Postmus D, Mavris M, Hillege H L, Salmonson T, Ryll B, Plate A, Moulon I, Eichler H-G, Bere N, Pignatti F
European Medicines Agency, London, UK.
University of Groningen, University Medical Center Groningen, The Netherlands.
Clin Pharmacol Ther. 2016 May;99(5):548-54. doi: 10.1002/cpt.332. Epub 2016 Feb 17.
Currently, patient preference studies are not required to be included in marketing authorization applications to regulatory authorities, and the role and methodology for such studies have not been agreed upon. The European Medicines Agency (EMA) conducted a pilot study to gain experience on how the collection of individual preferences can inform the regulatory review. Using a short online questionnaire, ordinal statements regarding the desirability of different outcomes in the treatment of advanced cancer were elicited from 139 participants (98 regulators, 29 patient or carers, and 12 healthcare professionals). This was followed by face-to-face meetings to gather feedback and validate the individual responses. In this article we summarize the EMA pilot study and discuss the role of patient preference studies within the regulatory review. Based on the results, we conclude that our preference elicitation instrument was easy to implement and sufficiently precise to learn about the distribution of the participants' individual preferences.
目前，向监管机构提交的上市许可申请中无需纳入患者偏好研究，而且此类研究的作用和方法尚未达成一致。欧洲药品管理局（EMA）开展了一项试点研究，以获取关于收集个人偏好如何为监管审评提供信息的经验。通过一份简短的在线问卷，从139名参与者（98名监管人员、29名患者或护理人员以及12名医疗保健专业人员）中引出了关于晚期癌症治疗中不同结果可取性的有序陈述。随后进行了面对面会议，以收集反馈并验证个人回答。在本文中，我们总结了EMA试点研究，并讨论了患者偏好研究在监管审评中的作用。基于研究结果，我们得出结论，我们的偏好诱导工具易于实施且足够精确，能够了解参与者个人偏好的分布情况。
