Patient Engagement Practices in Clinical Research among Patient Groups, Industry, and Academia in the United States: A Survey

ObjectivePatient-centered clinical trial design and execution is becoming increasingly important. No best practice guidelines exist despite a key stakeholder declaration to create more effective engagement models. This study aims to gain a better understanding of attitudes and practices for engaging patient groups so that actionable recommendations may be developed.MethodsIndividuals from industry, academic institutions, and patient groups were identified through Clinical Trials Transformation Initiative and Drug Information Association rosters and mailing lists. Objectives, practices, and perceived barriers related to engaging patient groups in the planning, conduct, and interpretation of clinical trials were reported in an online survey. Descriptive and inferential statistical analysis of survey data followed a literature review to inform survey questions.ResultsSurvey respondents (n = 179) valued the importance of involving patient groups in research; however, patient group respondents valued their contributions to research protocol development, funding acquisition, and interpretation of study results more highly than those contributions were valued by industry and academic respondents (all p ConclusionsDespite reported similarities among approaches to engagement by the three stakeholder groups, key differences exist in perceived barriers and benefits to partnering with patient groups among the sectors studied. This recognition could inform the development of best practices for patient-centered clinical trial design and execution. Additional research is needed to define and optimize key success factors.

研究背景与目的：以患者为中心的临床试验设计与实施正愈发受到行业重视。尽管关键利益相关方已提出构建更高效的患者参与模式的倡议，但目前尚未形成统一的最佳实践指南。本研究旨在深入了解当前患者群体参与临床试验相关的态度与实践现状，以期形成可落地的行动建议。
研究方法：本研究通过临床试验转化倡议（Clinical Trials Transformation Initiative）与药品信息协会（Drug Information Association）的成员名册及邮件列表，招募了来自工业界、学术机构及患者群体的参与者。研究首先开展文献调研以确定问卷的问题框架，随后通过线上问卷收集相关数据，内容涵盖在临床试验的规划、实施与结果解读阶段邀请患者群体参与的相关目标、实践举措及感知障碍。最后对调研数据进行描述性与推断性统计分析。
研究结果：本次调研共回收有效问卷179份。所有受访者均认可在临床试验中邀请患者群体参与的重要性；但相较于工业界与学术机构的受访者，患者群体受访者对自身在研究方案制定、经费获取及试验结果解读方面所做贡献的重视程度更高（所有p）。
研究结论：尽管三类利益相关方在患者参与模式上存在共性，但不同领域的受访者在与患者群体合作时感知到的障碍与收益存在显著差异。这一发现可为以患者为中心的临床试验设计与实施的最佳实践制定提供参考依据，未来仍需开展更多研究以明确并优化临床试验成功的关键影响因素。