科学家开发出基于价值来对药物进行定价的新型框架模型

  近日，发表在国际杂志JAMA Oncology上的研究论文中，来自埃默里大学等处的研究人员开发了一种新型的框架为所有进入市场的肿瘤药物建立了一种以价值为基础的定价，用以应对当前抗癌药物价格不断飙升的现状。文章中研究者利用了一种高度复杂的经济学模型利用肺癌药物作为例子进行了相关研究。
  研究者集中调查了一种名为necitumumab的药物，该药物目前正在等待FDA批准，此前有研究发现该药物可以延长转移性鳞状细胞肺癌患者寿命长达7周时间；因此研究者利用药物因素的经济学模型来对药物预期的价格、使用频率、副作用以及患者的生活质量进行了预测和管理，研究结果表明，以每三周为一个循环周期，药物necitumumab以价值为基础的价格范围在563美元至1309美元之间，相比已经进入市场的抗癌药物而言该价格明显降低了。
  研究者Daniel A. Goldstein说道，近些年来癌症药物的价格居高不下，一直处于增长之中，而且药物的价格并没有和药物预期的效益呈正向关联，很多患者服用癌症药物每月都超过了1万美元，而癌症药物价格的上升似乎并不能持续太长时间，潜在拯救生命的药物往往会被贴上高价的标签，但是诸如necitumumab的药物而言，其花费并不高而且还可以有效延长肺癌患者的生命。
  当前美国抗癌药物的偿还系统并没有为制药商和临床医生们考虑药物的价格和使用药物提供太多的动力，尽管本文研究以一种抗癌药物作为例子来进行研究，但研究中建立的模型也适用于评估其它癌症药物，这或许可以在未来可以真正帮助制定出以价值来进行药物定价的模式，当前的研究对于后期抗癌药物的开发以及药物价值、价格的评估提供了新的思路。（转化医学网360zhyx.com）
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转化医学网推荐的原文摘要：

Necitumumab in Metastatic Squamous Cell Lung Cancer: Establishing a Value-Based Cost.
JAMA Oncol        doi: 10.1001/jamaoncol.2015.3316
Goldstein DA1, Chen Q2, Ayer T2, Howard DH3, Lipscomb J3, Ramalingam SS1, Khuri FR1, Flowers CR1.
IMPORTANCE:
The SQUIRE trial demonstrated that adding necitumumab to chemotherapy for patients with metastatic squamous cell lung cancer (mSqCLC) increased median overall survival by 1.6 months (hazard ratio, 0.84). However, the costs and value associated with this intervention remains unclear. Value-based pricing links the price of a drug to the benefit that it provides and is a novel method to establish prices for new treatments.
OBJECTIVE:
To evaluate the range of drug costs for which adding necitumumab to chemotherapy could be considered cost-effective.
DESIGN, SETTING, AND PARTICIPANTS:
We developed a Markov model using data from multiple sources, including the SQUIRE trial, which compared standard chemotherapy with and without necitumumab as first-line treatment of mSqCLC, to evaluate the costs and patient life expectancies associated with each regimen. In the analysis, patients were modeled to receive gemcitabine and cisplatin for 6 cycles or gemcitabine, cisplatin, and necitumumab for 6 cycles followed by maintenance necitumumab. Our model's clinical inputs were the survival estimates and frequency of adverse events (AEs) described in the SQUIRE trial. Log-logistic models were fitted to the survival distributions in the SQUIRE trial. The cost inputs included drug costs, based on the Medicare average sale prices, and costs for drug administration and management of AEs, based on Medicare reimbursement rates (all in 2014 US dollars).
MAIN OUTCOMES AND MEASURES:
We evaluated incremental cost-effectiveness ratios (ICERs) for the use of necitumumab across a range of values for its cost. Model robustness was assessed by probabilistic sensitivity analyses, based on 10 000 Monte Carlo simulations, sampling values from the distributions of all model parameters.
RESULTS:
In the base case analysis, the addition of necitumumab to the treatment regimen produced an incremental survival benefit of 0.15 life-years and 0.11 quality-adjusted life-years (QALYs). The probabilistic sensitivity analyses established that when necitumumab cost less than $563 and less than $1309 per cycle, there was 90% confidence that the ICER for adding necitumumab would be less than $100 000 per QALY and less than $200 000 per QALY, respectively.
CONCLUSIONS AND RELEVANCE:
These findings provide a value-based range for the cost of necitumumab from $563 to $1309 per cycle. This study provides a framework for establishing value-based pricing for new oncology drugs entering the US marketplace.