Department of Neurology, University of California, San Francisco, CA 94143-0114, USA.
Stroke. 2010 Oct;41(10 Suppl):S59-62. doi: 10.1161/STROKEAHA.110.597351.
The societal costs and health benefits of tissue plasminogen activator (tPA) for ischemic stroke can be modeled and extended to the US population. Similarly, the societal impact of new thrombolytics with improved efficacy or safety or extending eligibility can also be modeled.
We previously modeled the impact of tPA on societal costs and health in the United States. Pertinent publications on utilization, societal cost, and health impact were identified by systematic review, updated to include studies describing the impact of extending the tPA time window. Information on utilization of tPA was integrated with published per-use data on costs and health impact (converted to 2004 dollars) to generate annual projections for the US population. Model inputs were modified to reflect various characteristics of new thrombolytics.
At its current price, tPA saves $6074 and adds 0.75 quality-adjusted life year (QALY) per use. If tPA were priced at $50,000/QALY, a standard benchmark for cost-effectiveness, it would cost $45,800 per dose and would be expected to generate $458 million in revenue annually for its manufacturer. Thrombolytics for stroke that extended the time window or improved efficacy could generate greater revenue if priced at an accepted level of cost-effectiveness. Given current clinical development costs, development of candidate drugs with 2.8% to 5.7% probability of ultimate FDA approval would be justified.
tPA produces substantial health and economic benefits in the United States. Better thrombolytics for stroke could have substantial impact on society, and potential returns to developers would appear to justify greater investment in new candidates.
组织型纤溶酶原激活剂（tPA）治疗缺血性脑卒中的社会效益和健康效益可以建模并推广至美国人群。同样，具有更好疗效或安全性或扩大适应证的新型溶栓药物的社会效益也可以建模。
我们曾对 tPA 在美国的社会效益和健康效益进行建模。通过系统评价确定与利用、社会成本和健康影响相关的研究文献，纳入描述延长 tPA 时间窗影响的研究。利用 tPA 的相关数据与发表的每例使用成本和健康影响数据（转换为 2004 年美元）相结合，对美国人群进行年度预测。模型输入根据新型溶栓药物的不同特征进行修改。
tPA 目前的价格为每例节省 6074 美元，增加 0.75 个质量调整生命年（QALY）。如果按照 50000 美元/QALY 的标准成本效益价格进行定价，tPA 的每个剂量将花费 45800 美元，其制造商每年预计可获得 4.58 亿美元的收入。如果按可接受的成本效益价格定价，延长时间窗或提高疗效的溶栓药物可能会产生更大的收益。考虑到当前的临床开发成本，具有最终获得美国食品和药物管理局（FDA）批准 2.8%~5.7%概率的候选药物的开发是合理的。
tPA 在美国产生了巨大的健康和经济效益。治疗脑卒中的更好溶栓药物可能会对社会产生重大影响，而开发者的潜在回报似乎表明，有必要对新的候选药物进行更多投资。
