患者及公众参与国际临床试验设计：真实世界经验

Patient and public involvement in the design of an international clinical trial: real world experience.

作者信息

Simons Gwenda, Jones Helen, Clarke Ian, Davies Firoza, Grealis Stacey, Insch Elspeth, Kahn Hameed, Lloyd Joanne, Richards Al, Rose Hayley, Williams Ruth, de Wit Maarten, Woodcock Clarissa, Romaniuk Leigh, Bardgett Michelle, Pratt Arthur G, Falahee Marie

机构信息

Department of Inflammation and Ageing, College of Medicine and Health, Rheumatology Research Group, University of Birmingham, Queen Elizabeth Hospital, Birmingham, UK.

PPI Partner, Patient and public Involvement and engagement in Musculoskeletal reSearch (PIMS) group, Newcastle upon Tyne Hospitals, Newcastle, UK.

出版信息

Res Involv Engagem. 2024 Nov 6;10(1):117. doi: 10.1186/s40900-024-00642-7.

DOI:10.1186/s40900-024-00642-7

PMID:39506862

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11539688/

Abstract

BACKGROUND

The value of patient and public involvement (PPI) during the earliest stages of clinical trial development, and prior to the award of substantive funding, is widely recognised. However, it is often under-resourced and PPI processes during this phase are rarely reported in detail. Having benefitted from seed funding to develop an international clinical trial proposal, we sought to describe and appraise PPI activities and processes that support pre-award co-development.

METHODS

A 12-month "accelerator" award facilitated development of a substantive funding application to deliver the Rheumatoid Arthritis Prevention PlatfORm Trial (RAPPORT), conceived to prioritise preventative interventions for people at risk of RA. PPI partners, including individuals at risk of rheumatoid arthritis (RA), RA patients, relatives and members of the public, provided feedback on key trial design issues through online meetings, a feedback form and emails. PPI processes employed during the one-year accelerator project were thereafter evaluated by PPI partners using an anonymous online feedback form with reference to National Institute of Health and Care Research (NIHR) UK standards for public involvement in research.

RESULTS

Sixteen out of the 25-strong PPI partner panel completed an online feedback form (64%). Respondents perceived PPI processes positively in relation to all NIHR standard domains. Several key facilitators and challenges were identified, including the need for adequate PPI funding during pre-award phases of research, strategies for creating an inclusive environment, flexibility around levels of involvement, and challenges in achieving representatively diverse participation, and the importance of communicating transparent processes for role-assignment and time-reimbursement.

CONCLUSIONS

In general, RAPPORT was considered an example of PPI well done, and in line with UK standards for public involvement in research. Facilitators and challenges of relevance for the development of future translational and clinical trial funding applications are highlighted.

摘要

背景

在临床试验开发的最早阶段以及获得实质性资金之前，患者和公众参与（PPI）的价值已得到广泛认可。然而，其资源往往不足，这一阶段的PPI过程也很少有详细报道。在获得种子资金以制定一项国际临床试验提案后，我们试图描述和评估支持预拨款共同开发的PPI活动和过程。

方法

一项为期12个月的“加速器”奖促进了一份实质性资金申请的制定，以开展类风湿性关节炎预防平台试验（RAPPORT），该试验旨在优先为有类风湿性关节炎（RA）风险的人群提供预防干预措施。PPI合作伙伴，包括有类风湿性关节炎风险的个体、RA患者、亲属和公众成员，通过在线会议、反馈表和电子邮件就关键试验设计问题提供了反馈。此后，PPI合作伙伴使用一份匿名在线反馈表，参照英国国家卫生与保健研究机构（NIHR）关于公众参与研究的标准，对为期一年的加速器项目中采用的PPI过程进行了评估。

结果

在由25名成员组成的PPI合作伙伴小组中，有16人填写了在线反馈表（64%）。受访者对PPI过程在所有NIHR标准领域的评价都是积极的。确定了几个关键的促进因素和挑战，包括在研究的预拨款阶段需要充足的PPI资金、营造包容环境的策略、参与程度的灵活性、实现具有代表性的多样化参与的挑战，以及明确角色分配和时间补偿的透明过程的沟通的重要性。