Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, MA, USA.
UCB Pharma, Brussels, Belgium.
Amyotroph Lateral Scler Frontotemporal Degener. 2023 Aug;24(5-6):436-448. doi: 10.1080/21678421.2023.2165947. Epub 2023 Feb 7.
This review sought to gain a comprehensive, up-to-date understanding of the epidemiology and cost and healthcare resource use (HCRU) burden of amyotrophic lateral sclerosis (ALS) in the US, at a patient and national level. A targeted literature review (TLR) to identify epidemiological evidence (prevalence, incidence, mortality, survival), and systematic literature review (SLR) to identify cost and HCRU data published since January 2016, were performed. MEDLINE databases and Embase searches were conducted in January 2021. Key congresses (2019-2020) and bibliographies of relevant SLRs were hand-searched. Two high-quality SLRs were reviewed for additional cost data published between January 2001-2015. Registry and database studies were prioritized for epidemiological evidence. To allow comparison between studies in this publication, only evidence from the US was considered, with costs inflated to the 2020/2021 cost-year and converted to US dollars. Eight studies from the epidemiology TLR, and eighteen from the cost and HCRU SLR, were extracted. Reported ALS incidence in the US was ∼1.5 per 100,000 person-years, and point prevalence ranged from 3.84-5.56 per 100,000 population. Total US national costs spanned ∼$212 million-∼$1.4 billion USD/year, and variably consisted of direct costs associated with HCRU and indirect costs. : The national cost of ∼$1.02 billion USD/year (estimated using a prevalence of 16,055 cases) best aligns with prevalence estimates found in the TLR (equating to ∼13,000-18,000 cases). However, large-scale, population-based studies are necessary to precisely assess US epidemiology of ALS and capture all costs needed to inform cost-effectiveness models and resource planning.
本综述旨在全面了解美国的肌萎缩侧索硬化症（ALS）在患者和国家层面的流行病学、成本和医疗资源使用（HCRU）负担。我们进行了有针对性的文献综述（TLR）以确定流行病学证据（患病率、发病率、死亡率、生存率），并进行了系统文献综述（SLR）以确定自 2016 年 1 月以来发表的成本和 HCRU 数据。2021 年 1 月进行了 MEDLINE 数据库和 Embase 搜索。还手检了相关 SLR 的重点会议（2019-2020 年）和参考文献。为了获取 2001 年至 2015 年期间发表的额外成本数据，还对两项高质量的 SLR 进行了审查。优先考虑登记处和数据库研究来获取流行病学证据。为了使本出版物中的研究之间能够进行比较，仅考虑了来自美国的数据，将成本膨胀到 2020/2021 成本年度，并转换为美元。从流行病学 TLR 中提取了 8 项研究，从成本和 HCRU SLR 中提取了 18 项研究。美国报告的 ALS 发病率约为每 10 万人年 1.5 例，时点患病率范围为每 10 万人 3.84-5.56 例。美国全国总成本范围为 2.12 亿至 14 亿美元/年，其中包括与 HCRU 相关的直接成本和间接成本。：使用 16055 例病例的患病率估计，美国 ALS 的全国成本约为 10.2 亿美元/年（估计），这与 TLR 中发现的患病率估计最为吻合（相当于 13000-18000 例）。然而，需要进行大规模的基于人群的研究，以准确评估美国 ALS 的流行病学情况并捕获所有用于为成本效益模型和资源规划提供信息的成本。
