Abstract：
UNASSIGNED: Etranacogene dezaparvovec (EDZ), Hemgenix, is a gene therapy recently approved for people with hemophilia B (PwHB).
UNASSIGNED: To estimate long-term clinical impact and cost of EDZ in the United States (US).
UNASSIGNED: A decision-analytic model was developed to evaluate the long-term impact of introducing EDZ for PwHB over a 20-year time horizon. Factor IX (FIX) prophylaxis comparator was a weighted average of different FIX prophylaxis regimens based on US market share data. We compared a scenario in which EDZ is introduced in the US versus a scenario without EDZ. Clinical inputs (annualized FIX-treated bleed rate; adverse event rates) were obtained from HOPE-B phase 3 trial. EDZ durability input was sourced from an analysis predicting long-term FIX activity with EDZ. EDZ one-time price was assumed at $3.5 million. Other medical costs, including FIX prophylaxis, disease monitoring, bleed management, and adverse events were from literature. The model estimated annual and cumulative costs, treated bleeds, and joint procedures over 20 years from EDZ introduction.
UNASSIGNED: Approximately 596 PwHB were eligible for EDZ. EDZ uptake was estimated to avert 11,282 bleeds and 64 joint procedures over 20 years. Although adopting EDZ resulted in an annual excess cost over years 1-5 (mean: $53 million annually, total $265 million), annual cost savings were achieved beginning in year 6 (mean: $172 million annually; total $2.58 billion in years 6-20). The total cumulative 20-year cost savings was $2.32 billion, with cumulative cost savings beginning in year 8.
UNASSIGNED: Introducing EDZ to treat PwHB is expected to result in cost savings and patient benefit over 20 years. Initiating PwHB on EDZ sooner can produce greater and earlier savings and additional bleeds avoided. These results may be a conservative estimate of the full value of EDZ, as PwHB would continue to accrue savings beyond 20 years.
This analysis assessed the long-term clinical and financial impact of introducing EDZ in the United States of America for people with severe or moderately severe hemophilia B. A decision-analytic model was developed comparing a scenario with EDZ and one without EDZ over 20 years. Introducing EDZ would avert 11,292 bleeds and 64 joint procedures over 20 years and would achieve cumulative cost savings in year 8, with a total cumulative 20-year cost saving of $2.32 billion.
UNASSIGNED: To estimate long-term clinical impact and cost of EDZ in the United States (US).
UNASSIGNED: A decision-analytic model was developed to evaluate the long-term impact of introducing EDZ for PwHB over a 20-year time horizon. Factor IX (FIX) prophylaxis comparator was a weighted average of different FIX prophylaxis regimens based on US market share data. We compared a scenario in which EDZ is introduced in the US versus a scenario without EDZ. Clinical inputs (annualized FIX-treated bleed rate; adverse event rates) were obtained from HOPE-B phase 3 trial. EDZ durability input was sourced from an analysis predicting long-term FIX activity with EDZ. EDZ one-time price was assumed at $3.5 million. Other medical costs, including FIX prophylaxis, disease monitoring, bleed management, and adverse events were from literature. The model estimated annual and cumulative costs, treated bleeds, and joint procedures over 20 years from EDZ introduction.
UNASSIGNED: Approximately 596 PwHB were eligible for EDZ. EDZ uptake was estimated to avert 11,282 bleeds and 64 joint procedures over 20 years. Although adopting EDZ resulted in an annual excess cost over years 1-5 (mean: $53 million annually, total $265 million), annual cost savings were achieved beginning in year 6 (mean: $172 million annually; total $2.58 billion in years 6-20). The total cumulative 20-year cost savings was $2.32 billion, with cumulative cost savings beginning in year 8.
UNASSIGNED: Introducing EDZ to treat PwHB is expected to result in cost savings and patient benefit over 20 years. Initiating PwHB on EDZ sooner can produce greater and earlier savings and additional bleeds avoided. These results may be a conservative estimate of the full value of EDZ, as PwHB would continue to accrue savings beyond 20 years.
This analysis assessed the long-term clinical and financial impact of introducing EDZ in the United States of America for people with severe or moderately severe hemophilia B. A decision-analytic model was developed comparing a scenario with EDZ and one without EDZ over 20 years. Introducing EDZ would avert 11,292 bleeds and 64 joint procedures over 20 years and would achieve cumulative cost savings in year 8, with a total cumulative 20-year cost saving of $2.32 billion.
摘要：
简介：Etranacogenedezaparvovec（EDZ），血友病,是最近批准用于血友病B（PwHB）患者的基因疗法。目的：评估EDZ在美国（US）的长期临床影响和成本。方法：开发了一个决策分析模型，以评估在20年的时间范围内将EDZ引入PwHB的长期影响。因子IX（FIX）预防比较是基于美国市场份额数据的不同FIX预防方案的加权平均值。我们比较了在美国引入EDZ的场景与没有EDZ的场景。从HOPE-B3期试验获得临床输入(年度FIX治疗出血率;不良事件率)。EDZ耐久性输入来自预测EDZ长期FIX活性的分析。EDZ的一次性价格假定为350万美元。其他医疗费用,包括FIX预防，疾病监测,出血管理,不良事件来自文献。该模型估计了年度和累计成本，治疗出血，和联合程序超过20年从EDZ引入。结果：约596个PwHB符合EDZ条件。EDZ吸收估计在20年内避免了11,282次出血和64次联合手术。虽然采用EDZ导致了1-5年的年度超额成本(平均：每年5300万美元，总计2.65亿美元），从第6年开始实现了年度成本节约(平均：每年1.72亿美元；第6-20年总计25.8亿美元)。累计20年累计节约成本23.2亿元,从第8年开始累计节省成本。结论：采用EDZ治疗PwHB有望在20年内节省成本并使患者受益。尽早在EDZ上启动PwHB可以产生更大和更早的节省，并避免了额外的出血。这些结果可能是EDZ全值的保守估计，因为PwHB将继续积累超过20年的储蓄。
该分析评估了在美国引入EDZ对重度或中度血友病B患者的长期临床和财务影响。开发了一个决策分析模型，比较了EDZ和没有EDZ的情况20年。引入EDZ将在20年内避免11,292例出血和64例联合程序，并将在第8年实现累计成本节约，20年累计成本节约总额为23.2亿美元。
该分析评估了在美国引入EDZ对重度或中度血友病B患者的长期临床和财务影响。开发了一个决策分析模型，比较了EDZ和没有EDZ的情况20年。引入EDZ将在20年内避免11,292例出血和64例联合程序，并将在第8年实现累计成本节约，20年累计成本节约总额为23.2亿美元。
