背景:尽管利益相关者参与政策制定正在引起医学和医疗保健领域的关注,尚未建立实用的方法。罕见疾病政策,专门为有限的研究资源分配确定研究优先级,是一个通过利益相关者参与生成证据的领域,预计将是有效的。我们通过利益相关者的参与为罕见疾病政策制定提供了证据,并探索了利益相关者之间的有效合作。
方法:我们构建了一个名为“证据生成共用空间”的空间,在那里,病人,家庭成员,研究人员,和前决策者可以分享他们的知识和经验,并参与关于证据生成的持续审议。因此出现了十种罕见疾病。在\'Commons\'中,连续举办了25次研讨会,主要是在线,从2019年到2021年。这些研讨会的重点是(1)澄清罕见病患者面临的困难,(2)制定和选择优先级设置标准,(3)通过应用标准确定优先级。第一步,举行了使用便签的现场研讨会。数据采用KJ法进行分析。第二步和第三步,举行了关于具体主题的讲习班,以建立共识。研讨会的议程和方法根据与会者的反馈进行了修改。
结果:由43名参与者组成的“下议院”成立,产生了能力建设等积极影响,互动的机会,相互理解,参与者之间的同理心。罕见疾病患者面临的困难分为10类。七个研究主题被确定为需要解决的优先问题,包括“日常生活障碍”,\'财务负担\',\'焦虑\',和“住院负担”。这是通过合成两个标准的应用结果来进行的,这两个标准对于加强对罕见疾病的未来研究特别重要。我们还通过使用患者和家庭成员比研究人员和前决策者更重视的标准来澄清高优先级的研究主题。和具有特定视角的标准。
结论:我们为罕见疾病领域的决策提供了证据。这项研究对利益相关者参与的见解可以增强循证决策。我们与政策制定者就政策实施和计划分析参与者在这个项目中的经验进行了全面的讨论。
利益相关者的参与对于罕见疾病领域的有效决策具有重要意义。然而,这种参与的实际方法尚未建立。因此,我们开发了“Commons项目”,以生成有价值的决策信息,并探索利益相关者合作的有效途径。本文解释了25个连续车间的过程和结果,从2019年到2021年,有43人参加,包括患者,家庭成员,研究人员,和前决策者。在“下议院”中进行的讨论的主要成就包括对罕见疾病患者面临的困难的概述以及高度优先研究主题的制定。首先,罕见病患者面临的困难分为10类.第二,七个研究课题被确定为优先问题,包括“日常生活障碍”,“经济负担”,'焦虑',和“医院就诊负担”。在项目过程中,能力建设等积极影响,互动的机会,相互理解,参与者之间的同理心,已确定。在罕见疾病和政策科学领域之外,这些发现对社会的未来很有用,包括利益相关者之间的共同创造以及患者和公众的参与。根据这项研究的结果,我们已经开始与罕见疾病领域的政策利益相关者进行沟通,以政策执行为目的。
BACKGROUND: Although stakeholder involvement in policymaking is attracting attention in the fields of medicine and healthcare, a practical methodology has not yet been established. Rare-disease policy, specifically research priority setting for the allocation of limited research resources, is an area where evidence generation through stakeholder involvement is expected to be effective. We generated evidence for rare-disease policymaking through stakeholder involvement and explored effective collaboration among stakeholders.
METHODS: We constructed a space called \'Evidence-generating Commons\', where patients, family members, researchers, and former policymakers can share their knowledge and experiences and engage in continual deliberations on evidence generation. Ten rare diseases were consequently represented. In the \'Commons\', 25 consecutive workshops were held predominantly online, from 2019 to 2021. These workshops focused on (1) clarification of difficulties faced by rare-disease patients, (2) development and selection of criteria for priority setting, and (3) priority setting through the application of the criteria. For the first step, an on-site workshop using sticky notes was held. The data were analysed based on KJ method. For the second and third steps, workshops on specific themes were held to build consensus. The workshop agendas and methods were modified based on participants\' feedback.
RESULTS: The \'Commons\' was established with 43 participants, resulting in positive effects such as capacity building, opportunities for interactions, mutual understanding, and empathy among the participants. The difficulties faced by patients with rare diseases were classified into 10 categories. Seven research topics were identified as priority issues to be addressed including \'impediments to daily life\', \'financial burden\', \'anxiety\', and \'burden of hospital visits\'. This was performed by synthesising the results of the application of the two criteria that were particularly important to strengthen future research on rare diseases. We also clarified high-priority research topics by using criteria valued more by patients and family members than by researchers and former policymakers, and criteria with specific perspectives.
CONCLUSIONS: We generated evidence for policymaking in the field of rare diseases. This study\'s insights into stakeholder involvement can enhance evidence-informed policymaking. We engaged in comprehensive discussions with policymakers regarding policy implementation and planned analysis of the participants\' experiences in this project.
Stakeholder involvement is significant for effective policymaking in the field of rare diseases. However, practical methods for this involvement have not yet been established. Therefore, we developed the ‘Commons project’ to generate valuable policymaking information and explore effective ways for stakeholders’ collaboration. This article explains the process and results of 25 continuous workshops, held from 2019 to 2021 with 43 participants, including patients, family members, researchers, and former policymakers. The main achievements of the discussion that took place in the ‘Commons’ included a presentation of the overview of the difficulties faced by patients with rare diseases and formulation of high priority research topics.First, the difficulties faced by patients with rare diseases were grouped into 10 categories. Second, seven research topics were identified as priority issues including ‘impediments to daily life’, ‘financial burden’, ‘anxiety’, and ‘burden of hospital visits’. During the project process, positive effects such as capacity building, opportunities for interactions, mutual understanding, and empathy among the participants, were identified. Beyond the context of the field of rare diseases and science of policy, these findings are useful for the future of society, including co-creation among stakeholders and patient and public involvement. Based on this study’s results, we have initiated communications with policy stakeholders in the field of rare diseases, with the aim of policy implementation.