Garrison Louis P, Towse Adrian, Bresnahan Brian W
Department of Pharmacy, University of Washington, Seattle, USA.
Health Aff (Millwood). 2007 May-Jun;26(3):684-95. doi: 10.1377/hlthaff.26.3.684.
Regulatory authorities make difficult risk-benefit decisions when approving new drugs. Food and Drug Administration (FDA) advisory committees and reviewers must consider a complex body of evidence, including efficacy and safety results of trials, disease epidemiology, potential side effects, and patients' needs. However, this menu of information is not usually presented in a consistent and integrated framework. The members of an FDA review panel vote with some unobserved, implicit weighting of the evidence. This paper argues that outcomes research tools for modeling long-term health outcomes, measuring health preferences, and establishing the value of additional information could provide a more structured, transparent, and quantitative process of assessing risk-benefit balance.
监管机构在批准新药时要做出艰难的风险效益决策。美国食品药品监督管理局（FDA）的咨询委员会和审评人员必须考虑一系列复杂的证据，包括试验的疗效和安全性结果、疾病流行病学、潜在副作用以及患者需求。然而，这些信息通常并非以一致且综合的框架呈现。FDA审评小组的成员在投票时会对证据进行一些未被观察到的、隐含的权衡。本文认为，用于对长期健康结果进行建模、衡量健康偏好以及确定额外信息价值的结果研究工具，可以提供一个更具结构化、透明度和定量性的评估风险效益平衡的过程。
