美国桑菲利波综合征的经济负担。

Frederick Ashby, Haesuk Park, Mikael Svensson, Coy D Heldermon
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引用次数: 0

摘要

简介:Sanfilippo综合征在美国是一种罕见的疾病和致命的遗传性疾病,没有美国食品药品监督管理局批准的治疗方法,也没有对经济疾病负担进行全面评估。目的:开发一个模型,从2023年起,使用有价值的无形资产(残疾调整后的寿命损失)和间接负担(护理人员生产力损失)来估计美国桑菲利波综合征的经济负担。设计和设置:使用关于Sanfilippo综合征残疾的公开文献和2010年全球疾病负担研究的14个残疾权重,生成了一个多阶段共病模型。还使用美国疾病控制与预防中心全国共病调查的数据、桑菲利波综合征护理人员负担的回顾性研究和联邦收入数据估计了护理人员心理健康负担和护理人员生产力损失的归因增加。货币估值调整为2023美元,并从2023年起给予3%的贴现率。主要结果指标:计算每个年龄组每年桑菲利波综合征的逐年发病率和患病率,并通过与健康调整预期寿命(HALE)进行比较来计算因患者残疾而损失的逐年残疾调整寿命(DALYs),同时考虑到因过早死亡而损失的寿命(YLLs)和残疾寿命(YLDs)。无形资产以2023美元计价,经通胀调整后贴现,以提供疾病的经济负担。结果:从2023-2043年,按照目前的护理标准,桑菲利波综合征在美国造成的总体经济负担估计为15.5亿美元。从出生时起,每个患有桑菲利波综合征的孩子给单个家庭带来的负担超过了586万美元的现值。这些数字也是一个保守的估计,因为它们没有考虑与该疾病相关的直接成本,因为文献中目前还没有关于Sanfilippo综合征直接医疗成本的大量原始数据。结论:Sanfilippo综合征是一种罕见的溶酶体储存性疾病,但与该疾病相关的个体家庭的严重负担显示出相当大的累积影响。我们的模型代表了第一个与桑菲利波综合征相关的疾病负担值估计,并强调了桑菲利波综合症的巨大发病率和死亡率负担。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Economic Burden of Sanfilippo Syndrome in the United States.

Economic Burden of Sanfilippo Syndrome in the United States.

Economic Burden of Sanfilippo Syndrome in the United States.

Economic Burden of Sanfilippo Syndrome in the United States.

Introduction: Sanfilippo syndrome is a rare disease and fatal genetic disorder with no FDA-approved treatment in the United States (US), and no comprehensive assessment of economic disease burden is available.

Objectives: To develop a model to estimate the economic burden associated with Sanfilippo syndrome in the US using direct costs, indirect costs and valued intangibles (disability-adjusted life years, or DALYs) from 2023 onward.

Design and setting: A multistage comorbidity model was generated based on Sanfilippo syndrome symptoms, and disability weights from the 2010 Global Burden of Disease Study. Attributable increase in caregiver mental health burden were estimated using data from the CDC National Comorbidity Survey and retrospective studies on caregiver burden. Direct costs were approximated from the 2019 EveryLife Foundation survey, and indirect costs were estimated from Federal income data. Monetary valuations were adjusted to USD 2023 and given a 3% discount rate from 2023 onward.

Main outcome measures: Incidence of Sanfilippo syndrome was calculated for each year, and year-over-year DALYs due to patient years lived with disability (YLDs) and years life lost (YLLs) were calculated by comparing to the health-adjusted life expectancy (HALE) in the US. Direct and indirect costs were calculated for each simulated patient from onset of symptoms to death.

Results: From 2023-2043, overall economic burden in the US attributable to Sanfilippo syndrome was estimated to be $2.04 billion USD present value (2023) with current standard of care. The burden to individual families exceeded $8 million present value from time of birth per child born with Sanfilippo syndrome.

Conclusion: Sanfilippo syndrome is a rare lysosomal storage disease, however the severe burden associated with the disease for individual families demonstrates a considerable cumulative impact. Our model represents the first disease burden value estimate associated with Sanfilippo syndrome, and underscores the substantial morbidity and mortality burden of Sanfilippo syndrome.

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