Division of Experimental Medicine, Department of Medicine, University of British Columbia, Vancouver, Canada.
Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.
JAMA. 2022 Jan 4;327(1):67-74. doi: 10.1001/jama.2021.22507.
Platform trials are a type of randomized clinical trial that allow simultaneous comparison of multiple intervention groups against a single control group that serves as a common control based on a prespecified interim analysis plan. The platform trial design enables introduction of new interventions after the trial is initiated to evaluate multiple interventions in an ongoing manner using a single overarching protocol called a master (or core) protocol. When multiple treatment candidates are available, rapid scientific therapeutic discoveries may be made. Platform trials have important potential advantages in creating an efficient trial infrastructure that can help address critical clinical questions as the evidence evolves. Platform trials have recently been used in investigations of evolving therapies for patients with COVID-19. The purpose of this Users' Guide to the Medical Literature is to describe fundamental concepts of platform trials and master protocols and review issues in the conduct and interpretation of these studies. This Users' Guide is intended to help clinicians and readers understand articles reporting on interventions evaluated using platform trial designs.
平台试验是一种随机临床试验，允许同时比较多个干预组与一个单一的对照组进行比较，该对照组基于预先指定的中期分析计划作为共同对照。平台试验设计使得在试验开始后可以引入新的干预措施，以便使用称为主（或核心）协议的单一总体方案以持续的方式评估多种干预措施。当有多种治疗候选药物时，可能会迅速发现科学治疗发现。平台试验在创建一种有效的试验基础设施方面具有重要的潜在优势，这种基础设施可以随着证据的发展帮助解决关键的临床问题。平台试验最近已用于研究 COVID-19 患者的不断发展的治疗方法。本《医学文献使用者指南》旨在描述平台试验和主方案的基本概念，并审查这些研究的实施和解释中的问题。本指南旨在帮助临床医生和读者理解使用平台试验设计评估干预措施的报告文章。
