The Comparative Health Outcomes, Policy & Economics (CHOICE) Institute, Department of Pharmacy, University of Washington, Seattle, WA.
Faculty of Pharmaceutical Sciences and Division of Respiratory Medicine, Department of Medicine, University of British Columbia, Vancouver, British Columbia, Canada.
Blood Adv. 2023 Feb 14;7(3):365-374. doi: 10.1182/bloodadvances.2021006281.
Sickle cell disease (SCD) is a severe monogenic disease associated with high morbidity, mortality, and a disproportionate burden on Black and Hispanic communities. Our objective was to estimate the total healthcare costs and out-of-pocket (OOP) costs attributable to SCD among commercially insured individuals over their nonelderly lifetimes (0 to 64 years of age). We constructed a retrospective cohort of individuals with diagnosed SCD using Truven Health Marketscan commercial claims data from 2007 through 2018, compared with matched control subjects from the Medical Expenditure Panel Survey. We estimated Kaplan-Meier sample average costs using previously reported survival curves for SCD and control subjects. Individuals with SCD (20 891) and control subjects (33 588) were included in our analysis. The SCD sample had a mean age of 25.7 (standard deviation, 17.4) years; 58.0% were female. Survival-adjusted costs of SCD peaked at age 13 to 24 years and declined at older ages. There was no significant difference in total medical costs or OOP costs between the sexes. SCD-attributable costs over 0 to 64 years of age were estimated to be $1.6 million (95% confidence interval [CI], $1.3M-$1.9M) and $1.7 million (95% CI, $1.4M-$2.1M) for females and males with SCD, respectively. The corresponding OOP estimates were $42 395 (95% CI, $34 756-$50 033) for females and $45 091 (95% CI, $36 491-$53 691) for males. These represent a 907% and 285% increase in total medical and OOP costs over control subjects, respectively. Although limited to the commercially insured population, these results indicate that the direct economic burden of SCD is substantial and peaks at younger ages, suggesting the need for curative and new medical therapies.
镰状细胞病（SCD）是一种严重的单基因疾病，与高发病率、高死亡率以及黑人和西班牙裔社区的不成比例负担有关。我们的目的是估计在非老年期（0 至 64 岁）内，商业保险个体中归因于 SCD 的总医疗保健成本和自付费用（OOP）。我们使用 Truven Health Marketscan 商业索赔数据，从 2007 年至 2018 年，构建了一个患有确诊 SCD 的个体回顾性队列，并与来自医疗支出面板调查的匹配对照个体进行了比较。我们使用先前报道的 SCD 和对照个体的生存曲线，估计了 Kaplan-Meier 样本平均成本。我们的分析纳入了 20891 名 SCD 个体和 33588 名对照个体。SCD 样本的平均年龄为 25.7 岁（标准差为 17.4）；58.0%为女性。SCD 患者的调整生存成本在 13 至 24 岁时达到峰值，之后随着年龄的增长而下降。男女之间的总医疗费用或 OOP 费用没有显著差异。在 0 至 64 岁期间，估计 SCD 归因于 SCD 的成本为女性患者 160 万美元（95%置信区间[CI]，130 万至 190 万美元）和男性患者 170 万美元（95%CI，140 万至 200 万美元）。相应的 OOP 估计值分别为女性患者 42395 美元（95%CI，34756 美元至 50033 美元）和男性患者 45091 美元（95%CI，36491 美元至 53691 美元）。这分别代表对照个体的总医疗和 OOP 成本的 907%和 285%的增加。尽管仅限于商业保险人群，但这些结果表明 SCD 的直接经济负担很大，并且在年轻时达到峰值，这表明需要新的治疗方法和疗法。
