Cost-effectiveness analysis of intensive and emerging rehabilitation therapies in children with cerebral palsy: an observational cohort study using real-world evidence and microsimulation modelling.

IF 3.4 3区 经济学 Q1 ECONOMICS
Diana Marcela Nova-Díaz, Sergio Aguilera-Albesa, Eduardo Sánchez-Iriso
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引用次数: 0

Abstract

Objective: To evaluate the short- and long-term cost-effectiveness of integrating Intensive and Emerging Rehabilitation Therapies into standard care for children with cerebral palsy (CP), compared to standard care alone, through a hybrid approach combining prospective real-world data analysis and individual-level microsimulation over a lifetime horizon.

Methods: A prospective observational cohort of 148 children with CP, stratified by Gross Motor Function Classification System levels, was followed over 12 months. Short-term incremental costs and quality-adjusted life years (QALYs) were estimated using seemingly unrelated regression equations (SURE) based on EQ-5D-Y scores. Costs were assessed from the Spanish public healthcare system perspective. To extrapolate long-term outcomes, an individual-level microsimulation model projected costs and QALYs over a 30-year horizon, applying a 3% annual discount rate.

Results: Compared with standard treatment, Therasuit and intensive physiotherapy demonstrated the most favourable cost-effectiveness profiles. Therasuit generated 0.222 additional QALYs at an incremental cost-effectiveness ratio (ICER) of €18,830/QALY, while intensive physiotherapy generated 0.216 additional QALYs at €31,772/QALY. Other therapies, including occupational therapy and hippotherapy, were dominated by standard care. Long-term microsimulation provided additional insights beyond short-term findings by capturing delayed benefits, which in some cases led to different cost-effectiveness rankings among therapies. Therasuit produced 5.49 additional QALYs at an ICER of €12,922/QALY compared to standard care, and intensive physiotherapy produced 4.92 additional QALYs at €25,789/QALY. Homeopathy and the Petö Method were cost-effective under broader willingness-to-pay thresholds but were less efficient.

Conclusions: Therasuit and intensive physiotherapy are high-value options when added to standard care for children with CP. Findings support prioritising Intensive and Emerging Rehabilitation Therapies using real-world evidence and modelling to guide sustainable healthcare decision-making.

脑瘫儿童强化和新兴康复治疗的成本-效果分析:一项使用真实世界证据和微观模拟模型的观察性队列研究。
目的:通过结合前瞻性真实世界数据分析和个体微观模拟的混合方法,评估将强化和新兴康复疗法纳入脑瘫(CP)儿童标准治疗的短期和长期成本效益,与单独标准治疗相比。方法:对148名CP患儿进行为期12个月的前瞻性观察队列研究,按大运动功能分级系统分级。使用基于EQ-5D-Y评分的看似无关的回归方程(SURE)估计短期增量成本和质量调整寿命年(QALYs)。从西班牙公共医疗系统的角度评估成本。为了推断长期结果,采用3%的年贴现率,个人层面的微观模拟模型预测了30年的成本和质量年增长率。结果:与标准治疗相比,Therasuit和强化物理治疗表现出最有利的成本-效果。治疗方案产生了0.222个额外的QALY,增量成本效益比(ICER)为18,830欧元/QALY,而强化物理治疗产生了0.216个额外的QALY,成本效益比为31,772欧元/QALY。其他疗法,包括职业疗法和海马疗法,以标准治疗为主。长期微观模拟通过捕捉延迟效益提供了短期发现之外的额外见解,这在某些情况下导致不同疗法的成本效益排名不同。与标准护理相比,Therasuit产生了5.49个额外的QALY, ICER为12,922欧元/QALY,强化物理治疗产生了4.92个额外的QALY, ICER为25,789欧元/QALY。顺势疗法和Petö方法在更广泛的支付意愿阈值下具有成本效益,但效率较低。结论:治疗和强化物理治疗是CP儿童标准护理的高价值选择。研究结果支持使用真实世界的证据和模型来优先考虑强化和新兴康复治疗,以指导可持续的医疗保健决策。
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来源期刊
CiteScore
3.90
自引率
4.20%
发文量
59
审稿时长
13 weeks
期刊介绍: Health Economics Review is an international high-quality journal covering all fields of Health Economics. A broad range of theoretical contributions, empirical studies and analyses of health policy with a health economic focus will be considered for publication. Its scope includes macro- and microeconomics of health care financing, health insurance and reimbursement as well as health economic evaluation, health services research and health policy analysis. Further research topics are the individual and institutional aspects of health care management and the growing importance of health care in developing countries.
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