Department of Kyosei Studies (Critical Studies in Coexistence, Symbiosis and Conviviality), Graduate School of Human Sciences, Osaka University, Suita, Japan.
Department of Biomedical Ethics and Public Policy, Graduate School of Medicine, Osaka University, Suita, Japan.
Front Public Health. 2022 Jul 19;10:915438. doi: 10.3389/fpubh.2022.915438. eCollection 2022.
Patient involvement (PI) in determining medical research priorities is an important way to ensure that limited research funds are allocated to best serve patients. As a disease area for which research funds are limited, we see a particular utility for PI in priority-setting for medical research on rare diseases. In this review, we argue that PI initiatives are an important form of evidence for policymaking. We conducted a study to identify the extent to which PI initiatives are being conducted in the rare disease field, the features of such initiatives, the trends in the priorities elicited, and the extent to which translation into policy is reported in the academic literature. Here, we report the results of this exploratory review of the English-language literature gathered through online databases and search engines, with the aim of identifying journal articles published prior to December 2020, describing PI initiatives focused on determining priorities for medical research funding in the rare disease field. We identified seven recently-published articles and found that the majority made use of structured methodologies to ensure the robustness of the evidence produced, but found little reported practical implementation or concrete plans for implementation of the results of the initiatives. We conclude that priority-setting initiatives are meaningful mechanisms for involving patients in determining research directions. However, we highlight the importance of translation into policy as a necessary next step to fully utilize the results and move beyond well-intentioned exercises. Finally, we draw attention to the benefits of involving patients throughout this process.
患者参与（PI）确定医学研究重点是确保有限的研究资金用于最好地为患者服务的重要途径。作为一个研究资金有限的疾病领域，我们认为 PI 在确定罕见病医学研究重点方面具有特别的作用。在这篇综述中，我们认为 PI 倡议是决策制定的重要证据形式。我们进行了一项研究，以确定 PI 倡议在罕见病领域的开展程度、这些倡议的特点、所引出的优先事项的趋势，以及在学术文献中报告转化为政策的程度。在这里，我们报告了通过在线数据库和搜索引擎收集的英文文献的探索性综述结果，旨在确定截至 2020 年 12 月之前发表的描述 PI 倡议的期刊文章，这些倡议侧重于确定罕见病领域医学研究资金的优先事项。我们确定了七篇最近发表的文章，发现大多数都采用了结构化的方法来确保所产生证据的稳健性，但很少有报告实际实施或具体实施倡议结果的计划。我们的结论是，确定优先事项的倡议是让患者参与确定研究方向的有意义的机制。然而，我们强调将其转化为政策是必要的下一步，以充分利用结果并超越良好的意图。最后，我们提请注意在整个过程中让患者参与的好处。
