Innovation Studies, Copernicus Institute of Sustainable Development, Utrecht University, Utrecht, The Netherlands.
Department of Health, Ethics and Society, Faculty of Health, Medicine and Life Sciences, Maastricht University, Maastricht, The Netherlands.
Health Expect. 2024 Feb;27(1):e13912. doi: 10.1111/hex.13912.
Recently, different actors have intensified their efforts to make drug development more participatory. They have produced many frameworks, tools and dedicated fora, where patients are portrayed as relevant stakeholders to be involved throughout the entire drug development trajectory. To better understand what such participatory efforts entail, in this article, we investigate how patient representation is configured in drug development and what patients can engage as representatives in this field.
This is a qualitative study based on the thematic analysis of 40 semistructured interviews with different stakeholders in the field and three patient engagement How-To guides (HTGs) complemented by observations of two sessions of the Patient Engagement Open Forum (PEOF) and a patient expert training of the European Patients' Academy on Therapeutic Innovation (EUPATI).
The emerging practices of patient engagement in drug development configure representation as hinging upon three types of knowledge-drug development knowledge, autobiographical knowledge and community knowledge-and a specific set of skills. We discern a new kind of representation based on these findings, termed 'knowledge-based representation', which appears to more accurately describe how patients are expected to represent others in drug development.
Even though knowledge-based representation may be understood as an attempt to downplay the political aspects of representation in favour of its epistemic elements, the political processes involved in patient representation in drug development cannot be ignored. The extent to which reliance on knowledge-based representation will contribute to democratic decision-making is likely to depend on the resources needed to develop the types of knowledge relevant to representation work and on how these types of knowledge are determined.
Patient representatives and practitioners in the field of patient engagement (including 13 interviewees, representatives of EUPATI and HTG developers) gave feedback on the interpretation of the findings during a multistakeholder workshop we organised. We also sent an interviewee an extended draft and discussed it during an online meeting. Claudia Egher presented these findings at a PEOF session in June 2023, which further contributed to their validation.
最近，不同的利益相关者加大了努力，使药物开发更加具有参与性。他们提出了许多框架、工具和专门的论坛，将患者描绘为在整个药物开发过程中应参与的相关利益攸关方。为了更好地理解这些参与性努力的具体内容，本文探讨了在药物开发中患者代表性是如何配置的，以及患者可以在该领域担任哪些代表。
这是一项基于对该领域 40 名不同利益相关者的半结构化访谈的主题分析的定性研究，以及对三个患者参与度指导（HTG）的补充，这些指导辅以对两次患者参与开放论坛（PEOF）会议的观察和对欧洲治疗创新患者学院（EUPATI）的一次患者专家培训。
药物开发中新兴的患者参与实践将代表性配置为取决于三种类型的知识——药物开发知识、自传知识和社区知识——以及一套特定的技能。根据这些发现，我们发现了一种新的代表性形式，称为“基于知识的代表性”，这似乎更准确地描述了患者在药物开发中代表他人的期望方式。
即使基于知识的代表性可以被理解为试图淡化代表性的政治方面，强调其认识论元素，但在药物开发中患者代表性所涉及的政治过程不容忽视。依赖基于知识的代表性是否有助于民主决策，可能取决于发展代表性工作相关知识类型所需的资源，以及这些知识类型是如何确定的。
在我们组织的一次多方利益相关者研讨会上，患者代表和参与患者参与领域的从业者（包括 13 名访谈者、EUPATI 和 HTG 开发者的代表）对研究结果的解释提供了反馈。我们还向一名访谈者发送了一份扩展的草稿，并在一次在线会议上进行了讨论。Claudia Egher 在 2023 年 6 月的 PEOF 会议上介绍了这些发现，这进一步验证了这些发现。
