雷珠单抗治疗新生血管性黄斑变性的比较疗效和成本效益再探讨。
The comparative effectiveness and cost-effectiveness of ranibizumab for neovascular macular degeneration revisited.
作者信息
Brown Gary C, Brown Melissa M, Lieske Heidi B, Turpcu Adam, Rajput Yamina
机构信息
The Center for Value-Based Medicine®, Box 6181, Hilton Head, SC 29938 USA ; The Eye Research Institute, Philadelphia, PA USA ; The Retina Service, Wills Eye Institute, Jefferson Medical College, Philadelphia, PA USA ; The Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA USA.
The Center for Value-Based Medicine®, Box 6181, Hilton Head, SC 29938 USA ; The Eye Research Institute, Philadelphia, PA USA ; The Research Department, Wills Eye Institute, Jefferson Medical College, Philadelphia, PA USA ; The Department of Ophthalmology, Emory University School of Medicine, Atlanta, GA USA.
出版信息
Int J Retina Vitreous. 2017 Feb 13;3:5. doi: 10.1186/s40942-016-0058-3. eCollection 2017.
BACKGROUND
To compare a near decade of follow-up, newer control cohort data, use of both the societal and third party insurer cost perspectives, and integration of unilateral/bilateral therapy on the comparative effectiveness and cost-effectiveness of intravitreal ranibizumab therapy for neovascular, age-related macular degeneration (AMD).
METHODS
Value-Based Medicine, 12-year, combined-eye model, cost-utility analysis employing MARINA and HORIZON clinical trial data. Preference-based comparative effectiveness outcomes were quantified in (1) QALY (quality-adjusted life-year) gain, and (2) percent improvement in quality-of-life, while cost-effectiveness outcomes were quantified in (3) the cost-utility ratio (CUR) and financial return-on-investment (ROI) to society.
RESULTS
Using MARINA and HORIZON trial data and a meta-analysis control cohort after 24 months, ranibizumab therapy conferred a combined-eye patient value (quality-of-life) gain of 16.3%, versus 10.4% found in 2006. The two-year direct ophthalmic medical cost for ranibizumab therapy was $46,450, a 33.8% real dollar decrease from 2006. The societal cost perspective CUR was -$242,920/QALY, indicating a $282,517 financial return-on-investment (ROI), or 12.3%/year to society for direct ophthalmic medical costs expended. The 3rd party insurer CUR ranged from $21,199/QALY utilizing all direct, medical costs, to $69,591/QALY using direct ophthalmic medical costs.
CONCLUSIONS
Ranibizumab therapy for neovascular AMD in 2015, considering treatment of both eyes, conferred greater patient value gain (comparative effectiveness) and improved cost-effectiveness than in 2006, as well as a large monetary return-on-investment to the Gross Domestic Product and nation's wealth. The model herein integrates important novel features for neovascular age-related macular degeneration, vitreoretinal cost effectiveness analyses, including: (1) treatment of both eyes, (2) a long-term, untreated control cohort, and (3) the use of societal costs.
背景
为比较近十年的随访情况、更新的对照队列数据、社会和第三方保险公司成本视角的使用，以及单/双侧治疗整合对玻璃体内注射雷珠单抗治疗新生血管性年龄相关性黄斑变性（AMD）的比较效果和成本效益的影响。
方法
基于价值医学的12年双眼模型，采用MARINA和HORIZON临床试验数据进行成本效用分析。基于偏好的比较效果结果通过以下方式量化：（1）质量调整生命年（QALY）增益，以及（2）生活质量改善百分比，而成本效益结果通过以下方式量化：（3）成本效用比（CUR）和对社会的财务投资回报率（ROI）。
结果
使用MARINA和HORIZON试验数据以及24个月后的荟萃分析对照队列，雷珠单抗治疗使双眼患者的价值（生活质量）增益达到16.3%，而2006年为10.4%。雷珠单抗治疗的两年直接眼科医疗成本为46,450美元，与2006年相比实际美元成本下降了33.8%。社会成本视角的CUR为-$242,920/QALY，表明财务投资回报率（ROI）为282,517美元，即社会为所支出的直接眼科医疗成本每年获得12.3%的回报。第三方保险公司的CUR范围从使用所有直接医疗成本时的21,199美元/QALY到使用直接眼科医疗成本时的69,591美元/QALY。
结论
考虑双眼治疗，2015年用于新生血管性AMD的雷珠单抗治疗比2006年赋予患者更大的价值增益（比较效果）并改善了成本效益，同时对国内生产总值和国家财富产生了巨大的货币投资回报率。本文中的模型整合了新生血管性年龄相关性黄斑变性、玻璃体视网膜成本效益分析的重要新特征，包括：（1）双眼治疗，（2）长期未治疗的对照队列，以及（3）社会成本的使用。
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