Co-creation of practical "how-to guides" for patient engagement in key phases of medicines development-from theory to implementation.
Co-creation of practical "how-to guides" for patient engagement in key phases of medicines development-from theory to implementation.
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DOI：
10.1186/s40900-021-00294-x
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发表时间：
2021-08-23
影响因子：
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通讯作者：
Patient Focused Medicines Development Working Groups 2B
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文献类型：
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作者：
Feldman D;Kruger P;Delbecque L;Duenas A;Bernard-Poenaru O;Wollenschneider S;Hicks N;Reed JA;Sargeant I;Pakarinen C;Hamoir AM;Patient Focused Medicines Development Working Groups 1;Patient Focused Medicines Development Working Groups 2A;Patient Focused Medicines Development Working Groups 2B
患者参与（PE）在整个药物开发过程中的有效影响得到了包括卫生当局在内的各种卫生利益相关者群体的广泛认可;然而，如何实施有意义和一致的PE的实际应用并不总是得到解决。实际执行PE的指南需要粒度，对此类指南的需求已被确定为优先事项。我们描述了合作生产，并总结了如何在药物开发的早期阶段专注于PE的指南的内容。以患者为中心的药物开发（PFMD）成立了多利益相关方工作组（WG），以指导开发。通过公众咨询和工作组，与患者共同制作了PE活动的操作指南，这些活动被确定为优先事项。指南是通过在迭代过程中应用PE质量指南和相关质量标准来开发的。如何指南由专家（即在特定的体育活动或重点领域具有相关经验的专家）在特定的焦点小组中进行内部审查和验证，并通过适当的活动和公众咨询进行外部审查和验证。总体而言，来自38个组织（代表8个利益攸关方群体，包括患者/患者组织）和14个国家的103名个人贡献者被组织到工作组和工作流程中。每个工作组由15-30名具有与具体操作指南相关的PE经验的贡献者组成。为早期发现和临床前阶段的PE、临床结局评估策略开发中的PE以及临床试验方案设计中的PE制定了操作指南。操作指南有一个标准化的格式和结构，以促进用户熟悉。它们提供了与个人体育活动相关的详细指导和示例，旨在促进体育的实际实施。操作指南形成了一系列全面的可操作和逐步的资源，这些资源基于并整合了整个药物连续体的PE质量标准。它们将通过PFMD的患者参与管理套件（pemsuite.org）免费提供，并广泛分享给不同环境中的各种受众，确保访问不同的患者人群。这些指南的实施将推动PE领域将新药推向市场，并最终使患者受益。开发药物是为了帮助患者改善健康和生活。许多组织和个人希望确保药物的开发能够满足患者的真实的需求，并解决对患者最重要的问题。了解患者需要什么和患者想要什么需要良好的患者参与，但知道如何进行患者参与并不总是清楚的。这是因为药物开发是复杂的，涉及许多不同的步骤，人员和组织。以患者为中心的药物开发（PFMD）成立于2015年，旨在将致力于为患者生产药物的个人和组织联系起来。为了做到这一点，PFMD将患者和其他具有相关经验的人群聚集在一起，并就如何在现实世界中实现患者参与提出了好的想法。PFMD共同为患者参与制定了“操作指南”，涵盖了药物开发过程中沿着的主要活动。这些指南可以免费使用，并提供实用的建议和示例，任何人都可以在患者参与活动中使用。指南还将帮助患者了解药物开发以及如何最好地参与这一过程以满足他们的需求。在线版本包含补充材料，可通过10.1186/s40900-021-00294-x获得。
The effective impact of patient engagement (PE) across the medicines development continuum is widely acknowledged across diverse health stakeholder groups, including health authorities; however, the practical applications of how to implement meaningful and consistent PE are not always addressed. Guidance for the practical implementation of PE requires granularity, and the need for such guidance has been identified as a priority. We describe the co-production and summarize the content of how-to guides that focus on PE in the early stages of medicines development. Multi-stakeholder working groups (WGs) were established by Patient Focused Medicines Development (PFMD) for how-to guide development. How-to guides were co-produced with patients for PE activities identified as priorities through public consultation and by WGs. Guides were developed by applying PE quality guidance and associated quality criteria in an iterative process. How-to guides underwent internal review and validation by experts (ie, those with relevant experience in the particular PE activity or focus area) in specific focus groups and external review and validation through appropriate events and public consultation. Overall, 103 individual contributors from 38 organizations (representing eight stakeholder groups, including patients/patient organizations) and from 14 countries were organized into WGs and workstreams. Each WG comprised 15–30 contributors with PE experience relevant to the specific how-to guide. How-to guides were developed for PE in the early discovery and preclinical phases; PE in the development of a clinical outcomes assessment strategy; and PE in clinical trial protocol design. The how-to guides have a standardized format and structure to promote user familiarity. They provide detailed guidance and examples that are relevant to the individual PE activity and aim to facilitate the practical implementation of PE. The how-to guides form a comprehensive series of actionable and stepwise resources that build from and integrate the PE quality criteria across the medicines continuum. They will be made freely available through PFMD’s Patient Engagement Management Suite (pemsuite.org) and shared widely to a variety of audiences in different settings, ensuring access to diverse patient populations. Implementation of these guides should advance the field of PE in bringing new medicines to the market and ultimately will benefit patients. Medicines are developed to help patients improve their health and lives. Many organizations and individuals want to ensure that medicines are developed to meet real patient needs and to address what is most important to patients. Finding out what patients need and what patients want requires good patient engagement, but knowing how to do patient engagement is not always clear. This is because medicines development is complicated, and a lot of different steps, people, and organizations are involved. Patient Focused Medicines Development (PFMD) was established in 2015 to connect individuals and organizations that are committed to making medicines not just for patients but with patients. To do this, PFMD brought together patients and other groups of people with relevant experience and good ideas on how to achieve patient engagement in the real-world setting. Together, PFMD has developed “how-to guides” for patient engagement that cover the main activities along the medicines development process. The guides are free to use and provide practical advice and examples that anyone can use in their patient engagement activities. The how-to guides will also help patients to understand medicines development and how best they can participate in this process to address their needs. The online version contains supplementary material available at 10.1186/s40900-021-00294-x.