Borup Gitte, Bach Karin Friis, Schmiegelow Merete, Wallach-Kildemoes Helle, Bjerrum Ole Jannik, Westergaard Niels
1 Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark.
2 Copenhagen Centre for Regulatory Sciences, Copenhagen, Denmark.
Ther Innov Regul Sci. 2016 May;50(3):304-311. doi: 10.1177/2168479015622668.
The Copenhagen Centre for Regulatory Science (CORS) and Biopeople at the University of Copenhagen held a workshop in May 2015 titled "Patient Involvement in Medicines Development and Approvals: A Paradigm Shift Towards True Patient Impact in Medicines Development and Regulatory Science" that acknowledged the importance of having patients more involved in the entire process of medicines research and development (R&D) and life cycle management. Four key stakeholders, representing patients, academia, industry, and regulatory authorities, each gave their view and perspective on the status and challenges of current patient involvement. From the 3 breakout sessions, it was concluded that patient-reported outcomes (ie, the report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else), was considered as an important tool when deciding endpoints. It was agreed that professionalization of the patient within medicine R&D to some extent would be necessary for obtaining influence. However, the industry should also seek to accommodate to the patient instead of waiting passively for patients to become educated. A much better organized and stronger involvement of patients was called for. However, this should not only rely on goodwill, but should preferably be implemented by legal requirements, so as to secure compliance by all stakeholders. An independent platform with the purpose of providing access to patient experience was proposed. A research and educational center such as CORS, which was founded on cross-sectorial and cross-disciplinary cooperation, is an example of an institution that could be a good starting point for hosting such a platform.
哥本哈根监管科学中心（CORS）与哥本哈根大学的生物人才组织于2015年5月举办了一场题为“患者参与药物研发与审批：向药物研发和监管科学中真正的患者影响转变的范式”的研讨会，该研讨会承认让患者更多地参与药物研发（R&D）和生命周期管理的整个过程具有重要意义。代表患者、学术界、产业界和监管机构的四个关键利益相关者分别就当前患者参与的现状和挑战发表了各自的观点和看法。从3个分组会议得出的结论是，患者报告的结果（即直接来自患者的关于其健康状况的报告，无需临床医生或其他任何人对患者的回答进行解读）在决定终点时被视为一项重要工具。大家一致认为，为了获得影响力，在某种程度上有必要使患者在药物研发中实现专业化。然而，产业界也应寻求适应患者，而不是被动等待患者接受教育。有人呼吁对患者进行更有序、更有力的参与。然而，这不应仅依赖于善意，而最好通过法律要求来实施，以确保所有利益相关者都能遵守。有人提议设立一个独立平台，目的是提供获取患者体验的途径。像CORS这样基于跨部门和跨学科合作建立的研究与教育中心，就是一个可以作为托管这样一个平台良好起点的机构的例子。
