Establishing the Cost-Effectiveness of New Pharmaceuticals under Conditions of Uncertainty—When Is There Sufficient Evidence?

Decisions about which health-care interventions represent adequate value to collectively funded health-care systems are as widespread as they are unavoidable. In the case of new pharmaceuticals, many countries now require formal cost-effectiveness analysis to inform this decision-making process. This requires evidence on parameters associated with health-related utilities, treatment effects, resource use, and costs, for which data from available regulatory trials are invariably absent or highly uncertain. This uncertainty results from a number of factors including the predominance of intermediate end points in the clinical evidence-base and the limited period of follow-up of patients in clinical studies. Despite these imperfections in the evidence base, decisions about whether new pharmaceuticals are sufficiently cost-effective for reimbursement cannot be side-stepped. Data limitations do, however, require the use of rigorous analytical methods to support decision making. Probabilistic decision models and value of information analysis offer a means of structuring decision problems, synthesizing all available data, characterizing the uncertainty in the decision, quantifying the cost of uncertainty, and establishing the expected value of perfect information. This analytical framework is important because it addresses two fundamental questions about new pharmaceuticals. First, is the product expected to be cost-effective on the basis of existing evidence? Second, is additional research concerning the product itself cost-effective? In addressing these questions, the analytical framework can establish when sufficient evidence exists to sustain a claim for a new pharmaceutical to be cost-effective.     

比较行胃镜和肠镜检查术的麻醉异同点

目的比较静脉麻醉下行胃镜和肠镜检查术病人麻醉管理要点及副反应。方法选择拟行无痛性胃镜、结肠镜检查的患者各120例,在静脉麻醉下行内镜检查。观察其间生命体征变化,比较两组病人对内镜插入的耐受性、副反应以及胃镜和肠镜麻醉管理的侧重点。结果接受胃镜检查的患者麻醉期间生命体征无显著性变化,接受肠镜检查的患者,麻醉期间部分可出现反射性心率减慢和血压下降,需予以对症处理。两组病人对插入胃、肠镜的刺激耐受性良好。结论成功的静脉麻醉虽可达到无痛目的,但应注重对病人呼吸和循环功能监测,确保麻醉安全。