Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA.
Pharmaceutical Outcomes Research and Policy Program, Department of Pharmacy, University of Washington, Seattle, WA, USA.
Value Health. 2019 Feb;22(2):161-167. doi: 10.1016/j.jval.2018.09.2841. Epub 2018 Oct 25.
The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration-approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC).
A 2-state Markov model, alive and dead, with a lifetime horizon.
Visual acuity (VA) and visual field (VF) were tracked to model quality-adjusted life-years (QALYs). VN led to an improvement in VA and VF that we assumed was maintained for 10 years followed by a 10-year waning period. The cost of VN was $850 000, and other direct medical costs for depression and trauma were included for a US healthcare system perspective. A modified societal perspective also included direct nonmedical costs and indirect costs.
VN provided an additional 1.3 QALYs over the remaining lifetime of an individual. The average total lifetime direct medical cost for individuals treated with VN was $1 039 000 compared with $213 400 for SoC, leading to an incremental cost-effectiveness ratio (ICER) of $643 800/QALY from the US healthcare system perspective. Direct nonmedical costs totalled $1 070 900 for VN and $1 203 300 for SoC, and indirect costs totalled $405 400 for VN and $482 900 for SoC, leading to an ICER of $480 100/QALY from the modified societal perspective.
At the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC. VN has important implications for both development and pricing of future gene therapies; therefore clinical and economic analyses must be carefully considered.
基因治疗药物 voretigene neparvovec（VN）是首个经美国食品和药物管理局批准用于治疗因超罕见 RPE65 介导的遗传性视网膜疾病导致的视力丧失的疗法。我们建立了 VN 与标准护理（SoC）相比的成本效用模型。
具有生存和死亡两种状态的两状态马尔可夫模型，具有终生的时间范围。
跟踪视力（VA）和视野（VF）以构建质量调整生命年（QALY）。我们假设 VN 可提高 VA 和 VF，且该疗效可维持 10 年，随后 10 年逐渐减弱。VN 的成本为 85 万美元，还包括了抑郁症和创伤的其他直接医疗费用，从美国医疗保健系统的角度来看。改良的社会视角还包括了直接非医疗费用和间接费用。
VN 在个体的剩余生命周期内提供了额外的 1.3 个 QALY。接受 VN 治疗的个体的平均终生直接医疗总成本为 103.9 万美元，而 SoC 的总成本为 21.34 万美元，这导致从美国医疗保健系统的角度来看，VN 的增量成本效益比（ICER）为 643800 美元/QALY。VN 的直接非医疗费用总计为 107.09 万美元，SoC 的直接非医疗费用总计为 120.33 万美元，VN 的间接费用总计为 40.54 万美元，SoC 的间接费用总计为 48.29 万美元，这导致从改良的社会视角来看，VN 的 ICER 为 480100 美元/QALY。
按照当前的价格，VN 不太可能达到传统的与 SoC 相比的成本效益标准。VN 对未来基因治疗的开发和定价都有重要影响；因此，必须仔细考虑临床和经济分析。
