Cost-Effectiveness of Newborn Screening for Spinal Muscular Atrophy in Australian Hospitals.

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-06-01 Epub Date: 2025-04-27 DOI:10.1007/s40120-025-00744-8

Ian R Woodcock, Didu S Kariyawasam, Maina P Kava, Eppie M Yiu, Damian Clark, Jane Adams, Matthias Bischof, Adrian Peacock, Colman Taylor, Nicholas J C Smith

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引用次数: 0

Abstract

Introduction: This analysis evaluated the cost-effectiveness of newborn screening (NBS) for spinal muscular atrophy (SMA) from the perspective of Australian state hospital payers.

Methods: A cost-utility analysis consisting of a decision tree and Markov cohort designed to calculate the difference in costs and health outcomes between two scenarios: (1) disease-modifying treatment (DMT) for SMA after diagnosis through NBS, and (2) DMT for SMA after diagnosis as symptoms appear. A population of 295,906 newborns was modeled, based on the total number of live births in Australia in 2023. Inputs included screening parameters, epidemiology inputs, SMA natural history data and DMT parameters (nusinersen and onasemnogene abeparvovec), costs, and health-related quality of life parameters. Assumed participation in NBS was 100%. A one-way sensitivity analysis and probabilistic sensitivity analysis were conducted to examine the impact of parameter uncertainty.

Results: There were 30 patients identified with SMA, of whom 25 patients would be eligible for presymptomatic treatment. NBS for SMA was dominant compared with no NBS for SMA. On a population level, NBS demonstrated a lifetime gain of 267 quality-adjusted life years (QALY) and incremental costs of -AUD$3,983,263 (i.e., cost savings). Every dollar invested in NBS would save hospitals $3.69. Deterministic and probabilistic sensitivity analyses demonstrated the robustness of the base-case results.

Conclusion: NBS for SMA was dominant compared with no NBS for SMA in Australia from a state and territory payer perspective. Universal implementation of NBS for SMA would support access equity, as well as early diagnosis and treatment in infants with SMA, potentially leading to improved outcomes.

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澳大利亚医院新生儿脊髓性肌萎缩症筛查的成本效益

本分析从澳大利亚州立医院支付者的角度评估了新生儿脊髓性肌萎缩症（SMA）筛查（NBS）的成本效益。方法：采用决策树和马尔可夫队列进行成本效用分析，旨在计算两种情况下的成本和健康结果差异：(1)通过NBS诊断后对SMA进行疾病改善治疗（DMT），(2)在诊断后出现症状时对SMA进行DMT。根据2023年澳大利亚活产婴儿总数，对295906名新生儿进行了建模。输入包括筛选参数、流行病学输入、SMA自然史数据和DMT参数（nusinersen和onasemnogene abeparvovec）、成本和与健康相关的生活质量参数。假设全国统计局的参与率为100%。通过单向灵敏度分析和概率灵敏度分析考察了参数不确定性的影响。结果：30例SMA患者，其中25例符合症状前治疗条件。与无NBS相比，SMA的NBS占主导地位。在人口水平上，NBS证明了终身增益267质量调整生命年（QALY）和- 3,983,263澳元的增量成本（即成本节约）。在国家统计局每投入1美元，医院将节省3.69美元。确定性和概率敏感性分析证明了基本情况结果的稳健性。结论：从澳大利亚州和地区支付者的角度来看，与没有NBS相比，NBS在SMA方面占主导地位。针对SMA的国家统计局的普遍实施将支持获得公平，以及对SMA婴儿的早期诊断和治疗，可能导致改善的结果。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

期刊介绍： Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. 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