Opportunity Cost of Funding Drugs for Rare Diseases: The Cost-Effectiveness of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
Opportunity Cost of Funding Drugs for Rare Diseases: The Cost-Effectiveness of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
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DOI：
10.1177/0272989x14539731
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发表时间：
2014-11-01
影响因子：
3.6
通讯作者：
Evans, Gerald A.
中科院分区：
文献类型：
--
作者：
Coyle, Doug;Cheung, Matthew C.;Evans, Gerald A.
背景关于罕见病药物的资金问题，已经提出了伦理和经济方面的关切。本文报告了依库珠单抗治疗阵发性睡眠性血红蛋白尿症（PNH）的成本-效果及其相关的机会成本。方法.从公共资助的医疗保健系统的角度分析比较了依库珠单抗加现行标准治疗与现行标准治疗。马尔可夫模型涵盖了PNH和治疗的主要后果。根据每生命年和每质量调整生命年（QALY）的增量成本评估成本效益。机会成本是通过放弃的健康收益和额外资源的替代用途来评估的。结果依库珠单抗与更大的生命年（1.13），Qs（2.45）和成本（524万加元）相关。每生命年和每质量调整生命年的增量成本分别为462万加元和213万加元。根据既定的阈值，资助依库珠单抗的机会成本为每名受资助患者102.3个折扣Qs。敏感性和亚组分析证实了结果的稳健性。如果依库珠单抗的采购成本降低98.5%，则可以认为具有成本效益。局限性。罕见疾病的性质意味着进行经济评估的数据往往很少。当数据有限时，假设偏倚结果有利于依库珠单抗。结论.这项研究证明了在罕见病背景下进行经济评估的可行性。依库珠单抗可在预期寿命和生活质量方面为PNH患者提供实质性益处，但增量成本高，机会成本大。决策者在做出积极的补偿决策之前，应充分考虑机会成本。
Background. Both ethical and economics concerns have been raised with respect to the funding of drugs for rare diseases. This article reports both the cost-effectiveness of eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and its associated opportunity costs. Methods. Analysis compared eculizumab plus current standard of care v. current standard of care from a publicly funded health care system perspective. A Markov model covered the major consequences of PNH and treatment. Cost-effectiveness was assessed in terms of the incremental cost per life year and per quality-adjusted life year (QALY) gained. Opportunity costs were assessed by the health gains foregone and the alternative uses for the additional resources. Results. Eculizumab is associated with greater life years (1.13), QALYs (2.45), and costs (CAN$5.24 million). The incremental cost per life year and per QALY gained is CAN$4.62 million and CAN$2.13 million, respectively. Based on established thresholds, the opportunity cost of funding eculizumab is 102.3 discounted QALYs per patient funded. Sensitivity and subgroup analysis confirmed the robustness of the results. If the acquisition cost of eculizumab was reduced by 98.5%, it could be considered cost-effective. Limitations. The nature of rare diseases means that data are often sparse for the conduct of economic evaluations. When data were limited, assumptions were made that biased results in favor of eculizumab. Conclusions. This study demonstrates the feasibility of conducting economic evaluations in the context of rare diseases. Eculizumab may provide substantive benefits to patients with PNH in terms of life expectancy and quality of life but at a high incremental cost and a substantial opportunity cost. Decision makers should fully consider the opportunity costs before making positive reimbursement decisions.