Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK.
Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK; National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, Birmingham, UK; NIHR Applied Research Collaboration (ARC) West Midlands, University of Birmingham, Birmingham, UK.
Contemp Clin Trials. 2022 Sep;120:106882. doi: 10.1016/j.cct.2022.106882. Epub 2022 Aug 13.
Real-world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE generation can be enhanced by collecting and using patient-reported outcomes (PROs), which can provide valuable information on the effectiveness, safety, and tolerability of health interventions from the patient perspective. This analysis aims to examine and summarise the utilisation of patient-reported outcomes measures (PROMs) in real-world studies.
Descriptions of phase IV trials were downloaded on July 22, 2021 from the Clinicaltrials.gov database since its inception. An automated algorithm was built to detect trials utilising PROMs and composite measures including patient-reported components. Search terms were developed based on the PROQOLID database.
Of 27,976 phase IV clinical trials posted on Clinicaltrials.gov between 1999 and July 2021, 21% and 4% used PROMs and composite measures, respectively. Recent years demonstrated a steady increase in the utilisation of PROMs in phase IV trials.
The use of PROMs in phase IV trials seems to be lower than its use in earlier phases of clinical research. Increased uptake of PROMs in RWE studies can be facilitated in a number of ways including the development of standards for their collection, analysis and use.
真实世界证据（RWE）在全球监管和报销流程中发挥着越来越重要的作用。通过收集和使用患者报告的结局（PROs）可以增强 RWE 的生成，这些结局可以从患者角度提供有关健康干预措施的有效性、安全性和耐受性的宝贵信息。本分析旨在检查和总结真实世界研究中使用患者报告结局测量（PROMs）的情况。
自 2021 年 7 月 22 日起，从 Clinicaltrials.gov 数据库中下载了自成立以来的四期试验描述。构建了一个自动算法来检测使用 PROMs 和包括患者报告成分的综合措施的试验。搜索词是基于 PROQOLID 数据库开发的。
在 1999 年至 2021 年 7 月期间在 Clinicaltrials.gov 上发布的 27976 项四期临床试验中，分别有 21%和 4%使用了 PROMs 和综合措施。近年来，四期临床试验中使用 PROMs 的比例稳步上升。
在四期临床试验中使用 PROMs 的比例似乎低于其在临床研究早期阶段的使用。可以通过多种方式促进 RWE 研究中 PROMs 的使用增加，包括制定用于收集、分析和使用 PROMs 的标准。
