Mario Negri Institute for Pharmacological Research, Milan, Italy.
Pharmacoeconomics. 2010;28(11):981-6. doi: 10.2165/11537590-000000000-00000.
Drug authorization, prescription and utilization are all based on benefit-risk assessment. This is made difficult by the apparent lack of objective means to measure the balance and by limitations regarding each of the two items. Benefit is sometimes measured by surrogate indicators of a real clinical advantage. It is assumed to be applicable to individuals even though it is measured in populations, and is represented in different ways that may convey different messages to physicians and patients. Risks are also hard to predict on an individual level. They may also be overlooked or revealed later than benefit, thus biasing the balance for a long time. Their causal relationship with the treatment is often not fully established. The benefit-risk balance itself has no generally recognized measure. This is why benefits and risks are hard to compare; either one or both may occur in single patients, and a risk-benefit profile that is acceptable in severe diseases may not be acceptable in diseases with a favourable prognosis. Pharmacoeconomics offers promising methods of health outcomes modelling using QALYs that take into consideration quality of life as well as survival. Primarily conceived as a guide for establishing the value of a treatment, they may prove useful as a means of trading efficacy and safety. However, quality of life is not always - or adequately - assessed in clinical studies. It is also not clear which is the most appropriate model to calculate QALYs for clinical purposes and how it can be used as a predictive tool at the individual level.
药物的批准、处方和使用均基于获益-风险评估。这一评估受到明显缺乏衡量平衡的客观手段以及两个项目各自的局限性的阻碍。获益有时通过真实临床优势的替代指标来衡量。虽然它是在人群中测量的,但它被认为适用于个体,并且以不同的方式呈现,这可能会向医生和患者传达不同的信息。风险也很难在个体层面预测。它们也可能被忽视或比获益晚出现,从而长期影响平衡。它们与治疗的因果关系往往尚未完全确定。获益-风险平衡本身没有公认的衡量标准。这就是为什么很难比较获益和风险的原因;在单个患者中可能会出现一种或两种情况,在严重疾病中可以接受的风险-获益特征在预后良好的疾病中可能无法接受。药物经济学提供了使用 QALYs 进行健康结果建模的有前途的方法,这些方法考虑了生活质量和生存。这些方法最初被认为是确定治疗价值的指南,它们可能被证明是在疗效和安全性方面进行权衡的有用手段。然而,在临床研究中,生活质量并不总是或充分地得到评估。目前也不清楚哪种模型最适合用于临床目的计算 QALYs,以及如何将其用作个体水平的预测工具。