Reaney Matthew, Bush Elizabeth, New Mary, Paty Jean, Roborel de Climens Aude, Skovlund Soren E, Nelsen Linda, Flood Emuella, Gater Adam
1 Sanofi, Guildford, United Kingdom.
2 University of Chichester, Chichester, United Kingdom.
Ther Innov Regul Sci. 2019 Sep;53(5):630-638. doi: 10.1177/2168479018807448. Epub 2018 Oct 22.
Benefit-risk assessment is the cornerstone of decision making in medical care, playing a critical role in bringing treatments to market by informing decisions regarding drug development, licensing and reimbursement, and informing treatment decisions made by health care professionals and patients in clinical practice. In regulatory approval decision making, benefit and risk attributes are identified and defined based on available, aggregated clinical data from registration trials. In the context of major developments in recent years for involvement of patients as partners in all phases of drug development and in health care improvement, decision makers increasingly recognize the importance of informing treatment decisions by patient needs, values, experiences, and preferences. Using this as a basis, a DIA workstream was convened to explore the potential of individual-level benefit-risk assessment as a supplement to traditional group-level benefit-risk assessment for evaluating treatment. Various approaches as to how this information could be collected, including via patient-reported outcome measures, open-ended questioning, and stated-preference methods are presented. The utility of this information for various stakeholders is discussed.
获益-风险评估是医疗决策的基石，在药物研发、许可和报销决策中发挥关键作用，从而推动治疗方法上市，并为医疗保健专业人员和患者在临床实践中做出治疗决策提供依据。在监管审批决策过程中，获益和风险属性是根据注册试验中可用的汇总临床数据来识别和定义的。近年来，患者作为合作伙伴参与药物研发和医疗保健改善的各个阶段有了重大进展，决策者越来越认识到根据患者的需求、价值观、经验和偏好为治疗决策提供信息的重要性。以此为基础，DIA召集了一个工作流程，探讨个体层面获益-风险评估作为传统群体层面获益-风险评估补充手段用于评估治疗的潜力。文中介绍了收集此类信息的各种方法，包括通过患者报告结局测量、开放式提问和陈述偏好方法。同时还讨论了这些信息对各类利益相关者的效用。
