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澳大利亚基于人群的扩大遗传性疾病生殖携带者筛查的成本效益:微观模拟分析

Cost-effectiveness of population-based expanded reproductive carrier screening for genetic diseases in Australia: a microsimulation analysis.

作者信息

Schofield Deborah, Lee Evelyn, Parmar Jayamala, Castelo Taboada Adriana, Hobbs Matthew, Laing Nigel, Shrestha Rupendra

机构信息

Centre for Economic Impacts of Genomic Medicine, Macquarie University, Macquarie Park, NSW, Australia.

Leeder Centre for Health Policy, Economics & Data (The Leeder Centre), University of Sydney, Sydney, NSW, Australia.

出版信息

Eur J Hum Genet. 2025 Apr 16. doi: 10.1038/s41431-025-01835-8.

Abstract

Using the Australian Census survey 2021 as base population, a microsimulation model, PreconMOD was developed to evaluate the cost-effectiveness of population-based expanded reproductive carrier screening (RCS) for 569 recessive conditions from the health service and societal perspectives. The model simulated the effect of expanded RCS including the downstream interventions for at-risk couples on cost and outcomes. The comparators were (i) no population screening (ii) limited screening for cystic fibrosis, spinal muscular atrophy, and fragile X syndrome and (iii) a 300 conditions screening panel. Averted affected births and health service cost with expanded RCS were projected to year 2061. At a 50% uptake, our model predicts that expanded RCS is cost saving (i.e., higher quality-adjusted life-years and lower costs) compared with other screening strategies in the model from the health service and societal perspectives. The number of affected births averted in a single cohort would increase from 84 [95% confidence interval (CI) 60-116] with limited screening to 2067 (95%CI 1808-2376) with expanded RCS. Expanded RCS was cost-saving compared to the 300-conditions screening panel. Indirect cost accounted for about one-third of the total costs associated with recessive disorders. Our model predicts that the direct treatment cost associated with current limited 3 genes screening would increase by 20% each year to A$73.4 billion to the health system by 2061. Our findings contribute insights on the cost burden of genetic diseases and the economic benefits of expanded RCS to better informed resource allocation decisions.

摘要

以2021年澳大利亚人口普查数据作为基础人群,开发了一种微观模拟模型PreconMOD,从卫生服务和社会角度评估针对569种隐性疾病的基于人群的扩大生殖携带者筛查(RCS)的成本效益。该模型模拟了扩大RCS(包括对高危夫妇的下游干预措施)对成本和结果的影响。比较对象为:(i)不进行人群筛查;(ii)对囊性纤维化、脊髓性肌萎缩症和脆性X综合征进行有限筛查;(iii)进行300种疾病的筛查组合。预计到2061年,扩大RCS可避免的患病出生数和卫生服务成本。在50%的接受率下,我们的模型预测,从卫生服务和社会角度来看,与模型中的其他筛查策略相比,扩大RCS可节省成本(即更高的质量调整生命年和更低的成本)。单个队列中避免的患病出生数将从有限筛查时的84例[95%置信区间(CI)60 - 116]增加到扩大RCS时的2067例(95%CI 1808 - 2376)。与300种疾病的筛查组合相比,扩大RCS节省了成本。间接成本约占与隐性疾病相关总成本的三分之一。我们的模型预测,到2061年,与目前有限的3基因筛查相关的直接治疗成本将以每年20%的速度增长,给卫生系统带来734亿澳元的成本。我们的研究结果为了解遗传疾病的成本负担以及扩大RCS的经济效益提供了见解,有助于做出更明智的资源分配决策。

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