Basque Health Service (Osakidetza), Debagoiena Integrated Healthcare Organisation, Research Unit, Arrasate-Mondragón, Guipúzcoa, Spain.
Kronikgune Institute for Health Service Research, Barakaldo, Spain.
Alzheimers Res Ther. 2020 Dec 11;12(1):166. doi: 10.1186/s13195-020-00737-9.
The LipiDiDiet trial showed that Souvenaid, a medical food, might delay progression to dementia in prodromal Alzheimer's disease (AD). The objective of this study was to assess the cost-utility of Souvenaid compared to placebo in patients with prodromal AD under the conditions applied in that trial.
A discrete event simulation model was developed based on the LipiDiDiet trial and a literature review to assess the cost-utility of Souvenaid from a societal perspective considering direct and indirect costs. For both intervention and control groups, patient trajectories in terms of functional decline on the Clinical Dementia Rating Sum of Boxes (CDR-SB) scale in LipiDiDiet were reproduced statistically with mixed models by assigning time until events to simulated patients. From the societal perspective, four scenarios were analysed by combining different options for treatment duration and diagnostic test cost. Univariate sensitivity analysis assessed parameter uncertainties.
Validation results at year 2 of disease progression fit with CDR-SB progression in LipiDiDiet. The incremental cost-utility ratio (ICUR) in the baseline case was €22,743/quality-adjusted life year (QALY). All scenarios rendered an ICUR lower than €25,000/QALY (the societal threshold). Moreover, the treatment option was cost-saving and increased health benefits when diagnostic costs were not considered and treatment was only administered during the prodromal stage.
Treating prodromal AD with Souvenaid is a cost-effective intervention in all scenarios analysed. The LipiDiDiet trial showed a modest improvement in disease course but as the social costs of AD are very high, the intervention was efficient. Assessing small benefits at specific stages of AD is relevant because it is reasonable to expect that no effective, safe and affordable disease-modifying therapies will become available in the short to medium term.
LipiDiDiet 试验表明，一种医学食品 Souvenaid 可能延缓前驱期阿尔茨海默病（AD）向痴呆的进展。本研究旨在评估在该试验条件下，与安慰剂相比，Souvenaid 在前驱期 AD 患者中的成本-效用。
基于 LipiDiDiet 试验和文献回顾，开发了一个离散事件模拟模型，从社会角度评估 Souvenaid 的成本-效用，同时考虑直接和间接成本。对于干预组和对照组，通过为模拟患者分配事件发生的时间，使用混合模型对 LipiDiDiet 中 CDR-SB 量表上的功能下降轨迹进行了统计学再现。从社会角度出发，通过结合不同的治疗持续时间和诊断测试成本选择方案，分析了四种情况。单变量敏感性分析评估了参数不确定性。
疾病进展 2 年的验证结果与 LipiDiDiet 中的 CDR-SB 进展相吻合。基线情况下的增量成本-效用比（ICUR）为 22743 欧元/质量调整生命年（QALY）。所有情况下的 ICUR 均低于 25000 欧元/QALY（社会阈值）。此外，当不考虑诊断成本且仅在前驱期进行治疗时，该治疗方案具有成本效益并增加了健康效益。
在分析的所有情况下，用 Souvenaid 治疗前驱期 AD 都是一种具有成本效益的干预措施。LipiDiDiet 试验表明疾病进程有一定改善，但由于 AD 的社会成本非常高，该干预措施是有效的。在 AD 的特定阶段评估小的效益是相关的，因为短期内不太可能出现有效、安全和负担得起的疾病修饰疗法。
