EURORDIS-Rare Diseases Europe, Paris, France.
Radcliffe Primary Care Building, Radcliffe Observatory Quarter, Woodstock Rd, Oxford, OX2 6GG, UK.
Ther Innov Regul Sci. 2021 Sep;55(5):936-953. doi: 10.1007/s43441-021-00282-z. Epub 2021 May 10.
There is increased recognition that incorporating patients' perspectives and insights into the medicines development process results in better health outcomes and benefits for all involved stakeholders. Despite the increased interest and the existence of frameworks and practical recommendations, patient engagement (PE) is not yet considered standard practice. The objective of this work was to provide a roadmap to support systematic change in all stakeholder organisations involved in medicines development across Europe, patients and patient organisations, medicines developers, academia, regulatory authorities, Health Technology Assessment bodies, payers, policy-makers and public research funders, to sustain PE practices.
A mixed-methods approach was used by the EU-funded Innovative Medicines Initiative PARADIGM Consortium to co-develop the sustainability roadmap including background work to identify success factors and scenarios for sustainable PE. The roadmap development was based on the Theory of Change concept and populated with findings from (1) interviews with national/ and international institutions with the potential to increase PE uptake by other stakeholders; (2) multi-stakeholder workshops and webinars; and (3) consultations with specific stakeholder groups, Consortium members and a consultative body formed by international PE initiatives.
This roadmap sets strategic goals for the PE community to achieve meaningful and systematic PE through changes in the culture, processes and resources of stakeholder organisations. It brings in key PARADIGM outputs to work in a coordinated fashion with existing frameworks and mechanisms to achieve system-wide sustained PE.
The roadmap provides a framework for all stakeholders to take collective action within their organisations and across Europe to implement PE in a sustainable manner.
越来越多的人认识到，将患者的观点和见解纳入药物开发过程中，会为所有相关利益攸关方带来更好的健康结果和收益。尽管人们的兴趣日益浓厚，并且已经存在框架和实际建议，但患者参与（PE）尚未被视为标准做法。这项工作的目的是为支持欧洲所有参与药物开发的利益相关者组织（患者和患者组织、药物开发商、学术界、监管机构、卫生技术评估机构、支付方、政策制定者和公共研究资助者）进行系统变革提供路线图，以维持 PE 实践。
欧盟资助的创新药物倡议 PARADIGM 联盟采用混合方法来共同制定可持续性路线图，包括确定成功因素和可持续 PE 情景的背景工作。路线图的制定基于变革理论的概念，并结合（1）对有潜力提高其他利益相关者参与度的国家/国际机构的访谈；（2）多利益攸关方研讨会和网络研讨会；以及（3）与特定利益攸关方群体、联盟成员和由国际 PE 倡议组成的咨询机构的磋商的调查结果。
该路线图为 PE 社区设定了战略目标，通过改变利益攸关方组织的文化、流程和资源，实现有意义和系统的 PE。它引入了关键的 PARADIGM 产出，以协调的方式与现有框架和机制合作，实现全系统的可持续 PE。
该路线图为所有利益攸关方提供了一个框架，以便在其组织内部以及整个欧洲采取集体行动，以可持续的方式实施 PE。
