Quartey George, Wang Jixian
Statistics Methods and Research Group, F. Hoffmann-La Roche, Welwyn Garden City, UK.
Pharm Stat. 2012 Jan-Feb;11(1):82-5. doi: 10.1002/pst.497. Epub 2011 Oct 13.
Benefit-risk assessment is a fundamental element of drug development with the aim to strengthen decision making for the benefit of public health. Appropriate benefit-risk assessment can provide useful information for proactive intervention in health care settings, which could save lives, reduce litigation, improve patient safety and health care outcomes, and furthermore, lower overall health care costs. Recent development in this area presents challenges and opportunities to statisticians in the pharmaceutical industry. We review the development and examine statistical issues in comparative benefit-risk assessment. We argue that a structured benefit-risk assessment should be a multi-disciplinary effort involving experts in clinical science, safety assessment, decision science, health economics, epidemiology and statistics. Well planned and conducted analyses with clear consideration on benefit and risk are critical for appropriate benefit-risk assessment. Pharmaceutical statisticians should extend their knowledge to relevant areas such as pharmaco-epidemiology, decision analysis, modeling, and simulation to play an increasingly important role in comparative benefit-risk assessment.
获益-风险评估是药物研发的一个基本要素，其目的是加强决策以促进公众健康。恰当的获益-风险评估可为医疗保健环境中的主动干预提供有用信息，这可以挽救生命、减少诉讼、提高患者安全性和医疗保健结果，此外，还能降低总体医疗保健成本。该领域的最新发展给制药行业的统计学家带来了挑战和机遇。我们回顾了比较获益-风险评估的发展并审视其中的统计问题。我们认为，结构化的获益-风险评估应该是一项多学科的工作，涉及临床科学、安全评估、决策科学、卫生经济学、流行病学和统计学等领域的专家。精心规划和实施的分析，同时明确考虑获益和风险，对于恰当的获益-风险评估至关重要。制药统计学家应将其知识扩展到药物流行病学、决策分析、建模和模拟等相关领域，以便在比较获益-风险评估中发挥越来越重要的作用。
