Royal Infirmary of Edinburgh, Edinburgh, EH16 4SA, UK.
Tumour Centre of Excellence, Cancer Research UK Edinburgh Centre, Brain, University of Edinburgh, Edinburgh, EH4 2XR, UK.
Trials. 2023 May 31;24(1):370. doi: 10.1186/s13063-023-07367-0.
Patient and public involvement (PPI) in clinical trial design contributes to ensuring the research objectives and outcome measures are relevant to patients. The minimal clinically important difference (MCID) in the primary outcome influences trial design and feasibility and should be predicated on PPI. We aimed to determine current practice of reporting PPI and the MCID in phase III/IV randomised controlled trials (RCTs).
Following a search of Medline, Embase, and the Cochrane Central Register of Controlled Trials, we included primary publications of phase III/IV RCTs, in English, inclusive of any medical specialty or type of intervention, that reported a health-related outcome. We excluded protocols and secondary publications of RCTs. We extracted RCT characteristics, the use of PPI, and use of the MCID.
Between 1 July 2019 and 13 January 2020, 123 phase III/IV RCTs matched our eligibility criteria. Ninety percent evaluated a medical rather than surgical intervention. Oncology accounted for 21% of all included RCTs. Only 2.4% (n = 3) and 1.6% (n = 2) RCTs described PPI and the MCID respectively.
PPI and the MCID are poorly reported, so it is uncertain how these contributed to trial design. Improvement in the reporting of these items would increase confidence that results are relevant and clinically significant to patients, contributing to improving the overall trial design.
Not registered.
患者和公众参与临床试验设计有助于确保研究目标和结果衡量指标与患者相关。主要结局的最小临床重要差异（MCID）影响试验设计和可行性，并且应该基于患者和公众参与。我们旨在确定在 III 期/IV 期随机对照试验（RCT）中报告患者和公众参与和 MCID 的当前实践。
在 Medline、Embase 和 Cochrane 对照试验中心注册库进行搜索后，我们纳入了报告与健康相关结局的 III 期/IV 期 RCT 的主要出版物，包括任何医学专业或干预类型，以英文发表，包含 RCT 的特征、患者和公众参与的使用以及 MCID 的使用。
2019 年 7 月 1 日至 2020 年 1 月 13 日，有 123 项符合我们纳入标准的 III 期/IV 期 RCT。90%评估的是医学干预而非手术干预。肿瘤学占所有纳入 RCT 的 21%。只有 2.4%（n=3）和 1.6%（n=2）的 RCT 分别描述了患者和公众参与以及 MCID。
患者和公众参与以及 MCID 的报告都很差，因此不确定它们如何影响试验设计。这些项目报告的改进将提高对结果与患者相关且具有临床意义的信心，有助于改进整体试验设计。
未注册。
