUA) is compromised. This paper presents and discusses existing and novel options for deriving utilities for paediatric health states for use in CUAs. While a direct elicitation may be preferred, a child's ability to complete a standard gamble or time trade-off task is hampered by cognitive and age limitations. The abstract notions contained in indirect instruments such as the EQ-5D and Health Utilities Index may also pose challenges for young children. Novel approaches to overcome these challenges include the development of age-appropriate instruments such as the EQ-5D-Y, the development of new child-specific utility instruments such as the Child Health Utility-9D and the re-calibration of existing adult instruments to derive preference weights for health states from children themselves. For children aged <6 years, researchers have little choice but to use a proxy reporter such as parents. While parents may be reliable reporters for physical activity limitations and externally manifest symptoms, their ability to accurately report on subjective outcomes such as emotion is questionable. Catalogues of utility weights for a range of conditions are increasingly becoming available but retain many of the same limitations as valuing health states from children or from proxies. Given the dynamic relationship in quality of life (QOL) between family members when a child is ill, it seems appropriate to consider a 'family perspective' rather than an individual perspective in child health state valuation. In a collective approach, health state utilities derived from multiple family members may be combined mathematically. Alternatively, in a unitary approach, a single utility estimate may be determined to represent the family's perspective. This may include deriving utilities through parent-child dyad estimation or by using a household model that combines the utility weights of the patient and family members, incorporating reciprocal QOL effects. While these various approaches to child health state valuation represent novel research developments, the measurement challenges and threats to validity persist. Given the importance of non-health benefits to child health, especially in the domains of education and public policy, it may be worthwhile to consider an approach that allows incorporation of externalities to produce a cost-benefit analysis. The use of discrete-choice methods to assess willingness to pay for novel child health interventions holds promise as a means to produce meaningful economic evidence. Regardless of the approach taken, the highest degree of methodological rigour is essential. The increasing attention being paid by health economic researchers to the measurement challenges of paediatric health state valuation can only increase the value of child health economic evidence for decision making.

摘要

随着健康经济评估在医疗资源配置决策中的应用不断增加,应用标准方法评估儿童健康所面临的挑战已经变得明显。一个独特的局限性是缺乏儿童专用的偏好基础衡量标准。目前还没有一种单一的、有效的、适用于所有年龄段儿童的基于偏好的效用衡量标准。因此,用于成本效用分析(CUA)的 QALY 的推导能力受到了影响。本文提出并讨论了现有的和新颖的方法,用于推导儿科健康状况的效用,以便在 CUA 中使用。虽然直接启发法可能是首选,但由于认知和年龄限制,儿童完成标准博弈或时间权衡任务的能力受到阻碍。间接工具(如 EQ-5D 和健康效用指数)中包含的抽象概念也可能对幼儿构成挑战。克服这些挑战的新方法包括开发适合年龄的工具,如 EQ-5D-Y;开发新的儿童专用效用工具,如儿童健康效用-9D;以及重新校准现有的成人工具,以便从儿童自身推导出健康状况的偏好权重。对于<6 岁的儿童,研究人员别无选择,只能使用父母等代理报告者。虽然父母可能是身体活动限制和外在表现症状的可靠报告者,但他们准确报告情绪等主观结果的能力值得怀疑。越来越多的适用于一系列疾病的效用权重目录正在出现,但仍保留了从儿童或从代理那里评估健康状况的相同局限性。考虑到儿童生病时家庭成员之间生活质量(QOL)的动态关系,在儿童健康状况评估中考虑“家庭视角”而不是“个体视角”似乎是合适的。在集体方法中,可以通过数学方法组合来自多个家庭成员的健康状况效用。或者,在单一方法中,可以确定一个单一的效用估计值来代表家庭的观点。这可能包括通过父母-子女二元关系估计来推导效用,或者使用结合患者和家庭成员效用权重的家庭模型,同时考虑互惠的 QOL 影响。虽然这些儿童健康状况评估的各种方法代表了新的研究进展,但测量挑战和有效性威胁仍然存在。考虑到非健康效益对儿童健康的重要性,特别是在教育和公共政策领域,考虑采用一种允许纳入外部因素以产生成本效益分析的方法可能是值得的。使用离散选择方法评估对新型儿童健康干预措施的支付意愿有望成为产生有意义的经济证据的一种手段。无论采用哪种方法,都必须采用最高程度的方法学严谨性。健康经济研究人员对儿科健康状况评估的测量挑战的日益关注只会增加儿童健康经济证据在决策中的价值。

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