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患者偏好科学的下一步是什么?互操作性、标准化和可转移性。

What Next for the Science of Patient Preference? Interoperability, Standardization, and Transferability.

作者信息

Marsh Kevin, Sepulveda Juan Marcos Gonzalez, Berlin Conny, Levitan Bennett, Boeri Marco, Groothuis-Oudshoorn Catharina G M, Crossnohere Norah L, Jimenez-Moreno Cecilia, Liden Barry, Stoeckert Isabelle, Veldwijk Jorien, Watt Stephen, Hauber Brett

机构信息

Evidera, London, UK.

Duke University, Durham, NC, USA.

出版信息

Patient. 2025 Mar;18(2):101-108. doi: 10.1007/s40271-025-00727-9. Epub 2025 Jan 28.

Abstract

Using patient preference information (PPI) to incorporate patient voices into the drug development lifecycle can help align therapies with the needs and values of patients. However, several barriers have limited the use of PPI, including a lack of clarity on its use by decision-makers, a need for greater decision-maker trust in PPI, and a lack of time, budgets, and access to specialist expertise. The value proposition for PPI could be enhanced by making it FAIR: Findable, Accessible, Interoperable, and Reusable. To support the development of a research agenda to deliver FAIR PPI, we reviewed related endeavors in the development of repositories of existing studies, disease models, benefit transfer, and common data standards. We concluded that developing FAIR PPI would require advances in the science of PPI, including the establishment of a consortium, mirroring the Clinical Data Interchange Standards Consortium (CDISC) or Observational Medical Outcomes Partnership (OPOM), to develop PPI data standards, and research into the sources of variation in patient preferences. This will require the science of PPI to graduate from being a body of empirical observations to developing theories that explain variations in patient preferences, simultaneously driving both efficiency in the generation of PPI and trust in PPI.

摘要

利用患者偏好信息(PPI)将患者声音纳入药物开发生命周期,有助于使治疗方法与患者的需求和价值观保持一致。然而,一些障碍限制了PPI的使用,包括决策者对其使用缺乏清晰认识、决策者对PPI的信任度有待提高,以及缺乏时间、预算和获取专业知识的途径。通过使其具备FAIR特性(即可查找、可访问、可互操作和可重用),可以提升PPI的价值主张。为了支持制定实现FAIR PPI的研究议程,我们回顾了现有研究库、疾病模型、效益转移和通用数据标准开发方面的相关工作。我们得出结论,开发FAIR PPI需要PPI科学取得进展,包括成立一个类似于临床数据交换标准协会(CDISC)或观察性医疗结局合作组织(OPOM)的联盟,以制定PPI数据标准,并对患者偏好的变异来源进行研究。这将要求PPI科学从一系列实证观察发展为能够解释患者偏好变异的理论,同时提高PPI生成的效率和对PPI的信任度。

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